Europaudvalget 2018
KOM (2018) 0051
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EUROPEAN
COMMISSION
Brussels, 31.1.2018
SWD(2018) 41 final
COMMISSION STAFF WORKING DOCUMENT
IMPACT ASSESSMENT
Strengthening of the EU Cooperation on Health Technology Assessment (HTA)
Accompanying the document
Proposal for a Regulation of the European Parliament and of the Council
on health technology assessment and amending Directive 2011/24/EU
{COM(2018) 51 final} - {SWD(2018) 42 final}
EN
EN
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1.
1.1.
1.2.
1.3.
1.4.
Introduction _________________________________________________________ 10
Context _________________________________________________________________ 10
What is HTA? ____________________________________________________________ 11
The role of HTA __________________________________________________________ 12
State of play _____________________________________________________________ 15
HTA in the Member States
___________________________________________________ 15
HTA at EU level
_____________________________________________________________ 21
1.4.1.
1.4.2.
1.5.
1.6.
Political context __________________________________________________________ 24
International outlook ______________________________________________________ 26
2.
Problem definition ____________________________________________________ 27
Problem 1. Impeded and distorted market access_____________________________________ 27
Problem 2. Duplication of work for national HTA bodies _______________________________ 32
Problem 3. Unsustainability of HTA cooperation ______________________________________ 39
3.
4.
5.
5.1.
5.2.
5.3.
Why should the EU act? ________________________________________________ 41
Policy objectives ______________________________________________________ 42
Policy options ________________________________________________________ 43
Discarded policy option ____________________________________________________ 44
Key characteristics of the policy options ______________________________________ 44
Description of the policy options ____________________________________________ 46
Policy option 1 (Baseline scenario). No Joint Actions after 2020
___________________ 46
Policy option 2. Project-based cooperation on HTA activities
______________________ 49
Policy option 3. Permanent cooperation on common tools, procedures and early
dialogues
__________________________________________________________________ 51
Policy option 4. Permanent cooperation on common tools, procedures, early
dialogues and joint REA______________________________________________________
53
5.3.1.
5.3.2.
5.3.3.
5.3.4.
6.
Impacts of the policy options ___________________________________________ 56
Policy option 2. Project-based cooperation on HTA activities ___________________________ 57
6.1.1.
6.1.2.
Economic impacts
___________________________________________________________ 57
Social impacts
______________________________________________________________ 60
6.2.
Policy option 3. Permanent cooperation on common tools, procedures and early
dialogues ________________________________________________________________ 62
Economic impacts
___________________________________________________________ 62
Social impacts
______________________________________________________________ 65
6.2.1.
6.2.2.
6.3.
Policy option 4. Permanent cooperation on common tools and procedures, early
dialogues and joint REA ____________________________________________________ 66
Economic impacts
___________________________________________________________ 67
6.3.1.
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6.3.2.
Social impacts
______________________________________________________________ 71
6.4.
Analysis of the governance structure and financing system _______________________ 72
Description of the governance arrangements
___________________________________ 72
Feasibility and efficiency of the governance arrangements
________________________ 76
Input from studies and stakeholders on the feasibility and efficiency of the
governance arrangements
___________________________________________________ 80
Costs related to the governance arrangements
__________________________________ 81
6.4.1.
6.4.2.
6.4.3.
6.4.4.
7.
8.
8.1.
Comparing policy options ______________________________________________ 84
Preferred policy option ________________________________________________ 91
Description of the preferred option __________________________________________ 91
Joint outputs
________________________________________________________________ 91
Technology scope
___________________________________________________________ 91
Instrument
__________________________________________________________________ 92
Governance and financing
____________________________________________________ 95
8.1.1.
8.1.2.
8.1.3.
8.1.4.
8.2.
8.3.
Justification of the preferred option __________________________________________ 97
Implications for Member States and other stakeholders ________________________ 101
9.
Monitoring and Evaluation ____________________________________________ 105
2
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ANNEXES TO THE IMPACT ASSESSMENT:
Annex I. Procedural Information
Annex II. Stakeholder Consultation: Synopsis Report
Annex III. Who is Affected by the Initiative
Annex IV. Analytical Models
Annex V. Health Technology Sectors
Annex VI. European Cooperation on HTA
Annex VII. International Outlook
Annex VIII. Earlier Market Access Calculations
Annex IX. Implementation Mechanisms and Policy Options
Annex X. Costs of Joint Outputs and Overall Costs per Policy Option and Implementation
Mechanisms
Annex XI. Implementation Tables for Preferred Policy Option
STUDIES CONDUCTED TO SUPPORT THE IMPACT ASSESSMENT
Accessible online at: https://ec.europa.eu/health/technology_assessment/policy_en
Mapping of HTA National Organisations, Programmes and Processes in EU and Norway.
2017, Contract nr. 17010402/2016/734820, ISBN: 978-92-79-77080-7
Mapping of HTA Methodologies in EU and Norway. 2017, DG SANTE Contract nr.
17010402/2016/736040, ISBN: 978-92-79-77074-6
Study on Impact Analysis of Policy Options for Strengthened EU Cooperation on HTA. 2017.
Sogeti, Austrian Public Health Institute, London School of Economics.
(CHAFEA/2016/Health/16), ISBN: 978-92-79-73966-8
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ABBREVIATIONS
CHAFEA
ED
EFPIA
EMA
EUnetHTA
EURORDIS
GÖG
HTA
ICT
IVD
JA
LSE
OECD
POP
RCT
REA
SEED
SMEs
WHO
Consumers, Health, Agriculture and Food Executive Agency
Early dialogue
European Federation of Pharmaceutical Industries and Associations
European Medicines Agency
European Network for Health Technology Assessment
Rare Diseases Europe
Austrian Public Health Institute / Gesundheit Österreich Forschungs-
und Planungs GmbH
Health Technology Assessment
Information and Communication Technology
In Vitro Diagnostics
Joint Action
London School of Economics
Organisation for Economic Co-operation and Development
EUnetHTA Planned and Ongoing Projects database
Randomised controlled clinical trials
Relative Effectiveness Assessment
Shaping European Early Dialogues for health technologies
Small and Medium Enterprises
World Health Organization
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GLOSSARY
1
Term or acronym
Additional data generation
Meaning or definition
Refers to the generation of additional clinical evidence in the course of
an HTA process and includes all studies and provision of data in
addition to the clinical studies carried out for the purpose of obtaining
marketing authorisation.
Refers to the drawing of conclusions on added value on the basis of the
scientific evidence presented in the HTA report, in order to inform
pricing and reimbursement decisions.
Clinical trials as defined in Regulation (EU) 536/2014
See 'REA' below
Refer to the areas of assessments covered by the HTA Core Model®.
Four are clinical domains (i.e. health problem and current use of
technology; description and technical characteristics of the technology;
safety; clinical effectiveness) and five are non-clinical (cost and
economic evaluation; ethical analysis; organisational aspects; patient
and social aspects; legal aspects). (EUnetHTA)
An early dialogue allows input from HTA bodies on the development
of the health technology. It focuses on development strategies and not
on pre-evaluation of data. The advice is prospective in nature (advice
on on-going trials is out of scope). Early dialogues can be requested
during the initial clinical development phase of the technology. For
pharmaceuticals, it should ideally be requested at the end of the phase
II to discuss the content of the planned Phase III i.e. planned
confirmatory trial(s) and the economic rationale. The objective of an
early dialogue is to reduce the risk of inadequate data when products
are presented for evaluation with the aim of reimbursement by national
health insurance.
(SEED project)
The European Network for Health Technology Assessment is a Joint
Action, co–funded by the Health Programme of the European
Commission (DG SANTE) and other participating actors. It gathers
mainly national and regional HTA bodies and also organisations using
HTA to support decision making. Its scope of activities is on scientific
and technical issues. (See Annex VI for more details on EUnetHTA's
activities)
Activities in which countries and/or organisations work together in
order to prepare shared products or agreed outcomes. These may
include, for example, literature reviews, structured information for
Appraisal
Clinical Trials
Clinical assessment
Domains
Early dialogue (see also 'Parallel
scientific advice/early dialogue'
below)
EUnetHTA
EUnetHTA joint work
1
The purpose of this glossary is to provide the reader with better understanding of the terms used in this IA. It
should in no way prejudge the terminology defined in the legal proposal.
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rapid or full HTAs, early dialogues or scientific advice on R&D
planning and study design. Joint work aims at supporting Member
States in providing objective, reliable, timely, transparent, comparable
and transferable information and enables an effective exchange of this
information. (HTA Network)
Economic assessment
The comparative analysis of the costs and consequences of two or more
possible options. Depending on whether the consequences are
expressed as monetary, physical or qualitative variables, the analysis
may be a cost-benefit, cost-effectiveness or cost-utility analysis.
(HTA Glossary.net)
The extent to which an intervention does more good than harm under
ideal circumstances, e.g. in a controlled clinical trial. (High Level
Pharmaceutical Forum, 2005
–
2008. Final Report)
The extent to which an intervention does more good than harm when
provided under the usual circumstances of health care practice. (High
Level Pharmaceutical Forum, 2005
–
2008. Final Report)
A (new) health technology that has not yet been adopted within the
healthcare system.
Note: pharmaceuticals in the Phase II or III clinical trial, or pre-launch
stage; medical devices are in the pre-marketing stage.
A health technology assessment covering not only the clinical domains
(i.e. REA), but also other non-clinical domains: cost and economic
analysis, ethical analysis, organisational aspects, patient and social
aspects, as well as legal considerations.
Health technologies refer to a pharmaceutical, a medical technology or
medical and surgical/radiation procedures as well as measures for
disease prevention, diagnosis or treatment used in healthcare (Directive
2011/24/EU).
Health technology assessment (HTA) is a multidisciplinary process that
summarises information about the medical, social, economic and
ethical issues related to the use of a health technology in a systematic,
transparent, unbiased, robust manner. Its aim is to inform the
formulation of safe, effective, health policies that are patient focused
and seek to achieve best value. (EUnetHTA)
The HTA Core Model is a methodological framework for collaborative
production and sharing of HTA information. It consists of three main
components:
The HTA ontology contains an extensive list of generic
questions that can be asked in an HTA. The ontology also
identifies relations between the questions
Methodological guidance helps researchers in finding answers
to the questions defined by the ontology. It recommends the
use of already existing, generally recognised guidance and
guidelines along with other methodological recommendations
and requires transparency on the methods used when applying
the HTA Core Model
Efficacy
Effectiveness
Emerging health technology
Full HTA
Health technologies
Health technology assessment
(HTA)
HTA Core Model
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A common reporting structure for presenting findings in a
standardised "question-answer pair" format
The Guiding Principles on the HTA Core Model Use provides the basic
principles on the Model's utilisation in various settings (EUnetHTA).
HTA methodologies
Should be understood as scientific and technical methodologies applied
by HTA institutions or groups of HTA researchers in the collection,
analysis and synthesis of evidence and information on health
technologies and their use in healthcare to inform decision making.
It is a voluntary Network set up under Article 15 of Directive 2011/24.
It gathers mainly Ministries of Health or competent authorities
responsible for HTA, appointed by Member States. Its scope of
activities is on strategic issues. The HTA Network works in synergy
and complementarity with the Joint Action(s) EUnetHTA, which
provides to the Network the technical and scientific expertise to foster
EU cooperation on HTA.
The systematic identification of health technologies that are new,
emerging or becoming obsolete and that have the potential to effect
health, health services and/or society.
(HTA Glossary.net)
Are collaborative projects specific to the Health Programme aiming to
develop / share / refine / test tools, methods and approaches to specific
issues or activities, and engage in capacity building in key areas of
interest for the Member States and countries participating to the
Programme. They are co-financed by the European Commission and
authorities of the Member States. This type of project was introduced
during the 2nd Health Programme (2008-2013) and continues under the
current one (2014-2020).
In this Impact Assessment, the term "Joint output" is used as an
umbrella term to cover any result of joint work in the context of the EU
cooperation. In particular it includes:
(1) “Technology Specific Reports" produced through cooperation (Joint
Early Dialogues, Joint Rapid Relative Effectiveness Assessments, Joint
Full Health Technology Assessments);
(2) “Common tools and procedures” essential for the cooperation,
including IT tools enabling exchanges and data gathering
(methodologies (e.g. EUnetHTA Core Model and Standard Operating
Procedures/SOP), horizon Scanning, submission templates and
templates for other key documents, training materials and other
capacity building activities).
"Joint reports" refer to REA and/or full HTA reports carried out jointly
by Member States HTA bodies according to jointly agreed HTA
methodologies and procedures.
Occurs for medicinal products after a market authorisation has been
granted (either at EU level or national level) and, for medical
technologies (i.e. medical devices, in vitro diagnostics), once the CE
marking is in place. It normally happens at Member State-level
following the conclusion of pricing and reimbursement negotiations or
when these are at an advanced stage. Market launch can subsequently
HTA Network
Horizon scanning
Joint actions
Joint output
Joint reports
Market launch
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occur in other Member States.
Medical Technologies
Medical devices and in vitro diagnostics as defined by Regulation (EU)
2017/745 and Regulation (EU) 2017/746 respectively
Multiple Technology Assessments are assessments which cover more
than one technology, or one technology for more than one indication
(cf. NICE technology appraisal guidance)
"National uptake" means that the joint output is used in national
decision making process (i.e. in the same way as an output carried out
at national level) and the joint activity is not duplicated (i.e. re-done) by
HTA bodies at national/regional level.
Currently the term is mainly used in the context of EUnetHTA output
(i.e. joint assessments, submission templates, guidelines, POP
Database, HTA Core Model®, etc.) where it refers to the general
implementation of a joint output in a local/national HTA setting.
Other health technologies
Other health technologies refer to interventions that typically involve
the use of pharmaceuticals, medical devices or diagnostics, but are
characterised by additional layers of complexity (e.g. use of one or
more technologies in the context of a medical procedure, or a
vaccination or screening programme).
It refers to the parallel/simultaneous scientific advice given by
regulators and HTA bodies to medicine developers on the appropriate
tests and studies to be carried out during the development of a new
medicine. It started as a pilot project in 2010 by the EMA.
As of July 2017 EMA offers consultations in parallel with EUnetHTA
in order to allow medicine developers to obtain feedback from
regulators and HTA bodies on their evidence-generation plans to
support decision-making on marketing authorisation and
reimbursement of new medicines at the same time.
Priority setting
The assignment of an order of priority based on explicit or implicit
criteria for selection of health technologies for assessment. (HTA
Glossary.net)
The POP database was set up by the EUnetHTA Joint Action and
allows HTA agencies to share information with each other on planned
and on-going projects conducted at the individual agency. The aim of
the database is to reduce duplication and facilitate collaboration among
HTA agencies.
Relative effectiveness can be defined as the extent to which an
intervention does more good than harm compared to one or more
intervention alternatives for achieving the desired results when
provided under the usual circumstances of health care practice (High
Level Pharmaceutical Forum 2005-2008, European Commission DG
Enterprise & Industry and DG Health & Consumers)
The Rapid Relative Effectiveness Assessment (REA) covers and is
limited to the clinical domains and measures the medical/therapeutic
Multiple Technology Assessments
(MTA)
National uptake
Parallel scientific advice/early
dialogue (see also 'early dialogue'
above)
Planned and Ongoing Projects
(POP) Database
Relative Effectiveness
Rapid Relative Effectiveness
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Assessment (REA)
SEED
added value of a technology. It is also called clinical assessment.
"Shaping European Early Dialogues for health technologies" was a
project running from 2013 to 2015, financed by the European
Commission for conducting pilots on early dialogues with health
technology developers (pharmaceuticals and medical devices) by
participating HTA bodies. The work was carried out based on
experience from and in synergy with the EUnetHTA Joint Action 2. In
total, eleven early dialogues were carried out are planned with an aim
to conduct 7 on medicinal products and 3 on medical devices.
Single Technology Assessment is an assessment of a single technology
for a single indication (NICE technology appraisal guidance)
Single Technology Assessment
(STA)
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1. Introduction
1.1. Context
The rapidly evolving health technology
2
market provides important opportunities to improve
public health by delivering better outcomes for patients and society as a whole. The health
technology market is also a key driver of economic growth and innovation in the Union.
Pharmaceuticals and medical technologies
3
are two large sectors of the Union’s health
technology market, contributing significantly and steadily to growth and job creation, even in
years of slower economic development. (For more information on these sectors, see Annex
V).
At the same time, in the EU, the total (public and private) health care expenditure amounts to
around EUR 1 300 billion per annum
4
(including EUR 220 billion for pharmaceuticals
5
and
EUR 110 billion for medical technologies).
6
Health care expenditure thus accounts on average
for approximately 10% of EU GDP.
7
This expenditure is likely to increase in the coming
years, given
inter alia
Europe’s ageing population, the increase of chronic diseases, and the
expected introduction of complex new technologies.
8,9
Simultaneously, Member States are
increasingly confronted with budgetary constraints which will require them to further improve
the efficiency of their health care systems in order to ensure the maximum benefit for
individual patients and public health in general.
10
In order to address the above mentioned challenges and opportunities and to balance various
interests, health technology assessment (HTA) has become an increasingly important tool
used to assist Member States in creating and maintaining sustainable health care systems and
to stimulate innovation that delivers better outcomes for patients
11
and cooperation is ongoing
at EU level.
This Impact Assessment has aimed to investigate opportunities for strengthening EU
cooperation on HTA, building on its achievements and addressing its shortcomings.
2
3
See section 1.2 for further details on the scope of health technologies.
For the purpose of this Impact Assessment, the term medical technologies comprises medical devices and in
vitro diagnostics as defined by Regulation (EU) 2017/745 and Regulation (EU) 2017/746 respectively (see
Annex V for further details).
4
Eurostat - expenditure of providers of health care using data from 2012 or latest data entry for all Member
States available. The figure is complemented by WHO Health data for the following countries: IE, IT, MT and
UK (ECB annual exchange rate).
5
Eurostat data, see DG GROW SWP 2014. Pharmaceutical industry: A Strategic Sector for the European
Economy.
6
Communication on Safe, effective and innovative medical devices and in vitro diagnostic medical devices for
the benefit of patients, consumers and healthcare professionals. COM(2012) 540 final. World Bank, EDMA,
Espicom and Eucomed calculations.
7
European Commission. European Semester Thematic Fiche: Health and Health systems, 2015.
8
DG ECFIN. The 2015 Ageing report, 2015. and DG ECFIN- EPC 2016. Joint Report on Health Care and Long-
Term Care Systems & Fiscal Sustainability, Institutional Paper 37. 7 October 2016
9
OECD. 2015. Pharmaceutical expenditure and policies: past trends and future challenges.
10
DG ECFIN. Cost-containment policies in public pharmaceutical spending in the EU, 2012.
11
Council conclusions on strengthening the balance in the pharmaceutical systems in the EU and its Member
States (2016/C 269/06).
10
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1.2. What is HTA?
In this Impact Assessment report, HTA is defined as
"a multidisciplinary process that
summarises information about the medical, social, economic and ethical issues related to the
use of a health technology in a systematic, transparent, unbiased, robust manner. Its aim is to
inform the formulation of safe, effective health policies that are patient focused and seek to
achieve best value".
12
The term ”health technology” is to be understood in a broad
sense
comprising pharmaceuticals, medical technologies (medical devices and in vitro
diagnostics)
13
and other technology-based tools for disease prevention, diagnosis or treatment
used in healthcare.
14
HTA is thus an evidence-based process that independently and objectively assesses a (new or
existing) technology and compares it to other/existing ones. A HTA can cover different
aspects (domains) ranging from clinical domains (e.g. safety, clinical effectiveness) to non-
clinical domains (e.g. economic, ethical, organisational etc.) (see Figure 1). Broadly speaking
two types of assessments can be distinguished: (1) the Rapid
Relative Effectiveness
Assessment (REA)
15
which covers the clinical domains and evaluates the medical/therapeutic
added value of a technology; and (2) the
full HTA,
which also includes other domains (e.g.
cost-effectiveness).
Whilst clinical assessments (REA) are often based on global evidence (e.g. global clinical
trials in the case of pharmaceuticals), full HTA assessments include domains that are more
sensitive to national/regional contexts.
Figure 1. HTA domains (based on EUnetHTA HTA Core Model)
12
EUnetHTA Joint Action definition.
Medical devices and in vitro diagnostics as defined by Regulation (EU) 2017/745 and Regulation (EU)
2017/746 respectively
14
This includes more complex health interventions that involve the use of pharmaceuticals, medical devices or
diagnostics (e.g. in the context of a medical procedure, or a vaccination or screening programme).
15
The generally accepted term is Relative Effectiveness Assessment. If REA is done at the time of marketing
authorisation, it is usually based on efficacy data from clinical trials. For re-assessments, the availability of data
on effectiveness is more frequent. Efficacy: is the extent to which an intervention does more good than harm
under ideal circumstances. Effectiveness is the extent to which an intervention does more good than harm when
provided under the usual circumstances of health care practice (High Level Pharmaceutical Forum, 2005
–
2008.
Final Report).
13
11
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1.3. The role of HTA
Before new health technologies are placed on the market, they are evaluated for their quality,
safety and efficacy (marketing authorisation of pharmaceuticals) or safety and performance
(CE marking of medical devices). While HTA builds on the evidence used for these
assessments, it focuses specifically on the potential benefits of a new health technology in
comparison to the existing standard of care in the health system. The box below discusses the
differences between marketing authorisation and HTA in more detail, including the added
value of HTA. Market access pathways for health technologies and relevant EU legislation
are described in detail in Annex V.
Differences between marketing authorisation and the clinical part of HTA (example of
pharmaceuticals)
Marketing authorisation and HTA have different remits and answer different questions, even if they
base their answers on some common evidence (e.g. pivotal clinical trials, typically phase III trials).
Marketing authorisation assesses the quality, safety and efficacy of an individual product. A marketing
authorisation is granted if a new product has a positive benefit-risk ratio in the sense that it is
efficacious and its safety profile is acceptable. It is not within the remit of the marketing authorisation
to determine the existing standard of care and to conduct a comparative assessment of the new
product against alternative products reflecting the standard of care. By contrast, the clinical part of
HTA (REA) assesses the added clinical value of a product, i.e. its relative effectiveness and relative
safety compared to one or more existing products (or other health interventions) reflecting the
standard of care. HTA therefore reviews and uses a broader evidence base than the assessment for
marketing authorisation: First of all, the evidence base on existing products/interventions needs to be
reviewed in order to determine the current standard of care. Subsequent steps of the HTA process
analyse in how far the pivotal clinical trials submitted for marketing authorisation purposes cover the
full spectrum of the standard of care. Frequently, these trials include one comparator, while the
standard of care includes more than one alternative pharmaceutical/intervention. HTA will therefore
review additional studies on other relevant pharmaceuticals/interventions and consider whether and
how this additional evidence can be assessed (e.g. via indirect comparisons or network meta-analysis
approaches).
Moreover, HTA aims to understand relative effectiveness under the conditions of usual clinical
practice (rather than under the ideal conditions of a controlled trial). Therefore, relevant data sources
for the clinical part of HTA go beyond the initial pivotal clinical trials and also include observational
(“real world”) data from clinical practice (e.g. disease-specific
patient registries, health data
recorded by health services and insurances). When HTA is conducted around the time of or shortly
after marketing authorisation, some limited effectiveness data may already be available (e.g. from
early access schemes in some EU Member States, or from another jurisdiction such as the U.S. where
the product was licensed earlier). Even if relative effectiveness data from clinical practice are not yet
available, HTA may use modelling approaches to predict relative effectiveness based on efficacy data
from pivotal trials. At later points in time (HTA re-assessments), relative effectiveness assessments can
typically draw on increasing sources and amounts of data from clinical practice.
Finally, the clinical part of HTA differs from the assessment for marketing authorisation in the way
that patient subgroups are considered and analysed. HTA aims to consider all patient subgroups that
are relevant for clinical practice (whether or not they may have been included and analysed in the
pivotal clinical trials for marketing authorisation purposes). To this end, HTA first considers whether
the existing standard of care differs for different patient subgroups within a particular therapeutic
indication. HTA then assesses whether and how the added value of the new pharmaceutical differs by
patient subgroup.
12
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It is also important to underline that HTA does not comprise pricing and reimbursement
decisions. However, HTA can substantially contribute to the
sustainability of health
systems
16
by providing scientific evidence/input for national decision-making on pricing and
reimbursement.
17
The link between HTA and the decision on pricing and reimbursement is
currently more pronounced in the case of pharmaceuticals than for medical technologies
(medical devices and in vitro diagnostics) and other health technologies (e.g. complex
interventions). For pharmaceuticals, pricing and reimbursement decisions are typically taken
at national/central level at times of market launch (or shortly thereafter), whereas for medical
and other health technologies such decisions are often taken in a more decentralised manner,
e.g. through local (hospital level) decisions or procurement processes, with more limited input
from HTA reports.
18
However, there is a growing trend towards applying HTA to support
decision-making also for medical technologies and other health technologies, including the
development of HTA methods and processes adapted to the specificities of these
technologies.
19,20
Figure 2 shows a schematic overview of the HTA step in the market access pathways for
pharmaceuticals and medical technologies. For further details, see Annex V.
Pharmaceuticals
Medical devices and in vitro diagnostics
Figure 2. HTA in the context of market access pathways for pharmaceuticals and medical
technologies
21
It should also be noted that in addition to informing national/local pricing and reimbursement
decision, HTA supports the development of evidence-based clinical guidelines (for diagnosis
European Commission
(DG
ECFIN- EPC), Joint Report on Health Care and Long-Term Care Systems &
Fiscal Sustainability
–
Volume 1, Institutional Paper 037 | October 2016.
17
A distinction also needs to be made between health technology “assessment” and “appraisal”. Assessment is
defined as compilation and critical evaluation of the available scientific evidence on all or selected domains,
whereas appraisal means that conclusions are drawn on the basis of the assessment results which are used to
support national or regional decision-making, typically on pricing and reimbursement. The scientific process of
assessment demonstrates potential for convergence and EU cooperation, whereas appraisals are not in line with
the explanation above.
18
See Annex V. Health Technology Sectors
19
Tarricone R., Torbica A., Drummond M. and MedtecHTA Project Group: Key Recommendations from the
MedtecHTA Project. Health Econ. 2017 Feb; 26, Suppl 1:145-152.
20
Lysdahl KB., Mozygemba K., Burns J., Brönneke JB., Chilcott JB., Ward S., Hofmann B.. Comprehensive
assessment of complex technologies: Integrating various aspects in health technology assessment. Int J Technol
Assess Health Care. 2017 Aug 7:1-7.
21
R&D: Research and development. P&R: pricing and reimbursement.
16
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and treatment of patients in particular therapeutic areas) and thus promotes evidence-based
healthcare. Finally, HTA is applied to both new technologies coming to the market and to
technologies which have been in use in healthcare for some time, i.e. it informs
(dis)investment decisions for new and existing health technologies. HTA thus helps to
prioritise health technologies with high added value and to de-prioritise technologies with no
or limited additional benefits (lower prices, disinvestment, and discontinued use). In
summary, HTA can facilitate evidence-based decision-making and efficient allocation of
resources in healthcare, ultimately supporting the optimisation of national healthcare systems.
Some individual studies offer a certain level of insight on the potential role of HTA in terms
of economic benefits.
22,23,24
A recent study from the UK,
25
focusing on 10 HTAs reached the
conclusion that a potential benefit of approximately GBP 3.0 billion/year could be achieved in
the UK if the recommendations from HTA reports were fully followed. Another study from
Austria found that for medical technologies in hospitals, HTA led to more reasonable
investments and saved several million euros at the level of a single hospital association.
26
In
the lower income countries, HTA is particularly important as these Member States have more
limited financial resources and the health status of their populations also tends to be lower.
27
A well-functioning HTA system also
improves business predictability
for industry and
creates and maintains a stimulus for innovation.
28,29
A predictable HTA system which rewards
innovations with added value for patients can influence longer-term R&D investment
decisions by industry and thus play an important role in incentivising
innovation for the
benefit of patients.
In particular, it can help to steer industry resources towards the
development of products that address unmet medical needs and significantly improve health
outcomes for patients.
30
HTA
can also contribute to greater transparency
and has the potential to improve the
involvement of key stakeholders,
such as patients and health professionals. Patients' input is
Schumacher I, Zechmeister I: Assessing the impact of health technology assessment on the Austrian healthcare
system. Int J Technol Assess Health Care 2013, 29:84–91.
23
Ognyanova, D, Zentner, A, and Busse, R. Pharmaceutical reform 2010 in Germany: striking a balance between
innovation and affordability. Eurohealth (Lond). 2011; 17: 11–13
24
Rosen, M. and S. Werko (2014). "Does health technology assessment affect policy-making and clinical
practice in Sweden?" International Journal of Technology Assessment in Health Care 30(3): 265-272.
25
Guthrie S, Hafner M, Bienkowska-Gibbs T and Wooding S, Returns on research funded under the NIHR.
Health Technology Assessment (HTA) Programme: Economic analysis and case studies. RAND Report RR-666-
DH, 2015. Estimated using assuming that recommendations of 10 HTA reports were followed in the UK during
the course of 1 year. Figure report the potential net-benefit including possible savings and health gains in terms
of QALYS using a value of £20,000 per QALY.
26
Schumacher I, Zechmeister I: Assessing the impact of health technology assessment on the Austrian healthcare
system. Int J Technol Assess Health Care 2013, 29:84–91.
27
Kaló, Z., Gheorghe, A., Huic, M., Csanádi, M., and Kristensen, F. B. (2016) HTA Implementation Roadmap
in Central and Eastern European Countries. Health Econ., 25: 179–192.
28
Kanavos, P., Manning M., Taylor D., Schurer W., Checch K., (2010): Implementing value-based pricing for
pharmaceuticals in the UK. London
29
OECD (2004): Increasing value for money in health systems: the quest for efficiency. In: Towards High-
Performing Health systems.
30
Rovira, J. et al, (2015) Health technology assessment and the Incentives to Innovation in the life Cycle of a
Health technology. In. Health Technology Assessment and Health Policy Today: A Multifaceted View of their
Unstable Crossroads.
22
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particularly relevant for assessing which treatment options improve their health-related
quality of life.
31
In the Open Public Consultation carried out by the Commission addressed to citizens, the
majority of the respondents (98%) indicated that they consider HTA useful for decision
making (see Annex II).
1.4. State of play
1.4.1. HTA in the Member States
In the last 20 years, all Member States have started to introduce HTA processes at national or
regional level (i.e. with 51 HTA bodies established in 26 Member States).
32
There are national
legal frameworks for HTA in place in 26 Member States and Norway; some Member States
are only at the initial phase of establishing HTA systems and/or have dedicated only limited
resources to HTA. Whilst there is some convergence in national HTA systems there are also
significant discrepancies. A summary of these discrepancies is set out below. For further
information see Annexes VIII, IX and X.
33
(a) Main differences in the procedural framework
1) The national HTA systems differ in the
scope of health technologies
that are being
assessed. Whilst the majority of Member States and Norway report national HTA activity for
pharmaceuticals,
two Member States have no such activities, but are in the process of
developing their national HTA systems. 20 Member States and Norway indicate having an
HTA system for
medical devices
(with five of these countries stating that it has not been
formalised yet). 17 Member States and Norway indicate that they have an HTA system for
other technologies,
whilst the remaining countries do not carry out such assessments.
2) National HTA organisations also differ in terms of
tasks
allocated. The main role of most
HTA organisations is to
carry out assessments and provide recommendations for decision
making
(i.e. pricing and reimbursement decisions). In addition to this main role, some HTA
bodies develop quality standards (12 Member States and Norway) and/or clinical guidelines
(14 Member States and Norway), perform horizon scanning
34
(10 Member States and
Norway), manage registries (11 Member States and Norway), or offer early
dialogues/scientific advice to health technologies developers (12 Member States and
Norway).
3) Concerning the
resources available
in the national HTA organisations, the study mapping
on HTA processes across the EU reveals that there are significant differences between MS. In
particular, the number of
staff
ranges from no human resources being dedicated to HTA
Patient Involvement in Health Technology Assessment in Europe. Results of the European Patients' Forum
Survey. 2013.
32
Mapping of HTA national organisations, programmes and processes in EU and Norway. 2017. Contract nr.
17010402/2016/734820, ISBN: 978-92-79-77080-7.
33
A short summary of such differences is also found in the ECP-ECFIN report on ageing. Getting more value for
money: the example of Health Technology Assessment (HTA). p. 100.
34
Horizon scanning refers to the systematic identification of emerging technologies that could have significant
effects on health care, and which might be considered for health technology assessment (WHO definition).
31
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activities (one Member State), to departments with less than 10 full-time equivalents (FTEs)
(seven Member States), to countries with more than 100 FTEs (four Member States) with a
maximum of 600 FTEs (one Member State). The available expertise of the core staff also
varies between HTA organisations.
35 36
As regards
financing,
HTA organisations from 26 EU
Member States and Norway
37
are public bodies, most of them being financed from public
money (annual budget allocated from governments). A combination of budget and service
fees directly received from industry is reported by five Member States. The data provided by
26 HTA bodies show that the annual budget allocated to HTA varies from no specific budget
(one Member State) or Member States with up to EUR 100 000 (four Member States) to
Member States with more than EUR 1 million (nine Member States and Norway), with a
maximum of EUR 70 million (one Member State). It has to be noted that the cost of one HTA
report varies considerably. In the survey conducted by the GÖG-LSE study
38
, the cost of an
HTA (single technology assessment) reported by HTA bodies ranged from EUR 4 000 to
EUR 135 000. This reflects differences in the resources available to different HTA bodies, but
also factors such as the scope and depth of the assessment (e.g. how much an HTA body
invests into conducting its own analysis to evaluate and contextualise the evidence generated
by industry)
39
.
4) In many Member States, HTA bodies consider a
dossier submitted by industry
in their
assessments. For
pharmaceuticals,
20 Member States reported that they carry out a review of
an industry submission of evidence. The extent of this review varies among HTA bodies and
can cover aspects such as missing evidence, errors in submitted evidence, internal and
external validity, as well as additional evidence analyses produced by the HTA body itself
(e.g. based on the scientific literature or clinical study registers). For
medical technologies
only 9 out 21 Member States reported that they review industry submissions, i.e. a greater
proportion of Member States carry out their own standalone assessments.
40
Gulacsi, L., Rotar A., Niewada, M., Loblova O., Rencz F., Petrova G., Boncz I., Klazinga, N.S. Health
technology assessment in Poland, the Czech Republic, Hungary, Romania and Bulgaria. Eur J Health Econ
(2014) 15 (Suppl 1):S13–S25
36
WHO, 2015 Global Survey on Health Technology Assessment by National Authorities - Main findings
37
Information from two Member States, Greece and Cyprus, which are currently in the process of setting up
their national HTA systems is not available.
38
Study on impact analysis of policy options for strengthened EU cooperation on HTA. 2017. Sogeti, Austrian
Public Health Institute, London School of Economics. CHAFEA/2016/Health/16. ISBN: 978-92-79-73966-8.
The GÖG-LSE Study is the main study supporting the Impact Assessment process by collecting evidence and
providing an in-depth analysis on the potential impacts of identified policy options for cooperation of the
European Commission (including the baseline scenario), and providing the relevant literature on HTA, with a
specific focus on the European Union. The study was carried out by a consortium consisting of Gesundheit
Österreich Forschungs- und Planungsgesellschaft (Austria), London School of Economics - LSE Health (UK)
and SOGETI (Luxembourg). For establishing the baseline scenario, a relevant sample of health technologies
which included 20 pharmaceuticals, 15 medical devices and 5 “other technologies” (including complex health
interventions) was analysed (i.e. HTA-process per type of technology and Member State, costs incurred by
technology developers/industry and HTA bodies per technology, influence of the legislative framework on
technology developers). The analysis of the impacts included a survey concerning the opinions of industry,
public administrations and other stakeholders on the potential economic and social impacts of the identified
policy options, complemented by focus groups, interviews and findings from literature review. A description of
the implementation mechanisms and an estimation of their costs were also provided. The study has been peer-
reviewed by leading experts in the field.
39
EUnetHTA WP7 draft report and study "Mapping of HTA national organisations, programmes and processes
in EU Member States and Norway"
40
EUnetHTA WP7 draft report
35
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5) As regards the
type of assessments,
all Member States carry out single technology
assessments (STA) (i.e. an assessment of a single technology compared with the standard of
care) and 13 Member States perform multiple technology assessments (MTA) (e.g. an
assessment of several technologies in use for a particular clinical indication).
41
For
pharmaceuticals
most of the Member States apply a single technology assessment (STA). Six
Member States and Norway indicate performing both STAs and MTAs.
For medical
technologies,
seven Member States and Norway indicate carrying out STAs and MTAs,
whilst only six Member States report carrying out only STAs for medical devices.
6) The
number of assessments
produced varies considerably between countries. The
mapping study of HTA procedures across Europe showed that the number of assessments
carried out by HTA bodies (single technology assessments of pharmaceuticals, medical and
other health technologies) ranged from about 5 HTA per year to up to 390 HTA per year. For
pharmaceuticals, some HTA bodies assess all new products (including generics, biosimilars)
and all licence extensions (including minor variations) of existing products. However, given
current national working practices, pharmaceutical topics that are most likely to be assessed
by many Member States across the EU are products with new active substances requiring
central marketing authorisation and major licence extensions of existing products.
42
For
medical devices, the number of assessments performed annually is lower than for
pharmaceuticals across all EU countries.
43
Innovative medical technologies with potentials to
transform the organisation of care (“transformative technologies”) and
medical technologies
subject to the scrutiny mechanism
44
are most likely to be covered in national assessments.
7) According to a survey of HTA bodies, the
time needed to complete
a health technology
assessment process (single technology assessment, from topic selection/identification to
delivery of HTA results) ranges from a few weeks to more than a year.
45
For pharmaceuticals
assessments based on industry submissions, the time from industry submission to completion
of the review ranges from less than 10 days to up to 200 days across HTA bodies, although
the majority of HTA bodies complete their review within 2-3 months.
46
In general the
timeframes for the assessment of medical technologies are longer than those for
pharmaceuticals across Member States.
47
41
For example, the assessment of a particular anti-cancer drug for the treatment of a specific type of cancer falls
into the category of STAs. The assessment of several anti-cancer drugs available for the treatment of a specific
type of cancer represents a MTA.
42
Recent EMA annual reports give an idea of the number of new active substances and new therapeutic
indications for existing products licensed per year. For example, in 2015, 39 new active substances and 54
extensions reflecting a new therapeutic indication of an existing product were licensed. The number of medical
devices receiving CE marking in 2015 is estimated to be around 4,500 (2015, MedTech Europe data)
43
Study "Mapping of HTA national organisations, programmes and processes in EU Member States and
Norway".
44
Mechanism for scrutiny of conformity assessments of certain class III and class IIb devices (as defined in
Regulation (EU) 2017/745, Article 55) and Mechanism for scrutiny of conformity assessments of class D
devices (as defined in Regulation (EU) 2017/746, Article 50)
45
Study "Mapping of HTA national organisations, programmes and processes in EU Member States and
Norway". Note that HTA bodies in some MS produce assessments which do not inform pricing and
reimbursement decisions (but are rather e.g. clinical guidelines).
46
EUnetHTA WP7 draft report (unpublished)
47
EUnetHTA WP7 draft report and study "Mapping of HTA national organisations, programmes and processes
in EU Member States and Norway"
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8) There are also differences across EU Member States in the
starting point
of the HTA
process. Figure 3 illustrates HTA timelines for pharmaceuticals in different EU Member
States compared with the EU marketing authorisation timeline (EMA process)
48
.
Figure 3. Stylised comparison between EUnetHTA and several national HTA timelines
(EFPIA/CRA Study)
The figure shows that depending on the Member State, HTA reports can be published after or
at the time of the last step of the marketing authorisation, while the HTA preparatory process
can already start in parallel
49
. However, in reality, HTA often takes place later, because HTA
submissions by industry are typically not initiated simultaneously or at the earliest possible
dates in all countries.
9) Another important aspect refers to
stakeholders' involvement
(patients, healthcare
providers, payers, etc.) throughout the HTA process which varies from country to country (for
further details, see Mapping study on HTA processes). It should be noted that even within the
Member States indicating that they engage stakeholders, there are significant differences in
the level of involvement
,
with stakeholders being consulted in one or more or all of the steps
of their HTA processes. In general for pharmaceutical assessments stakeholder involvement is
greatest towards the end of the assessment process when the assessment is reviewed and
advice/decision is made. For medical technologies there is greater stakeholder involvement in
the earlier stages (e.g. scoping, production of the assessment) than for pharmaceuticals, and
less involvement in the advice and decision making steps.
48
Note that the majority of new innovative medicines are authorised at EU level, via the centralised procedure.
For further details on the market access pathway for pharmaceutical, see Annex V.
49
EFPIA/Charles River Associates. 2017. Assessing the wider benefits of the EU’s proposal on strengthening
cooperation on health technology assessment from the industry perspective.
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(b) Main differences in methodologies
There are also divergences in the methodologies used by different HTA bodies when
assessing the evidence produced by technology developers.
50
For example, HTA bodies can
take different methodological approaches when assessing the acceptability of particular types
of studies and study design issues such as the comparator used, endpoints measured, the type
of patients enrolled and the duration of the study.
1) The choice of a
comparator
51
is decisive in any health technology assessment. A recent
study mapping HTA methodologies in EU Member States and Norway
52
showed that there
are commonalities but also differences in the criteria used for choosing a comparator. For
example, when assessing pharmaceuticals:
- 25 HTA bodies consider both whether the comparator reflects current healthcare practice
and whether its use is supported by evidence on its efficacy and safety, while 7 HTA bodies
consider only healthcare practice and 4 only the evidence base.
-
27 HTA bodies accept different technologies (i.e. also medical technologies and other
technologies) as possible comparators in a pharmaceuticals assessment, whilst 10 HTA bodies
compare pharmaceuticals only to other pharmaceuticals.
- 6 HTA bodies do not accept indirect comparisons
53
, whilst a large majority (i.e. 40 HTA
bodies) accepts such comparisons.
The process for choosing the comparator may also differ, for example the extent to which
proposals by the manufacturer or input from medical societies/healthcare professional
organisations are considered.
A similarly heterogeneous picture can be described for the selection of the comparator when
assessing medical and other technologies. In this regard, 20 HTA bodies reported using as a
comparator a technology likely to be replaced by the assessed technology if proven inferior
but also the comparator supported by evidence of its efficacy and safety profile. Six HTA
bodies reported using only the first type of comparator, whilst 4 other HTA bodies stated
using only the second one. Two HTA bodies informed that other criteria area also used when
choosing the comparator for assessing these categories of health technologies
52
.
However, there is scope for cooperation; in the GÖG-LSE case studies, in 68% of the cases,
the comparator included was the same across HTA bodies. Moreover, it should also be
50
References: Mapping study methods, GÖG-LSE study, Nicod 2017 (Eur J Health Econ), Nicod 2016
(International Journal of Technology Assessment in Health Care), Akehurst 2016 (Value in Health), Kleijnen
2016 (Annals of Oncology)
51
In the context of relative effectiveness assessment, a comparator is a health care intervention with which a
pharmaceutical is compared in order to establish if it has an added therapeutic benefit (in terms of clinical
effectiveness and/or safety). Such a comparator could be another pharmaceutical, a medical device, a procedure
or psychological approach, surgery or, if appropriate, providing advice, for example advice on diet or smoking,
any combination of these, or “watchful waiting” (no intervention).
52
Mapping of HTA methodologies in the EU and Norway. 2017. DG SANTE Contract nr.
17010402/2016/736040. ISBN: 978-92-79-77074-6.
53
The need for indirect comparisons arises when comparing treatments A and B, but the only available evidence
comes from studies comparing A with C and B with C. By using a common comparator, in this case treatment C,
it is possible to generate an indirect comparison with treatments A and B. For a variety of reasons, placebo-
controlled trials are commonly conducted in preference to head-to-head trials giving rise to the need for indirect
comparisons (EUnetHTA guidelines for comparators and comparisons).
19
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highlighted that, as noted in the study mapping HTA methodologies, HTA is closely linked to
the broader field of evidence-based medicine (EBM). Many developments in EBM are
already taking place at European level (e.g. development of evidence-based clinical guidelines
by European-level medical/scientific societies) or international level
54
. HTA bodies will thus
be increasingly able to draw on European-level or even international guidance when
considering the evidence-based standard of care.
2) Furthermore, HTA bodies have different methodological approaches with regard to certain
health outcomes and outcome measures.
For example, a minority of HTA bodies does not
accept surrogate endpoints
55
(one HTA body), composite endpoints
56
(nine HTA bodies) or
patient-reported outcomes (e.g. questionnaires on health-related quality of life) (10 HTA
bodies).
44
3) Another important difference in methodology relates to the
type of evidence/studies
accepted by HTA bodies.
Whilst the gold standard for all HTA bodies is randomised
controlled clinical trials (RCTs) and a small minority of HTA bodies (4) accept only RCTs,
the large majority of HTA bodies accept also other types of studies (e.g. observational
studies)
44
.
Case studies conducted in the context of the GÖG-LSE Study confirmed the differences but
also highlighted a tendency towards common methods in assessing products, which shows the
scope for cooperation. In particular the primary clinical trials considered were generally the
same type.
57, 58, 59
The diversity of approaches related to HTA methodology in the EU Member States is
confirmed by the input to the public consultation from both pharmaceutical and medical
technologies industry and described in section 2.
Regional cooperation on HTA
While this Impact assessment is being prepared some groups of countries have started to
develop stronger regional cooperation. Typically, this type of cooperation brings together
neighbouring Member States with similar socioeconomic situation, with the overall objective
of addressing the challenge of ensuring access to innovative technologies through possible
joint economic assessments, joint price negotiations and joint procurement. The
54
55
Examples include the work of Cochrane (http://www.cochrane.org/) and the Guidelines International)
In clinical trials, a surrogate endpoint (or marker) is a measure of effect of a specific treatment that may
correlate with a real clinical endpoint but does not necessarily have a guaranteed relationship. For example, the
serum cholesterol concentration may be considered a
surrogate endpoint
when assessing pharmaceuticals
aiming to prevent complications of cardiovascular disease. However, only about 10% of those with serum
cholesterol concentration above the reference range are going to have a stroke or heart attack. Therefore more
relevant clinical endpoints could be the number of nonfatal myocardial infarction or stroke cases.
56
Composite or combined endpoints
are defined as the combination of component (singleton) endpoints, each
of which has clinical significance in its own right. For example, a heart attack study may combine in a single
endpoint the number of patients who present with at least one clinical endpoint, either chest pain or myocardial
infarction, or death. The alternative would be to conduct RCTs with distinct clinical endpoints such as death and
nonfatal acute myocardial infarction (AMI).
57
GÖG-LSE Study, Section 7.1.9
58
Tarricone R, Torbica A, Drummond M, Schreyögg J. Assessment of medical devices: challenges and
solutions. Health Economics 2017; (S1):1-152.
59
Tarricone R, Boscolo P.R, Armeni P. What type of clinical evidence is needed to assess health technologies?
European Respiratory Review, 2016;25:259-265
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BENELUXA
60
, la Valletta cooperation
61
, the Nordic countries and the Visegrad cooperation
are examples of such regional cooperation recently set up (the first one about 18 months ago,
the most recent formalised its terms of references only a few weeks ago). The objectives of
the regional cooperation are different from the EU cooperation on HTA, which excludes
pricing and reimbursement. However, the tools and procedures developed by EUnetHTA are
being used to perform joint assessments. To date only BeNeLuxA has produced few Joint
assessments and La Valletta is in the process of identifying the technologies to assess in the
coming months.
Regional initiatives are also referred to in section(s) 5.2.1 and 6.1.1.
1.4.2. HTA at EU level
At EU level, the value of HTA and the fact that joint work could facilitate the implementation
of HTA processes and reduce redundancies regarding the assessment of technologies has been
recognised. Already in the 1980s, the Health Services Research Committee of the European
Commission began to assign contracts for economic appraisals and mechanisms for the
regulation of expensive health technologies in different countries. Between 1993 and 2002
three projects were funded by the European Commission to support collaboration on HTA
between Member States. In 2004, the European Commission and Council of Minsters
requested the establishment of a sustainable European network on HTA. This was initiated in
2005 when a group of 35 organisations started the EUnetHTA project, which explored
possibilities and key challenges for an enhanced transnational collaboration for the following
years.
Since then, to support cooperation between HTA bodies, the European Commission has made
substantial investments. Two Joint Actions (EUnetHTA JA) have been carried out together
with a number of projects
62
: (1) EUnetHTA 1 from 2010-2012 (budget EUR 6 million) and
(2) EUnetHTA 2 from 2012-2015 (budget EUR 9.5 million). A third Joint Action (EUnetHTA
3 - budget EUR 20 million) was launched in June 2016 and runs until 2020. Participation in
the Joint Actions has been very high and the latest Joint Action has more than 80 members
from all Member States and a number of observers from Member States, Norway and
Switzerland
63
The primary objective of the Joint Actions is scientific and technical cooperation, more
precisely to develop common methodologies, pilote and produce joint REA and full HTA
reports, and to develop common ICT tools. The Joint Action partners also piloted so called
early dialogues (i.e. a mechanism via which HTA bodies provide scientific advice to health
technology developers on the design of clinical trials
–
typically phase III or pivotal trials -
with a view to encouraging the generation of evidence that better meets the needs of HTA
60
BeNeLuxA is an initiative started by Belgium and the Netherlands (2015), later joined by Luxembourg and
Austria (2016). This group of countries intends to collaborate more closely across a range of areas: health
technology assessment; horizon scanning; exchange of information on pharmaceutical markets; prices and
disease-specific cross-border registries; and pricing and reimbursement, including joint negotiation.
61
Round table meeting for European Health Ministers and Heads of pharmaceutical companies (Malta, 9 May
2017)
62
Public Health Programme: EUR-ASSES 1994-1997; ECHTA/ECHAI 1999-2001; EUnetHTA 2006-2008.
Research Programme: AdHopHTA; INTEGRATE-HTA; MedtechHTA; Advance HTA.
63
Switzerland is an affiliated partner.
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agencies). All these activities in which HTA bodies prepare shared products/agreed outcomes
are referred to as “joint output”
64
.
More precisely, EUnetHTA 1 and 2:
- developed the HTA Core Model as a methodological framework for assessments, databases
for exchanging information (e.g. Planned and On-going Projects (POP) database allowing for
sharing of information on planned and on-going assessments in the Member States) and
- delivered a number of outputs, including 13 clinical assessments/REAs (7 on pharmaceutical
and 6 on non-pharmaceutical technologies such as medical devices), 5 Full HTA reports, 11
Early dialogues, 14 methodological guidelines, as well as common evidence submission
templates for pharmaceuticals and medical devices.
The ongoing Joint Action EUnetHTA 3 has planned to scale up the joint outputs, and aims to
deliver by the end of 2020 approximately 80 joint assessments and up to 35 early dialogues.
However, as the Joint Action 3 has so far only produced a small number of assessments, it is
currently uncertain if project will be able to deliver the target number of 80 joint assessments.
In addition, the importance of HTA cooperation at EU level is emphasised in Directive
2011/24/EU on the
application of patients’ rights in cross-border
healthcare, which states that
'cooperation in the evaluation of new health technologies can support Member States through
economies of scale and avoid duplication of effort, and provide a better evidence base for
optimal use of new technologies to ensure safe, high-quality and efficient healthcare'.
65
To
further strengthen the technical cooperation between HTA bodies, the Directive provides for
the establishment of a network connecting national authorities or bodies responsible for HTA
(HTA Network). Such a Network was set up in 2013 to provide strategic guidance and policy
orientation for the scientific and technical cooperation.
66,67
While participation in the
Network is voluntary, all Member States have applied for membership and participate. The
HTA Network develops policy papers
68
and discusses areas of potential collaboration
69
,
which are then implemented by the Joint Action, in accordance with its work plan.
The current cooperation model described above, in particular the Joint Actions as an
instrument to implement cooperation on HTA at scientific and technical level, is meant to
develop/share/refine/test tools, methods and approaches for specific issues or activities and
involve a degree of capacity-building
70
. In this respect the EUnetHTA Joint Actions have
been successful, as outlined by the Mid-term evaluation of the Public Health programme
71
.
Their usefulness has mainly been associated with an increased level of trust between HTA
bodies and stakeholders involved; increased knowledge of working procedures and
Or joint work in some EUnetHTA and HTA Network documents
Cross-border Healthcare Directive 2011/24/EU, RECITAL 58
66
Cross-border Healthcare Directive 2011/24/EU, Article 15.
67
Commission Implementing Decision of 26 June 2013 providing the rules for the establishment, management
and transparent functioning of the Network of national authorities or bodies responsible for health technology
assessment
68
HTA Network. Strategy for EU Cooperation on Health Technology Assessment. 2014
69
HTA Network
Reflection Paper on “Synergies Between Regulatory and HTA Issues on Pharmaceuticals.
2016.
70
Extract from the evaluation report on the second Public health programme
71
It should be noted that Joint Action EUnetHTA have not be subject to a specific evaluation in the context of
the Report, but only used as an example of growing interest in a specific policy area.
65
64
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methodologies in Member States and capacity building and sharing of best practices (see
report on the public consultation).
On the other hand, the current cooperation model has demonstrated important shortcomings
including:
Delays.
The current Joint Action has started with significant delays, due to a complex
and long negotiation process between the high number of beneficiaries and the funding
Agency. The process from the evaluation to the signature of the contract lasted nearly
one year. The late start was further aggravated by implementation challenges due to
non-delivery by certain beneficiaries and/or misunderstandings between beneficiaries
of the task(s) at stake. One important example is the delay in securing an efficient and
reliable IT infrastructure which would enable the cooperation to function.
Changes in Human Resources.
During and after the negotiation process some
beneficiaries which had agreed to take up important responsibilities and had been
allocated corresponding resources, have undergone reorganisations leading to changes
in priorities. This has caused dramatic decrease in the expertise and human resources
which were expected to be available from that beneficiary. While funding was
available to recruit the necessary staff, changes in priorities of the beneficiary
organisation prevented the recruitment. To date, after more than 12 months from the
start of the Joint Action, organisations leading key workplaces have not yet a full team
with the relevant expertise in place, with important consequences on the progress of
the activities.
High number of beneficiaries and heterogeneous profile/roles in national HTA
activities.
In the Joint Action model the Member States appoint organisations which
have an interest in the subject of the Action. Due to the high interest which
cooperation on HTA has generated in the EU the EUnetHTA Joint Action included at
its start 79 beneficiaries and at the time this report is written the number increased to
82. The large number also implies very heterogeneous profiles between the
beneficiaries, ranging from national HTA bodies with a statutory function in informing
decision makers for pricing and reimbursement decisions, to HTA bodies which has a
remit oriented toward development of clinical guidelines, to regional HTA agencies,
and also academic institutions with an interest in HTA but with no (official) role in the
national HTA/decision-making process. The high number of beneficiaries and their
heterogeneous profile and role in the decision-making process in the respective
countries increases significantly overheads to the coordinating agency, leading to
inefficiencies in the use of resources and creates challenges in identifying relevant
tasks for the appropriate profiles.
Uncertain delivery.
To produce a high quality and useful joint assessments the
involvement of technology developers has proven to be extremely important. Under
the current cooperation model such involvement is often seen as an add-on activity for
technology developers, which have to prepare submissions for national HTA
assessments and for joint assessments carried out under the Joint Action. This situation
is likely to persist as long as the uptake by national HTA bodies of Joint Assessments
is not happening to the desired extent. Both in Joint Action 2 and Joint Action 3, the
Commission in cooperation with the Joint Action secretariat and relevant trade
associations had to organise activities to trigger interest and commitment from
technology developers to submit technologies for Joint Assessments. While the
activities were successful, the engagement is not certain as it will depend on the ability
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of the Joint Action to secure uptake of joint assessments in national processes. This
situation brings an additional element of uncertainty to the ability of the Joint Action
to meet the planned target(s) within the necessary timeline.
Inconsistency of quality and timely delivery.
The examples outlined above result in
very uneven progress in the activities of the Joint Action, which in turn affects the
ability to deliver both in terms of time and quality of the output. The Joint Action is
highly dependent on the organisation responsible for the delivery of the task and even
on the technology developer whose technology is being assessed. While this issue can
be handled in an action which aims at developing and testing a proof of concept, it is
not acceptable when the objective is to timely deliver high quality output to be used in
national decision-making processes.
1.5. Political context
In recent years,
many key players have called for reinforced EU cooperation
in the area of
HTA. As regards
Member States,
a clear orientation was
contained in the “Strategy for EU
Cooperation on HTA”
72
, which was adopted by the Member States representatives in the
HTA Network in October 2014. In this document, the HTA Network called upon the
Commission to explore how to secure support for the joint work in the long-term. Moreover,
the
Council,
in its conclusions on “Innovation for the benefits of patients”
73
adopted in
December 2014, acknowledged the key role of HTA and called on the Commission to
continue to support sustainable cooperation. Furthermore, in the Council conclusions on
“Personalised medicines for patients” of December 2015
74
, the Member States and the
Commission were invited to reinforce HTA methodologies applicable to personalised
medicine. The Council conclusions on “Strengthening the balance
in the pharmaceutical
systems” in June 2016
75
confirmed again that Member States see a clear added value of EU
HTA cooperation. The joint report of DG ECFIN and the Economic Policy Committee calls
for further developing European cooperation on HTA.
76
The European Parliament
has also asked for a reinforcement of HTA cooperation at EU
level. In its joint motion for a resolution on the Commission Work Programme 2016, the
Parliament called for "a
step forward towards a common European Health Technology
Assessment"
77
. Moreover, in its resolution of 2 March 2017 on EU options for improving
access to medicines
78
, the Parliament calls on the Commission to "propose legislation on a
European system for health technology assessment as soon as possible, to harmonise
72
73
HTA Network. Strategy for EU Cooperation on Health Technology Assessment. 2014
Council conclusions on innovation for the benefit of patients (2014/C 438/06) .
74
Council conclusions on Personalised medicine for patients (2015/C 421/03),
75
Council conclusions on strengthening the balance in the pharmaceutical systems in the EU and its Member
States (2016/C 269/06)
76
DG ECFIN- EPC 2016. Joint Report on Health Care and Long-Term Care Systems & Fiscal Sustainability,
Institutional Paper 37. 7 October 2016
77
European Parliament resolution on the Commission Work Programme for 2016 (2015/2729(RSP)
78
European Parliament resolution of 2 March 2017 on EU options for improving access to medicines
(2016/2057(INI)
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transparent HTA criteria in order to assess the added therapeutic value of medicines". The
Parliament has also commissioned a study on HTA, highlighting its interest in the subject.
79
The
Commission
has on several occasions referred to HTA, including as a key part of
supporting other important Commission/EU initiatives. For example, the Commission
Communication on effective, resilient and accessible health systems
80
suggested HTA as one
way to build resilience. In a recent Staff Working Document, the lack of
“binding
mechanisms for mutual recognition of joint assessments”
was identified as one of the major
shortcomings of the current HTA system.
81
The Staff Working Document “Better Regulation
for innovation driven investment at EU level” pointed out that the fragmentation
of HTA
systems in the EU is currently “very
high”
and that the rise of personalised medicine
82
will
accelerate the concerns of fragmentation.
83
The recent Commission Communication
“Upgrading
the Single Market: more opportunities for people and business”
contained a
commitment that the Commission will introduce an initiative on HTA with a view to
improving the functioning of the Single Market of health technologies, in particular in order
to avoid duplication of efforts for Member States and industry.
84
Stakeholder views
A thorough stakeholder consultation has been carried out in the context of this Impact
Assessment in order to collect stakeholders’ views on EU cooperation on HTA beyond 2020
(see synopsis report, Annex II).
The usefulness of EU cooperation on HTA was underlined by the input provided by
stakeholders in response to the public consultation. Most of the contributors (69%) consider
EU cooperation on HTA useful or to some extent useful, with most benefit seen by public
administrations, payers and academia (100%) and less benefits typically seen by the medical
technologies industry (for more information, see Annex II). The most cited benefits of the EU
cooperation on HTA were the opportunity to share knowledge and best practices, contribute
to HTA capacity building in the Member States, contribute to building trust between
participating organisations and increase awareness (Figure 4).
Van Wilder, P. (Vrije Universiteit Brussel and SMART&BI), Mabilia V. (Milieu Ltd.), Kuipers CavacoY.
(Milieu Ltd.) and McGuinn J.(Milieu Ltd.), "Towards a Harmonised EU Assessment of the Added Therapeutic
Value of Medicines", Report commissioned by the Committee of Environment, Public Health and Food Safety at
the European Parliament, 2015.
80
Commission Communication "On effective, accessible and resilient health systems", COM (2014) 215 final
81
Commission Staff Working Document, "A Single Market Strategy for Europe
–
Analysis and Evidence",
SWD(2015) 202 final
82
Personalised medicine refers to a medical model using characterisation of individuals' phenotypes and
genotypes (e.g. molecular profiling, medical imaging, lifestyle data) for tailoring the right therapeutic strategy
for the right person at the right time, and/or to determine the predisposition to disease and/or to deliver timely
and targeted prevention (see Council conclusions on personalised medicine for patients, 2015/C 421/03).
83
Commission Staff Working Document "Better Regulation for innovation driven investment at EU level" SWD
(2015) 298 final.
84
Commission Communication “Upgrading the Single Market: more opportunities for people and business”
(COM (2015) 550 final.
79
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Figure 4. Benefits of EU cooperation on HTA as reported by the contributors to the public
consultation (Total number of replies = 186)
In general, Member States’ public authorities are in favour of continuing EU cooperation on
HTA beyond 2020. Some Member States have indicated a preference for voluntary
cooperation, while others support a system with mandatory elements. Most contributors
highlighted that in case of a mandatory framework, uptake of joint work should be limited to
clinical matters, whereas assessment of non-clinical domains (e.g. economic, ethical) should
be carried out individually or jointly by interested Member States on a voluntary basis. The
idea of a phase-in approach was also raised by some contributors.
1.6. International outlook
There is a growing world-wide recognition of the significant benefits of HTA.
85
The World
Health Organization (WHO) suggested that a global cooperation of HTA bodies would seem
useful, in particular on the clinical domains
86
and the World Bank supports the development
of national HTA programmes around the globe. A recent OECD report
87
assesses the state of
play and gives recommendations for the use of HTA. The International Monetary Fund also
promotes the development of national HTA systems.
88
A number of countries outside the EU
have developed well established HTA systems. The Canadian example of centralisation or the
regular review of the HTA system in Australia, are interesting examples (see Annex VII). It is
also interesting to note the setting up of networks between HTA bodies in other parts of the
world, following the model of EUnetHTA
89
.
85
Ciani O, Wilcher B, Blankart CR, et al. Health Technology Assessment of Medical Devices: A Survey of
Non-European Union Agencies. International Journal of Technology Assessment in Health Care.
2015;31(3):154-165.
86
WHO resolution on Health intervention and technology assessment in support of universal health coverage.
87
OECD (2017), New Health Technologies: Managing Access, Value and Sustainability, OECD Publishing,
Paris.
88
Examples include the Memoranda of Understanding for Greece, Portugal, Cyprus or Romania.
89
Regional Network for HTA for the Americas RedETSA.
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2. Problem definition
While HTA is considered a valuable tool for ensuring sustainability of health systems and
stimulating innovation and cooperation at EU level, evidence shows that a series of
shortcomings affect the exploitation of the benefits for Member States and economic
operators, with subsequent negative consequences also for EU patients and healthcare
professionals.
Figure 5. Main problems, their drivers and consequences
Problem 1. Impeded and distorted market access
The
different national processes and methodologies of national and regional HTA bodies
illustrated in section 1.4.1 (and Annex V) mean that economic operators who want to
introduce a health technology in multiple Member States are confronted with various data
requests. This in turn contributes to an impeded and distorted market access, leading to lack of
business predictability, higher costs, and in the long run negative effects on innovation.
Differences in national processes and methodologies also lead to differences in how evidence
is considered in assessments and to potentially different HTA conclusions, which can
contribute to delays and inequalities in patient access to technologies
90
.
The differences in HTA processes and their effects were underlined by various stakeholders in
the public consultation. The most significant impact was reported by representatives of the
pharmaceutical industry who pointed out that this diversity constitutes a hurdle for companies,
as they have to
adapt to multiple and various national requirements
(e.g. regarding the
starting moment of the assessment, data requirements, length of the procedures, scope of HTA
and type of assessment carried out). With regard to differences in HTA methodologies,
90
Valzania C, Torbica A, Tarricone R, Leyva F, Boriani G. Implant rates of cardiac implantable electrical
devices in Europe: a systematic literature review. Health Policy 2016;120(1):1-15
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representatives of the pharmaceutical industry emphasised (among others) differences with
respect to the acceptability of particular comparators, endpoints and data other than
randomised clinical trials, which contribute to the
lack of business predictability due to
different outcomes of national HTA clinical reports
91
.
Duplication of assessments also
increases costs for industry,
which needs to prepare dossiers
for multiple national systems with potentially different data requirements. Requirements for
additional evidence are a key cost component, with potential delays/risks in market access.
Costs related to additional evidence requirements are particularly high if they necessitate
carrying out new trials.
92
The GÖG-LSE study provides an indication of current costs for
industry related to the meeting current national HTA requirements (see text box below).
Current costs for industry related to the preparation of national HTA dossiers
On the basis of the figures reported in the survey carried out by the GÖG-LSE study
93
, the costs for
industry related to national pharmaceutical HTA processes are summarised in the table below.
The survey carried out by the GÖG-LSE Study indicates that earmarking the costs for one single
assessment is difficult as manufacturers prepare a global value dossier for each product, which is
usually the main source of input for their HTA departments/teams. Moreover, certain costs can pertain
also to the regulatory (e.g. related to evidence) or pricing and reimbursement processes (e.g. in-
country staff costs).
HTA costs are particularly relevant for SMEs as they typically do not have structures or resources
dedicated to HTA, or the in-country capacities needed to adapt to multiple national requirements and
formats. In the open public consultation carried out by the Commission, a higher proportion of SMEs
indicated that differences among EU Member States regarding HTA procedures and/or methodologies
contributed to high costs/expenses for their organisation. In the dedicated SME consultation, 75% of
pharmaceutical and 71% of medical technologies companies indicated that differences among EU
91
Nicod E, Why do health technology assessment coverage recommendations for the same drugs differ across
settings? Applying a mixed methods framework to systematically compare orphan drug decisions in four
European countries. Eur J Health Econ. 2017 Jul;18(6):715-730.
92
Martin L, Hutchens M, Radnoy A, How much do clinical trials cost?, Nature Reviews Drug Discovery, 16,
381–382 (2017). Note that this study estimated the average cost of a phase III clinical trial at USD 21.4 million.
93
The GÖG-LSE survey asked respondents to (1) indicate their current HTA related costs and (2) express their
expectations regarding the impacts in a number of areas of the 5 policy options described in the Inception Impact
Assessment on strengthening EU cooperation on HTA. These areas cover economic and social aspects and are
defined by Better Regulation Guidelines of the European Commission; for each area, the study defined specific
indicators. 120 stakeholders of the medical technologies industry, 20 stakeholders of the pharmaceutical industry
and 37 stakeholders representing public administration and other organisations (total 177) participated.
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Member States regarding HTA procedures and/or methodologies may contribute to high
costs/expenses for their organisation. The same figure was 44% for non-SMEs in the public
consultation, confirming the expectation that differences are more difficult to handle by SMEs.
With regard to additional data generation, it should be noted that it is mainly the requirements of the
largest markets that companies take into account when designing clinical trials or generating
additional evidence.
In their input to the online public consultation it was highlighted that while most of the larger
pharmaceutical companies resort to national affiliates to engage with national authorities and
ensure adequate preparation of the documentation requested by HTA bodies,
smaller
companies with limited resources
may face difficulties in putting in place such a
mechanism, which could create a discriminatory environment and discrepancies in the speed
of market access. This situation is confirmed by a recent report from EuropaBio showing that
SMEs have limited experience in working with HTA bodies and may not have staff dedicated
to HTA work. According to EuropaBio, this has led to some products getting marketing
authorisation but not being recommended by the HTA bodies because the data was not
sufficient to establish the required added clinical and/or economic value.
94
The variety of HTA processes and methodologies is also reflected in the
HTA spending by
industry.
For example, the HTA spending by the
pharmaceutical sector
ranges from EUR 73
000 and EUR 1 700 000 per HTA submission
95
(depending also on the type of assessment).
Extra evidence generation is responsible for additional expenditure (between EUR 50 000 and
EUR 20 000 000/submission), with 85% of companies reporting such costs.
96
Furthermore,
the pharmaceutical sector actively engages in early dialogues with HTA bodies (69% of
responses) with an average cost of EUR 55 750 per case, but which have the potential to
alleviate costs in the next phases of development/approval/access to market. With regards to
the
medical technology industry,
HTA submission dossiers range from EUR 1 000 to EUR 3
400 000
97
. Additional evidence generation in the context of a HTA submission dossier had a
range of EUR 17 000 - EUR 12 800 000, with 37% of companies reporting such costs
98
. Early
dialogues are not a routine procedure in the sector.
99
While the costs related to HTA
submissions may not always be significant (seen in the context of overall industry spending) it
is important to note that the parallel submissions and assessments by national HTA bodies,
entail a significant risk of divergent outcomes in different Member States, which has a
negative impact on business predictability.
Insufficiently predictable, fragmented and
94
Deerfield Institute - EuropaBio Report on regulatory and HTA scientific advice for small and medium
enterprises, 2015.
95
These values represent a sum of average costs per HTA submission per company as reported to the GÖG-LSE
survey. Companies reported staff costs, consultant costs, in-house model costs, external model costs, and other
costs. Section 7.1.12.
96
Other calculations confirm this range: the Ecorys report on European Cooperation on Health Technology
Assessment: Economic and governance analysis of the establishment of a permanent secretariat, (2013)
estimates the total costs for industry at 200.000 €. These cost figures do not capture the full costs, as they only
refer to the human resources needed to prepare the submission dossiers (for industry)
97
These values represent a sum of average costs per HTA submission per company (comprising staff costs,
consultant costs, in-house model cost, external model costs, and other costs) through the survey carried out by
the GÖG-LSE Study.
98
It should be noted that obligations for additional evidence generation for medical technology is much less
frequent than for pharmaceuticals, due to the different regulatory process.
99
GÖG-LSE Study Section 7.1.12
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delayed market access is therefore the most important shortcoming resulting from the HTA
fragmentation across the EU (see impact estimate in chapter 6.4.1.1).
As confirmed in interviews and focus groups with pharmaceutical industry representatives,
poor business predictability and high fragmentation of HTA systems across Europe constitute
barriers to investment by industry in development programmes for innovative
technologies.
100
Finally, as described in section 1.4.1 (and
GÖG-LSE Study Section 7.1.7 and 7.1.14
) the high
variability in the timing (in terms of both the starting point and the duration) of assessments
contributes to
differences in the availability of health technologies for EU patients.
101
Moreover, divergences in the conclusions of HTA reports on added value, which are due to
different approaches taken by HTA bodies as described above, contribute to differences in
availability of medicines to EU patients
102
. As illustrated by Table 1 for a sample of cancer
drugs, there are common trends but also discrepancies in the conclusions reached by different
HTA bodies on the same product. The observed divergences between HTA bodies are due to
differences in the clinical part of HTA (REA) and/or the economic part of HTA. In several
examples, divergences in HTA conclusions were influenced by how different HTA bodies
assessed effects seen for particular clinical endpoints (when assessing the new drug against
the same comparator). The authors note the potential for European consensus-building on
these clinical aspects, which may be informed by ongoing scientific initiatives of
European/international medical societies. Other case studies for other pharmaceutical
products and therapeutic areas show similar results, confirming both current HTA divergences
but also the potential for improved scientific consensus-building on the clinical part of HTA
103,104
.
GÖG-LSE Study, Section 7.1.13
GÖG-LSE Study, Mapping HTA procedures, Akehurst 2016 (Value in Health)
102
References: Impacts study, Nicod 2017 (Eur J Health Econ), Nicod 2016 (International Journal of
Technology Assessment in Health Care), Akehurst 2016 (Value in Health), Kleijnen 2016 (Annals of Oncology)
103
Impacts study, Nicod 2017 (Eur J Health Econ), Nicod 2016 (International Journal of Technology
Assessment in Health Care), Akehurst 2016 (Value in Health)
104
Spinner DS, Birt J, Walter JW, Bowman L, Mauskopf J, Drummond MF, Copley-Merriman C, Do different
clinical evidence bases lead to discordant health-technology assessment decisions? An in-depth case series
across three jurisdictions, Clinicoecon Outcomes Res. 2013; 5: 69–85.
101
100
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Table 1. Conclusions of HTA reports across a sample of cancer drugs
105
For
medical technologies,
the case studies conducted by the GÖG-LSE study indicate an
overall lower number of assessments produced by HTA bodies across Europe than for
pharmaceuticals (see Table 2). However, the case studies also showed several examples
where different HTA bodies reached divergent conclusions on the same medical technology
due to differences in the clinical and/or economic parts of their assessments (see GÖG-LSE
study for further details
106
). Increased HTA activities in Member States on medical
technologies (see problem 2) have the potential to increase further the number of parallel
assessments on the same technologies. Considering the different national processes and
methodologies, this is likely to lead to increased divergence across the EU and thus
fragmentation of the market.
The considerations above are confirmed by the overall input received from the online public
consultation. According to the respondents, the main consequences of the differences in HTA
processes and methodologies across the EU are the diverging outcomes of HTA reports which
may
affect patients' access to new technologies
(e.g. delays, restricted access) (81% of
contributions), followed by
duplication of work for both HTA bodies and industry
(54%),
decrease in business predictability
(53%), higher costs for the actors (38%) and
negative
effect on innovation
(37%)(Figure 6). Further details on the public consultation, including
results by stakeholder group, are presented in Annex II.
105
Figure courtesy of W. Goettsch (Presentation to EHFG 2016). Based on data published in "Relative
effectiveness assessments of oncology medicines for pricing and reimbursement decisions in European
countries.", Kleijnen S, Lipska I,,Leonardo
Alves T, Meijboom K, Elsada A, Vervölgyi V, D’Andon A, Timoney
A, Leufkens HG, de Boer A, Goettsch WG. Ann Oncol (2016) 27 (9): 1768-1775 (see Supplementary Table 4 to
the publication for further details on comparators and clinical endpoints considered).
106
GÖG-LSE Study (Table 26 and Annexes to the study)
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Figure 6. Consequences of differences in HTA process and methodologies across the EU as
identified by public administrations, organisations and associations responding to the online
public consultation (Total number of replies = 186)
Problem 2. Duplication of work for national HTA bodies
The duplication of work refers to assessments of the same technology being conducted in
parallel or within a similar time frame by HTA bodies in different Member States. The text
box below illustrates current costs for HTA bodies related to the production of REAs at
national level, as reported by the GÖG-LSE study.
Current costs for HTA bodies related to the production of REAs at national level
According to the figures reported in the survey carried out by the GÖG-LSE study, costs for HTA
bodies range from an average of EUR 35 000 for a REA produced mainly by an HTA body and EUR
20 000 per REA produced by industry and reviewed by an HTA body to EUR 95 000 for a full HTA
produced by an HTA body and EUR 40 000 for the cases in which the full HTA is produced by
industry and reviewed by HTA body. Further research and benchmarking of these figures with
additional HTA bodies not included in the survey indicate that these costs may be underestimated.
This means that the duplications expected to continue under this scenario would have an impact on the
spending/budget of Member States and public administrations
In addition, the current low uptake of joint REA undertaken by the EUnetHTA results in
duplication and incurs
additional work and costs.
The duplication may be associated with
different outcomes/conclusions (depending on the type of assessment and applied
methodology), which negatively
affects business predictability
and contributes to
delays
and inequalities in patient access.
The GÖG-LSE Study
107
found that in a sample of 20
pharmaceuticals,
8-15 HTA reports
were conducted by different Member States for each individual product.
108
This indicates that
107
108
Section 7.1.2
The study aimed to build a representative sample; however in order to have sufficient data for analysis, one of
the criteria for inclusion for a pharmaceutical was to have been assessed by at least 5 MS between 2012-2016.
Therefore the result cannot be generalised to conclude that all pharmaceuticals undergo on average X HTA
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there is a group of pharmaceuticals (typically innovative, centrally authorised, approximately
40 new molecules/year) where there is a significant duplication of work for HTA bodies and
industry. At the same time, the report concludes that not all Member States have adequate
capacities to produce HTAs on all relevant products.
Although a lower number of Member States assess
medical and other technologies
than
pharmaceuticals
109
(Table 2) there is still considerable duplication of efforts also in these
sectors; albeit to a more limited extent than in the case of pharmaceuticals (3-8 reports per
technology in the sample).
110 111 112
processes, but it can be concluded that there is a group of pharmaceuticals (typically innovative, centrally
authorised) where there is an important duplication of HTA.
109
Study "Mapping of HTA national organisations, programmes and processes in EU Member States and
Norway": 23 MS indicated assessing pharmaceuticals, 20 MS for medical technologies and 17 MS for other
technologies. Moreover while all 23 MS conducting pharmaceuticals use it for pricing and reimbursement
decision, only 19 do so for medical devices and 9 for other technologies.
110
Section 7.1.2 Just as in the case of pharmaceuticals, having sufficient number of HTA reports was a criterion
for selection. Therefore it should not be concluded that this figure is representative for all medical technologies
but that there is a group of medical technologies where important duplications take place.
111
Tarricone R, Callea G, Ogorevc M, Prevolnik Rupel V. Improving the methods for the economic evaluation
of medical devices. Health Economics 2017;26(Suppl S1):70-92
112
Rummel, P, Hawlik, K, Wild, C. Health Technology Assessments on Medical Devices in Europe. Final
Report. LBI-HTA Rapid Assessment Nr. 12; 2016. Vienna: Ludwig Boltzmann Institute for Health Technology
Assessment.
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TOTAL ASSESSED
TOTAL ASSESSED
Pharmaceuticals
Included
Medical Devices
Included
Other Technologies
Included
Abiraterone
Aclidinium Bromidium
Alemtuzumab
Apremilast
Ataluren
C anagliflozin
Dapagliflozin
Defibrotide
Ivacaftor
Mirabegron
Nivolumab
Nintedanib
Ocriplasmin
Ofatumumab
Omalizumab
Pasireotide
Ramucirumab
Rilpivirine
Riociguat
Sofosbuvir
average
13
12
12
10
10
14
13
8
11
10
12
13
11
12
10
8
14
10
12
15
11,5
Endovascular stents
Home haemodialysis
device
Transcatheter
implantable devices
Balloon Eustachian
Tuboplasty
Oscillometric blood
pressure monitor
High intensity focused
ultrasound (HIFU)
Self-monitoring
coagulometers
Positron emission
tomography (PET)
C ochlear implants
Left ventricular assist
devices
LASER KTP
Gene expression
profiling diagnostics
Nucleic acid
amplification tests
(NAATs)
Duodenal-jejunal
bypass sleeve
In-vitro fertilisation
(IVF)
average
6
5
6
4
4
8
7
6
8
6
8
7
3
7
5
6
HPV Vaccination
C olorectal C ancer Screening
Pneumococcal Vaccination
Rotavirus Vaccination
C ervical cancer screening
average
10
4
3
6
6,2
Table 2. Number of assessments carried out by HTA bodies in the Member States reflecting
duplication of assessments - case studies
In the public consultation, MedTech Europe argued that the duplication of efforts is not so
prominent for medical technologies. However, recent publications have shown increasing
duplication of assessments also for medical technologies.
113, 114
This is in line with current
trends for increased HTA activities on medical technologies in Member States (see box
below).
113
Tarricone R, Callea G, Ogorevc M, Prevolnik Rupel V. Improving the methods for the economic evaluation
of medical devices. Health Economics 2017;26(Suppl S1):70-92.
114
Hawlik K, Rummel P, Wild C, Analysis of duplication and timing of health technology assessments of
medical devices in Europe, International Journal of Technology Assessment in Health Care (accepted for
publication in October 2017)
TOTAL ASSESSED
8
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Examples of Member States confirming the growing trend towards applying HTA to support
decision-making for medical technologies
Spain.
The HTA Spanish Network (which includes the regional HTA agencies of seven autonomous
regions and representatives from the regional health-care administrations of the remaining regions) is
fully operational since few years and relies on regional HTA agencies to perform and coordinate HTA
work with a focus on medical technologies. The network has been instrumental to increase the
evidence-based information available to local health authorities to take decision on access. It has
contributed to build capacity, increase consistency and quality of HTA within Spain and avoid
115
duplication of assessments on medical technologies .
Italy.
A recently adopted "national programme for HTA on medical technologies" is the response to
the growing need of HTA for medical technologies in Italy. The programme has established a Steering
Committee, coordinated by the Ministry of Health and gathering key national agencies and the
regional HTA bodies which have expertise and perform HTA and foresees an active engagement of
stakeholders’ organisations. The final objective of the Programme, similarly to the one of the Spanish
HTA Network, is to increase the availability of HTA for medical technologies, to provide guidance to
decision makers, increase consistency and avoid duplication of assessments for better use of
resources
116
.
United Kingdom.
While the National Institute for Health and Care Excellence/NICE has traditionally
focused on pharmaceuticals, in the last years it established a dedicated programme for assessments of
117
medical technologies .
The issue of duplication was confirmed by the input received in response to the public
consultation, where 57% of all respondents and 53% of participating public administrations
indicated that differences in HTA processes and methodologies also resulted in duplication of
work for their organisation.
Differences in methodologies and procedures are considered significant obstacles for EU
cooperation on HTA,
limiting also the possibility of pooling resources
and of a full benefit
from the potential efficiency gains at EU level
118
.
The
uptake of joint EU outputs
(i.e. joint tools
–
EUnetHTA Core Model, guidelines, joint
early dialogues, joint REA, joint full HTA)
at national level has remained low.
Despite the
fact that a joint European report was prepared, most Member States still performed
assessments of the same technology. The low uptake of joint reports was confirmed by the
evaluation report of EUnetHTA Joint Action 2. Looking at the 11 joint assessments carried
out under this second Joint Action, on average each European assessment was used in the EU
Member States 6.4 times: twice related to direct decision-making; 3.3 times for cross-
checking evidence or as a source of information; 0.7 times related to the category 'other' and
for 0.4 times no data was indicated.
119
115
L. Sanpietro-Colon, J. Martin Eds, Hospital-Based Health Technology Assessment. The Next Frontier for
Health Technology Assessment. Springer International Publishing 2016, pg.78
116
http://www.salute.gov.it/portale/temi/p2_6.jsp?id=1202&area=dispositivi-medici&menu=tecnologie
117
HTA National Programme for medical technologies - Strategy Document 19 September 2017
https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-medical-technologies-guidance
118
Kleijnen S, Toenders W, de Groot F, Huic M, George E, Wieseler B, Pavlovic M, Bucsics A, Siviero PD, van
der Graaff M, Rdzany R, Kristensen FB, Goettsch W. European collaboration on relative effectiveness
assessments: What is needed to be successful? Health Policy. 2015 May;119(5):569-76.
119
http://www.eunethta.eu/national-uptake
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The low uptake of joint outputs is confirmed by the HTA GÖG-LSE Study which points out
that most national HTA bodies, despite taking part in the joint assessments, did not adequately
make use of the resulting output.
Barriers to uptake have been analysed in several documents, including the HTA Network
reflection paper on “Reuse of Joint Work in National HTA Activities”
120
, a survey carried out
by EUnetHTA Joint Action 2
121
a study commissioned by EFPIA
122
, and publications in peer-
reviewed journals
118, 123,124
. Barriers to uptake were also discussed by national organisations in
their replies to the public consultation.
Across the above-mentioned sources, the following main hurdles to uptake were identified:
Legal uncertainty:
Uncertainty around the status/relevance of the joint outputs in the
context of national HTA frameworks constitutes a major reason for the current low
uptake. As discussed in section 1.4.1., there are national legal/procedural frameworks for
HTA in place in 26 Member States (also see Annexes VIII and IX for further details). As
part of these national frameworks, the preparation and uptake of e.g. national clinical
assessments is regulated at national level. While there is some diversity due to the
different legal systems, in general, key provisions related to the roles and responsibilities
of the HTA bodies and the HTA assessments are outlined in national law, while further
details are elaborated in administrative provisions (e.g. in procedural rules). By contrast,
the legal status of joint outputs stemming from the EUnetHTA Joint Action and their
relevance for national HTA processes is not defined, making difficult for national decision
makers to adapt their national legal framework to joint outputs.
Concerns around timeliness:
The timely availability of the joint output (e.g. joint REA
report) for national decision making process has been underlined as another important
limitation leading to low uptake. While in national HTA systems, timelines are enforced
as defined in respective legal/procedural HTA frameworks, the EUnetHTA Joint Actions
have so far not been able to ensure timeliness of joint outputs (e.g. joint REA reports) to
meet Member States needs and feed into national decision-making processes.
Concerns around quality:
As was highlighted in the public consultation, Member States
will only use a joint REA report if it is of high quality. Some respondents in the public
consultation considered that the first reports prepared under the first two EUnetHTA Joint
Actions were of sub-optimal quality. While national HTA systems have established
standard operating procedures (SOP) and dedicated quality assurance mechanisms for
national work, it has so far not been possible to ensure comparable procedural and quality
standards for the production of joint work under the EUnetHTA Joint Actions.
Furthermore, there continue to be a number of methodological differences between HTA
HTA Network. 2015.
Reflection paper on “Reuse of Joint Work in National HTA Activities”.
EUnetHTA JA2. WP3 DELIVERABLE Report on evaluation of project completion including assessment of
impact on secondary users of HTA information p. 23.
122
Charles Rivers Associates. 2017.
EU REA
–
A discussion of barriers for adoption and possible actions to
overcome them Main findings.
123
Lo Scalzo A, Vicari N, Corio M, Perrini MR, Jefferson T, Gillespie F, Cerbo M. Collaborative models for the
joint production of core health technology assessments: negative and positive aspects for the joint work of
different European agencies. Int J Technol Assess Health Care. 2014 Nov;30(5):536-43.
124
Vondeling H, Sandvei M. Is the planned and ongoing project (POP) database a suitable tool to reduce
duplication in the process of assessing new health technologies in the European Union? Preliminary experience
in the context of the EUnetHTA Joint Action Project Framework (2010-2012). Int J Technol Assess Health Care.
2014 Nov;30(5):504-7.
120
121
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bodies (described in detail in section 1.4.1). Some HTA bodies have therefore expressed
concerns that joint work may not be fully in line with their national methodologies (as
defined in national legal/procedural frameworks and methods papers), impeding national
uptake.
Topic prioritisation for joint work:
The relevance of the topics selected for joint work (in
particular for joint REAs) to national work plans and priorities is another important
element to ensure national uptake. A number of Member States have noted that there have
been insufficient mechanisms for topic prioritisation in the EUnetHTA Joint Actions so
far. Some topics may have been relevant to the authors or other partners involved in
particular Joint Action work packages, but have not met the needs and priorities of all
HTA bodies.
Other issues:
Language barriers have been identified as a hurdle to uptake of joint outputs.
In some HTA systems, use of the national language (e.g. in national HTA reports or early
dialogues) is currently defined by legal/procedural frameworks. Some HTA bodies also
noted that they would need to adapt their current human resources, to have staff with the
right profile (e.g. language skills) for facilitating uptake of joint outputs. Moreover, a
number of HTA bodies expressed the need for more training related to joint outputs (e.g.
on the process and methods underlying the production of joint REAs), in order to facilitate
incorporation in national work.
While the points above refer to specific elements hindering the uptake of joint work, all
of them are related to the first point on the legal uncertainty of the status of joint
outputs.
The current cooperation model via the Joint Action EUnetHTA is trying to address
some of the challenges outlined above on an ad hoc basis to improve uptake. For example,
with support from the European Commission and EMA, arrangement have been put in place
to facilitate contacts between CHMP
125
rapporteurs of the dossier for marketing authorisations
(once finalised) and authors of the Joint REA to allow an early start of the joint REA work
which could facilitate more timely availability of the joint assessment report. Criteria for
selection of authors and co-authors are also being developed by the Joint Action to contribute
to secure quality output; quality management procedures are being developed and stricter
commitment requirements are being explored to engage national HTA bodies in delivering
what was promised. However, from the input received so far in the context of EUnetHTA
and the HTA Network discussions, it is clear that as long as the issue of the legal status of
joint outputs is not resolved, it is very challenging for national HTA bodies to ensure uptake
of joint output in a systematic and continuous manner.
In addition to the above, it should also be noted that low uptake
decreases the readiness of
industry to submit new technologies
for a joint assessment. The preparation of a submission
file costs financial and human resources. Such investments are not worthwhile and will not be
done if the joint assessments have no relevance for national procedures. The current model of
cooperation mainly relies on industry voluntary submission of joint assessments. Without the
certainty that such assessments will be used for national decision making (i.e. uptake) the
willingness of industry to continue submission for joint assessments is expected to decrease.
Also from industry's perspective the key problems with the joint reports are the same as those
referred to above, i.e. inappropriate timing, insufficient assurance of consistent quality and
lack of uptake in national/regional decision-making. In addition, from the medical
125
The Committee for Medicinal Products for Human Use (CHMP) is the European Medicines Agency's (EMA)
committee responsible for human medicines.
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technologies perspective, not sufficiently addressing the specificities of the sector is also seen
as a problem.
Duplication of efforts and low uptake contribute to
increased work and costs for HTA
bodies and to sub-optimal use of their resources,
as they repeat identical/similar
assessments. By contrast, current experience suggests that sharing the work can lower the
costs for HTA bodies significantly (in one case where only two agencies agreed to cooperate
on clinical guidelines they were able to save 30% respectively).
126
However, if joint
assessments are not taken up, they can actually add an additional layer of duplication, as an
HTA body may end up working both on a joint assessment and a national assessment for the
same technology.
To conclude, the current duplication and low uptake imply that investments into the
cooperation both in terms of
resources from the EU budget and the human resources from
the Member States are not used optimally.
The example of EU pharmaceutical legislation
The issue of duplication of national efforts was an important aspect triggering the
development of the pharmaceutical legislation and the set-up of the European Medicines
Agency (EMA) as illustrated in the box below. While it should be noted that the scope and
technical content of HTA differs from marketing authorisation of pharmaceuticals, the set-up
of EMA provides a useful example of how scientific and technical cooperation can be
organised at European level and of the type of benefits it can lead to.
The development of the pharmaceutical legislation and the setup of the European Medicines
Agency (EMA)
Despite considerable efforts at harmonisation, at the beginning of the 1990s the European
pharmaceutical market remained more fragmented, along Member State boundaries, than any other
market for consumer products, as the granting of marketing authorisations were entirely in the hands
of Member States.
In November 1990, the Commission proposed to Council and Parliament a major overhaul of the
European authorisation system for medicinal products, including the introduction of a 'centralised'
EU authorisation procedure for technologically advanced medicinal products and a 'decentralised
procedure', operated by the Member States with EU arbitration if needed, for all other products. To
support the operation of the new system, the Commission proposed setting up a 'European Medicines
Evaluation Agency' (EMEA)
–
later renamed EMA.
EMA was set up in 1995 to harmonise the work of existing national medicine regulatory bodies.
The centralised procedure was a success, as it effectively allowed access to the entire EU market in
little over a year, closely matching the review time of the Food and Drug Administration of the USA.
Previously, it had taken on average six years for a new medicine to be authorised in a significant
number of Member States. In turn, despite a slow start, the decentralised procedure proved to be a
real alternative to the centralised procedure.
EMA has a 20+ year track record of ensuring efficacy and safety of human and veterinary medicines
across Europe, and promoting research and innovation in the development of medicines.
126
GÖG-LSE Study. Annex: HTA Bodies Focus group minutes
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EMA’s success is based on cooperation within the European medicines regulatory network –
a unique
partnership between the European Commission, the medicines regulatory authorities in the European
Economic Area countries, and EMA. Working together has encouraged the exchange of knowledge,
ideas and best practices, in order to ensure the highest standards in medicines regulation. This works
because EU legislation requires that each Member State operates to the same rules and requirements
regarding the authorisation and monitoring of medicines. By working closely together, Member States
reduce duplication, share the workload and ensure the efficient and effective regulation of medicines
across the EU. Today, seven EMA scientific committees and more than 30 working parties provide
scientific expertise for the regulation of medicines by drawing on a pool of several thousand European
scientific experts from the network.
Problem 3. Unsustainability of HTA cooperation
The current EU cooperation on HTA is project-based. This means its funding needs to be
secured and re-negotiated in every financial cycle and there is no guarantee for continuing
activities in the long term. During the initiation and closing of such a large project (the Joint
Action 3 involves 81 participants and benefits from an EU contribution of approximately
EUR 16 000 000) substantial time and resources are spent on organisational issues. For
instance, more than one year after the launch of Joint Action 3, there are still positions to be
filled in key work packages and in the coordinating institution. This has resulted in drops of
joint outputs linked to project cycles (Figure 7) as well as in inefficiencies (e.g. development
of a new IT system for every new project).
127
The evaluation of EUnetHTA Joint Action 2
also highlights that finalising a project and starting a new one at the same time stretched the
resources of the participating organisations and caused delays.
128
127
128
http://www.eunethta.eu/joint-assessments
EUnetHTA JA2. Report on evaluation of project completion including assessment of impact on secondary
users of HTA information. 2016 p. 39.
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Figure 7. EUnetHTA Joint Action outputs
129
The need for sustainability was highlighted by many of the contributors to the public
consultation. Among the limitations of the current model of cooperation most cited by public
administrations were: the lack of flexibility of the framework for EU-funded projects which
require high efforts for the preparation of a proposal, difficulties to put in place a sustainable
IT platform (including IT tools) for the use of all participants and access of joint work, delays
in performing joint work which affected the availability of joint reports, insufficient
commitment from all partners to use the output, uncertainties about the quality of joint work,
insufficient coordination and agreement on topic selection, lack of knowledge on the impact
on decision-making and the limited participation of some categories of stakeholders such as
health professionals and patients.
Organisations representing stakeholders other than HTA bodies (e.g. academia, patients and
consumers representatives) expressed concerns related to the limited duration in time and the
lack of a sustainable funding mechanism of the current EU cooperation on HTA.
The following case study illustrates the problems described this section.
A case study for metastatic cancer treatment
The pharmaceutical received marketing authorisation in mid-2011. HTA was performed on the same
product by 12 HTA bodies in 11 countries, resulting in considerable duplication of work for both HTA
bodies and industry. The timeframe of the reports is between 2011 and 2016, indicating the differences
of the time of market access.
129
Own calculation http://www.eunethta.eu/joint-assessments and SEED project final report 2017.
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There are commonalities but also important methodological differences in the clinical assessment
(REA) regarding the comparator and the evidence used. In all HTA reports, the primary evidence
stemmed from the same randomised, double-blind clinical trial. Three agencies used indirect
comparison to assess the pharmaceutical against another comparator; four others considered another
phase III study, an observational study or referred directly to the marketing authorisation report; one
agency also considered another comparator. A number of social value judgements (e.g. end of life
criteria or advantages related to the method of administration) were identified in the report; in some
countries they were considered in a systemic, in others in a less standardised manner. Half of the
agencies considered the treatment important because of unmet medical needs. Three pointed out its
innovativeness. Nonetheless, the fact that the treatment improves the quality of life of patients was an
important consideration in all HTA reports. Patients were involved in varying degrees in the
processes.
Following the HTA process, most of the countries reimbursed the pharmaceutical with criteria, in the
rest with no criteria. It should be noted that in one country the product was rejected due to its cost-
effectiveness; the decision was eventually reversed due to a reduced price. During this period the
product was available on a case-by case basis through a special fund, which gives a good example of
the complexity of the relationship between HTA and access. In the majority of the countries
confidential pricing or a risk sharing pricing agreement is in place, which often requires the collection
of real world evidence.
3. Why should the EU act?
Article 114 of the Treaty on the Functioning of the European Union (TFEU) allows for the
adoption of measures for the approximation of the provisions laid down by law, regulation or
administrative action in the Member States, provided they are necessary for the establishment
or functioning of the internal market, whilst at the same time ensuring a high level of public
health protection. Article 168(4) provides for the adoption of measures setting high standards
of quality and safety for medicinal products and devices for medical use. Article 168(1) TFEU
states that a high level of human health protection shall be ensured in the definition and
implementation of all Union policies and activities.
Most health technologies are products which benefit from the free movement of goods within
the internal market. Despite this, a number of obstacles to their free movement have been
outlined in section 2 of this report. The procedural and methodological differences, along with
the considerable duplication of HTA across the EU Member States, have a significant
negative impact on when and where health technologies reach the market, thus reducing
business predictability for companies, particularly SMEs. This, in turn, contributes to
differences in patient access to innovative health technologies. These divergences and
duplication also result in considerable additional costs for HTA bodies and industry alike.
The aims of this initiative cannot be achieved sufficiently without strengthened cooperation at
EU level. As described in section 2, the diversity and multitude of approaches to HTA across
the Member States means that, due to their scale and effect, only action at Union level can
eliminate the obstacles described. Without action at EU level it is unlikely that national rules
on how HTAs are carried out would be harmonised and thus the current fragmentation of the
single market would persist.
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While the on-going cooperation, namely the Joint Actions and the HTA Network, has
illustrated benefits of EU cooperation, in terms of establishing the professional network, the
tools and methodologies for cooperation and piloting joint reports, the current cooperation
model has not contributed to the removal of the fragmentation of the national systems and the
duplication of efforts. Without an EU initiative, it is unlikely long-term cooperation on HTA
between Member States would be significantly strengthened through bilateral or regional
cross-border initiatives. There are clear additional costs for HTA bodies and industry from
carrying out HTA on the same health technology in multiple Member States. By carrying out
a HTA only once at EU-level, economies of scale, greater business predictability, increased
quality and consistency of HTA and improved transparency for patients would be achieved in
the long run.
This impact assessment report has identified a division between the HTA domains (the
clinical domains) which lend themselves to a common assessment at EU-level and those (the
non-clinical domains) which have more country-specific elements. By making this distinction,
this initiative will maximise the EU added value while at the same time ensuring an approach
to HTA assessment that is proportionate and in keeping with the principle of subsidiarity by
leaving Member States to continue carrying out the parts of HTA better achieved at national
level.
The report has also underlined the differences between the pharmaceutical and the medical
technologies sectors, not only in relation to the different market access path, but also in
relation to the role HTA plays in the two sectors and the lower level of duplication/parallel
processes compared with pharmaceuticals. In order to ensure a proportionate approach is
taken, such differences are reflected in the design and comparison of the identified policy
options along with the different categories of products within sectors (centrally authorised v.
national authorisation etc) which are reflected in the product scopes considered in the various
policy options. The proportionality of the initiative is further considered in the design and
comparison of policy options, and the measures envisaged do not go beyond what is necessary
to remove obstacles to the free movement of goods within the internal market.
Creating a system of HTA at EU-level would necessitate some financial and administrative
costs for the Union and for EU Member States. Such costs also need to be considered in light
of the current EU-level HTA cooperation and its lack of sustainability as outlined in section 2.
The principle of subsidiarity is furthered ensured in the initiative by fully respecting Article
168(7) TFEU which stipulates that the Union shall respect the responsibilities of Member
States for the definition of their health policies and for the organisation and delivery of health
services and medical care. In particular, Member States are responsible for decisions on
pricing and reimbursement, which are not within the scope of this initiative.
4. Policy objectives
The general, specific and operational objectives of the initiative are listed below. Figure 8
provides an overview linking the objectives to the problems discussed in detail in section 2.
General objectives
The general objectives of the initiative are:
Ensure a better functioning of the internal market
Contribute to a high level of human health protection
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Specific objectives
The specific objectives of the initiative are:
Improve the availability of innovative health technologies for EU patients
Ensure efficient use of resources and strengthen the quality of HTA across the EU
Improve business predictability
Operational objectives
The operational objectives of the initiative are:
Promote convergence in HTA tools, procedures and methodologies
Reduce duplication of efforts for HTA bodies and industry
Ensure the uptake of joint outputs in Member States
Ensure the long-term sustainability of EU HTA cooperation
Figure 8. Intervention logic
5. Policy options
Based on identified shortcomings, experience with the current cooperation and comments
from stakeholders, the following
key principles
were identified for constructing the policy
options:
The need to build on existing structures, activities and achievements and maintain a
Member States driven approach;
The need to address the specificities of the different sectors: pharmaceuticals, medical
and other technologies;
Ensure a high level of quality, transparency and independence (scientific and
financial);
Ensure the engagement of stakeholders, in particular patients, health care
professionals and payers;
Support the development of HTA capacities at national level.
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A number of policy options (PO 1-5) were identified already in the Inception Impact
Assessment
130
. Of these, policy option 5 was discarded upfront (as discussed in section 5.1
below), while policy options 1-4 were further developed and are discussed in more detail in
subsequent sections.
5.1. Discarded policy option
The inception impact assessment put forward a legislative option which includes joint full
HTA reports ("option 5"), i.e. joint production of HTA reports which cover clinical and non-
clinical (e.g. economic, organisational, ethical) HTA domains. This option was also included
in the analysis of options conducted in the GÖG-LSE Study and in the public consultation.
However, it has become clear from the input received in the public consultation and other fora
that such an option is not realistic. While there is broad agreement that voluntary cooperation
on methodologies to develop full HTA reports would be useful to increase consistency and
predictability of assessments, the development of EU legislation mandating joint full HTA
reports at EU level would bring more challenges than benefits. This is mainly due to the fact
that full HTA reports rely to a large extent on context-specific information (e.g. economic,
organisational, ethical) in order to serve national decision-making. These issues have been
highlighted by public authorities, experts as well as industry representatives. This implies that
a joint full HTA report could not in practice at this point in time support improved governance
or sustainability.
Option 5 as analysed in the GÖG-LSE study therefore raises concerns as regards its
proportionality, Member States' responsibilities under Article 168(7) TFEU and its feasibility.
This option is therefore discarded upfront and is not discussed further in subsequent sections
of this Impact Assessment.
5.2. Key characteristics of the policy options
The different policy options for EU cooperation on HTA after 2020 are defined along several
key characteristics focusing on:
(1)
Joint outputs,
(areas in which EU cooperation seems possible/useful) which could be
included in the initiative:
Technology Specific Reports
Early dialogues with health technology developers;
131
Joint Relative Effectiveness Assessments (REA) .
Common tools and procedures
Methodologies to formulate the contents and design of assessments (e.g. EUnetHTA
Core Model and Standard Operating Procedure/SOP);
Horizon Scanning;
Inception Impact Assessment on Strengthening of the EU cooperation on Health Technology Assessment
(HTA),
see
http://ec.europa.eu/smart-regulation/roadmaps/docs/2016_sante_144_health_technology_
assessments_en.pdf
131
REA can take place at the time of market launch, or later.
130
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Procedural framework (clarifying inter alia the rights, obligations and involvement of
stakeholders, such as patients and health care professionals; transparency;
independence etc.);
Submission templates and templates for other key documents, including assessment
reports and summaries;
Database for horizon scanning, planned, ongoing and finalised early dialogues;
planned, ongoing assessments and finalised assessments;
IT tools for exchanging (confidential) information, for supporting the collection of
Real World Data and for training and other capacity building activities.
The common tools and procedures should build on the work already undertaken in Union-
funded actions on HTA, including EUnetHTA, Horizon 2020-funded actions. Preliminary
results of regional initiatives such as the BeNeLuxA, and Valletta Declaration initiatives
could also be taken into account. The distinctive characteristics of the different sectors
(i.e. pharmaceuticals, medical devices and others) should be taken into account in the
development of common tools and procedures.
(2)
Technologies
which could be covered by the initiative:
Pharmaceuticals;
Medical technologies (medical devices and in vitro diagnostics)
132
;
Other technologies (e.g. Screening or vaccination programmes, surgical procedures).
(3)
The choice of instrument
used to establish and maintain the cooperation, including:
Voluntary cooperation of national bodies outside any EU agreement or framework (i.e.
an intergovernmental approach relying exclusively on the resources and political
commitments of Member States, as is the case in the BeNeLuxA or other similar
initiatives);
Contractual obligations between (some/all) Member States with possible co-financing
by EU programme/funding but no dedicated legal framework (i.e. project based
approach);
Common legal framework (Regulation or Directive)
Also combinations of these instruments could be envisaged. For example, some outputs could
be governed by a legal framework, while others could be produced on a voluntary basis.
(4)
The choice of governance structure
for the cooperation, including:
Project-based secretariat: a coordinating secretariat is set up and run for the
duration of a project by a Member State HTA body in agreement with the other
participants of the consortium;
Member State secretariat: a secretariat is established and hosted in a national/
regional HTA body;
132
Medical devices and in vitro diagnostics as defined by Regulation (EU) 2017/745 of the European Parliament
and of the Council of 5 April 2017 on medical devices and Regulation (EU) 2017/746 of the European
Parliament and of the Council of 5 April 2017 on in vitro diagnostic medical devices
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Central secretariat: a secretariat is established and hosted in the European
Commission, an existing EU Agency or a new EU agency
133
.
(5)
The financing
(i.e. possible sources of funding):
EU funding, either through the Public Health Programme or another EU financial
instrument
134
;
Funding by Member States participating in the cooperation, in cash and/or in kind;
Funding of joint assessments through industry fees have also been considered, but
has been discarded in the first phase of this initiative. This consideration is based
on a proportionality assessment weighing the relatively limited size of the structure
foreseen for cooperation against the burden of setting up a fee structure. It was also
considered appropriate to evaluate the cooperation after a certain period of time
based on experience gathered and assess, at that point in time, whether the
introduction of a fee structure for joint assessments would be appropriate.
From these elements, four policy options have been constructed, based on input from Member
States, stakeholders and experts through the various forms of consultation activities (for more
details on consultation activities, see Annex II). The study supporting the Impact Assessment
process has also further discussed and validated the proposed Policy Option (See GÖG-LSE
study, Chapter 4).
The policy options were constructed according to their feasibility (e.g. mandatory uptake
considered only for legislative options) as well as logical and coherent combinations of
elements (see section 5.3. below). The governance structure is assessed separately in section
6.5.
5.3. Description of the policy options
5.3.1. Policy option 1 (Baseline scenario). No Joint Actions after 2020
Rationale
The baseline scenario supposes that after the current EUnetHTA Joint Action 3 will end in
2020 there would be no further Joint Action on this topic. The EU funding to support
scientific and technical cooperation among Member States would be discontinued.
135
The choice of the baseline scenario is justified by the fact that although the Joint Actions have
been successful in demonstrating a proof of concept, a continuation in the form of a fourth
133
Two further possibilities were considered and disregarded: 1) a rotating MS Secretariat and 2) a fully
centralised model where both the support function and scientific expertise are integrated. Both models were
considered not feasible due to the limited support received in the public consultation and the limitations they
would pose in terms of implementation (e.g. losing know-how for the rotational model, and the challenge in
building and maintaining the necessary know-how in a fully centralised model) and acceptance (e.g. the link to
national HTA processes is lost in case of a fully centralised model).
134
For all policy options the source and the amount of funding are dependent on the negotiations of the
Multiannual Financial Framework (within the European Commission and the other relevant EU institutions) and
subject to renewal every budgeting period.
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Joint Action is deemed to be both ineffective and unrealistic (see section 1.4.2 and section 2,
problem 2). This was considered the most likely and most-evidence based baseline also in
light of the indications from the Court of Auditors, which considers that this type of
actions/projects is not supposed to be renewed too many times.
The Joint Actions have created the necessary trust to enable HTA bodies to work together,
have developed common methodologies and tools and have demonstrated by pilots that joint
outputs, including joint assessments can be done. This demonstration of "proof of concept" is
in line with the purpose of Joint Actions (see box below for further information).
The role of Joint Actions as an EU funding instrument
Joint Actions are collaborative projects specific to the Health Programme aiming to develop / share /
refine / test tools, methods and approaches to specific issues or activities, and engage in capacity
building in key areas of interest for the Member States and countries participating to the Programme.
They are co-financed by the European Commission and authorities of the Member States. This type of
project was introduced during the 2nd Health Programme (2008-2013) and continued under the
current one (2014-2020) to cover specific health-policy needs and aimed at supporting EU
cooperation in the field of health with as many partners as possible from all countries participating in
the Programme
136
.
Joint actions are often started, after several years of cooperation between relevant stakeholders and
participants are designated by Member States authorities, in a bid to secure political endorsement and
optimise policy coordination. Joint Actions are grants, assigned through a non-competitive negotiated
procedure (i.e. for each possible topic only one proposal for a Joint Action will be submitted), and
they are an exception to the Financial Regulations. The Commission, in consultation with the relevant
Member State Programme Committee defines the policy area and provides a general outline of the
aim, the final objective and the budget of the Action. All Member States are invited to appoint the
organisations they consider relevant for the Action and propose a detailed work plan, which the
Commission is expected to accept. As emphasised also in the Commission's Ex-post Evaluation
Report of the 2nd Health Programme 2008-2013
137
, "joint actions build on previous achievements
made possible through project grants started sometimes 10 or more years ago". EUnetHTA was
singled out as an example where a third joint action even though possible, was not seen as an
undisputable fact.
However, while constituting a good tool for testing and validating new ways of cooperation,
the EUnetHTA Joint Actions have showed important shortcomings in terms of disruptions
(regular renegotiations/reallocations of work packages, renewal of staff etc.), and difficulties
136
137
http://ec.europa.eu/chafea/documents/health/ja-2017-guide-applicants_en.pdf
https://ec.europa.eu/health/sites/health/files/programme/docs/ex-post_2nd-hp-2008-13_comm-swd.pdf
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in ensuring consistent delivery of high quality, timely output and uptake (see sections 1.4.2
and 2, problem 2).
In addition, as described above the objective of a financing through Joint Actions is not to
support permanent/long-term/recurrent actions. This was confirmed by the Court of Auditors
in its Report on cross-border threats to health, stating that
“…given their significant size, they
(NB. Joint Actions) take more time to prepare and also require political backing and national
co-funding. This means that, despite their potential for increasing the EU-wide take up of
outputs produced with health programme funding, there cannot be too many subsequent joint
actions in one policy area
."
138
In conclusion, Joint Actions are only intended to kick-start policy coordination between
Member States and are not intended to run indefinitely and have proven to be not effective in
feeding in to national decision making processes (see section 1.4.2)
139
.
In view of the above, a fourth Joint Action is not considered a realistic and credible option to
address the problems and achieve the objectives of the initiative.
Description
Under the baseline scenario (policy option 1), the European cooperation would be limited to
the high-level strategic policy discussions within the HTA Network, which mainly consists of
meetings between Ministries of Health and/or national HTA agencies to discuss policy
developments which are relevant to HTA both a national and/or European level. However,
without a continuous and sustainable support to coordinate and develop technical/scientific
activities, including the developments of joint outputs, it is not expected that Member States
will devote resources to continue the cooperation at EU level in a broader and more organised
way. This will be particularly true for Member States with limited resources and less
developed HTA systems, which will need to focus their resources on performing assessments
or identify reports from other agencies and/or possible regional networks that can be adapted
and used at national level.
No dedicated
governance
structure is foreseen under this policy option. Member States are
relying on national
resources
as illustrated above (section 1.4.1). The trend of more Member
States developing their own specific HTA systems (mostly in Central and Eastern Europe) is
also expected to continue, with different procedural frameworks and methodologies, and
variability in practices, procedures and methodologies.
140
The differences as regards good
governance principles, e.g. transparency of the HTA processes, stakeholders' participation/
involvement and quality control mechanisms would also continue to exist. HTA bodies would
continue the trend of duplicating assessments of the same technology carried out in other
Member States, in particular for pharmaceuticals. The capacity of HTA bodies to cover all
relevant innovative technologies would remain limited: higher for the already well-established
European Court of Auditors, Special Report Dealing with serious cross-border threats to health in the EU:
important steps taken but more needs to be done, 2016
https://www.eca.europa.eu/Lists/ECADocuments/SR16_28/SR_HEALTH_EN.pdf
138
EFPIA/ Charles River Associates. 2017. Assessing the wider benefits of the EU’s proposal on
strengthening
cooperation on health technology assessment from the industry perspective.
140
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HTA systems, while the others would have to continue to limit themselves to assessing only a
lower number of technologies. Member States with limited resources would therefore not be
able to fully benefit from HTA in their efforts to address challenges related to rising health
care expenditures, evolving health technologies, ageing populations and increasing burden
from chronic diseases
141,142
. Pooling of resources and re-use of jointly developed HTA reports
are not expected to a significant extent, as there would be no EU-wide mechanism and no
specific resources dedicated to EU-wide activities by Member States. Adaptation of reports
from other HTA bodies may continue, especially in Member States with limited HTA
capacity, as this would be the main source to build capacity and know-how.
Without EU stable cooperation, joint early dialogues would be limited to HTA bodies
participating to the parallel scientific advice procedure offered to developers by EMA. The
achievements of the current Joint Action, such as the single platform put in place by
EUnetHTA and EMA which ensures not only a coordinated advice from regulators, but also a
coordinated opinion from several HTA bodies, would likely be jeopardised if the process is
not sustained overtime.
Regional cooperation
is expected to continue on a voluntary basis, in particular in relation to
the production of some joint assessments to be used in possible joint price negotiations and
procurements efforts. On the other hand, as different regional cooperation networks are
developing across the EU, duplications between the regional networks are likely to occur, and
divergences as regards processes and methodologies can be expected between these networks
in addition to the continued national divergences. Moreover, as recent discussions around a
potential joint horizon scanning activity in the context of the BeNeLuxA initiative have
shown
143
, there are significant challenges in identifying a suitable organisational, legal and
financial framework to perform joint activities in a regional cooperation setting.
The HTA Network established under Directive 2011/24/EU is expected to continue to meet
twice per year to share some high level national experiences.
5.3.2. Policy option 2. Project-based cooperation on HTA activities
This option foresees voluntary cooperation supported by EU funding organised in the form of
project(s)
144
other than Joint Actions. The project(s) would be funded under the Health
Programme or any other EU financing instruments (e.g. Horizon 2020).
The
instrument
to implement this option would be project(s) through competitive calls for
proposals in line with the priorities/EU added value criteria identified by the Commission
following lessons learnt from EUnetHTA and other projects/initiatives.
The calls would support the development of a defined number of joint outputs (e.g. joint
assessments and/or early dialogues) in a given timeline. The selected project(s) is expected to
last 36-48 months, during which it would need to deliver the planned output. To address the
shortcoming of EUnetHTA, the eligibility criteria would be more specific and prescriptive
than the ones which can be used for Joint Actions. For example, the call would specify the
OECD. 2015. Pharmaceutical expenditure and policies: past trends and future challenges.
European Commission, DG ECFIN - EPC. (2016) Joint Report on Health Care and Long-Term Care Systems
& Fiscal Sustainability. Brussels: Publication Office of the European Union, p. 1-13.
143
For more information on BeNeLuxA initiative, see http://www.beneluxa.org/.
144
This could be done as one project, subsequent projects or multiple parallel projects. The assessment of PO2 in
this report is based on the assumption of one project in line with the GÖG-LSE study.
142
141
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minimum number of participants from different Member States to ensure a sufficient EU
coverage, the maximum number from each Member States (e.g. two agencies per Member
States) to ensure an efficient and manageable consortium. Their profile and role in the
national decision-making process will also need to be well defined to ensure that the most
relevant HTA Agencies are included and the necessary expertise participates. Conditionality
clauses would also be included to encourage uptake of results (i.e. final payment would be
subject to the level of uptake of the joint output).
To facilitate commitment from technology developers, their European trade associations
could also be included in the project. Involvement of patients, clinical societies and/or
healthcare professional organisations would also be foreseen to increase participation of these
stakeholders in the development of joint outputs.
Such a model differs from a Joint Action because it would be based on competitive calls
which may result in more than one group (i.e. Consortia) of Member States competing with
each other. The number of beneficiaries in the selected project will be lower and with a
homogenous profile and role in the national decision-making process. Engaging in a
competitive process with relatively high level of co-financing from beneficiaries (normally
50-40%) is expected to ensure a higher commitment to the objectives and outputs of the
project than what is the case in a Joint Action, including the uptake of results.
The evaluation process and the subsequent negotiation with the winning Consortium is
expected to result in a focused and defined work plan, which will be limited to the output for
which cooperation has demonstrated a clear EU added value (e.g. focus on clinical aspects of
the HTA process for joint REA). This is different from negotiations within Joint Actions
typically focusing on solving administrative issues due to the complexity of the different legal
status of the appointed beneficiaries and the Commission's limited possibilities to modify the
proposed work plan.
A similar project-based model was tested for Early Dialogues through the project SEED
(2012-2015). It could address some of the shortcomings identified in the Joint Action
EUnetHTA (delays, high number and heterogeneous profile and number of participants,
inconsistency of the quality and timely outputs).
The scope of the cooperation could cover
all categories of health technologies:
pharmaceuticals, medical technologies and other health technologies (see 5.1.2). For joint
technology-specific reports (joint assessments and early dialogues) the activity would be
limited to clinical domains. For other types of activities (cooperation on methodologies and
procedures), the projects could cover also non-clinical domains (e.g. economic,
organisational, ethical assessment). This approach would contribute to ensure best use of
resources and focus on outputs which have demonstrated major EU added value.
No EU legal framework is foreseen under this policy option and the
governance
model would
be a project secretariat managed by one of the beneficiaries of the winning
consortium/consortia. It is expected that similarly to a Joint Action, a national HTA body
would take up the coordination role and distribute and monitor tasks and responsibilities
between partners to ensure the delivery of the agreed joint outputs. IT tools will be managed
by the project throughout its duration. IT tools developed by EUnetHTA could be possibly
reused and built upon, provided the winning consortium would have the right to exploit them.
The cooperation foreseen under this option could also continue to benefit from the HTA
Network, which would be a platform to share practices of the different activities and would
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become an important mechanism to ensure inclusiveness of the cooperation to Member States
which may not be directly involved in the cooperation in the winning project(s).
This option foresees a top-down approach with the Commission in the lead, identifying
priorities, launching the call for proposal, monitoring the projects and disseminating the
results.
The
financing
of this option would rely on the EU budget and Member States' co-financing
(normally by providing in kind contributions).
5.3.3. Policy option 3. Permanent cooperation on common tools,
procedures and early dialogues
Table 3. Overview of policy option 3.
In option 3, the
joint outputs
would cover early dialogues (the only technology specific
reports under this option) and various common tools and procedures (listed in Table 3). As
described in section 5.2, the common tools would be based on work already carried out by
existing cooperation mechanisms and the specific characteristics of different health
technologies (e.g. pharmaceuticals and medical technologies) would be taken into account
when developing the common tools and procedures. The option foresees upwards
harmonisation of a basic set of tools and procedures (see Table 3 and further details in section
5.2), to ensure a high level of quality throughout the EU. Common procedures will be aimed,
in particular, at ensuring the involvement of patients and external expert (e.g. healthcare
professionals) in the HTA process, avoiding conflicts of interest and ensuring transparency
(e.g. via publication of joint outputs).
Option 3 would in principle cover
all types of health technologies,
subject to selection and
prioritisation criteria in accordance with the needs of Member States.
145
It should also be
In principle Member States will select and prioritise health technologies which shall undergo early dialogue
based on given criteria.
145
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noted that, as early dialogues would be initiated by the industry, it cannot be guaranteed that
all technologies will benefit from them.
The
instrument
used to implement this option would be a (new) EU legislative framework
which would ensure the mandatory uptake by HTA bodies of the common tools and
procedures and of joint early dialogues. These tools, procedures and early dialogues would
relate to the clinical aspects of HTA, thereby supporting the assessment at Member State level
of the clinical domains of HTA (REA). Mandatory uptake of the common tools (e.g.
methodologies, templates, IT tools) and common procedures (e.g. for stakeholder
involvement) implies that Member States shall use these basic tools/procedures when
conducting joint early dialogues and clinical HTA work at national level to ensure high
quality and to facilitate cooperation and use of each other's assessments. The development of
these common tools and procedures is expected to rely mainly on results of the Joint Actions
EUnetHTA and to be further developed by experts nominated by national HTA bodies.
Mandatory uptake of early dialogues means that Member States shall use the joint early
dialogues in the same way that they would use a national early dialogue, i.e. they should not
repeat at national level an early dialogue which has already been conducted jointly.
The
governance
of the cooperation under this option would be ensured by a central structure
which could provide administrative, scientific and IT support to deliver joint outputs of a
consistent high quality, in a transparent, independent and timely manner and with appropriate
involvement/consultation of stakeholders. To deliver the joint output, the cooperation shall
rely on HTA experts from Member States HTA bodies organised in dedicated
committees/groups covering the outputs.
The
financing
of this option is expected to rely mainly on the EU budget and in kind
contributions from Member States, which would be asked to provide expertise through their
experts. For early dialogues, depending on the governance structure chosen, a fee from
industry would cover the costs of the experts and the overheads needed to support the
production of this specific joint output.
146
Article 15 of Directive 2011/24/EU would be deleted under this option as it would not be
compatible with the legislative approach suggested above: the HTA Network foresees fully
voluntary cooperation, the output of the cooperation have no legal status. While Art 15 would
be deleted from Directive 2011/24/EC, the foreseen new Legal framework would maintain
and further develop the objectives defined by the article, and add provisions to ensure their
achievements, which is currently limited (see section 2). It will also re-introduce key elements
already foreseen by the article such as the involvement of stakeholders in the cooperation and
it will use similar working methods already applied such as the setting up of dedicated
Member States experts' groups/subgroups to develop the specific outputs, it will further
develop its good governance principles in a dedicate governance structure. In addition the
new Legal framework would provide a more stable framework for granting aid to support the
cooperation.
146
Industry fees would only be possible if the tasks are carried out by an EU Agency.
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5.3.4. Policy option 4. Permanent cooperation on common tools,
procedures, early dialogues and joint REA
Table 4. Overview of policy option 4
Option 4 comprises the
joint outputs
included in option 3 (common tools/procedures and
joint early dialogues, as described more detail in section 5.3.3) and in addition joint REAs
(i.e. assessments of the clinical HTA domains).
For
pharmaceuticals,
the joint REAs would comprise centrally authorised pharmaceuticals
and other pharmaceuticals prioritised by Member States due to their high value, high budget
impact, or their impact on public health / addressing unmet medical needs.
147
For
medical technologies,
the scope for joint REAs comprises those that are prioritised by
Member States based on their potential high risk (i.e. devices undergoing the EU scrutiny
mechanism) or potential impact on public health and health systems (e.g. addressing unmet
medical need, potential to transform the organisation of care, high budget impact).
148
The option also foresees a phase-in approach for joint REAs, i.e. a gradual introduction of the
full product scope while the system is built up.
The
instrument
used to implement this option would be a (new) EU legislative framework
which would not only ensure the mandatory uptake of the common tools and procedures, and
joint early dialogues as in option 3, but also of joint REAs. Mandatory uptake of joint REAs
implies that Member States would use the joint assessment reports in the same way as a
national assessment report is used today and that the REA should not be repeated at national
level. Member States would however continue to be free to assess other (non-clinical HTA)
147
148
See GÖG-LSE study (Table 7, section 4.3 and annexes 3 and 4).
See GÖG-LSE study (Table 7, section 4.3 and annexes 3 and 4).
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domains at national or regional level and would continue to draw the overall conclusions on
the basis of the (joint) clinical and (national) non-clinical assessment parts
149
.
The
governance structure
would be similar to option 3 but taking into consideration the
extended scope in terms of joint outputs (joint REA). In this respect, Member States experts
will be supported by a central secretariat providing administrative support (e.g. organisation
of meetings, travel arrangements etc.), scientific/technical and IT support. A management
board including representatives of Member States' HTA bodies would manage the overall
governance and would meet regularly to discuss topic prioritisation, progress with outputs
(e.g. quality, timeliness), provide guidance and steer the cooperation. The scientific-technical
work of producing the joint outputs would be carried out by experts nominated by Member
States' authorities.
150
For joint REAs, Member States' experts acting as author/rapporteur and
co-author/co-rapporteur would carry out the clinical assessment of the application/dossier
submitted by industry (complying with common tools and procedures as described in section
5.2) and prepare a joint assessment report. A committee/group including experts nominated
by Member States would thereafter examine the draft and approve the joint report which
would then be incorporated in national HTA processes (see more detailed explanations on
mandatory uptake above).
The
financing
solution is the same as the one foreseen in option 3.
Article 15 of Directive 2011/24/EU would be deleted under this option as it would not be
compatible with the legislative approach suggested. As under option 3, some key components
would be maintained and further developed in the new legal framework.
Option 4 could be divided in two sub options:
Option 4.1- an 'opt-in' system: For joint REA, such a system would allow Member
States, without prejudice to the need to achieve the stated specific objectives and
ensure legal feasibility, some flexibility to decide if / when to start participating in the
EU-level system of joint REA depending on their individual situation in terms of
needs of adjusting national law and practice etc. This decision to participate would be
system-based (i.e. Member States would decide whether or not to participate in the
system for all joint REA conducted at EU level) and not on a product-specific basis
(i.e. Member States would not decide for each product submitted for joint REA
whether to participate or not). Member States not participating in the joint REA
system would still be obliged to use the common tools and procedures (option 3) when
carrying out their own REA.
Option 4.2: is essentially the same as option 4.1 with the difference that this option
would be applicable to all Member States with no possibilities to opt in later or stay
out.
As discussed in sections 1.2, 1.4.1 and the Mapping study on HTA processes across the EU, all current
national HTA processes include an assessment of clinical evidence (REA), but in addition may also assess
various non-clinical domains (e.g. economic, organisation, ethical) on the same health technology.
150
This would be similar to the model implemented by EMA for the central marketing authorisation procedure
for medicinal products.
149
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Table 5. Overview of policy options
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6. Impacts of the policy options
This chapter will identify and describe the expected impacts of the policy options 2, 3 and 4,
compared to the baseline scenario (option 1) described in section 5.3.1. The following impacts
have been identified as most relevant for the key stakeholders.
Member States/Public
Administrations
Economic Impacts
- Costs
- Efficient allocation of
resources
- Administrative burden
- EU budget
Patients/consumers
Industry (pharmaceutical and
medical technologies)
- Functioning of the internal
market
- Costs
- Business predictability
- Innovation, research and
competitiveness
- Administrative burden
Social/health
impacts
- Governance, participation
and good administration
- Sustainability of health
systems
- Public health
- Participation/
involvement
- Availability of
innovative health
technologies
Table 6. Summary of key impacts for stakeholders
The key challenge in assessing and quantifying the impacts has been the fact that HTA is an
(often advisory) input for decision making; the access to health technologies and their prices
are set by the national pricing and reimbursement decisions.
151
Therefore, many impacts of
the HTA cooperation, in particular on sustainability and public health are
indirect.
Quantitative assessments have been completed with qualitative assessments when necessary.
None of the options are likely to have considerable impact on the
overall
demand for health
technologies. Therefore, no substantial changes in the production and distribution in the
pharmaceutical and medical technologies sector are expected. As regards HTA-related
employment, no major effects in HTA staffing in Member States HTA bodies are expected.
Some efficiency gains for REA could be envisaged, but resources are likely to be shifted to
increasing demands in assessing additional technologies, re-assessments etc. No impact on the
overall employment in the sector is therefore expected
136
. No impacts have been identified on
trade
152
, on environment and on fundamental rights.
The common horizontal aspects of the governance system and financing are analysed in a
dedicated section (6.5).
151
It should also be noted that there is a difference between the actual prices of pharmaceuticals paid by social
insurances and the publicly available, official list prices. Actual prices tend to be lower due to arrangements
between industry and payers. However, such discounts and rebates are typically strictly confidential and
therefore the actual prices are not known. WHO. Background Paper 8.3 Pricing and Reimbursement Policies:
Impacts on Innovation. 2013.
152
GÖG-LSE Study
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The estimations of costs included in this Impact Assessment report were provided by the
G
Ö
G-LSE Study and took into account the following type of costs:
- costs for the implementation mechanism/governance (e.g. project-based, permanent
structure; include costs of human resources, IT tools, travel, premises etc.). The personnel
costs and costs resulting from Member States’ expert committees were estimated based on the
average salaries of EUnetHTA JA 3 partners (for project-based cooperation), and the Staff
Regulation of Officials of the European Communities (for a permanent structure) and
Commission expert fees;
- costs of the output production are based on the assumption that annual joint output will
increase gradually from option 2 to 4, from 13 joint early dialogues (EDs) and 12 REAs in
option 2 to 40 joint EDs and 65 joint REAs in option 4. The number of joint EDs was
estimated taking into account the number of requests for scientific advice received by EMA
per year, the number of requests received by EUnetHTA JA2 and 3 and the SEED project, as
well as the average value of early dialogues performed at national level by the Member States
offering this service to technology developers. The number of joint REAs was approximated
based on the average number of centrally authorised medicinal products per year (approx. 40
new molecules) and the average number of assessments for medical technologies carried out
at national level (taking into account that in some Member States assessments of certain
medical technologies are also mandatory).
Policy option 2. Project-based cooperation on HTA activities
6.1.1. Economic impacts
Member States/Public administrations
Compared to the baseline scenario (described in section 5.3.1), a large part of the duplication
of work is expected to persist under policy option 2. The problem of duplication of work for
national HTA bodies (discussed in detail under section 2, problem 2) would largely remain,
entailing continued significant costs for HTA bodies e.g. related to the production of REAs at
national level (see section 2, problem 2, text box).
In particular, the legal uncertainty around the status/relevance of the joint outputs stemming
from the project foreseen under this option is expected to remain. It cannot be expected that
Members States will adapt their national HTA legal frameworks to ensure
consistency/compatibility with uptake of joint work (e.g. changes to language requirements,
formats, procedures) produced in the context of time-limited, voluntary EU-funded projects.
Even if contractual arrangements are foreseen under this option aimed to promote/require
national uptake, it can be expected that HTA frameworks defined in national law or
administrative provisions (e.g. the social code) would prevail over any contractual
arrangements in the context of a project. As the GÖG-LSE study points out, while
theoretically such contractual arrangements would be possible if parties agree, there is no
effective possibility for the European Commission to enforce these obligations and as such
address the issue
153
.
153
GÖG-LSE Study Section 4.3
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In addition, a contractual obligation to promote/require national uptake may discourage
participation in project(s), especially in those Member States where HTA process and
procedures are well developed and defined in national law. These are normally the Member
States which are less in need of EU cooperation to satisfy their national HTA obligations, and
as such they may find the contractual constraints of the project not proportionate for the
benefit they may gain.
Since there is no guarantee of uptake of joint outputs in national HTA systems, buy-in,
commitment and resource investment by Member States into an EU project for production of
joint outputs are also expected to remain limited. If uptake of joint outputs is not ensured, the
joint work cannot be fully effective in terms of reducing duplication of efforts for HTA bodies
and creating efficiency gains through joint work.
The duplication of work under option 2 will consist both in national HTA activities
continuing in parallel and in addition to joint work stemming from the project. Such
duplication is expected to result in additional
costs
and further negative impacts on the
efficient use of resources.
In addition, the same considerations made for policy option 1 in relation to the potential
impact of regional cooperations would apply also under this policy option. But contrary to the
option 1, in option 2 regional cooperations may have a negative impact on ensuring efficient
use of resources as they may add further to the duplication described above as Member States
could be active in both regional and/or European cooperation efforts, as it is currently the case
with EUnetHTA.
Nonetheless, compared with option 1, due to some requirements to improve the quality and
consistency of the joint work that the option would put in place (e.g. strict conditions in the
Terms of Reference of the project on requirements for number of participants, their role in
national decision making and profile/expertise, see section 5.3.2), this option is expected to
improve the situation compared to the baseline and facilitate to some extent the possibility to
make use of each other's work.
Some efficiency gains in the allocation of resources could be foreseen and quantified. More
specifically, option 2 is expected to provide modest cost savings compared to option 1 for
HTA bodies resulting from the voluntary production and uptake of joint REAs and full HTAs,
estimated at a total of EUR 383 000 (EUR 256 000 for pharmaceuticals and EUR 127 000 for
medical technologies) per year.
154
This estimation is confirmed by the results of the survey on the expectations of HTA bodies,
which indicate that the overall costs are expected to slightly decrease/remain stable for option
2. The additional workload caused by the joint work in addition to the national activities is
expected to be partly offset by the gains of the cooperation.
154
GÖG-LSE Study Section 7.2.3.2 While there are uncertainties in the cost calculation, the overall estimates
confirm that the impact on costs is neutral/mildly positive for all options of continued HTA cooperation.
Particular challenges include the high variation of HTA-related
costs both from the HTA bodies’ and industry's
side; or the assumptions related to the uptake of joint reports. Moreover, a number of impacts could not be
quantified, e.g. the reduction of national assessment due to better cooperation, alignment of methodology, and
data availability; reduction of costs of evidence generation; reduction of costs due to a lower number of early
dialogues. Sensitivity analysis was conducted.
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It should be noted that in its estimate the GÖG-LSE study assumed that any new EU project
would be of similar size as the EUnetHTA Joint Action (2016-2020). The requirements to
limit the number and define the profile of participants foreseen under this option may further
optimise cost savings due to a more homogenous, smaller and possibly more efficient
Consortium.
The differences caused by HTA bodies developing their own practices, processes and
methodologies independently as described under option 1 are expected to remain, but slightly
decrease under option 2 due to the project-based cooperation at EU level.
This option is expected to have some negative impact on
administrative burden
resulting
from the requirements related to the reporting obligations associated to EU co-funded
projects.
Concerning the
governance system
and the
financing,
this option is expected to have an
impact on the
EU budget
for running the project which is estimated at EUR 5 300 100
155
.
Industry
Duplication of efforts for industry to comply with different Member States' requirements, i.e.
for parallel assessments, early dialogues and additional evidence generation (as discussed in
detail in section 2, problem 1), are expected to continue under policy option 2. However, a
focused and efficient project-based cooperation, which has addressed some of the
shortcoming of the current cooperation model, may reduce differences in approaches and
facilitate some joint reports which may then be used in national decision-making processes.
This persisting but slightly improved fragmentation of the
internal market
is in line with the
pharmaceutical industry's own expectations as revealed by their replies to the survey carried
out by the GÖG-LSE Study and the public consultation.
Regarding costs for the
pharmaceutical industry
under policy option 2 compared to the
baseline scenario, modest savings were identified for the entire pharmaceutical sector across
the EU compared with the estimates under the baseline scenario. The cost savings resulting
from the production of joint REAs and full HTAs are estimated at EUR 3 700 000 per year
compared with the baseline
156
. This corresponds with the expectations of the pharmaceutical
industry which indicated that option 2 could lead to a slight reduction of costs and
administrative burden. On the other hand, in the public consultation and the EFPIA/CRA
study it was highlighted that without mandatory uptake, the benefits of the joint work would
not materialise for industry and therefore it would be more and more challenging to identify
companies which would be willing to engage in voluntary submissions for Joint
Assessments
157
.
Business predictability
is expected to remain low under policy option 2 and the benefits in
terms of
innovation and competitiveness
would not materialise. This is well in line with the
responses provided by the pharmaceutical industry to the public consultation stating that the
current model of cooperation based on voluntary joint work, which has limited effect on the
155
156
GÖG-LSE Study Section 7.2.3.2
GÖG-LSE Study Section 7.2.3.2
157
It should be noted that under a voluntary framework of cooperation, experience has demonstrated that for
pharmaceuticals Joint assessments are most efficiently carried out with the active engagement of the Industry,
which however also remain voluntary.
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convergence of HTA processes and methodologies across EU, leads to low business
predictability (93%) and discourages innovation (74%).
For the
medical technologies sector,
the economic impacts of option 2 would differ given that
the current market access path is more diverse and the role of HTA in the process is
substantially less developed than for pharmaceuticals.
Modest
cost
saving for the sector are expected compared to the baseline scenario related to
the preparation and submission of joint REAs and full HTAs, estimated at EUR 92 000 per
year. According to the results of the survey conducted by the GÖG-LSE Study, the medical
technologies sector indicated that stable/slightly increased costs are expected under option 2
compared with the baseline scenario, but with efficiency gains in terms of administration.
The medical technologies sector, both the large companies and SMEs, perceives this option
very positively. Industry representatives from this sector have expressed support for a
voluntary, non-legislative system
158
and argued in favour of a process that is demand-driven
by decision-makers responsible for the coverage and funding of health technologies. The
medical technology industry also expects that option 2 would reduce fragmentation and
increase predictability, competitiveness and innovation. However, such expectations have not
been substantiated by any specific evidence and from our analysis of the past and current EU
cooperation on HTA, such benefits are not expected, especially taking into account the issues
identified in section 2.
Overall, commitment and resource investment by industry to an EU system for production of
joint outputs are expected to remain limited, as there is no mandatory national uptake of joint
outputs foreseen under this option, nor any obligation to industry to submit technologies for
joint assessment. Continued low uptake by national HTA systems is expected to limit the
impacts of this option on reduced duplication of efforts, increased efficiency gains and
improved business predictability for industry. This option is therefore only expected to have
limited positive impact on improving the functioning of the single market compared to the
baseline.
6.1.2. Social impacts
Member States/Public administrations
As described above, joint reports such as early dialogues and joint REA would be done in the
framework of a project. Considering that participation in the project is voluntary in
combination with high requirements as regards profile role and number of participants and
conditionality for uptake, it is likely that not all Member States will be able or willing to meet
the conditions identified in the Terms of Reference/Call. This together with other hurdles to
national uptake described above is expected to preserve the significant differences throughout
the EU as regards the number of HTAs performed, e.g. only in some well-developed systems,
HTA bodies would be able to assess all newly authorised medicines. Therefore, this policy
option is expected to bring no or very limited benefits on
sustainability of the health
systems
and
public health
in general.
In addition,
t
he limitations in participants and their profile, which would be necessary for
improving the likelihood of timely output of consistent quality in a project based cooperation,
158
Public consultation, GÖG-LSE Study
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is likely to negatively affect the inclusiveness of the option and as such not provide EU wide
capacity building opportunities, which are particularly necessary in Member States with less
developed HTA systems which are often the ones more in need of enhanced sustainability.
The role of the HTA Network in disseminating the knowledge across the EU could mitigate
this, but only to a limited extent.
The impact of the joint work is expected to be limited for
decision-making
by Member States
due to the difficulties to enforce any contractual obligations. Also, the intermittent/disruptive
nature of the cooperation based on a renewable project, is expected to have negative impact
on
governance
as it would prevent planning and overall sustainability.
Since, as discussed above, uptake of joint outputs in national HTA systems cannot be ensured,
this option is expected to have only limited impacts on strengthening evidence-based
decision-making for the benefit of Member States health systems.
Patients
As regards
participation and good administration,
the stakeholders' involvement and
transparency would slightly improve compared with option 1. The requirement which could
be introduced in the call for proposals/terms of reference to include patient representatives in
the project(s) to increase their involvement in the preparation of Joint outputs, while may be
implemented correctly in Joint output, is unlikely to have significant impact on national
practices. Stakeholder involvement, while valued by many HTA bodies, is seen as a major
challenge to implement due to the resources needed. Current experience of patients and other
stakeholders is that the Joint Actions did not sufficiently address the issue of patients’
involvement in the joint HTA evaluations.
159
This is confirmed by the results of the survey
carried out by the GÖG-LSE Study, showing that HTA bodies do not expect stakeholder
involvement to increase.
The currently observed delays and divergences in the
availability of innovative new health
technologies to patients
across Europe would likely remain unchanged under this policy
option. As emphasised by patients in the public consultation, current differences in HTA
methodologies/procedures contribute to diverging outcomes of HTA reports (83% of patient
replies) and disincentives to innovation (58% of patient replies), with negative consequences
for the availability to patients.
However, while the Option foresees a number of requirements to ensure more timely and
consistent quality output (see description in policy option 2), it should be noted that there are
important
risks and issues of feasibility
related to a project-based cooperation model as
suggested under this policy option. The success of joint REA would continue to largely
depend on the use of the report in national decision-making and the voluntary industry
submission. Therefore, there would be no guarantees for fully realising these benefits.
160
As
159
E.g. contribution to the public consultation by European Cancer Patient Coalition, European Organisation for
Research and Treatment of Cancer (EORTC) Association Internationale de la Mutualité, European Social
Insurance Platform
160
While there are uncertainties in the cost calculation, the overall estimates confirm that the impact on costs is
neutral/mildly positive for all options of continued HTA cooperation. Particular challenges include the high
variation of HTA-related
costs both from the HTA bodies’ and industry's side; or the assumptions related to the
uptake of joint reports. Moreover, a number of impacts could not be quantified, e.g. the reduction of national
assessment due to better cooperation, alignment of methodology, and data availability; reduction of costs of
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uptake of joint outputs in national HTA systems is not made mandatory under this option, the
impacts of this option on strengthening evidence-based decision-making for the benefit of
patients will remain limited.
As stated by EURORDIS in their contribution to the public consultation as long as
"work is
voluntary for both HTA and industry, there is no virtuous cycle: industry may hesitate to
participate in a joint assessment, HTA bodies may hesitate to participate as authors, other
hesitate to use the HTA reports in parts or in totality, so convincing evidence that such joint
work is useful to all is difficult to generate."
6.2. Policy option 3. Permanent cooperation on common tools,
procedures and early dialogues
6.2.1. Economic impacts
Member States/Public administrations
This option is expected to have a moderate positive impact for Member States/public
administrations.
For HTA bodies, the overall
costs
are expected to slightly decrease due to the joint early
dialogues which would largely replace national dialogues as well as the stronger convergence
of processes and methodologies (including horizon scanning, IT tools etc.), which is expected
to facilitate reuse of national HTA reports. Such reuse of national HTA reports can be
particularly beneficial to Member States that have limited HTA resources. It may not
primarily result in resource savings, but in an increase of quality.
This option may also enable further voluntary cooperation on joint outputs among Member
States outside the framework (e.g. regional cooperation with participation of a limited number
of Member States and on specific activities), which may also contribute to some cost savings
and
more efficient use of resources
for those involved.
On the other hand, Member States would be faced with some
administrative costs/burden
to
implement and adapt their systems to the common tools and procedures. This is a one-off
cost/burden appearing at the start of the cooperation which is expected to affect all Member
States but to different extent depending on the HTA rules/procedures in place at national level
(as described in annexes VIII and IX). Member States with more detailed national rules will
have to adapt more, but at the same time would benefit from already developed rules/policy at
national level as regards quality, transparency, stakeholder involvement etc. Member States
with a less developed system and legal framework will have to adopt new rules according to
the proposed common rules and procedures. Considering that they have little in place this is
not expected to be over demanding. In any case, these administrative costs are expected to be
more than compensated by work-sharing arrangements, especially through the facilitated
reuse of national HTA reports due to the common tools and procedures.
Impacts would differ across the EU depending on the current activities of the national HTA
body. For instance, for smaller agencies which currently conduct only a limited number of
HTA activities, cooperation could increase the scope of their activities.
evidence generation; reduction of costs due to a lower number of early dialogues. Sensitivity analysis was
conducted.
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It has to be noted that the main drivers of cost changes in policy option 3 are difficult to
quantify due to the lack of data (e.g. on the number of national early dialogues conducted
across Europe), as further explained in the GÖG-LSE study. In the survey and the focus group
discussion, the conclusion was that stronger EU cooperation would lead to a cost decrease per
product. Current experience suggests that sharing the work decreases the costs for national
agencies significantly (in one case where only two agencies agreed to cooperate on clinical
guidelines they were able to save 30% respectively).
161
Public administrations' response to the public consultation confirmed the usefulness of the
joint tools (responded very much (75%) or to some extent (25%) to their needs) and
guidelines (responded very much (55%) or to some extent (37%) not at all (8%) to their
needs).
Industry
Under option 3 the expected economic impacts and their magnitude differ for the
pharmaceutical and medical technologies sectors.
Industry would face fewer differences when dealing with multiple national systems, as the
tools and procedures (e.g. submission templates, data requirements, early dialogues) would be
streamlined. This is expected to remove part of the current distortion on the
internal market
and facilitate more equal market access for health technologies throughout the EU.
For the
pharmaceutical industry,
the most important economic impact is related to the
expected benefits in terms of
predictability,
leading to better
innovation and increased
competitiveness.
The joint early dialogues would have an important positive impact on
predictability in so far as they could optimise the selection and design of the clinical trials and
reduce the risk of investing in costly trials that do not produce relevant acceptable data for all
HTA bodies, i.e. improve value for money.
According to industry
162,163
, early dialogues are very useful, improving transparency on
evidence requirements and thereby also business predictability. It represents an opportunity
for companies to receive a clear ‘red light’ message on certain aspects of medicine
development and reduce risks associated with the development of the product. Therefore, this
exercise can guide developers to invest their resources in viable developments from both
regulatory and HTA perspectives. Carrying out joint early dialogues in a systematic way
would remove the possibility of parallel national dialogues with conflicting messages for the
developers, thus contributing to a more effective design and more efficient funding of clinical
trials which would better meet the needs of HTA bodies as well as improved capacity to bring
innovations to market and increased competitiveness.
The relevance of early dialogues and increased predictability are particularly relevant for
SMEs
164
. According to SME interviews conducted by the Deerfield Institute, SMEs confirm
the potential of early dialogues and parallel scientific advice in reducing risks in the overall
development of a health technology. The report also points out the particular relevance for
SMEs creating the right clinical trial design from the beginning, due to the typically less
161
162
GÖG-LSE study, Annex 8
EFPIA/CRA Study
163
GÖG-LSE Study, survey and interviews Section 7.1.13
164
Deerfield Institute. 2015. EuropaBio survey on Regulatory and HTA advice for SMEs.
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available funding. This is underlined by the fact that that more and more SMEs request
parallel scientific advice with HTA. For example, in 2014 no such procedure was requested,
whilst in 2016, 26 % of such advice was finalised for SMEs.
165
In addition, the total number of national early dialogues and the associated costs due to fees,
are expected to decrease under this option. Nevertheless, the actual cost saving of this
decrease is estimated to be low due to the limited number of early dialogues currently carried
out and their costs compared to the overall investment on the product development.
166
Also, the alignment of procedures and tools foreseen under this policy option is expected to
improve efficiencies for industry and reduce the costs of complying with multiple systems
under the baseline scenario. These cost savings are relatively limited looking at the broader
perspective and would materialise once the necessary adaptations to the common format have
been made.
Regarding
administrative burden,
this policy option would not impose any mandatory
obligation for industry. Early dialogues would be initiated by manufacturers and they would
be voluntary. Streamlining tools and methodologies means that there is better alignment in the
information that manufacturers need to provide in the national processes (i.e. reduced need to
adapt to multiple national requirements) with some limited initial costs in order to adapt to the
common format. The alignment is expected to be particularly beneficial for SMEs with
limited resources and typically no national affiliates to engage with national authorities.
In the public consultation, over 90% of the respondents from the pharmaceutical industry
(non-SMEs) considered joint tools, guidelines and early dialogues useful to very much
respond to their needs. SMEs were also positive, around 75% responding that such joint work
would respond to their needs.
The positive impacts described above may be more limited for the
medical technologies
industry
due to the fact that HTA processes are less prevalent and in particular that early
dialogues are much less frequent in this sector. Only 28% of the companies reported
participating in early dialogues vs 70% of pharmaceutical companies, and even when
participants responded yes, they clarified that their experience was limited to one procedure.
The
medical technologies industry
also indicated strong concerns that a legislative
framework imposing mandatory uptake at EU level is expected to substantially increase their
costs
and administrative burden, which may have a strong negative impact on
predictability,
innovation and competitiveness.
Medical technologies representatives interpreted policy
options 3-5 as (leading to) legally mandating REA (or full HTA) at the time of market launch,
and as such they felt that it would substantially increase HTA activities in Member States for
medical technologies and fundamentally change the current business model, which is based
largely on public procurement at local level. As clarified in the description of the policy
options, option 3 is limited to cooperation on common tools, procedures and early dialogues
and does not foresee Joint REA at market launch for any technology in the scope of the
option. The statement that this option would fundamentally change the current business model
of the sector is thus unfounded. In addition, it should be noted that, in any case, HTA is
currently being developed and broadened at national level, including on medical technologies,
165
European Medicines Agency. SME Office annual report 2016.
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(see section 1.4.1) although in a divergent manner and that the joint early dialogues foreseen
under policy option 3 would only take place at the initiative of industry. Therefore the
concerns expressed by industry in this sector do not appear to be fully justified. This analysis
is also supported by the fact that in the public consultation, most respondents from the
medical technologies sector, both large companies and SMEs, considered that HTA tools
somewhat respond to their needs; they considered guidelines most relevant, followed by joint
tools and early dialogues. The responses from SMEs showed more variations.
However, it is understandable that, considering the recent changes to the Union legislation on
medical technologies, which is still in the process of being implemented, the
medical
technologies industry
is particularly sensitive to any further changes in processes which may
have any impact on the predictability of the market access pathway, their well-established
practices and business models. In this context it should be kept in mind that this initiative is
envisaged post 2020.
6.2.2. Social impacts
Member States/Public administrations
For the HTA bodies, the joint horizon scanning, joint early dialogues and the reuse of their
national assessments developed based on a common methodology mean that better evidence
would be available for
national decision-making.
This is particularly relevant for smaller
agencies or agencies that are still developing their capacities.
An increase in the consistency of the methods used to assess a technology through reliance on
common tools and an expected increase in the relevance of the evidence generated via joint
early dialogues would also have a positive impact on the
sustainability of health systems
and ultimately
public health.
This is particularly important in lower income Member States
as the opportunity costs of making a 'bad' decision are higher.
167
This is well in line with the
feedback received from research organisations (such as EORTC), and industry who have
pointed out that harmonised clinical data requirements across European HTA agencies would
lead to a stronger expression of the European data needs and thereby ensure that requirements
of HTA bodies and decision-makers would be adequately reflected by drug developers in the
design of the clinical trials.
On
governance and as far as good administration
is concerned, setting up a permanent
structure to enable HTA bodies to cooperate on a continuous basis on agreed joint outputs, is
also expected to contribute to building capacities in Member States, ensure and efficient use
of the resources devoted to the cooperation and ultimately have a positive impact on the
quality of the output.
Regarding the
risks and issues of feasibility,
option 3 has stronger guarantees for the
implementation than option 2 due to the legal framework foreseeing the common tools and
procedures. The political buy-in, particularly from Member States with well-developed HTA
systems is a key success factor as they are the ones that might need to adapt their systems
more, which entails upfront investment.
Patients
167
Kaló, Z., Gheorghe, A., Huic, M., Csanádi, M., and Kristensen, F. B. (2016) HTA Implementation Roadmap
in Central and Eastern European Countries. Health Econ., 25: 179–192.
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The partial removal of the current market distortion envisaged above (see economic impact on
industry) and the resulting improved market access for health technology products throughout
the EU would mean an improved
availability of innovative health technologies
for patients.
This option is also expected to improve patient
participation
to the HTA process and
transparency.
As highlighted by patient organisations such as Eurordis and the European Patient Forum,
early dialogues, if conducted with the involvement of the patients, not only improve
transparency but also provide valuable input to the discussion with technology developers
(e.g. on patient needs and preferences, relevance of particular health outcomes and quality of
life). Patients should be aware of the plans to develop a new technology for their disease and
have an opportunity to be part of the dialogue with the developers. Moreover, early dialogues
can reduce futile clinical research, and maximise the chances that the end results of the
development are relevant for HTA, which can then result in more timely decisions on patient
access.
168
Joint early dialogues together with common templates and common processes
would bring benefits in the 21 Member States currently without such a process, but also
improve transparency and patient involvement in the Member States where early dialogues
are currently conducted at national level. Patient organisations noted in the public consultation
that joint early dialogues would be more efficient for them than participation in several
national early dialogues, considering the time, financial resources and training needed for
patient participation. Joint early dialogues at European level would also give Member States
and patients more influence in steering R&D investment decisions by industry towards health
technologies with added value for patients.
The use of common templates implies that elements of value for patients are also adopted in
Member States where they are not currently captured; common processes can increase patient
involvement and transparency. In particular, patient involvement would substantially improve
in over half of the Member States as currently only 12 Member States indicated involving
patients in the assessment process of pharmaceutical (11 for medical technologies).
169
6.3. Policy option 4. Permanent cooperation on common tools and
procedures, early dialogues and joint REA
As explained in section 5.3.4., policy option 4 includes two sub-options:
Option 4.1 allowing Member States to decide if and when to start applying the joint
REA;
Option 4.2 requiring all Member States to apply the joint REA.
Option 4.1 would have the advantage of allowing Member States to select the time at which
they would wish to join the system which would ensure a strong political commitment and
willingness to participate. HTA bodies and stakeholders would have time to adapt to the new
system, thus minimising disruptions which are inevitable when moving from established
national processes to a Union approach. At the same time, this option implies a risk that some
168
EURORDIS public consultation contribution, , EPF position paper "Core Principles from the Patients'
Perspective on the Value and Pricing of Innovative Medicines."
169
Patient Involvement in Health Technology Assessment in Europe. Results of the European Patients' Forum
Survey. 2013.
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Member States would choose to stay out for a very long time, or permanently, which could
have a significant adverse impact on the internal market, an outcome which would be in
contradiction with the underlying objective of the measure as defined. Such a risk therefore
raises concerns as regards its legal feasibility.
Considering, however, the high interest in cooperation in joint REA indicated by Member
States in the public consultation and bilateral discussions as well as the wide interest from all
Member States in the EUnetHTA Joint Action (2016-2020), it is expected that over time most
Member States would join the system, but impacts would be spread over a longer period of
time than under option 4.2. The opt-in approach in option 4.1 thus makes it difficult to predict
which and how many Member States would benefit from the new system and in which time
frame and raises the question as to whether the specific objectives of reducing duplication and
increasing uptake of joint work could be fully met. On the other hand, option 4.2 would
ensure full coverage within a shorter period of time.
As the impacts of these two sub-options are expected to be similar, they are assessed together.
Differences are expected to be limited to the timing/moment when they occur and the
coverage throughout the EU.
6.3.1. Economic impacts
Member States/Public administrations
In addition to the economic impacts described under option 3, option 4 is expected to have
further positive impacts for Member States/public administrations, which are due to the joint
production and mandatory uptake of joint REA foreseen under this option. The mandatory
uptake foreseen under this option will be ensured by requiring Member States to use the joint
REA in the national system in the same way as they are currently using a national REA and to
report/notify their uptake to the Commission and to other Member States.
The
cost
savings related to the joint REA have been estimated at EUR 1 560 000 per year for
HTA bodies for option 4.1 and EUR 2 670 000 for option 4.2. This is in line with the results
of the focus group meeting with public administrations, who agreed that a stronger EU
cooperation would lead to a cost decrease per joint output (once the system is well established
and running).
170
Moreover, a high quality joint REA is also expected to contribute to broader
economic benefits for Member States which result from more efficient healthcare investments
decisions. However, such broader economic benefits are more difficult to quantify.
In addition to the adaptation to common tools and procedures foreseen under option 3,
Member States would be faced with some
administrative costs/burden
related to the
national participation in the joint REA. In the first phase, this is expected to mainly affect
those Member States with a more developed system as experts from those Member States are
most likely to be selected as assessors/co-assessors for the joint REA (see section 7.3.2.1. of
GÖG-LSE study). In these cases, the joint work will replace the national assessment and be
used in the same way to inform national pricing and reimbursement decisions. As an
additional benefit, national HTA bodies providing experts will be compensated for their work.
Member States with less developed resources will mainly benefit from work carried out by
170
Section 7.2.3.2 in contrast to this finding, the survey performed by GÖG-LSE Study, indicated a slight
increase of costs. Such contradictory results were discussed in the focus group and could be explained by
overheads and coordination costs at the beginning of the cooperation.
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others in the initial phase until they have built up own expertise. In a longer term, the work
sharing will be more equal and expertise developed across the EU. In addition, Member States
will need to ensure the use of the joint REA, e.g. to align processes and notify uptake. In any
case, these costs/burden are expected to be more than compensated by the long-term work-
sharing arrangements foreseen under this option, i.e. on joint REA, early dialogues and
common tools and procedures. The piloting of joint REAs under EUnetHTA has already
indicated the potential for efficiency gains from work-sharing arrangements: e.g. in one case
reported, a national HTA assessor was able to prepare a national REA in 5 days by adapting a
EUnetHTA pilot joint REA, whereas preparation of a national REA from scratch usually
takes 25 days in the respective HTA body
171
. It is expected that these efficiency gains would
increase further in the more streamlined joint REA preparation foreseen under option 4. The
efficiency gain estimated here is dependent on the selection of health technologies where joint
assessment is of EU wide/common interest. This option may also foresee some additional
administrative burden for Member States which have HTA provisions set out in national
legislation; these are normally the ones with more developed HTA systems. However, such a
burden, which is inevitable when moving from established national processes to a European
approach, is likely to be compensated for by the benefits from work sharing agreements (cost
savings, efficiency gains) as described above, in particularly for the Member States with more
developed HTA systems, as they are the ones most likely to perform Joint Work, at least in an
initial phase of the cooperation.
While there are efficiencies to be attained for HTA bodies, this option is not likely to lead to
an overall reduction in
employment,
as existing staff would be able to engage in further HTA
related activities (including economic assessments) that are relevant at national/regional level.
As indicated in the survey carried out by the GÖG-LSE Study, participating HTA bodies even
expect that the increase in output production and the mandatory uptake of REA would be
accompanied by an increase in staff.
The extent and timing of these impacts would vary between sub option 4.1 and 4.2 in the
sense that they would occur at a slower pace in option 4.1 with the risk of never reaching the
full impact described, contrary to option 4.2. Option 4.1 would allow Member States to decide
when, and even if, to join the system. However, a strict implementation of option 4.2 may not
fully accommodate Member State requests for time to adapt their national HTA processes to
the new EU system.
Industry
Both sub option 4.1 and 4.2 are expected to bring further positive economic impacts for the
industry compared with option 3, in particular for the pharmaceutical sector.
For the
pharmaceutical industry,
the introduction of joint REA introduced under options 4.1
and 4.2 would greatly reduce parallel clinical assessments for pharmaceuticals, as joint REAs
are produced jointly and taken up at national level, thereby contributing to improved
timeliness and convergence of outcomes of national HTA assessments. This is expected to
further reduce the current
internal market
distortion and improve market access for
innovative health technologies. Whilst improved conditions for the functioning of the single
market are expected under both policy options 4.1 and 4.2, it should be noted that considering
the opt-in possibility under option 4.1 there is no guarantee under this option– in contrast to
171
Example from presentation by Wim Goettsch (CAPR Meeting, Malta, 2017)
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option 4.2
–
that the internal market distortion would be significantly reduced across all
Member States.
The joint REA is also expected to result in
cost
savings estimated for policy option 4.1 at
EUR 35 000 000 and for policy option 4.2 at EUR 64 000 000 annually for the sector.
172
Scope for savings related to a joint REA was confirmed in the focus group meetings, where it
has been estimated that a joint report could recover 20-25% of the local HTA costs if there is
no requirement for translation/adaption. Also, no fees are foreseen for the joint REAs. Costs
of national submissions are also expected to slightly decrease due to aligned methodologies
and tools (see option 3).
However, companies would still have to address national requirements not pertaining to the
EU cooperation (i.e. provide information for the assessment of non-clinical HTA domains), as
well as national reimbursement procedures; and there are costs related to the production of
joint REA.
173
These potential savings for industry are minor if considered in the broader
context of R&D for pharmaceuticals. The overall drug development per drug is estimated at
EUR 1 926 000 000.
174
Overall, the pharmaceutical industry does not expect any significant
changes in their current costs, as confirmed by both the HTA GÖG-LSE Study (baseline,
interviews, study, focus group) and the EFPIA/CRA study. In their view, the benefits from
this option would manifest themselves in other indicators (such as business predictability)
rather than costs related to the production of HTA submissions (see below).
Industry
Joint REA
Estimated impacts
Joint Early Dialogues
Additional evidence
generation following joint
early dialogues
Savings due to single Joint REA per product estimated at
EUR 35 117 000 (option 4.1) and EUR 63 833 000 (option 4.2)
175
Costs of national submissions can slightly decrease due to aligned
methodologies and tools
Improved timeframe for market access for certain countries
Improved predictability, potentially very limited saving, one
central process could replace more national processes (~ option 3)
Improved predictability, no cost change (~ option 3)
Table 7. Summary of economic impacts for pharmaceutical industry option 4 (source: GÖG-
LSE Study)
A more important positive impact for the
pharmaceutical industry
relates to the timelines for
market access. The CRA/EFPIA study found that within a sample of 16 countries, the
availability of joint REA at the time of marketing authorisation can speed up market access in
12 of them from 2 to 6 weeks. The impacts of this 2-6 weeks of earlier access on a group of
originator pharmaceuticals launched in Q2 2008 (see Annex VIII) was estimated. Results
show that this (one-month) earlier access for innovative products launched in one quarter can
mean EUR 130 315 010 additional revenue over 12 years; which is a 1 % increase. A similar
172
173
Estimations for 30 EDs, 40 REAs, without industry fees.
Costs of joint REA for industry is estimated at EUR 140 000.
174
DiMasi et al, Journal of Health Economics, January 2016
175
GÖG-LSE Study. Cost estimations for 40 joint REAs for pharmaceuticals/year
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gain can be expected for other innovative products launched. In the long run, this may reduce
differences in access to market, improve the functioning of the internal market and have a
positive impact on
business predictability, competitiveness and innovation.
176
Accessing
first markets quickly is particularly relevant for SMEs that depend on fast access to first
revenues. Again, the uncertainty of full coverage and timeframe under sub option 4.1 needs to
be taken into account compared with sub option 4.2.
It should be noted that, less heterogeneity among EU markets would not necessarily translate
immediately into higher revenues since the negotiations on the pricing and reimbursement of
pharmaceuticals will still take place and the overall public budget allocated to
pharmaceuticals is not expected to increase.
Despite the efficiencies it is unlikely that in-country or central HTA-related human resources
would be reduced, although it may be necessary to relocate staff to a central level, so no
overall impact on
employment
is expected.
177
In the public consultation, the vast majority of
pharmaceutical industry
respondents
considered joint REA very useful and indicated a preference for mandatory uptake. SMEs
were slightly less positive, still over 75% considered joint REA very useful or useful.
By contrast, representatives of the
medical technologies industry
anticipate significant
negative economic impacts if there is a legally mandated joint REA at the time for market
launch. The representatives of this sector argue that a joint REA at time of launch would not
support decision-making but would delay market access.
178
If HTA is mandatory at market
launch, this would negatively affect innovators, the 'first movers', because they are obliged to
generate comprehensive evidence. As innovations in medical technologies are incremental
and do not receive the same patent protection as pharmaceuticals, the early followers can use
the generated evidence and enjoy the benefits of quicker market access. Thus, a situation is
created where the first mover has a considerable disadvantage, with a potential negative
impact on
innovation.
179
Moreover, due to the currently limited role of HTA before market access of medical
technologies, the extent of current duplications (therefore the scope for efficiencies) is more
limited than for pharmaceuticals. However as described in section 2 (problem 2), current
trends for increased use of HTA for medical technologies may increase the scope for
efficiency gains also on this sector. The
cost
calculation estimated little potential for savings
for the
medical technologies industry
due to reduction of duplication: EUR 3 000 000 (option
4.1) and EUR 7 000 000 (option 4.2) annually for the sector. It is unlikely that in the first
years of collaboration the number of joint assessments would reach 25 so even this modest
saving would be reduced. Similarly to the pharmaceutical sector, these costs are not
significant compared with the size of the sector.
It should be noted that the GÖG-LSE study team concluded in the context of its overall study
findings that some of the anticipated negative impacts expressed by
medical technologies
industry
representatives seem to be overestimated, which may reflect the lower level of actual
176
177
GÖG-LSE Study. Section 7.2.3.2.
GÖG-LSE Study Survey, focus group interviews Annex VI-VII.
178
Online Public consultation report. Strengthening of the EU cooperation on Health Technology Assessment.
2017.
179
GÖG-LSE Study Focus group interviews Annex VI-VII.
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experience with HTA among respondents from the medical technologies industry compared
with respondents from the pharmaceutical sector where HTA is already more established.
Also, the medical technologies industry seems to challenge conducting HTA as such, not
specifically joint EU assessments and as explained above (see also section 7.3.4.7 of the
GÖG-LSE study), the mapping shows an increase of national HTA on medical technologies
regardless of an EU initiative or not. Furthermore, the medical technologies industry
statement mainly relates to concerns with mandatory HTA at market launch.
In addition, there are reasons to believe that, contrary to what is suggested by industry as
referred to above, a joint REA on medical technologies can have benefits in terms of
innovation as it may rationalise investment decisions for developers, clarify the EU-wide data
needs, reduce uncertainties as regards procedures and ensure performance of health
technologies.
6.3.2. Social impacts
Member States/Public administrations
Sub options 4.1 and 4.2 are expected to further strengthen the positive social impacts of
option 3 for Member States/public administrations although the impacts are more certain and
predictable under option 4.2 as under option 4.1 some Member States could choose to stay out
in long term or permanently.
The availability of timely and good quality joint REAs means better evidence available for the
national
decision-making, sustainability of health systems and ultimately public health.
Member States with less developed HTA systems and/or less capacities and stronger
pressures on their health budget can particularly benefit from such evidence. Focusing the
joint assessment and mandatory uptake on the clinical aspects will avoid duplication and
ensure that the work is relevant for decision-makers, while at the same time not interfering
with Member States' subsequent decisions on making available certain health technologies to
patients or on pricing and reimbursement. The earlier market access referred to above
(economic impact on industry) would also increase the positive impact on health.
It would also allow pooling of expertise, with potential specialisation of HTA bodies in
certain therapeutic areas or types of health technologies, with a subsequent increase in the
quality of joint outputs.
It is expected that joint REAs would provide a stronger evidence base for price negotiation
with industry especially in Member States with less developed HTA systems.
In terms of
risks and issues of feasibility,
key factors for the success of this option are (1) the
timely and good quality joint REA and (2) the uptake of the joint work - especially as some
well-developed HTA systems would need to make necessary adaptations. These risks would
need to be addressed through a well-developed implementation mechanism, the legal
guarantees and the monitoring and evaluation.
Patients
The joint REA will further improve the
participation
of patients, transparency and
availability of innovative health technologies
compared with option 3.
Patient involvement in joint REA can enhance the quality and relevance of the HTA report by
improving the understanding of the impact of technologies in a real-life context (e.g. barriers
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to complying with current therapy, side-effects etc.), providing input related to quality of life
aspects and can lead to a higher accuracy in assessing the needs and preferences of patients.
180
Compared with the multiple parallel procedures for patient involvement in national
assessments under option 3, joint REAs would improve streamlining of patient involvement
by creating one procedure with the necessary dedicated resources. This is also expected to
lead to efficiency gains for patient representatives (e.g. in terms of time and training needs, as
already discussed in the context of joint early dialogues under option 3).
Regarding
availability of innovative health technologies
to EU patients, as explained in the
economic impact section above, a high quality and timely joint REA has the potential to speed
up assessment timelines and thereby reduce delays in the availability of innovative medicines.
For patients, an accelerated access by 2-6 weeks can be important
181
, because in many
diseases, earlier therapy is associated with better health outcomes. High quality and timely
evidence on the added therapeutic value of a product provided by the joint REA can also
contribute to promoting high quality and improved coherence of national full HTA reports.
Joint REAs would also facilitate the involvement in the HTA process and awareness of HTA
results of other relevant stakeholders such as health professionals. From the perspective of the
health professionals, a joint REA would facilitate their access to reliable, timely and objective
information on medical technologies and support them in taking better informed decisions
with their patients on the best treatment.
182
Timely uptake of positive HTA results in
evidence-based clinical treatment guidelines (which are often developed by scientific/learned
societies at European level) can further contribute to facilitating patient access in clinical
practice
183
.
The public consultation showed strong support from patient organisations to not only use
common tools and methodologies and perform joint early dialogues (see option 3), but to also
carry out the joint REA as foreseen by option 4. 87% of respondents in the category "patient
and consumers" noted that joint REAs would very much meet their needs, while the
remaining respondents in this category replied that it would to some extent respond to their
needs. Of note, some patient representatives even expressed support for a joint full HTA
report, although they also recognised the inherent complexities in implementing such a joint
full HTA.
6.4. Analysis of the governance structure and financing system
6.4.1. Description of the governance arrangements
As indicated in section 5, the following governance arrangements have been considered:
180
Project secretariat
Member State secretariat
Central secretariat
Patient Involvement in Health Technology Assessment in Europe. Results of the European Patients' Forum
Survey. 2013.
181
EFPIA/CRA Study 2017
182
Standing Committee of European Doctors (CPME) public consultation
183
Input from the European Society of Cardiology to the European Commission online public consultation
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o
established and hosted in a new EU Agency;
o
established and hosted in an existing EU Agency;
o
established and hosted in the European Commission.
No specific governance structure is foreseen for the baseline scenario and the project
secretariat is suitable only for policy option 2. Policy options 3 and 4 could, theoretically, be
supported by any of the remaining structures (a Member State secretariat or a central
secretariat with different locations) although some of the governance structures are much
more adequate than others as illustrated below.
Project secretariat
This type of secretariat refers to a governance/coordination structure responsible for managing
the day-to-day operations of a project, making sure the participants respect their tasks and
achieve their objective by agreed deadlines. A project consortium is chosen by the European
Commission or an EU Executive Agency (depending on the funding instrument) following a
call for tender /call for proposals and evaluation procedure.
In EU-funded projects a project management and coordination work package is considered
pivotal for achieving the project' objectives. Its aim is to establish the management structure.
It includes the day-to-day management and the quality supervision of the project as well as
reporting to the European Commission/Executive Agency. The main expected result is to
ensure a smooth coordination of the different steps of the project so they are realised on time
within the budget limits and according to the predefined objectives. Another expected result is
the coordination with partners so they are properly involved and regularly updated on the
implementation of the project.
Figure 9. Coordination of an EU-funded project
Member State secretariat
The secretariat is set up, hosted and run by a national/regional HTA body in one Member
State for an agreed duration.
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Figure 10 Diagram for the organisation of the Member States secretariat (based on GÖG-
LSE study)
The Member State secretariat would be organised and have the following
tasks:
1) Administrative support
•
Organisation of meetings, travel arrangements and other administrative issues relevant to
the overall coordination and to the operation of Member States representatives and Experts
organised in working parties (WPs);
•
Managing financial issues, especially important with regards to handling reimbursement of
national experts and any other financial issues;
•
Communication
•
Providing support to the Member States representatives overseeing the overall EU
cooperation on HTA.
2) Scientific/technical support
•
Support the production of outputs (Standard Operating Procedures for identifying and
organising the work of experts from national authorities in WPs; provide scientific/technical
support to authors and co-authors of the joint outputs);
•
Quality management (both from a scientific and editorial perspective);
•
Liaison with stakeholders (patients, industry, health professionals, academia, payers etc.).
3) Provide IT support
•
intranet, communication tools, database etc.
Central secretariat
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Figure 11. Diagram for the central secretariat (based on GÖG-LSE study)
Irrespective of its location in an EU agency or in the Commission, the
tasks
of this central
secretariat would be similar to those of the Member State secretariat. Depending on the
ambition of the policy option and foreseen outputs, it would include:
1) Administrative support
•
Organisation of meetings, travel arrangements and other administrative issues relevant to
the overall coordination and to the operation of the Management Board and Expert
committees;
•
Managing financial issues, especially important with regards to handling reimbursement of
national experts, any industry fees (only possible if an agency), and legal aspects;
•
Communication and
•
Providing support to Management Board.
2) Scientific/technical support (scientific secretariat to output-producing HTA bodies and MS
expert Committees)
•
Support the production of output (Standard Operating Procedures for identifying and
organising the work of experts from national authorities in Member States Expert
Committees; provide scientific/technical support to authors and co-authors of the joint
outputs);
•
Quality management (both from a scientific and editorial perspective);
•
Liaison with stakeholders (patients, industry, health professionals, academia, payers etc.);
•
Provide support for national implementation (e.g. training).
3) Provide IT support
•
Submission system, intranet, communication tools, database etc.
It has to be emphasised that the secretariat has no tasks related to the production of the
different joint outputs, which is ensured by experts nominated by Member States HTA bodies
organised in Committees.
As regards human resources, the staffing depends on the type and number of planned joint
outputs, as shown below and thus also depends on the policy option chosen.
Central
Secretariat
(PO3)
Central coordination management -
Total 14 FTE
-
Head (1 FTE)
-
Administrative support (total 4 FTE)
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Central
Secretariat
(PO4.2)
o
Head of administration (1 FTE)
o
Project Manager (1 FTE)
o
Administrative staff (2 FTE)
-
Scientific/technical support (total 6 FTE)
o
Head (1 FTE)
o
Scientific officers (2 FTE)
o
Methodology, guidelines, templates (2 FTE)
o
Administrative staff (1 FTE)
-
IT (total 3 FTE)
o
Internal support l (1 FTE)
o
Maintenance of tools and databases (2 FTE)
Output production contracted to HTA bodies
Central coordination management - Total
34,5 FTE
Head (1 FTE)
Administrative support (total 11 FTE)
o
Head of administration (1 FTE)
o
Project Manager (4 FTE)
o
Administrative (6 FTE)
-
Scientific/technical support (total 18,5 FTE)
o
Head (1 FTE)
o
Scientific officers (9,5 FTE)
o
Methodology, guidelines, templates (2,5 FTE)
o
Administrative (5,5 FTE)
-
IT support internal (4 FTE)
o
Internal support (1,5 FTE)
o
Maintenance of tools and databases (2,5 FTE)
Output production contracted to HTA bodies
Table 9. Characteristics and staff of the central secretariat as estimated for policy options 3
and 4.2. (based on GÖG-LSE study, for 65 joint REA/year)
6.4.2. Feasibility and efficiency of the governance arrangements
In order to choose the most appropriate solution, the pros and cons of the above mentioned
governance arrangements are described below, including their feasibility to support the
various policy options assessed within this Impact Assessment.
Project secretariat
Pros:
- It is an adequate mechanism for running projects with defined objectives for a limited
duration in time.
- Allows for flexibility, does not oblige Member States to commit for a long period of time.
Cons:
- Contractual obligations cannot guarantee uptake of the results/outputs because they do not
supersede national legal provisions.
- Does not provide guarantees for full EU geographic coverage.
- Member States have limited influence in prioritisation of projects and determining scope.
Conclusions:
It is suitable only for option 2.
Member State secretariat
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Pros:
- It is close to national expertise and processes and can ensure a strong Member State driven
approach.
- Staff from HTA bodies has experience with national HTA processes and the needs for
national decision-making.
Cons:
- Uncertainties/challenges related to hosting (location, decision mechanism for the nomination
of the hosting Member State).
- Uncertainties/challenges related to funding (recurrent contribution from EU budget).
- Uncertainties/challenges related to the possibility of collecting and redistributing fees from
industry to other HTA bodies and to enforce and ensure uptake will persist.
- Risk to steer the cooperation towards the model of one Member State, which may not be
suitable to all.
- It received low support from stakeholders in the public consultation (see Fig. 12).
Conclusions:
Particular challenges are related to the selection of the Member State hosting the
secretariat, possible rotation, political acceptability and sustainability of financing.
Would be difficult to reconcile with a legal framework as suggested in option 3 and
especially option 4, therefore this governance structure was not considered in the
following section assessing the costs.
184
Central secretariat
a) Central secretariat established and hosted in a new EU Agency
Pros
- An EU agency for HTA would preserve independence of HTA bodies from other influences
(e.g. regulatory, industry)
- Staff would be recruited in order to ensure expertise in all areas of HTA (i.e. EDs, REA, full
HTA) and for all type of health technologies
- It would be a permanent structure, with no additional administrative burden related to
renewal of the coordination structure, and allowing for a continuous production of joint
outputs.
Cons
- Currently there are important political constraints not to create new EU agencies
- It requires a longer start-up phase and higher costs due to start-up costs and over heads
(human resources, financial etc.)
- It requires a selection mechanism and a decision on location
- It would be less relevant if human resources and estimated output the agency would be very
limited in size.
Conclusion:
184
However, the costs were estimated in the GÖG-LSE study (section 7.2.)
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Suitable for EU cooperation encompassing a broader number of activities for which
no existing EU agency has appropriate expertise and a considerable size.
Allows for collection of fees in case of services provided to industry (i.e. ED)
Currently not a feasible governance arrangement due to important political
constraints, therefore this governance structure was not considered in the following
section assessing the costs.
185
b) Central secretariat established and hosted in an existing EU Agency
The pros and cons of the two EU Agencies in the field of health (i.e. European Medicines
Agency/EMA and European Centre for Disease Control/ECDC) are presented below.
EMA
Pros
- It is an established agency, so it would require less start-up costs
- It has experience running Member States' expert committees with a rapporteur-co-rapporteur
system (similar to the current mechanism for carrying out joint assessment by EUnetHTA
with author and co-author)
- It has already developed well established cooperation with HTA bodies and has already
some capacity and expertise in the area of HTA for pharmaceuticals.
- It can collect industry fees and has a fee structure in place
- It received some support from public consultation, especially from patient organisations
- It could ensure synergies in the area of pharmaceuticals between regulatory and HTA issues
- Would ensure continuous production of joint outputs
Cons
- Some Member States expressed concerns or a clear opposition (because of perceived
conflict of interest between authorisation and HTA processes)
- The medtech sector expressed opposition due to the lack of mandate, expertise and
experience in the field of medical devices and IVDs
- Uncertainty over future location and future capacity
- Requires a change in mandate for carrying out tasks in the area of HTA
ECDC
Pros
- It is an established agency, so it would require less start-up costs
- In principle ECDC could collect industry fees, but has currently no fee structure set up
Cons
- Requires a change in mandate for carrying out tasks in the area of HTA
- Need to expand the agency's mandate with associated risks of lengthy discussions on its
mandate or its further expansion.
- It would require to set up a structure for collecting fees from industry (e.g. for ED)
Conclusion:
185
However, the costs were estimated in the GÖG-LSE study (section 7.2.)
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Suitable for EU cooperation encompassing most types of joint outputs (i.e. common
tools and methodologies, horizon scanning, ED, REA).
Central functions are already in place.
It allows for collection of fees in case of services provided to industry (i.e. ED).
It would require changes in the mandate and staffing of both agencies.
Concerns expressed by some key stakeholders
c) Central secretariat established and hosted in the European Commission.
Pros
- It would avoid the debate on which agency should be more appropriate to take over the
HTA tasks and the discussions related to the changes of its mandate
- It is an honest broker
- It has experience with running Member States expert Committees and scientific Committees
(e.g. Scientific Committee on Health, Environmental and Emerging Risks/SCHEER,
Scientific Committee on Consumer Safety/SCCS)
- Several DGs already employ staff with scientific profile
186
(e.g. DG JRC, SANTE, RTD,
CNECT) and experts from Member States HTA bodies could be seconded for an agreed
period of time.
- It has broad support from the public consultation (see Figure 12)
- It would have the necessary infrastructure to facilitate the cooperation in relatively short
time.
Cons
- It cannot collect and redistribute fees from industry.
Conclusion:
Suitable for EU cooperation encompassing most types of joint outputs (i.e. common
tools and methodologies, horizon scanning, ED, REA).
It is a reasonable solution as long as human resources and estimated outputs are
limited
In addition to the pros and cons,
the contribution of the most feasible governance
arrangements to the achievement of the operational objectives
is outlined below (table
10).
Operational objectives
Project Secretariat
Central Secretariat
EC
EU Agency
Yes
Yes
Promote convergence in Possible, but with no
HTA tools, procedures and guarantees
methodologies
Reduce
duplication
of No
efforts for HTA bodies and
Yes
Yes
186
Scientific profiles for both European Commission staff and national experts include: HTA assessors,
pharmacists, pharmacologists, biologists, doctors, experts in biotechnology, engineers with expertise in the
development of medical devices and in vitro diagnostics, statisticians, researchers in the field of health
technologies.
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industry
Ensure the uptake of joint No
outputs in Member States
Ensure
the
long-term No
sustainability of EU HTA
cooperation
Yes
Yes. But
collection of fees
from industry is
not possible
Yes
Yes. Can contribute
to the financial
sustainability by
collecting fees from
industry.
Table 10. The contribution of the most feasible governance arrangements to the achievement
of the operational objectives
6.4.3. Input from studies and stakeholders on the feasibility and
efficiency of the governance arrangements
The study supporting the Impact Assessment carried out by GÖG and LSE concluded that a
central permanent governance structure supported by legislative cooperation could overcome
the current fragmentation of European HTA systems regarding both HTA processes and
related outcomes. Support functions can be more readily centralised in a permanent
cooperation model as compared to a temporary/short-term one. Such a secretariat is expected
to increase the efficiency of processes and ensure greater consistency in outcomes. It would
also enable national agencies and their experts to keep a primary focus on the scientific work
and not on the administrative and coordination functions, which supports production of high
quality joint outputs (e.g. organisation of meetings, interaction with experts from other
countries and/or stakeholders etc.).
The majority of the stakeholders who contributed to the online public consultation expressed
support for a stable, central secretariat.
Figure 12. Analysis of the overall replies provided to the online public consultation regarding
the governance mechanism of the future EU cooperation on HTA
In relation to the governance model, representatives of
public administrations
responding to
the public consultation emphasised the importance of separating the regulatory and HTA
functions and ensuring the independence of HTA agencies. Many respondents indicated that a
structure/unit to support HTA at EU level could be seen as a practical solution, especially if
EUnetHTA structures and tools (such as POP database, intranet) could be easily incorporated.
While some respondents were against the creation of a new EU agency, others expressed their
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preference for this governance mechanism which would better reflect the specific needs of the
HTA sector, with competencies clearly and transparently defined.
Academia
(e.g. EORTC)
and
patients' representatives
(e.g. EURORDIS, EPF) advocated for
a centralised HTA system, similar to the central marketing authorisation model involving
EMA, in order to ensure harmonised assessment of new technologies, especially if it
addresses the clinical assessment. This system should entail permanent administrative and
technical staff interacting with standing committee(s) of Member States. The system should
benefit from strong governance and appropriate resources, ensuring its independence and
guaranteeing high scientific standards developed and agreed by Member States experts. The
high-quality and transparency of the assessments should be maximal, and stakeholders
including clinicians, patients and industry, should be involved through appropriate permanent
mechanisms regulated by solid and well defined conflict of interest provisions, which are
considered key for the successful implementation of any of the policy options. A centralised
system was seen as the appropriate mechanism for ensuring adequate funding for patients'
involvement (e.g. training activities, developing methods to obtain relevant patients' views,
coordination activities, and contribution to guidelines development.
Representatives of pharma industry
(e.g. EFPIA, EuropaBio, Leem - Les Entreprises du
Medicament) emphasised that any secretarial/organisational support function should be based
on high scientific standards, should receive appropriate resources, and joint scientific
assessments should be carried out by committee(s) of Member States experts.
Representatives of the
medical technologies' industry
observed that setting up a new EU
agency does not seem feasible and, while EMA is a good model for a successful agency in the
field of pharmaceuticals, due to its limited/lack of expertise would be an inappropriate host
for the EU cooperation on HTA on medical technologies. In this context, an existing structure
within the European Commission was seen as a potential solution for providing support from
a secretarial and organisational point of view.
6.4.4. Costs related to the governance arrangements
Irrespective of the governance arrangements, two types of costs were estimated and analysed.
This section describes first the
running costs
followed by the description of the
costs directly
related to the joint outputs.
The following section refers to costs when the system is fully
operational.
Running costs
The key drivers of the running costs are the following:
-
-
-
The scope and number/volume of joint outputs foreseen in the policy options. These
costs would mainly be related to the services provided directly to experts drafting the
joint reports (e.g. committees for early dialogues and/or Joint REAs);
The geographical location of the secretariat. Depending on the price level of the
location, the costs of the central secretariat can change by 30%, driven mainly by the
cost of premises and the indexation of the salaries;
The size of the secretariat; a smaller secretariat would typically have higher
overheads;
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Table 11 summarises the running costs per policy option(s) and implementation
mechanism(s).
187
Table 11. Summary of running costs
188
. All costs are compared to the baseline scenario which
entails only minor expenses from EU budget (i.e. financing on average of two meetings of the
HTA Network per year
–
approximately EUR 120 000).
Cost of the joint outputs
The costs of the joint outputs range are presented in Table 12.
187
Option 2 was not included in the tables summarising the costs, because it does not have an overall governance
arrangement. A new EU Agency as potential governance arrangement was also discarded based on the political
constraints mentioned in section 6.5.2.3.
188
Adapted from tables 20, 52, 53 in the GÖG-LSE Study
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Table 12. Summary of joint outputs' costs per POs and implementation mechanism
189
. All
costs are compared to the baseline scenario which does not include EU budget earmarked for
production of joint outputs (i.e. cost for joint outputs = 0).
These costs are presented separately from the running costs of the secretariat for two reasons:
- These costs depend on the number of joint outputs estimated to be carried out under each
policy option and are therefore directly linked to the policy option;
- These costs correspond to a fully operational system, with a broad scope and a high number
of joint outputs (i.e. ED, REA). In practice it is expected that these costs will not reach the
values presented in the table from the beginning, but will gradually increase to the maximum
foreseen in the calculation as the cooperation produces the foreseen number of joint products.
The costs of the joint outputs include also the remuneration of HTA bodies carrying out the
joint work (i.e. joint REA, joint early dialogues) as authors, co-authors and reviewers. Fees
from industry could be foreseen to cover part of these expenses (i.e. for early dialogues)
depending on the governance structure chosen. However, the proportion and the mechanism
of the industry fees should be carefully considered to prevent any conflicts of interest and
guarantee the scientific independence of the work.
190
189
190
Adapted from tables 20, 53 and 56 in the GÖG-LSE Study
Fees for joint assessments are not foreseen in the first stage as it has been considered disproportionate to the
relatively limited size of the structure envisaged. However, it is suggested to evaluate the situation after a certain
period of time to consider if fees would be appropriate. A dedicated impact assessment and appropriate proposal
would be foreseen to examine industry fees.
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Overall costs
The overall costs (adding the running to the joint outputs' costs) are presented in Table 13
below.
Table 13. Summary of the overall costs per POs and implementation mechanism. All costs are
compared to the baseline scenario which entails only minor expenses from EU budget (i.e.
financing on average of two meetings of the HTA Network per year
–
approximately EUR 120
000), with no EU budget allocated for production of joint outputs (i.e. cost for joint outputs =
0).
7. Comparing policy options
The policy options presented above are compared against the criteria of
effectiveness
(the
extent to which the option would achieve the objective),
efficiency
(balance between costs vs
benefits) and
coherence
(with the overarching objectives of EU policies).
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General
Objectives
Specific
Objectives
Operational
Objectives
Policy
Option 1
(baseline)
No Joint
Actions after
2020
Effectiveness
Ensure
better functioning of the internal market
Contribute to a
high level of human health protection
Improve the
availability of innovative health technologies for EU patients
Improve
business predictability
Ensure
efficient use of resources
and
strengthen the quality of HTA across the
EU
Promote
Reduce
Increase the
Ensure long term
convergence in
duplication of
uptake of joint
sustainability of
HTA
efforts for HTA
output in MS
EU HTA
Procedures
bodies Industry
cooperation
methodologies
---
---
N/A
---
Persisting or even
Duplication of
No joint outputs
EU cooperation is
increasing
work, parallel
limited to the high-
divergences.
national/regional
level strategic
processes
policy discussions
within HTA
Network. No
comprehensive
scientific and
technical EU
cooperation is
foreseen. If
cooperation takes
place, is expected
between cluster of
MS.
Efficiency
(benefit to cost)
Coherence
- A deeper and fairer
internal market
- Support health systems
- Foster research and
innovation
Subsidiarity and
Proportionality
---
High costs (parallel
systems) with no or
limited benefit for both
HTA bodies and
industry.
---
Fragmented internal
market.
Low business
predictability. No positive
stimulus for innovation or
EU competitiveness.
No support to health care
systems to address
challenges.
--
MS cannot address
current fragmentation
at national level.
The output of the
national effort would
not be proportionate to
the investments.
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General
Objectives
Specific
Objectives
Operational
Objectives
Policy
Option 2
Project-based
cooperation
on HTA
activities
Effectiveness
Ensure
better functioning of the internal market
Contribute to a
high level of human health protection
Improve the
availability of innovative health technologies for EU patients
Improve
business predictability
Ensure
efficient use of resources
and
strengthen the quality of HTA across the
EU
Promote
Reduce
Increase the
Ensure long term
convergence in
duplication of
uptake of joint
sustainability of
HTA
efforts for HTA
output in MS
EU HTA
Procedures
bodies Industry
cooperation
methodologies
0
--
--
--
Some convergence
Some pooling of
Limited uptake of No long term
on methodologies
resources may
joint assessments sustainability is
may appear but
appear but
expected to
ensured as the
differences in
duplication of
persist as no
approach is project-
assessment practices work due to
enforcement is
based.
and their outcomes
parallel
foreseen.
are expected to
national/regional
remain.
process is
expected to
continue.
Efficiency
(benefit to cost)
Coherence
- A deeper and fairer
internal market
- Support health systems
- Foster research and
innovation
Subsidiarity and
Proportionality
--
Limited benefits for
patients, industry and
health care systems due
to the scattered approach
to the cooperation and
limited use of joint
assessments at national
level.
--
Fragmented internal
market for health
technologies persists.
Business predictability is
expected to remain low as
well as the stimulus to
innovation and
competitiveness.
Support to health care
systems remains
inefficient.
--
MS cannot fully
address current
fragmentation through
a project-based
approach.
The necessary
resources invested are
not expected to be
proportionate to the
output.
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General
Objectives
Specific
Objectives
Operational
Objectives
Policy
Option 3
Permanent
cooperation
on:
common
tools
procedures
early
dialogues
Effectiveness
Ensure better functioning of the internal market
Contribute to a high level of human health protection
Improve the availability of innovative health technologies for EU
patients
Improve business predictability
Ensure efficient use of resources and strengthen the quality of HTA
across the EU
Promote
Reduce
Increase the
Ensure long term
convergence in
duplication of
uptake of joint
sustainability of
HTA
efforts for HTA
output in MS
EU HTA
Procedures
bodies Industry
cooperation
methodologies
+
+
++
+++
Ensured
EU MS can
Mandatory
Long term
convergence in
benefit/rely on
uptake by HTA
sustainability is
terms of tools and
each other's
bodies of
ensured by the
procedures used by
assessments in an common tools,
permanent structure
EU MS.
easier way due to
procedures and
and the stable
the use of
conclusions of
funding from EU
common tools and joint early
budget + MS kind
procedures.
dialogues is
contribution.
However,
ensured.
duplication of
work due to
parallel
national/regional
HTA is expected
to remain
Efficiency
(benefit to cost)
Coherence
- A deeper and fairer
internal market
- Support health systems
- Foster research and
innovation
Subsidiarity and
Proportionality
+
Patients are expected to
benefit from some
convergence in HTA
methodology and also
improved participation
in the HTA process.
For HTA bodies' better
evidence is available and
more efficient use of
resources. For industry
improved predictability.
Slight costs savings
expected.
+
Moderate positive
performance concerning
the contribution of this
option to a fairer internal
market of health
technologies. and patients
are expected to benefit
from it. Business
predictability is expected
to improve Health care
systems benefit from better
quality evidence.
+
This option provides
for a pooling of
expertise and resources
in the area of common
tools, procedures and
EU early dialogues,
providing an EU added
value to MS activities.
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General
Objectives
Specific
Objectives
Operational
Objectives
Policy
Option 4.1
Permanent
cooperation on:
common tools
methodologies
early dialogues
joint REA (MS
opt-in)
Effectiveness
Ensure better functioning of the internal market
Contribute to a high level of human health protection
Improve the availability of innovative health technologies for EU
patients
Improve business predictability
Ensure efficient use of resources and strengthen the quality of HTA
across the EU
Promote
Reduce
Increase the
Ensure long term
convergence in
duplication of
uptake of joint
sustainability of
HTA
efforts for HTA
output in MS
EU HTA
Procedures
bodies Industry
cooperation
methodologies
++
++
++
+++
Ensured
No duplication of Mandatory
Long term
convergence
work expected
uptake by HTA is sustainability is
between the MS that between the
ensured for
ensured by the
opt-in.
participating MS.
participating MS
permanent structure
Lower risk of
However absence but does not
and the stable
divergent outcomes, of duplication
cover the MS not funding from EU
therefore business
cannot be ensured joining.
budget + MS in
predictability
if some EU MS
kind contribution.
increases and
do not join.
ultimately patients
will benefit from the
availability of HTA.
Efficiency
(benefit to cost)
Coherence
- A deeper and fairer
internal market
- Support health systems
- Foster research and
innovation
Subsidiarity and
Proportionality
++
Patients from
participating EU MS are
expected to benefit from
an improved availability
of innovative health
technologies and also
improved participation in
the HTA process.
For HTA bodies' better
evidence is available and
more efficient use of
resources. For industry
improved business
predictability.
Costs savings expected
for the participating MS.
++
Moderate positive
performance concerning
the contribution of this
option to a fairer internal
market of health
technologies depending on
the number of EU MS
participating and patients
are expected to benefit
from it. Business
predictability is expected
to improve according to
the number of EU MS
participating. Health care
systems of EU MS
participating benefit from
better quality evidence and
efficiency gains.
++
This option provides
for a pooling of
expertise and resources
providing an EU added
value to MS activities
in the area of HTA.
However the full
potential of EU
cooperation is
exploited in a limited
manner.
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General
Objectives
Specific
Objectives
Operational
Objectives
Policy
Option 4.2
Permanent
cooperation on:
common tools
methodologies
early dialogues
joint REA
(all MS from the
start)
Effectiveness
Ensure better functioning of the internal market
Contribute to a high level of human health protection
Improve the availability of innovative health technologies for EU
patients
Improve business predictability
Ensure efficient use of resources and strengthen the quality of HTA
across the EU
Promote
Reduce
Increase the
Ensure long term
convergence in
duplication of
uptake of joint
sustainability of
HTA
efforts for HTA
output in MS
EU HTA
Procedures
bodies Industry
cooperation
methodologies
+++
+++
+++
+++
Ensured
No duplication of Mandatory
Long term
convergence in
work. Efficient
uptake by HTA
sustainability is
HTA procedures
pooling of
bodies is ensured. ensured by the
and methodologies
resources and
permanent structure
in all EU MS.
expertise.
and the stable
No risk of divergent Expected increase
funding from EU
outcomes for the
of quality of
budget + MS in
clinical assessment,
HTAs.
kind contribution +
therefore business
industry fees for
predictability
early dialogues.
considerably
improves and
ultimately patients
will benefit from the
availability of HTA.
Efficiency
(benefit to cost)
Coherence
- A deeper and fairer
internal market
- Support health systems
- Foster research and
innovation
Subsidiarity and
Proportionality
+++
EU Patients: improved
availability of innovative
health technologies and
also improved
participation in the HTA
process.
For HTA bodies: better
evidence is available,
efficient allocation and
use of resources
/expertise. For industry
business predictability
considerably improves.
Costs savings are
expected. Benefit to cost
ratio is expected to be
the most advantageous
compared to the other
options.
+++
Positive performance
concerning the
contribution of this option
to a fairer and deeper
internal market of health
technologies and EU
patients are expected to
benefit from it.
The identified obstacles
impeding a well-
functioning internal
market are addressed.
Business predictability is
expected to improve.
Health care systems of EU
MS will benefit from
better quality evidence and
efficiency gains.
+++
This option provides
for a pooling of
expertise and resources
providing an EU added
value to MS activities
in the area of HTA.
Table 14. Comparison of policy options
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Legend:
Significant positive performance
Positive performance
Moderate positive performance
Neutral
Moderate negative performance
Negative performance
Significant negative performance
+++
++
+
0
-
--
---
PO
+
++
+++
0
-
--
*
1
*
******
2
*****
*
*
3
******
*
4.1
*******
4.2
5
Table 15. Overall scoring (Stars indicate the number of positive and negative scores the option has received)
---
*****
n/a
*
Total
-17
-12
+10
+15
+21
*******
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8. Preferred policy option
8.1. Description of the preferred option
As illustrated in section 7, option 4.2 receives the highest scores when comparing the other
options. However, this option implies a certain risk considering the view of some Member
States that they need adequate time to adapt to the system. This has been addressed by
integrating elements from other policy options (in particular policy option 2 and 4.1) and
allowing for some adjustments based on the assessment carried out in section 6 and comments
received from stakeholders. Such adjustments are discussed in more detail below.
8.1.1. Joint outputs
The preferred option comprises all types of joint outputs related to clinical aspects of HTA,
i.e. common tools/procedures, joint early dialogues and joint REAs (see option 4 and more
detailed descriptions of the different types of outputs in sections 5.3.3 and 5.3.4).
8.1.2. Technology scope
Pharmaceuticals
The preferred option defines the scope of joint REAs as follows:
Pharmaceuticals undergoing the central marketing authorisation procedure which in addition
meet one of the following criteria:
new products containing new active substances
existing products for which the marketing authorisation is extended to a new therapeutic
indication
The timing of the procedure for joint REAs will be linked to that of the central marketing
authorisation procedure (i.e. the joint REA will be available at the time of or shortly after
marketing authorisation), ensuring timeliness for supporting Member States decision-making
at the time of market launch. This approach is consistent with current HTA timelines in
Member States, i.e. around or shortly after the time of marketing authorisation of
pharmaceuticals (see section 1.4, Figure 3).
The scope takes into account the level of current duplication among Member States HTA
bodies (which is most prominent around or shortly after marketing authorisation), the EU
added value of a joint approach and stakeholder views (see sections 8.2 and 8.3). It also
respects the different remits of marketing authorisation and HTA (see section 1.3, text box),
while supporting synergies where possible (see section 8.2, coherence).
The preferred option will also allow for updates of joint REAs, i.e. re-assessments at a later
point in time, if significant additional evidence becomes available (e.g. from post-
authorisation studies).
191
Medical technologies
The preferred option limits the scope of joint REAs as follows:
191 Note that such updates of joint REAs could also draw on increased availability of real-world data, as
explained in more detail in section 1.3, text box.
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Medical technologies in the highest risk classes
192
and which in addition have been selected
by Member States for a joint REA based on the following criteria:
unmet medical need
potential impact on patients, public health, or healthcare systems (e.g. burden of disease,
budget impact, transformative technology)
significant cross-border dimension/ Union wide added value (e.g. relevance to a large
number of Member States)
Taking into account the more decentralised market access pathway for medical technologies,
the timing of the joint REA will not be linked to the timing of the conformity assessment, i.e.
it will not necessarily be at the time of market launch. Instead, Member States will define the
most appropriate time point for a joint REA as part of the selection process mentioned above.
Relevant considerations for the selection of an appropriate time point are expected to include
market access of the technology in a significant number of Member States and availability of
evidence for HTA purposes.
This limited scope for medical technologies takes into account the level of current duplication
among Member States HTA bodies, the EU added value of a joint approach and stakeholder
views and concerns (see sections 8.2 and 8.3).
Other technologies
For other technologies (i.e. pharmaceutical and medical technology products not covered by
the above-described scope, or other health technologies), the legislative framework will
include provisions for voluntary cooperation. Production and uptake of joint outputs related to
these technologies would be fully voluntary, but Member States could benefit from the
organisational framework (e.g. committee and secretariat structures) established by the
legislation.
193
By leaving joint work on other technologies at a voluntary level, the preferred option
incorporates elements of policy option 2 (voluntary cooperation between Member States and
cooperation on a broader scope of technologies).
8.1.3. Instrument
Type of legal instrument
The preferred option envisages the adoption of a new Union legislative act that could take the
form of a directive or regulation. In considering the most appropriate form of instrument, it is
necessary to assess the nature of the harmonisation intended to be achieved by the proposed
measure, the nature and extent of implementing measures that such harmonisation entails,
including, in particular, the extent of discretion afforded to Member States in the choice and
application of harmonised norms.
Mechanism for scrutiny of conformity assessments of certain class III and class IIb devices (as defined in
Regulation (EU) 2017/745, Article 55) and Mechanism for scrutiny of conformity assessments of class D
devices (as defined in Regulation (EU) 2017/746, Article 50)
193
Note the provisions for voluntary cooperation in the new legislative framework would replace the existing
voluntary cooperation on HTA as defined in Article 15 of the Cross-border Healthcare Directive 2011/24/EU,
i.e. the respective Article 15 would be repealed.
192
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In this context, it is notable that a key element of the preferred option is the establishment of
procedures and structures for cooperation on joint REAs at Union level. While inevitably such
a transition to a Union wide approach will require some adjustments to national rules, for
example, as regards allowing for the use of joint REAs at national level as part of the overall
HTA, that transition does not result in a need for significant implementing measures
establishing those procedures and structures at national level.
The study mapping of HTA processes across the EU shows that 26 Member States currently
have legal/procedural frameworks in place for HTA. Typically, key aspects related to the
HTA bodies roles and responsibilities and the HTA assessments are outlined in national
legislation, while much of the details e.g. on procedures are contained in the administrative
provisions of
Member States’ HTA bodies (see sections 1.4.1 and Mapping study on HTA
processes across the EU).
This suggests that a suitable adaptation period before the date of application of a regulation
would be a more adequate and proportionate approach than the transposition needed for a
directive, in ensuring uptake of joint REA and use of common tools.
Thus, in the light of the aims of the initiative, and the nature and level at which harmonisation
intended to be achieved, it is considered that a regulation would be the most appropriate form
of instrument for the proposed measure.
The new regulation foreseen under the preferred option will amend Directive 2011/24/EU, i.e.
Article 15 will be deleted from the Directive. The HTA Network, which currently provides
strategic guidance and policy orientation for the scientific-technical cooperation under
EUnetHTA (see section 1.4.2), will be replaced by an HTA high-level group composed by
experts from the Member States as part of the governance system defined in the new
regulation (see section 8.1.4 for further details on governance). The new regulation will
ensure that the good governance principle (including transparency and independence of
expertise), which is referred to in Article 15 of Directive 2011/24/EU, is re-introduced in the
new legislative framework. Current provisions for granting Union aid under Article 15 of
Directive 2011/24/EU will also be replaced by provisions for Union funding under the new
regulation foreseen by the preferred option (see section 8.1.4 for further details).
Ensuring mandatory uptake
The legislative framework foreseen by the preferred option will ensure the mandatory uptake
of the common tools/procedures, joint early dialogues and joint REAs for pharmaceuticals
and medical technologies that are within the scope as defined in section 8.1.2. Mandatory
uptake of joint REAs implies that Member States shall not repeat the REA at national level.
Member States shall use the joint REA in the same way as they would use a national clinical
assessment, i.e. they shall incorporate it in their overall national HTA process. This means
that Member States continue to be free to assess non-clinical HTA domains (e.g. economic,
organisational, ethical) at national level and to draw conclusions on the overall added value of
the technology.
Enforcement of mandatory uptake will be supported by a requirement on Member States to
share with the Commission / other Member States the national HTA report which incorporates
the joint REA carried out at EU level. This will be facilitated by IT tools developed as part of
the common tools of the EU cooperation. Moreover, as for any EU legislation, the
Commission may take the necessary action foreseen by the Treaty (e.g. infringement
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proceedings), if informed of failure to comply with the mandatory uptake or any other
requirement of the legislation foreseen by the preferred option.
Allowing time for adaptation and adequate safeguard provisions
The EU legislative framework foreseen by the preferred option will include provisions to
ensure that Member States have adequate time to adapt their national HTA frameworks to the
new EU system. This takes into account the diversity of HTA frameworks across the EU (see
section 1.4.1 and Mapping study on HTA processes across the EU) and the views of public
administrations in the public consultation (discussed further in section 8.3.). In particular, a
deferred application from the date of entry into force will allow for the alignment of
procedures and processes. After the date of application, a transitional period is foreseen
during which Member States can delay their participation in joint REAs and joint early
dialogues.
The legislative framework foreseen under the preferred option will also include a safeguard
clause allowing a Member State to carry out a national REA in addition to a joint REA if this
can be justified by a need to protect public health which is specific to that Member State.
Progressive implementation of joint work
The preferred option will include provisions for stepwise build-up of the new EU system for
joint work during its first years until it becomes fully operational.
In particular, the product scope for
pharmaceuticals
foreseen by the preferred option (see
section 8.1.2 and Annex XI) will be implemented in a progressive manner. This implies that
all pharmaceuticals identified in the scope are expected to go through a joint REA once the
system is fully operational. The numbers of e.g. joint REAs are expected to increase gradually
during the first years of joint work under the new EU system, taking into account the
capacities and priorities of Member States. Selection criteria (same as those used permanently
for medical technologies, see section 8.1.2) will be listed in the proposed legislation and
Member States will use these to agree which health technologies will be subject to joint work
during the building-up phase.
194
For
medical technologies,
the system will remain based on a prioritisation mechanism to
ensure that joint REA are only performed on medical technologies selected by Member States
according to agreed criteria within a limited well identified scope (see section 8.1.2).
It should be noted that by allowing adequate time for adaptation and progressive
implementation as discussed above, the preferred policy option combines elements of policy
option 4.1 and 4.2 in an optimal way. It allows sufficient flexibility for Member States to
adapt over a period of several years, while at the same time ensuring that all Member States
join the new EU system. The need for both sufficient time to adapt and join the system was an
important point which has been raised by several Member States in bilateral meetings when
considering a mandatory uptake of the output.
194
More details on the progressive implementation, including number of expected outputs (joint early dialogues,
joint REA ets.) over the first years before the system becomes fully operational are set out in Annex XI.
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8.1.4. Governance and financing
Governance
As shown in section 6.4. the analysis of the pros and cons of each possible governance
arrangement together with their potential contribution to the achievement of the operational
objectives of this initiative shows that at this stage the most feasible governance option is a
central secretariat hosted by the European Commission. Compared to the other governance
options, it offers a reasonable solution for an initial phase which requires a relatively limited
number of human resources while providing a stable structure for the EU cooperation on HTA
encompassing most types of joint outputs (i.e. common tools and methodologies, horizon
scanning, early dialogue, REA). The main disadvantage indicated in section 6.4, i.e. the
impossibility to collect and redistribute fees from industry, may be addressed in the future by
a possible transfer of the secretariat to an EU Agency. In addition, the governance option of a
European Commission secretariat received high support in the public consultation (see section
6.4.3).
On the basis of the analysis carried out (see section 6.5.), the most suitable governance
arrangement for the preferred option is considered a central secretariat hosted by the European
Commission, at least in an initial phase building up the cooperation.
As presented in section 5.3.4 and 6.5, the central secretariat will provide:
- administrative support (e.g. organisation of meetings, travel arrangements etc.),
- scientific/technical support (e.g. support for the technical meetings of the experts,
preparation of the documentation, manage procedures for involving stakeholders, ensure
quality management, support implementation of joint tools and procedures, etc.) and
- IT support (e.g. host and maintain the electronic submission system, the intranet and
internet, the databases/repositories of joint and national HTA reports, etc.).
A high level group/management board including representatives of Member States' HTA
bodies would manage the overall governance and would meet regularly to discuss the annual
work programme, topic prioritisation, progress with outputs (e.g. quality, timeliness) and
provide guidance and steer the cooperation.
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Figure 13. Diagram of the governance arrangement (i.e. EC Central secretariat) for the
preferred option
The scientific-technical work of producing the joint outputs would be carried out by experts
nominated by Member States' authorities organised in Committees/groups dedicated to the
various types of joint work (e.g. for Joint REA, for joint early dialogues, for horizon scanning,
for common tools and methodology etc.). For example, for the joint REAs, Member States'
experts acting as author/rapporteur and co-author/co-rapporteur would carry out the clinical
assessment of the application/dossier submitted by industry (complying with common tools
and procedures as described in section 5.2) and prepare a draft opinion. The HTA High Level
Group would thereafter examine the draft and approve the joint report which would then be
used in national HTA processes (see more detailed explanations on mandatory uptake above).
Financing
As regards financing, the costs of the secretariat should be supported from the EU budget
Approx. EUR 17 000 000 overall costs, of which EUR are 8 000 000 running costs/year, and
EUR 9 000 000 are costs of joint outputs/year. The remuneration of Member States HTA
bodies carrying out the joint work as authors and co-authors is included in the costs of the
joint outputs. Travel expenses for Member State experts contributing to the activities of the
dedicated committees/working groups (e.g. for joint REA, for joint early dialogues) are also
covered by the EU budget and are included in the running costs. An in-kind Member States
contribution would be possible in the form of a) seconded national experts
195
from
experienced HTA bodies who would also ensure the smooth transfer of the know-how
Seconded national experts are national civil servants or persons employed in the public sector who are
working temporarily for an EU Institution. They remain in the service of that employer throughout the period of
secondment and receive a daily allowance from the European Commission in line with the provisions in the Staff
Regulation.
195
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developed by EUnetHTA to the central secretariat and the interoperability between the central
secretariat and Member States HTA bodies and b) staff in national HTA bodies, who will
contribute to the activities of the relevant committees/working groups (e.g. on joint REA, on
joint early dialogues).
More detailed figures, including costs per year during the building-up phase are set out in
Annex XI.
196
Review clause
The EU legislative framework foreseen by the preferred option will also include a review
clause. This will enable a review of the new system once it has been fully operational for a
sufficient period of time, e.g. in terms of the need for any changes to the financing and
governance provisions, with a view to a possible transfer of the central secretariat to an
appropriate EU body. This could include an evaluation of the need to introduce a fee-for-
service system (e.g. industry fees to contribute to the cost of conducting joint REAs).
8.2. Justification of the preferred option
The preferred option is expected to provide for the best combination of effectiveness and
efficiency, while ensuring proportionality, subsidiarity and coherence with related EU
policies. It also considers the need to address potential risks and implementation challenges.
Effectiveness and efficiency
The EU legislative framework foreseen by the preferred option will ensure mandatory uptake
of joint work (see section 8.1.3 for further details). It will provide legal clarity and certainty
with regard to the legal status of joint outputs and enable and justify the necessary adaptations
in national legal/procedural HTA frameworks. As discussed in more detail in section 2
(problem 2), uncertainty around the legal status of joint outputs in the context of existing
national legal/procedural frameworks is a major reason for the low uptake under the current
voluntary, project-based Joint Action EUnetHTA. As explained in sections 5.3.2 and 6.2,
these hurdles could also not be sufficiently addressed by other forms of project-based
cooperation (even if contractual arrangements to promote uptake were envisaged). An EU
legislative framework which ensures mandatory uptake of joint work is therefore necessary to
effectively address the current problem of low uptake.
In addition, the EU legislative framework will also address other factors that currently hinder
uptake, in particular concerns around quality and timelines (discussed in more detail in
section 2, problem 2). The EU legal framework will define and ensure, inter alia, governance
and work-sharing mechanisms, high scientific standards of methodologies and assessments
(e.g. via pooling of expertise, input by external experts and stakeholders, quality assurance
mechanisms), and standardisation and timeliness of procedures for the production of joint
outputs. This will enable Member States to perform clinical HTA work at consistently high
quality and in a timely and efficient manner.
The figures included in the Annex are estimates. The contribution from the EU budget post 2020 will be
discussed within the framework of the preparation of the Commission's proposals for the next Multiannual
Financial Framework (MFF) and will reflect the outcome of the negotiations on the MFF post 2020.
196
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The preferred option will allow for the best possible achievement of the objectives of the
initiative. In particular, the preferred option will ensure that all Member States participate in
the new EU system, maximising the positive impacts of the initiative on the functioning of the
internal market and public health across the EU. Mandatory uptake of common tools,
procedures and high quality joint work on clinical aspects of HTA will promote evidence-
based decision-making, contributing to improved availability of innovative health
technologies to patients. The preferred option will also provide Member States with a
sustainable framework for cooperation and enable them to use their HTA resources more
efficiently. Industry will benefit from efficiency gains and from improved business
predictability with regard to evidence requirements and HTA outcomes.
The preferred option is cost efficient in the sense that the costs are significantly outweighed
by savings for Member States and industry, as a result of pooling of resources and avoiding
duplications (see sections 6.4.1, 6.5 and 8.3).
Proportionality and subsidiarity
Ensuring mandatory uptake by an EU legislative framework as foreseen by the preferred
option is proportionate in that it is an effective and necessary response to addressing the
current problem of low uptake of joint work (see previous section on effectiveness).
The inclusion of a provision for mandatory uptake is deemed necessary, as without this
obligation, there would be a high risk that the EU legislative framework foreseen by the
preferred option could not fully deliver on the objectives of the initiative (see section 4).
While improvements in quality assurance mechanisms and timeliness of procedures compared
to the current project-based cooperation may encourage and facilitate spontaneous (voluntary)
uptake by Member States, there would be no guarantee that all Member States would
consistently take up the joint outputs. In fact, there would be a risk that some Member States
may choose to decide on uptake of joint outputs (e.g. joint REAs) on a case by case basis, and
possibly only once the joint output has been produced. Such an approach would run counter to
the work-sharing, scientific consensus-building and decision-making mechanisms foreseen by
the preferred option (see section 8.1.4 for further details on governance). If there is no
obligation to take up the joint outputs, some Member States may not fully invest their
capacities and resources into the scientific consensus-building on joint outputs at EU level. If
some Member States continue to repeat jointly conducted work again at national level, the
objectives of the initiative to ensure efficient use of resources (both for Member States and the
EU) and reducing current duplication of efforts by HTA bodies would not be fully achieved.
Moreover, the current problems of duplication of efforts for industry and lack of business
predictability could not be fully addressed. Without mandatory uptake for Member States,
manufacturers may be confronted with situations where after submission of a dossier for joint
REA, they are requested by individual Member States to submit additional dossiers for
national REAs (with possibly different requirements and outcomes). In fact, mandatory
submission by industry for joint REAs (as foreseen by the preferred option) would be difficult
to justify and could even be considered disproportionate, if uptake by Member States
continued to be voluntary. Finally, without an obligation for mandatory uptake by Member
States, the expected benefits of the initiative in terms of quality and efficiency gains and
associated benefits for patients and public health may be unevenly distributed across the EU
(depending on the extent to which individual Member States take up joint work). In
conclusion, there would be a considerable risk that the initiative does fully deliver on its core
objectives of improving both the functioning of the single market and public health across the
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EU. This is substantiated by the current legal framework for the cooperation provide by article
15 of Directive 24/2011/EU, which defines ambitious objectives but has not been able to
achieve them.
The mandatory uptake foreseen by the preferred option is also proportionate in that it does not
go beyond what is necessary to ensure that joint outputs (e.g. joint REAs) are incorporated
into national HTA processes (see sections 8.1.3, 6.4.1, 5.3.3 and 5.3.4 for more detailed
explanations on mandatory uptake).
Proportionality and subsidiarity are further ensured by focusing the joint work to clinical
aspects of HTA, where EU cooperation can bring both quality and efficiency gains. The
assessment of more context-specific HTA domains (e.g. economic, organisational, ethical)
and decision-making on pricing and reimbursement remain at Member States level.
Mandatory uptake of e.g. a joint REA will not interfere with the assessment at national level
of non-clinical HTA domains. Moreover, mandatory uptake of a joint REA does not pre-empt
the national appraisal process which will continue to conclude on the presence/absence or
extent of added value (based on the scientific assessment of clinical evidence presented in the
joint REA and any additional non-clinical assessments conducted at national level).
The preferred option is also proportionate in that it limits the scope of mandatory production
and uptake of joint work to specific types of pharmaceuticals and medical technologies, and
allows flexibility concerning the timing of joint REAs for medical technologies (see section
8.1.2). This takes into account the differences between technology sectors and their market
access pathways (see section 1.3 and Annex V) and the EU added value of a joint approach
also in terms of focusing on the type of products where current duplication of work among
HTA bodies is most prominent (see section 2, problem 2). For other technologies, the
preferred option facilitates further voluntary cooperation (see section 8.1.3).
Finally, the preferred option respects the principle of proportionality by allowing sufficient
time for both Member States and industry to adapt to the new EU system (see section 8.1.3)
and by providing adequate safeguard provisions allowing Member States to carry out national
REAs in addition to joint REAs in duly justified circumstances related to the specific situation
in those Member States.
Coherence
The identified option constitutes a coherent approach, well in line with the EU's overarching
objectives, including a smooth functioning of the internal market, sustainable health systems
and an ambitious research and innovation agenda. In addition to coherence with these EU
policy objectives, the option is also coherent with and complementary to existing EU
legislation related to pharmaceuticals and medical technologies
197
. For example, there are
opportunities for mutual information-sharing and better alignment of the timing of procedures
between the joint REA and the centralised marketing authorisation for pharmaceuticals (see
Figure 3)
198
. Synergies are also expected between joint REAs for medical technologies and
197
Relevant legislation includes Regulation (EC) No 726/2004, Regulation (EU) 536/2014, Regulation (EU)
2017/745 and Regulation (EU) 2017/746 (see section 1.3 and Annex V for further details)
198
Note that the need for improved synergies, while respecting the different remits of marketing authorisation
and HTA, has been recognised by Member States in the HTA Network Reflection Paper "Synergies between
regulatory and HTA issues on pharmaceuticals" as well as by EUnetHTA and EMA in their joint "Report on the
implementation of the EMA-EUnetHTA three-year work plan 2012-2015".
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some of the provisions foreseen by the new EU Regulations for medical devices and
in vitro
diagnostics (e.g. strengthened rules on clinical evaluation and clinical investigation; EU-level
expert panels for certain high-risk medical devices)
199
. Moreover, the joint early dialogues
foreseen under the preferred option will contribute to the objectives of related EU legislation
on clinical trials to ensure that the evidence generated in clinical studies is robust and benefits
patients and public health. The option could also provide useful input to and synergies with
the Digital Single Market agenda by encouraging innovation and research of high-tech/digital
medical products and by supporting the development of a European IT infrastructure
supporting EU cooperation on HTA. The initiative is expected to play an important role in
supporting innovation for the benefit of patients by influencing longer-term R&D investment
decisions by industry (see section 1.3).
Addressing potential risks and unintended consequences
A potential risk to the implementation of the preferred option could be posed by challenges in
achieving scientific consensus on joint outputs on clinical HTA aspects. As discussed in
section 1.4.1, there are still divergences in the methodologies currently used by HTA bodies
in their national clinical HTA work. However, EUnetHTA and other European projects
(SEED project, EU-funded research projects) have already delivered the proof of concept that
convergence in methodologies and production of joint outputs at European level is possible
(e.g. common methodological guidelines, joint early dialogues and joint REAs produced by
EUnetHTA, see section 1.4.2). The improved governance structure foreseen by the preferred
option is expected to further facilitate consensus-building. In particular, it will ensure the
involvement of all Member States, both in the selection/prioritisation of the technology
undergoing a joint REA and in the preparation of outputs by technical staff and the final
approval of joint outputs by high-level representatives (see section 8.1.4). Such a governance
structure is expected to ensure that outputs are relevant and acceptable to all Member States.
For example, there may be situations where a minority group of Member States would prefer
a different comparator in a joint REA. Such needs can be accommodated by including
analyses against several comparators in a joint REA (as has already been discussed in the
context of EUnetHTA). Finally, consensus-building will be facilitated by limiting the content
of the joint REA to a scientific analysis and discussion of the clinical effects observed for
different health outcomes, in light of the available clinical evidence. HTA appraisals, i.e.
conclusions on the presence/absence or extent of added value (e.g. therapeutic, economic,
societal), will continue to remain at Member States level (see section 8.2, proportionality and
subsidiarity).
Another potential risk to the production of joint work under the preferred option relates to the
sharing of the work load among Member States. Particularly in the initial build-up phase of
the new EU system, some Member States may hesitate to take a leading role (e.g. as lead
authors of a joint REA) because of lack of experience with the new EU system. However, as
the joint work under the preferred option will build on best practices among Member States
HTA bodies and experiences with joint work under EUnetHTA, it is expected that HTA
bodies will be able to quickly familiarise themselves with the new system. Within the Joint
Actions agencies with experiences and capacities in HTA have been leading main work
Wild C, Sauerland S, Schnell-Inderst P. Closing the gap between regulatory and HTA requirements for
approval and reimbursement of high-risk medical devices in Europe, Journal of Medical Device Regulation,
Volume 14 (4), 2017
199
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packages and there is no reason to believe that they may change approach once the
cooperation becomes more structured and permanent, rather the contrary. Moreover, as
discussed in more detail in section 8.3, it is anticipated that Member States with advanced
HTA systems will take on the leading role particularly in the first years of the new system.
Member States with currently limited resources and capacities for HTA may initially
primarily participate by providing input and comments during the drafting process of e.g. a
joint REA. However, as confirmed by public authorities in the public consultation, joint work
at EU level can be expected to result in increased capacity building on HTA over time,
particularly for countries with more limited resources. It also provides opportunities for
building capacities and developing a leading role in specific areas (e.g. particular therapeutic
areas or types of technologies). The benefit of such a specialisation of expertise among HTA
bodies has been recognised by Member States representatives in the HTA Network
200
. It
should also be noted that such trends for specialisation have been observed among national
regulatory authorities in the context of the EU marketing authorisation of pharmaceuticals.
Finally, the preferred option will foresee dedicated administrative, scientific and financial
support by the central secretariat (including special allowances for lead authors), to further
encourage and incentivise active participation by all Member States in the production of joint
work (see sections 8.1.4 and 8.3).
Provisions of the preferred option to address the above-mentioned challenges are further
discussed in section 8.3.
8.3.Implications for Member States and other stakeholders
Member States
The production of joint outputs and their mandatory uptake foreseen by the preferred option
will require some adjustments to the legal/procedural HTA frameworks currently in place in
Member States. Individual Member States will be affected to different extents, depending on
whether they already have established HTA systems and how detailed the provisions in their
current legal/procedural frameworks are (see section 1.4.1 for further information on HTA
systems and processes across the EU). However, allowing adequate time for adaptation of
national frameworks and the progressive implementation of joint work during the first years
of the new EU system (see section 8.1.3) will ensure sufficient time for all Member States to
make the necessary adjustments.
With regard to resource implications for Member States, it is expected that any initial costs
related to the above-mentioned adjustments to national HTA framework will be more than
off-set by the efficiency gains from joint work (see sections 6.3.1 and 6.4.1). As explained in
section 8.1.4, the preferred option foresees that most of the costs of producing joint work
(support functions of the EU secretariat, travel of Member States experts to meetings, special
allowances to remunerate Member States bodies carrying out the work as lead authors of joint
outputs) will be covered from the EU budget. Member States may contribute in kind by: a)
sending seconded national experts to support the EU secretariat function, and b) through staff
of national/regional HTA bodies, who will contribute to the activities of the relevant
committees/working groups (e.g. on joint REA or joint early dialogues, see section 8.1.4). As
discussed in section 8.1.4., for each joint output, some Member States will assume a leading
200
HTA Network. Reflection paper on "Reuse of joint work in national HTA activities". 2015
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role (e.g. author and co-author drafting a joint REA) while the other Member States have
opportunities to review and comment, and the final output is then approved by all Member
States.
It is expected that Member States with advanced HTA systems (significant institutional
capacities, resources and expertise) will assume a larger part of the workload (e.g. by serving
as lead authors in joint assessments), particularly in the first years. This would be similar to
existing experiences with sharing of work load among national regulatory authorities in the
context of the EU marketing authorisation of pharmaceuticals, where some Member States
take a more prominent role in assuming the roles of main authors/rapporteurs
201
. It would also
be consistent with experience from the project-based EU cooperation on HTA (Joint Actions
EUnetHTA, SEED project, research projects on HTA, see section 1.4.2), where participants
from Member States with advanced HTA systems have been particularly active (e.g. serving
as work package leaders). Under the preferred option, Member States with advanced HTA
systems which assume a leading role in the joint work will also benefit from efficiency gains,
as part of the REAs which they currently have to produce on their own at national level will
be replaced by joint REAs with other Member States as lead authors. It should be noted that
Member States with advanced HTA systems already routinely conduct national REAs for the
technologies covered by the mandatory scope of the preferred option (as described in section
8.1.2) and some of them conduct national REAs for an even broader scope of technologies
(see section 1.4.1). Member States with advanced HTA systems are therefore expected to
have both the capacity and willingness to take a leading role in the production of joint work,
in particular in the first years of the new EU system. In addition, the EU secretariat will
provide compensation to Member States’ bodies carrying out the joint outputs as lead authors
in the form of special allowances (and will also cover the travel expenses of the other Member
States experts attending the meetings of the relevant committees/working groups providing
input to and approving the joint work, see section 8.1.4).
Member States with less advanced HTA systems and limited capacities, the efficiency gains
from joint work will be even greater: While they are currently only able to perform HTA for a
limited number of technologies due to resource constraints, joint REAs will enable them to
take better informed decisions on larger number of technologies. Member States with limited
resources could also benefit from efficiency gains through increased specialisation in an EU
system for joint work. For example, they could build up capacity in specific areas (e.g.
therapeutic indications, types of technologies) and over time assume an increasing role as lead
author for e.g. joint REAs in these areas.
202
A well-defined product scope focusing on health
technologies with EU added value and cross border relevance with appropriate
selection/prioritization criteria will allow Member States to focus on joint work relevant for
all.
In addition to the above-mentioned efficiency gains, Member States will also benefit from
quality gains through joint work. As already discussed in section 8.2., the EU legislative
framework foreseen by the preferred option will include common procedures such as quality
assurance mechanisms, procedures for consulting external experts (e.g. in the development of
methodologies or the production of joint REAs), and conflict of interest rules to ensure
See EMA Annual Report 2016, Section on European medicines regulatory network
Note that similar specialisation trends have been observed among national regulatory authorities in the
context of the EU marketing authorisation of pharmaceuticals.
202
201
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independence of expertise. These common procedures will build on current best practices
among HTA bodies across the EU. Member States with less advanced HTA systems and
limited resources will particularly benefit from the ensured high quality of joint outputs. But
even Member States with advanced HTA systems are expected to benefit from further quality
gains, by pooling their expertise and resources, and by drawing on the best available external
expertise across Europe (e.g. specialists for particular therapeutic areas, including rare
diseases, or experts on new and complex technologies).
The preferred option takes into account the views expressed by Member States representatives
in the HTA Network and by public administrations in the public consultation. Member States
have expressed their willingness, in principle, to take up joint work on clinical assessments
(REA), provided that current hurdles such as the legal uncertainty around the status of joint
outputs and concerns around quality assurance and timeliness are addressed. For example, the
HTA Network (which includes representatives at policy level from all Member States, see
section 1.4.2) has called for the strengthening of joint work (e.g. via improved quality
assurance and process management), for addressing administrative/legal hurdles and for
increasing the uptake of joint work in national activities.
203,204
In the public consultation,
public administrations also expressed support for the principle of uptake of joint work. In
particular, the majority of public administrations expressed support for the notion that once
institutions participate in joint work, uptake should be mandatory for them. With regard to
participation in joint work, a number of public administrations stressed the need to allow time
to build up the new EU system and to adapt national processes accordingly. The preferred
option takes into account this need for time to adapt by foreseeing both a deferred application
and a transitional period during which Member States may delay their participation in joint
work (see section 8.1.3).
Moreover, the provisions foreseen by the preferred option for improved transparency (see
sections 5.2, 5.3.3 and 5.3.4) are consistent with the general acknowledgement among
Member States of the importance of the good governance principle. For example, the HTA
Network has recognised that "cooperation is based on the principle of good governance,
including: transparency, objectivity, independence of expertise, fairness of procedure and
appropriate stakeholder involvement"
203
. Moreover, the HTA Network has supported
specifically that "authorities responsible for HTA should aim at ensuring that HTAs are
electronically accessible and understandable to stakeholders"
203
. The importance of
transparency has also been highlighted by stakeholders in the public consultation (also see
following section on patients and healthcare professionals).
The preferred option does not prevent Member States from continuing or initiating work at
national or regional level as far as non-clinical domains are concerned, thus mutual support
between EU cooperation, regional cooperation and national work can be envisaged.
Patients and healthcare professionals
The EU legislative framework foreseen by the preferred option will ensure adherence to
common procedures for stakeholder involvement and transparency (see sections 5.2, 5.3.3 and
5.3.4), building on current best practices of HTA bodies across the EU. Considering that some
HTA bodies currently do not involve patients and external experts such as healthcare
203
204
HTA Network. Strategy for EU Cooperation on Health Technology Assessment. 2014
HTA Network. Reflection paper on "Reuse of joint work in national HTA activities". 2015
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professionals or involve them only to a limited extent (see section 1.4.1), the preferred option
is expected to lead to significant improvements. For example, common procedures for patient
involvement will ensure that patients can express their views and provide their disease-related
experience when issues such as quality of life or importance of particular symptoms and side
effects are discussed in the context of a REA.
Patients and healthcare professionals will also benefit from increased access to high-quality
HTA reports. It should be noted that, as highlighted by the GÖG-LSE study, some HTA
bodies currently do not publish their HTA reports. The preferred option will contribute to
improving transparency in HTA across the EU, e.g. by ensuring publication of joint REAs and
other joint outputs.
In addition to pooling the expertise of HTA bodies across the EU, the preferred option also
foresees common procedures for ensuring input by external experts which can contribute up-
to-date specialist knowledge (e.g. on complex therapeutic areas, new technologies, methods in
clinical study design/analysis and evidence-based medicine) to the development of joint
outputs such as joint REAs or common methodologies. This will lead to improved quality and
more consistency in work on clinical aspects by HTA bodies across the EU. It is also in line
with the responses received in the public consultation from scientific/medical societies and
healthcare professional associations, who have expressed their willingness to become more
involved and contribute their expertise to the EU cooperation. Finally, improved quality and
consistency of work on clinical aspects of HTA across the EU will respond to the views
expressed by patient organisations in the public consultation: While patients acknowledged
that HTA bodies may reach different conclusions on non-clinical aspects of HTA (e.g. due to
economic differences between countries), they also noted that is does not make sense to them
that HTA bodies currently differ in their scientific assessments of the same clinical evidence
in the context of the clinical part of HTA (see section 2, problem 1). Patient representatives
have therefore called for this issue to be addressed by the EU cooperation.
Industry
As already discussed in detail in section 6.4.1., the
pharmaceutical industry
is expected to
overall benefit from the preferred option, which is in line with views expressed by the
industry representatives.
In the public consultation and the survey conducted by the GÖG-LSE study, representatives
of the
medical technology industry
expressed a number of concerns (see section 6.4.1). In
particular, they considered that their industry would be negatively affected if joint REAs were
applied to the full range of medical technologies (rather than focusing on technologies with
particular relevance to health systems across the EU) and if joint REAs were conducted at the
time of market launch (causing potential delays to market access). By limiting the product
scope of mandatory production and uptake of joint REAs and allowing flexibility with regard
to time point of the assessment (see section 8.1.2.), the preferred option takes into account
these concerns and at the same time ensures proportionality and EU added value (see section
8.2.).
In addition to the benefits already discussed,
SMEs
from both sectors are expected to benefit
from the fact that no industry fees are foreseen under the preferred option. This is expected to
increase participations of SMEs in early dialogues, which are particularly beneficial for
smaller companies with limited HTA expertise in the medical technology sector.
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Finally, industry representatives of both the pharmaceutical and the medical technologies
sectors have expressed a need for sufficient time to adapt to the new EU system, which will
be ensured by the preferred option (see section 8.1.3).
For further details on impacts of the preferred option on different stakeholders, see Annex III.
9. Monitoring and evaluation
Monitoring and evaluation of the specific objectives will have to use several means of data
collection, as not all objectives are equally quantifiable and some monitoring may depend on
a qualitative evaluation based for example on feedback from stakeholders obtained through a
survey.
This section will mainly suggest possible indicators on how to measure the effectiveness of
the preferred option in relation to the specific objectives stated in the impact assessment. As
much as possible quantitative indicators will be considered but qualitative ones may also be
used. As regards the implementation of the proposed legislation, data collection can be
qualitative by gathering information on the legal and administrative measures taken by
Member States to implement the legislation. Table 16 below illustrates different alternatives
for monitoring the outputs and to assess further impacts of the preferred policy option using
indicators on effectiveness.
Operational
Objectives
1. Promote
convergence
in HTA tools,
procedures
and
methodologies
Core indicators
Adjustment made by
Member States to their
legal/procedural HTA
frameworks, to enable
use of common tools,
procedures, and
methodologies and
joint REA
(qualitative)
Use of methodologies
and tools in MS HTA
activities. (qualitative)
Source of data
Member State National
Contact Points (NCP).
Member State authorities
Target
All Member States within
the foreseen application
(and possible transitional)
period.
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2.
Reduce
duplication of
efforts for
HTA bodies
and industry
3.
Ensure the
uptake of joint
output in
Member
States
Number of joint REA
for innovative
pharmaceutical
products (quantitative)
Number of national
REA for innovative
pharmaceutical
products (quantitative)
Number of joint ED
(quantitative)
Number of national
ED (quantitative)
HTA reports used by
other Member States
in national/regional
HTA activities
(quantitative)
Number of FTE
needed to produce a
national full HTA
report (quantitative)
Timeliness or joint
REAs
Number of joint REA
used in MS HTA
activities.
(quantitative)
Use of methodologies
and tools in MS HTA
activities. (qualitative/
quantitative)
Permanent secretariat of
the EU cooperation.
Member State authorities
National Contact Points
Qualitative evaluation
(e.g. comparison Member
State report with EU
assessment)
Initial target 65 REA
Long term target:
joint REA on all
pharmaceuticals
receiving a positive
recommendation by
EMA
No duplication of
joint REA.
No additional
Member State REA
(unless duly justified
dependent on
legislation).
Joint REAs for
pharmaceuticals
available at or shortly
after the time of
marketing
authorisation
Member State HTA
authorities.
Secretariat of the EU
cooperation
National Contact Points.
Full uptake of joint
REA if MS decides to
start assessment
procedure on the
same technology.
Comprehensive use
of methodologies and
tools provided by the
EU cooperation in
national HTA
activities.
4.
Ensure the
long-term
sustainability
of EU HTA
cooperation.
Budget devoted to
support EU
cooperation
Production rate of the
cooperation (number
of joint outputs/year)
Legislation
Member State HTA
authorities.
Timeline to be indefinite
(subject to review) to
ensure sustainability.
Table 16. Suggested core indicators for monitoring
Effectiveness indicators of actions and outputs in relation to the specific objectives (illustrated
in Table 16) will be part of the development of a broader monitoring programme, which will
also include specific indicators related to efficiency and coherence with other policies (e.g.
EU legislation on medicinal products and medical devices).
In addition to the monitoring programme, it is suggested that an evaluation will be carried out
at later point in time (when the system has been fully operational for a sufficient period of
time to enable a meaningful evaluation), to assess the wider impacts of the implementation of
the preferred policy option. In particular, the evaluation will look at addressing the problems
identified in the impact assessment. In addition, cost benefit analyses on the performance of
the implementation mechanism should be performed as part of future evaluations. According
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to the financial regulation, an evaluation will also be required, as the cost of the
implementation mechanism is expected to be above the EUR 5 000 000 threshold.
205
In summary, there are a number of quantitative indicators to assess the future cooperation, but
assessment will also depend on qualitative data sources. Monitoring and evaluation of the
wider impacts will require a number of qualitative tools such as desk research, surveys, focus
groups and Delphi surveys to assess many of the potential impacts. However, as regards
monetary costs of the cooperation, quantifiable data is expected to be available at a greater
extent.
205
Financial Regulation Article 30 and Rules of Application article 18
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Annexes
Annex I. Procedural Information
1. Identification.
The Directorate for Health and Food Safety (DG SANTE) is the lead DG on the initiative on
Strengthening of EU cooperation on Health Technology Assessment.
The initiative is in the European Commission's Work Programme for 2017, in Annex I New
Initiatives, under the heading A Deeper and Fairer Internal Market with a Strengthened
Industrial Base. The initiative has received the validation in the Agenda Planning on the 15
th
September 2016 (reference 2016/SANTE/144) when the Inception Impact Assessment was
published.
2. Organisation and timing
An Inter-Service Steering Group was set up and met on the 15
th
September 2016, 24
th
March, 6
th
June, 1
st
and 11
th
of September 2017. Next to the SG (Secretariat-General) and LS (Legal Service)
the following Commission services took part in the ISSG: BUDG (Budget), GROW (Internal
Market, Industry, Entrepreneurship and SMEs), RTD (Research and Innovation), CNECT
(Communications Networks, Content and Technology), ECFIN (Economic and Financial Affairs),
EMPL (Employment), TRADE (Trade), COMP (Competition) and the JRC (Joint Research
Centre). The members of the Inter-Service Steering Group were regularly informed on the
progress of the initiative and invited to relevant meetings.
In addition, there were close contacts with the European Medicines Agency and the Consumers,
Health, Agriculture and Food Executive Agency (CHAFEA) on this file and related projects and
studies.
3. Consultation of the RSB
The current initiative of DG SANTE was the first one to benefit from the new opportunity for
an upstream meeting with the Regulatory Scrutiny Board. The meeting took place on the 7
th
December 2016.
A first version of this Impact Assessment Report was submitted to the RSB on the 27
th
of
September 2017, the meeting took place on the 25
th
of October 2017 and the RSB written
opinion was received on the 27
th
of October 2017.
The Board concluded that the draft report required further work and asked for a resubmission.
The Opinion of the Board acknowledged the efforts to conduct an analysis of Member States'
structures, resources and processes in place for the development and use of HTAs. It also
acknowledges the quality of the stakeholder consultation.
However it identified shortcomings that need to be addressed, concerning the following key
aspects:
(1)
Justification of why considering the continuation of the current joint actions is
unsustainable.
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(2)
Justification of the choice of the baseline, and definition of the options.
(3)
Demonstration that the initiative is a proportionate and effective response to low HTA
uptake.
(4)
Explanation of what the proposed measures would imply for Member States with
regard to resources or adjustments to national regulatory frameworks and practices. Specify
the measures to improve patients' and consumers' participation in HTAs.
(5)
Further analyse the preferred option and the delivery mechanisms of the initiative,
including related resource implications.
DG SANTE carefully addressed all the comments received, including the technical ones
submitted directly to the DG in order to improve the quality of the IA Report.
A second version of the Impact Assessment Report was submitted to the RSB on the 21
th
of
November 2017 and the second RSB written opinion was received on the 4
th
of December
2017. The Opinion of the Board acknowledged the improvements to the previous version and
issued a positive opinion, while noting a number of remaining adjustment requirements to be
made by DG SANTE. DG SANTE carefully addressed the comments received in the final
version of the Impact Assessment Report. An overview of the main adjustments made in
response to the main considerations of the RSB is shown in the Table below.
RSB main considerations
Adjustments made in final version of IA
report
1) The baseline is treated as an option and not The final version of the IA report ensures that
as a comparator for the options.
policy options are consistently compared to
the baseline scenario. This has also been
clarified for figures related to governance and
budget. Adjustments were made accordingly
in sections 5.3.1 and 6.5.
2) The report provides indications that the
mandatory uptake of joint work would be
sufficient to address many of the current
shortcomings. However, it does not
convincingly demonstrate that it is necessary.
It is not clear what the resulting amendments
to the existing Directive are.
Further clarifications have been provided on
the proportionality of the preferred option,
elaborating why mandatory uptake of joint
work is considered necessary (see section
8.2) and clarifying the issue of
legal/procedural hurdles to uptake (sections 2
and 8.1). Moreover, the final version of the
IA report clarifies that some of the principles
referred to in the current Article 15 of
Directive 2011/24/EU (e.g. good governance,
transparency) will also be present in the new
legislative framework proposed under the
preferred option (sections 3 and 8.1).
3) The report provides insufficient indications Further details have been provided on
of Member States' support for key aspects of expected Member States support for key
the options.
aspects of the initiative, including
acceptability of mandatory uptake of joint
work, willingness and capability to take a
leading role in an EU framework and support
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for transparency measures (section 8.3). The
choice of a Commission-hosted secretariat is
also further elaborated (section 8.1.4).
4) The revised report insufficiently discusses
the uncertainties, risks, trade-offs and
implementation challenges associated with
the preferred option.
Risks and possible unintended consequences
of the initiative have been further discussed,
to better contextualise/qualify the expected
benefits of the initiative (sections 8.2 and
8.3).
4. Evidence
The Impact Assessment has strongly built on the consultation and experience of Member
States, through the two arms of the current HTA cooperation: the HTA Network and the
EUnetHTA Joint Action 3 as well as dedicated bilateral meetings. Input from stakeholders
was gathered through the Open Public Consultation, which was open between 21st October
and 13th January. SMEs were targeted through the European Medicines Agency and DG
GROW's Enterprise Europe Network. A number of bilateral meetings took place with
stakeholders. For more information, see Annex 2 Synopsis Report.
5. External expertise
The Impact Assessment was supported by three studies:
- Study on impact analysis of policy options for Strengthened EU cooperation on HTA
206
,
which was undertaken by SOGETI Luxembourg S.A., the Austrian Public Health Institute
(GÖG) and the London School of Economics (LSE), referred to as GÖG-LSE study. The
study provided an in depth analysis of (1) the role of HTA in the market access (baseline
scenario), (2) the potential impacts of identified options; (3) systematic literature review on
HTA, with a specific focus on Europe. The final report of the study is available (Annex X)
- Mapping of HTA national organisations, programmes and processes in EU Member States
and Norway
207
by Julia Chamova (Annex VIII)
- Mapping of HTA methodologies in EU Member States and Norway by Finn Borlum
Kristensen
208
(Annex IX)
In addition the draft deliverable from EUnetHTA Joint Action 3, Descriptive write up of HTA
processes (Work Package 7 National Implementation deliverable - unpublished) was also
used.
The main sources from the literature used are listed in the final report of the GÖG-LSE study
(please see Annex X).
The following table summarises the surveys and questionnaires supporting the Impact
Assessment
on
the
future
of
the
EU
HTA
cooperation.
206
207
CHAFEA Framework Contract 2016/Health/16
DG SANTE Contract 17010402/2016/734820
208
DG SANTE Contract 17010402/2016/736040
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Surveys and questionnaires supporting the Impact
Assessment on the future of the EU HTA cooperation
Name
Public consultation
By
DG SANTE
Aim
To gather
views and
opinions
related to
the future of the EU
cooperation on HTA,
as proposed in the
IIA. The results of this
public consultation
will feed into the
impact assessment
process
Timing of
survey
21/10/2016
-13/1/2017
Target group
All citizens and
organisations. Patient
organisations, public
authorities, HTA
bodies, payers,
companies developing
pharmaceuticals,
medical devices and
other technologies as
well as associations
representing
stakeholders. SME
outreach through EMA
SME office.
SMEs, outreach
through the Enterprise
Europe Network
national contact points
Two targeted surveys
1. Patient
organisations, public
authorities, HTA
bodies, payers
2. Technology
developers (industry)
Selected technology
developers (of the 40
technologies)
SME panel
DG
GROW/SAN
TE
Study on
the
Impacts
of Policy
Options
Survey
on the
Impacts
of Policy
Options
Austrian
Public
Health
Institute
(GÖG)
Case
studies
London
School of
Economics
(LSE)
HTA process
mapping study
Julia
Chamova
A simplified and
targeted version of
the open public
consultation for
SMEs
Gather
data and
evidence on the
impacts of the policy
options
per
stakeholder to
support the Impact
Assessment process
of the EC
Gather
evidence to
map the role of HTA
in market access
process through a
selection of 40
technologies (20
pharma, 15 med
tech, 5 other)
Map the HTA
processes in EU MS
and EEA
Map the HTA
methodologies in EU
MS and EEA
20/12/2016
-20/1/2017
18/12/2016
-
22/12/2017
3/1/2017-
31/1/2017
6/2016-
2/2017
HTA methodology
mapping study
Finn Børlum
Kristensen
7/2016-
2/2017
HTA bodies of MS and
EEA countries - survey
distributed via
EUnetHTA JA3 WP7
HTA bodies of MS and
EEA countries - survey
distributed via
EUnetHTA JA3 WP7
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Annex II. Stakeholder Consultation: Synopsis Report
1.
Introduction
The synopsis report documents all the consultation activities accompanying the Impact
Assessment on the initiative for strengthening EU cooperation on HTA.
The aim of the stakeholder consultation was to collect all stakeholders' views on the EU
cooperation on HTA, encompassing their experience with the on-going cooperation
mechanisms, their specific needs and their opinion on the proposed approaches described in
the Inception Impact Assessment.
For reaching all interested stakeholders and in order to ensure a high quality and balanced
input, a combination of consultation methods was used:
- The main channel to collect the opinions of stakeholders was the
open public consultation.
Besides the feedback received in response the publication of the Inception Impact
Assessment, DG SANTE launched a broad online public consultation to which all categories
of stakeholders provided input. In addition, position statements from different organisations
were received by email.
-
Bilateral meetings with interested stakeholder representatives
were organised to allow in-
depth discussion on specific topics and the expression of non-organized interests.
-
Experts
consultation
was carried out through the existing cooperation mechanisms,
EUnetHTA Joint Action and the HTA Network. Since these are the stakeholders who are
already participating in the cooperation and they will be the ones who continue cooperating
after 2020, these meetings allowed to openly discuss the options proposed in the Inception
Impact Assessment for the future EU cooperation in HTA.
- Presentations to
external events
were used to reach out to stakeholders, to explain the main
elements of the initiative, to invite them to participate in the public consultation, and listen to
their views and opinions.
A summary of all the above mentioned consultations is presented in the subsequent sections.
2.
2.1.
Open public stakeholder consultation
Feedback on the inception Impact Assessment
Following the publication of the Inception Impact Assessment on "Strengthening of the EU
cooperation on Health Technology Assessment (HTA)" on 14 September 2016
209
and up to
now, the Commission received a number of 9 positions and statements. Three were submitted
by national authorities, 4 by trade organisations, and 2 by industry
210
.
Most of the contributions were supportive, highlighting the need for continuing efforts to
facilitate EU cooperation on HTA, but also pointing out the challenges of the current
209
http://ec.europa.eu/smart-
regulation/roadmaps/docs/2016_sante_144_health_technology_assessments_en.pdf
210
https://ec.europa.eu/health/technology_assessment/consultations/cooperation_hta_en
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collaboration for the national uptake of the joint work. It was acknowledged that neither
option 1 nor 2 provide for appropriate means not only to preserve but also to further develop
achievements to date, whilst an EU legislative framework could enable a permanent,
systematic collaboration. In contrast, contributors emphasised that they are not supporting
joint work on full HTA reports (proposed as option 5) which was seen as having direct impact
on national pricing and reimbursement processes which are under Member States
competence. One national authority expressed reservations about the legal base of the
initiative, and reminded about the legal provisions in the Cross border Healthcare Directive
which limit EU cooperation on HTA to voluntary cooperation.
On the other hand, contributors representing medical technologies' industry were more
negative, calling for an in depth analysis of the medtech sector and expressing their concerns
that the particularities of their sector would not be taken into account. They also questioned
the potential added value of EU cooperation on HTA for medical technologies, arguing that
decision making is often local and that currently HTA is rarely used on these types of health
technologies. One of the contributors (i.e. manufacturer of medical devices) pointed out that
depending on the existing regulations in different Member States, HTA might become a
barrier to innovation and limit patient access to quality care. They were in favour of solutions
based on improved voluntary cooperation among HTA bodies.
2.2.
Online public consultation
2.2.1. Description of the questionnaires and of the respondents
The Commission launched an online public consultation which ran from 21 October 2016
until 13 January 2017
211
.
Due to the technical nature of health technology assessment, and in order to cover all
interested stakeholders, the online public consultation was carried out via two questionnaires.
One questionnaire was dedicated to citizens and was made available in all EU official
languages. A second one, available only in English was directed to administrations (both
public and private administrations with a public service obligation), economic stakeholders
(in particular pharmaceutical and medical technologies
212
' industry), as well as associations
and organisations representing stakeholders (e.g. patients and consumers, healthcare
providers, payers
213
, industry and service providers, academia and scientific societies). A
simplified version of the questionnaire dedicated to administrations, associations and
organisations, tailored for SMEs was circulated via the SME Network of DG GROW. This
questionnaire was also made available in all EU official languages.
Citizens' consultation
focused on their general awareness of HTA and national HTA systems,
EU cooperation on HTA, as well as usefulness and need to access HTA information by
patients, consumers and healthcare professionals. The
consultation of administrations,
211
212
https://ec.europa.eu/health/technology_assessment/consultations/cooperation_hta_en
Medical technology, or medtech, encompasses a wide range of healthcare products and is used to diagnose,
monitor or treat diseases or medical conditions affecting humans. In this report medical technologies refer to
medical devices and in vitro diagnostics (IVD).
213
For the purpose of this report, payers should be understood as insurance organisations or organisations
acting on behalf of a public authority responsible for the payment of healthcare services.
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economic stakeholders, associations and organisations
was directed to get their opinions on
the current state of play and EU cooperation on HTA, and on EU cooperation on HTA beyond
2020.
The online public consultation and the SME consultation gathered a total of 249 replies. Of
these responses, 25% (63 submissions) were from individuals/citizens and the rest from
administrations, economic stakeholders, associations or organisations ("non-individual
respondents") (75%, 186 submissions). Of the 186 non-individual contributions, 36 replies
were received in response to the questionnaire dedicated to SMEs distributed to the SME
Network in DG GROW. The distribution of responses per Member State and type of
organisation is shown below.
Profile of individual respondents
As regards the geographical distribution of individual responses, contributions from
citizens/individuals came from 21 EU Member States (62) and Switzerland (1). The highest
number of replies came from citizens in Germany and Netherlands (8/MS), followed by
Spain, France and Italy (6/MS), Portugal and United Kingdom (4/MS), Belgium and Sweden
(3/MS) and Greece, Ireland and Poland (2/MS). Only one reply came from citizens in Austria,
Bulgaria, Cyprus, Estonia, Finland, Malta, Romania and Slovakia.
Analysis of the information provided in relation to the level of education, work experience
and sector of employment showed that the large majority of the individual respondents are
well-educated, with expertise and work experience in either or both public and private sectors,
in areas relevant for this consultation (e.g. healthcare sector, HTA sector, public
administration, and health technologies 'industry). A large majority (78%) of the respondents
also indicate knowing how their national HTA system is organised and being aware of the
current EU cooperation on HTA (63%), confirming the contributors' interest and expertise in
this field.
Profile of respondents to the questionnaire dedicated to administrations, economic
stakeholders, associations and organisations
Concerning the contributions to the questionnaire dedicated to administrations, economic
stakeholders, associations and organisations, industry was the major contributor with 52 % of
all replies, followed by public administration (14%), patients and consumers associations
(13%) healthcare providers' organisations and scientific societies (13%) and payers (3%).
More details concerning the profile of each category of respondents are presented below:
- Concerning input from industry, most of the contributions were submitted by SMEs (46%)
followed by big commercial operators (27%), trade associations (26%) and consultancies
(1%). Additionally, most of the companies contributing to the public consultation are
European or international companies, active in more than one Member State or beyond the
EU (64%). A similar number of contributions were received from both pharmaceutical and
medical technologies' industry (32 and 35 contributions respectively).
- As regards public administrations, most of the contributions were provided by HTA bodies
(10 replies), as well as organisations with multiple responsibilities (8), Ministries of Health
(4), payers (1) and other national or regional organisations (3). Concerning the geographical
distribution of responses from public administrations, contributions came from 15 EU
Member States (Italy with 5 contributions, Germany, Finland and Spain with 3
contributions/Member State, Slovenia with 2 contributions, and Austria, Belgium, Czech
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Republic, Croatia, France, Hungary, Ireland, Poland, Portugal and United Kingdom with 1
contribution per country) and Norway (1 contribution).
- Patients and consumers were represented by an equal number of national (12 organisations)
and European patients' associations (12). Most of these associations (63%) acknowledged
their interest for both pharmaceuticals and medical technologies. Additionally 4% of these
organisations specified being interested in all health technologies.
- Healthcare providers were represented in the consultation by national associations (50%),
followed by European (31%) and regional organisations (19%). Fifty per cent of the
respondents in this category indicated representing hospitals and the rest provided input on
behalf of doctors, community pharmacists, optometrists and public health trusts.
- Respondents from academia were mostly European organisations (63%), but also national
and international ones (i.e. ISPOR).
- Payers were mostly represented by national associations (60%).
- The category "other" was selected by non-profit organisations promoting public health,
information on pharmaceuticals and therapeutic and diagnostic strategies, improved access to
medicines and their rational use, or the development of therapies in specific areas such as
cancer or regenerative medicine.
The results of the online public consultation were published in May 2017
214
and a summary is
included below.
2.2.2. Citizens opinions
Almost all individual respondents (98%) consider that it is useful to compare new health
technologies with existing ones and assess whether they work better, equally well or worse, in
order to provide guidance to decision makers. Citizens emphasised that patients should have
access to the best possible treatment, with the least possible cost, with HTA supporting
"rational decision making and control the health care budget". The usefulness of HTA as a
tool supporting disinvestment of obsolete technologies, allowing for and better allocation of
resources for truly innovative health technologies was also highlighted.
Almost all individual respondents confirmed the need to ensure key elements when carrying
out assessments: 1) transparency of HTA processes, 2) independence from industry or other
influences 3) appropriate expertise of the assessors, and last but not least 4) timely delivery of
assessments for informed decision making.
As regards the possibility of performing joint EU clinical and economic assessments, 57% of
the citizens indicated that national/regional HTA bodies should not perform clinical/medical
assessments of the same health technologies in parallel, independently from each other.
However, a similar percentage of the respondents (56%) were against carrying out EU joint
economic assessments.
The survey also showed that most individuals believe that HTA information should be easily
accessible to doctors to enable an informed decision when prescribing the treatment of their
patients (95%) and also to patients (84%). The involvement of relevant stakeholders and
representatives of the public (i.e. patients who can provide good understanding of the patients'
214
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point of view, especially on topics such as unmet needs, quality of life data and patients'
preferences) by HTA bodies when preparing and/or reviewing HTA reports was strongly
advocated.
2.2.3. Opinions from administrations, economic stakeholders, associations and organisations
Firstly the questionnaire aimed to verify whether the issues identified in the Inception Impact
Assessment are shared by stakeholders. In this respect, most of the respondents strongly
agreed or agreed with the existence of differences in HTA processes and methodologies (80%
confirmed the existence of different HTA methodologies for clinical assessments, 85%
acknowledged the differences in HTA methodologies for economic assessments, and 91%
agreed with the existence of differences in national HTA procedures).
Fig. 1. Overview of the opinions expressed by administrations, organisations and associations on the
existence of differences in HTA processes and methodologies across EU
Furthermore, the contributors to the public consultation confirmed that differences in HTA
processes and methodologies across the EU translate into diverging outcomes of HTA reports
which may affect patients' access to new technologies (e.g. delays, restricted access) (81% of
contributions), duplication of work for both HTA bodies and industry (54%), decrease in
business predictability (53%), higher costs for the actors (38%) and even affect innovation in
a negative way (37%).
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Fig. 2. Consequences of differences in HTA process and methodologies across EU as identified by
public administrations, organisations and associations
As regards the current EU cooperation on HTA, the consultation showed that 32% of the
respondents participated in EU-funded projects and joint actions and 47% of the contributors
state that even though they did not directly participate, they were aware of EU cooperation on
HTA. Whilst participation and awareness were relatively high among public administrations,
payers, industry, healthcare providers and academia, it was very low for SMEs. From the
respondents who confirmed their participation in or awareness of EU funded activities, 69%
considered EU cooperation on HTA useful or to some extent useful, with most benefit seen by
public administrations, payers and academia (100%) (Fig. 3). Among the most cited benefits
of the EU cooperation on HTA are sharing knowledge and best practices among participating
organisations, capacity building as well as increased trust between participants and increased
awareness on HTA issues (Fig. 4).
Fig.3. Usefulness of EU cooperation on HTA (i.e. EU-funded projects and joint actions) per category
of respondents
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Fig.4. Benefits of the current EU cooperation on HTA as reported by public administrations,
organisations and associations (Total number of replies = 157)
A negative opinion about the EU cooperation on HTA was reported by medical technologies
industry, SMEs, and a minority of respondents from the pharmaceutical industry (Fig. 3).
The survey showed that despite participation and/or awareness, the uptake of joint work
remained low, with significant variations in the estimations provided by different category of
respondents. Overall, less than 11% of the respondents estimated that joint tools, EUnetHTA
guidelines and joint early dialogues and joint reports (either REA or full HTA) were used to a
great extent and up to 51% estimated they were used to a limited extent (Fig. 5).
Fig. 5. Uptake of joint work from EU-funded projects and joint actions per type of joint activities
–
all
categories of respondents (Total number of replies = 150)
With regard to the future EU cooperation on HTA, a large majority of the respondents (87%)
consider that EU cooperation on HTA should continue beyond 2020 when EUnetHTA Joint
Action 3 will end.
Concerning the
scope
of the future cooperation, a large majority of the respondents found
useful and to some extent useful to continue EU cooperation on HTA in the field of
pharmaceuticals (80%), but also in the areas of medical technologies (72%) and other
technologies (54%) (Fig. 6).
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As regards the
policy options,
the questionnaire outlined three options with focus on the type
of participation (i.e. voluntary or mandatory) and uptake by participating Member States'
HTA bodies (i.e. voluntary or mandatory). In this regard, the "voluntary participation with
mandatory uptake option" was generally favoured, being at the same time the option with
overall lowest opposition and the highest percentage of neutral opinions. In contrast the
options voluntary participation with voluntary uptake and mandatory participation with
mandatory uptake registered a significantly higher opposition (50% or more) and less support.
(Fig. 7).
In relation to the potential
funding mechanisms
of the future EU cooperation on HTA, more
than half of the respondents (53%) pointed towards a mix of contributions from EU budget,
industry fees and Member States contributions (Fig. 8).
Fig. 6. Overview of the opinions regarding the
scope of EU cooperation on HTA beyond 2020Fig.
Fig.7. Overview of the opinions on the
policy options for continuing EU
cooperation on HTA
Fig. 8. Overview of the opinions on the funding
mechanism of the future EU cooperation on HTA
Fig.9. Overview of the opinions on the
governance mechanism of the future EU
cooperation on HTA
With respect to the
governance mechanism,
the consultation shows an overall preference
towards an existing EU agency, followed by the European Commission. Respondents
expressed similar preferences for a new EU agency, Member States HTA bodies on rotational
basis and other mechanisms, most of them consisting of elements of the current voluntary
cooperation and/or EMA-like models (Fig. 9).
Overall, the contributors to the online public consultation were positive in regards to the
future of EU cooperation on HTA beyond 2020, pointing out to the outputs responding to
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their needs and the benefits of harmonising the use of common tools, and of outputs such as
early dialogues and clinical assessments/REA. It was highlighted that in the process of
shaping the future EU cooperation on HTA, consideration should be given to the following
issues: distinguish between assessment and appraisal (the latter being the responsibility of
national health care services and local insurance bodies), clear separation between regulatory
assessment (which informs decision on marketing authorisation) and HTA (that informs
decisions on added value, use of technologies and reimbursement and pricing), step-wise
approach as potential key success factor, appropriate selection and prioritisation, focus on
selected technologies, clear and strong coordination/governance/secretarial support,
extension of the scope of early dialogues to guidelines on technology development, and
appropriate stakeholder involvement. Reduced duplication of efforts and costs, better
allocation of resources and last, but not least, the contribution to a faster and more equitable
access to new health technologies for EU patients were among the most quoted benefits.
However some categories of stakeholders, including medtech industry, SMEs and some
national authorities were mostly in favour of a voluntary cooperation.
3.
Bilateral meetings with stakeholders
Commission services organised senior level meetings with 10 Member States' Ministries of
Health and HTA bodies interested in receiving clarifications and providing an early input to
the initiative. Bilateral meetings took place at the request of all interested stakeholders:
payers (5 meetings), patients' and consumers' representatives (8 meetings), with healthcare
providers and academia representatives (8 meetings), as well as with industry representatives
and their trade associations (20 meetings)
215
.
4.
Experts Consultation
Following the publication of the Inception Impact Assessment, several meetings with Member
States HTA experts were organised. Two closed sessions with Member States representatives
to HTA Network took place on 10 November 2016
216
and 23 March 2017
217
. In addition,
brainstorming sessions with members of the EUnetHTA Executive Board were also organised
in December 2016, March and May 2017. These discussions were open and constructive, and
showed engagement by HTA Network members and EUnetHTA experts to contribute to the
development of the policy initiative.
5. External events
The initiative was presented during several meetings such as the EUnetHTA Forum (Brussels,
October 2016), the annual meeting of the International Society for Pharmacoeconomics and
Outcomes Research/ISPOR (Vienna, October 2016), and the European Health Forum Gastein
(Bad Gastein, September 2016), which allowed constructive discussions with stakeholders
(i.e. HTA experts, healthcare professionals, public health specialists, patients representatives,
industry representatives) and represented good opportunities for raising awareness about the
Commission's online public consultation.
215
216
Minutes available at
https://ec.europa.eu/health/technology_assessment/events_en#anchor3
https://ec.europa.eu/health/sites/health/files/technology_assessment/docs/ev_20161110_mi_en.pdf
217
https://ec.europa.eu/health/sites/health/files/technology_assessment/docs/ev_20170329_mi_en.pdf
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6. Conclusions
The consultation activities showed that there is broad support for the initiative and a high
level of stakeholder interest in concrete implementation of a pragmatic solution, from which
will benefit not only public authorities or industry but also patients and healthcare
professionals.
A large majority of stakeholders emphasise that EU cooperation beyond 2020 is needed to
ensure a constant exchange of information and knowledge between HTA institutions in
Europe, to increase synergies between Member States, to streamline HTA methodologies, to
increase transparency and evidence-based decision making, as well as business predictability.
The possibility to access a larger number of HTA reports with less duplication of work and
better allocation of resources by HTA bodies was highlighted. Patients' organisations,
healthcare professionals and academia stress that EU cooperation can enhance access to added
value and affordable technologies in a timely manner and in the long run can also lead to
savings, improving resilience and contributing to the sustainability of health systems. Several
stakeholders note that significant public resources have been invested in EU cooperation on
HTA and all the results achieved so far should be capitalised to support sustainable healthcare
systems and guarantee equitable access to technologies with added value to all patients in
Europe.
While all representatives of public administrations are in favour of continuing EU cooperation
on HTA beyond 2020, some indicate a preference for voluntary cooperation while others
support a system with mandatory elements (i.e. legal framework for EU cooperation on HTA
to streamline interoperability of HTA national systems). Most contributors highlight that in
case of a mandatory system, uptake of joint work should be limited to clinical and technical
matters, whereas assessment of non-clinical domains (e.g. economic, legal, ethical) should be
carried out individually or jointly by interested Member States/HTA bodies on a voluntary
basis. The idea of a phase-in approach was also raised.
Citizens, patients and consumers representatives, as well as healthcare providers and
academia were extremely positive, with most of them in favour of a collaboration covering
both clinical and economic assessments. They underline the need for involving patients and
healthcare professionals in the HTA process, the need of transparency (e.g. summary of HTA
reports publicly available, including criteria and rationale for evaluation), and last but not
least the need to ensure independence of HTA bodies from industry and other interests.
As regards industry, pharmaceutical industry and their trade associations support the
harmonisation of European relative efficacy assessments at time of launch, accompanied by
an alignment at EU level of the evidence requirements between regulators, HTA bodies and
payers. Many representatives of the pharmaceutical industry advocate for voluntary
participation for both Member States and manufacturers until the process of joint work has
proven itself, however with mandatory uptake of joint work. It was stressed that economic
assessments should remain the responsibility of Member States. Medical technologies'
operators and their trade associations reiterate the importance of taking into account the
particularities of their sector and the need for a Member States-driven approach (i.e. timing
and selection of technologies to be assessed should be decided by HTA bodies and not
centrally at EU level). It was underlined that HTA should focus on products that are
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innovative and address high unmet patient needs in disease areas where appropriate clinical
and economic evidence has been or can be generated (e.g. transformative in-vitro diagnostics
and medical devices).
These results are fully taken into account in the proposed preferred option presented in the
Impact Assessment, particularly with regard to:
- subsidiarity (i.e. focus on joint early dialogues and joint clinical assessments, with economic
assessments to be performed at national level), and
- proportionality (i.e. national uptake understood as implementation of joint work within
national HTA processes, addressing the differences between the pharmaceuticals and the
medical technologies sector in relation to the different market access path and the role HTA
plays in the two sectors, progressive implementation of the product scope).
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Annex III. Who is Affected by the Initiative
The aim of this annex is to set out the
practical implications of the preferred policy option
for the main stakeholders affected by it.
Who is
affected?
Public
administrations
How are they affected?
Main positive outcomes include:
Better evidence for national decision-makers
(i.e. due to high quality
and timely joint reports) to support sustainability of national health
systems and foster public health. Member States with less HTA
capabilities and higher pressure on their healthcare budgets will
particularly benefit from such evidence. Furthermore, focusing joint
reports on clinical data makes them relevant to all decision-makers,
without affecting national competences on pricing and reimbursement
decisions.
Cost savings and optimisation of resources.
The foreseen work sharing
is expected to result in cost savings for public administrations in the long
run, also allowing for a better allocation of resources.
Pooling expertise
and
enhanced capacity
to address more health
technologies. HTA bodies in the EU will be able to specialise in different
topics, rather than to keep a general profile of both their tasks and staff.
Therefore, the existing staff is expected to specialise (e.g. orphan
medicines, medical devices), developing complementary expertise which
would ensure the desired high quality of joint REA reports.
The introduction of an EU system may be accompanied by an
initial increase in
costs (mainly human resources)
related to the initial implementation of new
procedures and methodologies at national level. However, this is expected to be
compensated by the work-sharing arrangements and avoidance of duplication
foreseen under the preferred policy option. Member States' HTA bodies carrying
out the joint assessments as authors and co-authors would be remunerated for
their work. The HTA bodies which will not be actively participating as authors
and co-authors will benefit from the work produced by the EU system and could
therefore save the relevant resources that they would have had to invest.
This is expected to counterbalance the initial increased spending due to the
adaptation to the new EU system.
Patients
An EU HTA system would provide for a framework for the
involvement
of patients in the HTA processes at national and EU level
(i.e.
common procedures for involving patients, financial support to cover
participation of patients to the meeting of the expert Committees carrying
out the joint work).
The publication of the joint assessment/REA reports will also increase
the
transparency
in of decision-making in relation to the availability of
health technologies and the consistency of the clinical HTA assessments
across the EU as they will be based on common procedures and
methodologies.
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Healthcare
professional
and providers
Pharmaceutical
industry
An EU HTA system would provide for a framework for their
involvement in the HTA process
(i.e. common procedures for involving
healthcare professional and providers).
Additionally, the publication of the joint assessment/REA reports would
facilitate access to reliable, timely and objective information on medical
technologies allowing for
better informed decisions
on the best
treatment for their patients.
The introduction of a joint REA with mandatory uptake will be accompanied by
positive economic impacts for the pharmaceutical industry:
Improved business predictability.
A high quality and timely joint
assessment report should reduce divergent decisions related to access on
the market of new innovative technologies.
Potential positive impact on time to market.
A high quality and timely
joint assessment report has the potential to accelerate the next steps to be
carried out at national level (i.e. appraisal, pricing and reimbursement).
In this regard, quick first access to markets is particularly relevant for
SMEs
which depend on first access to first revenues.
It will
reduce duplication of work
through harmonisation of tools and
methodologies and one submission for joint early dialogues/joint REA.
A more predictable HTA system has the potential to
increase
investments in R&D activities in Europe.
Regarding
costs,
in an initial phase the introduction of joint REA at the time of
market authorisation is expected to lead to certain costs associated with the
adaptation to the new EU system. However, the implementation of the product
scope in a progressive manner during the first years of the new EU system is
expected to allow a
smoother transition to the new system.
Additionally, while the duplication of certain tasks/requirements for the staff of
pharmaceutical companies in EU Member States will be reduced, the costs
associated with national pricing and reimbursement procedures will persist.
Overall, according to the GÖG-LSE study the, the pharmaceutical industry does
not expect
any significant savings,
but the gains in terms of business
predictability are expected to overcome the costs related to the implementation of
an EU system.
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Medical
technologies
industry
Cost calculations estimate that reduction of duplications could lead to tangible
savings for the medical devices industry.
When representatives of this sector expressed the opinion that
no positive
economic impact
if there is any obligatory REA at the time of CE marking,
many assumed that HTA would become mandatory for all type of medical
technologies at the time of market launch.
It is important to highlight that the preferred option
foresees joint REAs only for
medical technologies which have undergone the scrutiny mechanism in the
context of their conformity assessment
and which in addition have been
selected by Member States for a joint REA based on specific criteria (e.g. EU
wide added value, potential impact of the technology on patients, public health
and healthcare systems across the EU).
Additionally, taking into account the more decentralised market access pathway
for medical technologies,
the timing of the joint REA will not be linked to the
timing of the conformity assessment,
i.e. it will not necessarily be at the time of
market launch. This will avoid putting an additional burden on the manufacturers
at market launch.
Contrary to what is foreseen for pharmaceuticals, for which the product scope is
expected to be fully covered over time (all pharmaceuticals identified in the
scope are expected to go through a joint REA at the end of transitional
arrangements), for medical technologies the system will remain based on a
prioritisation mechanism to ensure that joint REA are only performed on medical
technologies in which Member States identify that EU cooperation brings an
added value.
Overall, a predictable HTA system is expected to re-direct medtech industry
resources towards development of and investment in products which would for
instance address unmet medical needs and lead to the improvement of health
outcomes for patients.
SMEs
In the area of pharmaceuticals SMEs are mostly engaged in the discovery phase
of new molecules, and a very low number apply for central marketing
authorisation and undergo HTA process. The number of applications for joint
REA from SMEs is expected to be very low, and since no fees are foreseen for
this type of joint outputs, the compliance costs are expected to be low. As regards
joint early dialogues, no fees will be foreseen; therefore this should become a
very advantageous service provided to SMEs developing products in the area of
pharmaceuticals.
A similar treatment would be applied for SMEs in the field of medical
technologies (no fee in case of voluntary application for a joint early dialogues,
no compliance fee in case of a joint REA). SMEs manufacturing medical
technologies which will have to undergo the scrutiny mechanism in the context
of their conformity assessment are expected to benefit from the improved
convergence in procedures and methodologies and reduced duplications/parallel
REA.
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Annex IV. Analytical Models
To support the analysis of the impact assessment three studies were contracted. Firstly, two
smaller studies were launched: 1) Mapping of HTA national organisations, programmes and
processes in EU and Norway
218
, and 2) Mapping of HTA methodologies in EU and
Norway
219
. Thereafter, a study with a larger scope was launched with the following
objectives:
To collect data and evidence and provide an in-depth analysis of what would happen
in the absence of further action at EU level including its impacts (baseline scenario);
To collect data and evidence and provide analysis on the potential impacts of
identified policy options for cooperation of the European Commission;
To collect relevant literature on HTA, with a specific focus on the European Union, to
understand the way how it is used across EU Member States (MS).
The study has been performed by a consortia consisting of SOGETI Luxembourg S.A., the
Austrian Public Health Institute (GÖG) and the London School of Economics (LSE)
(henceforth the GÖG-LSE study).
In the GÖG-LSE study, to establish the baseline scenario, a case study comprising a product
sample of health technologies was analysed which included
20 pharmaceuticals, 15 medical
devices
and 5 “other technologies”
(including complex health interventions). The study team
collected detailed information on the HTA-process each technology underwent in Member
States. The case study also analysed the influence of the regulatory framework on technology
developers. In addition, the costs of performing a HTA were identified for both, the
technology developer and the HTA body through desk research and an online survey
disseminated to all stakeholder groups, where mainly public administrations and health
technology developers responded.
To analyse possible impacts of identified policy options of the European Commission, a
survey was performed on the economic and social impacts of the identified policy options,
complemented by focus groups, interviews and findings from literature review. The study
investigated the following impacts, for which one or more indicators were defined:
The impacts investigated included economic (EC) and social health (SH) criteria:
Mapping of HTA national organisations, programmes and processes in EU and Norway, 2017, DG SANTE
Contract 17010402/2016/734820 (Annex VIII)
219
Mapping of HTA methodologies in EU and Norway, 2017, DG SANTE Contract 17010402/2016/736040
(Annex IX)
218
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EC1: Costs
EC2: Administrative Burden
EC3: Competitiveness of EU health technology
sector
EC4: Innovation and Research
EC5: International Trade Innovation and
Research
EC6: Functioning of the internal market and
competition
EC7: Consumers and households
EC8: Macroeconomic environment
SH1: Employment
SH2: Governance, participation and
good administration
SH3: Access to social protection and
health systems
SH4: Sustainability of Health Systems
SH5: Public Health
*Environmental impacts were considered not to be relevant to the analysis at an early stage.
The study also provides a description of the implementation mechanisms and an estimation of
the costs based on data gathered from desk research (including data from existing agencies),
and responses in relation to the online survey.
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Annex V: Health Technology Sectors
In this Impact Assessment, the term health technology is understood in a broad sense,
comprising pharmaceuticals, medical technologies (medical devices and in vitro
diagnostics)
220
and other technology-based tools for disease prevention, diagnosis or treatment
used in healthcare.
This annex provides a description of the pharmaceutical and the medical technologies sectors
as the two key health technology sectors, by giving an overview of the size of each sector, the
actors, products and regulatory framework. Both sectors are highly innovative and play an
important role in the European growth and competitiveness.
The pharmaceutical sector is
characterised by stronger concentration of actors compared to the medical technologies,
where
95% of the companies are small and medium enterprises (SMEs). The regulatory
approval process and market access path (including pricing and reimbursement decision) for
pharmaceuticals is more centralised. The medical technologies sector is more heterogeneous
in terms of the products and the market access path. When a pharmaceutical arrives on the
market after a lengthy, costly and risky research and development process it is protected by a
patent. This is different for medical technologies, where a quick innovation cycle can be
observed due to the rather short lifecycle of products (18-24 months). Last but not least, the
Health Technology Assessment methodologies have been primarily developed for assessing
pharmaceuticals; addressing the particular methodological challenges relevant to the medical
technologies sector is still on-going.
A simplified diagram showing the HTA step within the lifecycle of pharmaceuticals and
medical devices in the context of the EU legal framework is presented below. As illustrated
below, HTA typically takes place following pharmaceutical market authorisation or CE
marking under the medical devices legislation. Enhanced cooperation on HTA as suggested in
this impact assessment is fully coherent with the legislation on clinical trials (Regulation (EU)
536/2014), pharmaceuticals (Regulation (EC) No 726/2004), medical devices and in vitro
diagnostics (Regulations (EU) 2017/745 and 2017/746) and the Transparency Directive
Council Directive 89/105/EEC).
220
Medical devices and in vitro diagnostics as defined by Regulation (EU) 2017/745 and Regulation (EU)
2017/746.
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Pharmaceuticals
Medical devices and in vitro diagnostics
Figure 1. HTA step within the lifecycle of pharmaceuticals and medical devices
I. Pharmaceutical Industry
1. Sector overview
The pharmaceutical sector in the EU employs approximately 800,000 people. It is one of the
industries with the highest labour productivity. The research and development investment is
high in the sector, thus it plays an important role in the European competitiveness.
221
The pharmaceutical sector is characterized by a strong R&D activity and a high level of
regulation. On the supply side, the market is divided between
originator companies
222
and
generic manufacturers.
The life cycle of a drug can be divided into 3 phases:
1) From R&D to market launch
2) Time on market under patent protection
3) Expiration of patent and access to the market of the generic drug.
1.1. Actors
The following table shows the top 10 pharma companies in million EUR 2016.
COMPANY
Pfizer
Novartis
Roche
Merk&Co
Sanofi
Gilead Sciences
Johnson & Johnson
GlaxoSmithKline
AstraZeneca
Abbvie
REVENUE
$ 43 112
$ 42 467
$ 38 733
$ 35 244
$ 34 896
$ 32 151
$ 29 864
$27 051
$23 264
$ 22 724
221
222
Pharmaceutical Industry: A Strategic Sector for the European Economy. SWD(2014) 216 final/2
Companies active in research, development, production and marketing of products protected by patents.
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1.2. Products
223
All new medicines introduced into the market are the result of lengthy, costly and risky
research and development (R&D) conducted by pharmaceutical companies:
o
By the time a medicinal product reaches the market, an average of 12–13 years would
have passed since the first synthesis of the new active substance;
o
The cost of researching and developing a new chemical or biological entity is
estimated at € 1 926 million ($ 2 558 million
in year 2013) in 2016 (DiMasi et al,
Journal of Health Economics, January 2016);
o
On average, only one to two of every 10 000 substances synthesised in laboratories
will successfully pass all stages of development required to become a marketable
medicine.
2. The Regulatory Framework
Medicinal products are (largely) regulated at EU-level throughout their lifecycle with the dual
objectives of facilitating their free movement within the single market and ensuring a high
level of public health protection. Directive 2001/83/EC and Regulation (EC) No 726/2004
are considered the core pieces of legislation covering the placing on the market, production,
labelling, classification, distribution and advertising of medicinal products for human use.
They are supplemented by a number of other Acts which focus on a particular step in the
product lifecycle or on specific types of medicinal products. The regulatory framework for the
main steps in this lifecycle is outlined below with a particular focus on marketing
authorisation procedures due to their links with HTA.
2.1 Clinical Trials
Once the initial research phase on a prospective new medicine has been completed, such
products need to undergo clinical trials, which are governed by Regulation (EU) 536/2014
(which replaces the former Directive 2001/20/EC). The clinical trials legislation lays down
rules on the protection of patients, informed consent, the manufacturing and clinical practice
of those medicines to be used in clinical trials, and the authorisation of trials and the
information available on them. The regulation is designed to ensure a streamlined application
procedure with defined deadlines. An EU clinical portal will provide for a single entry point
for sponsors and a single contact point by Member States. When making an application a
single set of documents will be needed and the applications will be assessed based on the new
harmonised assessment procedure consisting of two parts
–
a joint assessment by all Member
States concerned and a second part of separate assessments by those Member States. These
new rules will become applicable once the new portal and database run by the European
Medicines Agency are fully functional, which is expected in 2018.
223
EFPIA Key data 2016. Link: the-pharmaceutical-industry-in-figures-2016.pdf
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2.2 Marketing Authorisation Procedures
According to the rules governing medicinal products in the EU, such products may only be
placed on the market once a marketing authorisation has been issued, either by a Member
State authority or at Union level. These authorisations are granted based on an assessment of
the quality, safety and efficacy of the product in question and, in addition to a purely national
authorisation limited to one Member State, can be issued through one of three distinct
procedures:
1. Centralised procedure;
2. Mutual recognition procedure;
3. Decentralised procedure.
Whichever procedure is used the application is made using the Common Technical Document
consisting of the information on quality, non-clinical data, and clinical data. In addition to
these standard procedures it should be pointed out that special rules exist for the authorisation
of medicinal products for paediatric use, orphan drugs, traditional and herbal medicinal
products, and homeopathic medicinal products which provide for simplified rules for the
authorisation of such products.
Centralised procedure
The majority of new innovative medicines in the EU are authorised via the centralised
procedure. This procedure is governed by Regulation (EC) No 726/2004 and allows
applicants to obtain a marketing authorisation that is valid throughout the EU. It is
compulsory for medicinal products manufactured using biotechnological processes, for
orphan medicinal products and for human products containing a new active substance which
was not authorised in the EU before 20 May 2004 and which are intended for the treatment of
AIDS, cancer, neurodegenerative disorder or diabetes. The centralised procedure is optional
for any other products containing new active substances not authorised before 20 May 2004
or for products which constitute a significant therapeutic, scientific or technical innovation or
for which a central authorisation is in the interests of patients or animal health at Union level.
When a company wishes to place on the market a medicinal product that is eligible for the
centralised procedure, it sends an application directly to the European Medicines Agency, to
be assessed by the Committee for Medicinal Products for Human Use (CHMP). The
procedure results in a Commission Decision, which is valid in all EU Member States.
Centrally authorised products may be marketed in all Member States. Full copies of the
marketing authorisation application file are sent to a rapporteur and a co-rapporteur
designated by the competent EMA scientific committee. They co-ordinate the EMA's
assessment of the medicinal product and prepare draft reports. Once the draft reports are
prepared, they are sent to the CHMP, whose comments or objections are communicated to the
applicant. The rapporteur is therefore the privileged interlocutor of the applicant and
continues to play this role, even after the marketing authorisation has been granted.
The rapporteur and co-rapporteur then assess the applicant's replies, submit them for
discussion to the CHMP and, taking into account the conclusions of this debate, prepare a
final assessment report. Once the evaluation is completed, the CHMP gives a favourable or
unfavourable opinion as to whether to grant the authorisation. When the opinion is favourable,
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it shall include the draft summary of the product's characteristics, the package leaflet and the
texts proposed for the various packaging materials.
The time limit for the evaluation procedure is 210 days. The EMA then has fifteen days to
forward its opinion to the Commission. This is the start of the second phase of the procedure:
the
decision-making process.
The Agency sends to the Commission its opinion and
assessment report, together with annexes containing:
the summary of product characteristics (Annex 1);
the particulars of the manufacturing authorisation holder responsible for batch release,
the particulars of and the manufacturer of the biological active substance and the
conditions of the marketing authorisation (Annex 2); and
the labelling and the package leaflet (Annex 3).
During the decision-making process, the Commission services verify that the marketing
authorisation complies with Union law. The Commission has fifteen days to prepare a draft
decision. The medicinal product is assigned a Community registration number, which will be
placed on its packaging if the marketing authorisation is granted.
The draft decision is then sent to the Standing Committee on Medicinal Products for Human
Use. Member States have fifteen days to return their linguistic comments and 22 days for
scientific and technical ones. This procedure is conducted in writing but if a duly justified
objection is raised by one or more Member States, the committee holds a plenary meeting to
discuss it. When the opinion is favourable, the draft decision is adopted via the
empowerment
procedure.
The Commission then notifies the Commission Decision to the marketing
authorisation holder. The decision is then published in the
Community Register.
Marketing
authorisations are valid for five years. Applications for renewal must be made to the EMA at
least six months before this five-year period expires.
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Fig. 2. Centralised procedure
Mutual recognition procedure
As laid down in Directive 2001/83/EC, the mutual recognition procedure is compulsory for all
medicinal products to be marketed in a Member State other than that in which they were first
authorised. Any national marketing authorisation granted by an EU Member State's national
authority can be used to support an application for its mutual recognition by other Member
States.
The mutual recognition procedure is based on the principle of the mutual recognition by EU
Member States of their respective national marketing authorisations. An application for
mutual recognition may be addressed to one or more Member States. The applications
submitted must be identical and all Member States must be notified of them. As soon as one
Member State decides to evaluate the medicinal product (at which point it becomes the
"Reference Member State"), it notifies this decision to other Member States (which then
become the "Concerned Member States"), to whom applications have also been submitted.
Concerned Member States will then suspend their own evaluations, and await the Reference
Member State's decision on the product.
This evaluation procedure undertaken by the Reference Member State may take up to 210
days, and ends with the granting of a marketing authorisation in that Member State. It can also
occur that a marketing authorisation had already been granted by the Reference Member
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State. In such a case, it shall update the existing assessment report in 90 days. As soon as the
assessment is completed, copies of this report are sent to all Member States, together with the
approved summary of product characteristics (SPC), labelling and package leaflet. The
Concerned Member States then have 90 days to recognise the decision of the Reference
Member State and the SPC, labelling and package leaflet as approved by it. National
marketing authorisations shall be granted within 30 days after acknowledgement of the
agreement.
Should any Member State refuse to recognise the original national authorisation, on the
grounds of potential serious risk to public health, the issue will be referred to the coordination
group. Within a timeframe of 60 days, Member States shall, within the coordination group,
make all efforts to reach a consensus. In case this fails, the procedure is submitted to the
appropriate EMA scientific committee (CHMP or CVMP, as appropriate), for arbitration. The
opinion of the EMA Committee is then forwarded to the Commission, for the start of the
decision making process (described below). As in the centralised procedure, this process
entails consulting various Commission departments and the Standing Committee on Human
Medicinal Products.
Decentralised procedure
Introduced by Regulation (EC) No 726/2004, the decentralised procedure is similar to the
mutual recognition procedure in terms of procedural steps. The key difference is that there is
no existing marketing authorisation in any Member State before an application is made. An
identical application for marketing authorisation is submitted simultaneously to the competent
authorities of the Reference Member State and of the Concerned Member States. At the end
of the procedure, the draft assessment report, SPC, labelling and package leaflet, as proposed
by the Reference Member State, are approved. The subsequent steps are identical to the
mutual recognition procedure.
2.3 Manufacturing and distribution
Once a marketing authorisation has been granted, companies then require a manufacturing
authorisation before medicinal products can be manufactured in the Union based on the rules
laid down in Directive 2001/83/EC as well as the detailed rules on Good Manufacturing
Practice (GMP) set out in Directive 2003/94/EC. Once a manufacturer has made an
application, an inspection is carried out prior to the national competent authority issuing a
GMP certificate. Applicants are assessed on the suitability of their premises, their technical
equipment and control facilities, staff, and compliance with GMP including the use of active
substances complying with GMP. A manufacturing authorisation is also necessary for
companies importing medicinal products into the EU from third countries.
Distribution of medicinal products is governed by the rules on Good Distribution Practice
(GDP). A distribution authorisation is necessary in order to distribute medicinal products.
This allows either manufacturers to distribute products themselves or to sell products to those
in possession of a distribution authorisation. In turn distribution authorisation holders can sell
medicinal products to entities entitled by the MS to sell medicinal products to the public. The
retail of medicinal products is regulated at Member State level.
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2.4 Pharmacovigilance
Once medicinal products have been placed on the EU market pharmacovigilance is used to
ensure that the safety of medicinal products is fully monitored and that action is taken to
reduce the risks and increase the benefits of such products. The system requires data from
healthcare professionals working with medicinal products to be collected, managed, and
evaluated and decisions taken where necessary to protect public health. When granting a
marketing authorisation, certain conditions may be made requiring the collection of further
data post-authorisation, particularly on the efficacy of a product. Where safety issues do arise
this can result in further action being taken including the suspension, withdrawal, revocation
or non-renewal of the marketing authorisation. The basic rules and requirements for
pharmacovigilance are laid down in directive 2001/83/EC and Regulation (EC) No 726.2004.
2.5 Market access
In most Member States, pricing and reimbursement decisions for pharmaceuticals are taken at
national level at the time of market launch (or shortly thereafter). While pricing and
reimbursement are a Member States competence, the Transparency Directive (Council
Directive 89/105/EEC) defines procedural requirements to ensure the transparency of these
processes for pharmaceuticals, including the setting of timeframes for decision-making.
It is important to note that HTA does not comprise pricing and reimbursement decisions.
Rather, the role of HTA is to provide a scientific assessment to inform evidence-based
decision-making on pricing and reimbursement. In line with this distinction, the principle of
subsidiarity and Article 168(7) TFEU, decisions on pricing and reimbursement are not within
the scope of the initiative analysed in this impact assessment. Nevertheless, strengthened EU
cooperation on HTA can be considered coherent with the objectives of the Transparency
Directive in terms of supporting timely and transparent decision-making by Member States
224
.
II. Medical Technology Industry
1. Sector Overview
There are about 25 000 medical technology companies in Europe. About 95% of them are
Small and Medium-sized Enterprises (SMEs). The market is estimated to employ about 575
000 people. Total sales amount to EUR 100 billion per year, of which 8 to 10% are invested
in R&D activities
225
. On average, the European medical technology market has been growing
by 4% per annum over the past seven years. Demand fell in 2009 due to the global economic
crisis, resulting in a growth rate of only 1% in that year. The market recovered in 2010, but
growth rates fell back in 2011
226
. The sector is expected to grow, given the technological
Note that this relates in particular to supporting decision-making on clinical aspects of HTA (compare policy
option 3 and 4).
225
Medical devices, DG GROW
https://ec.europa.eu/growth/sectors/medical-devices_en
226
WHO; G. Donahoe and G. King
–
Estimates of Medical Device Spending in the United States, F.S.A.,
M.A.A.A., AdvaMed, 2012
224
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advancements and improvements in healthcare, combined with the steady increase in life
expectancy rates over the last decades.
Two main underlying characteristics of the industry is - a high variety of products and a quick
innovation cycle
227
. There are more than 500, 000 medical technologies currently available on
the market. Additionally, on average, products have a lifecycle of between 18 and 24 months
before an improved product enters the market, which is a strong driver for research and
innovation.
228
1.1. Actors
Most of the 25 000 medical technology companies in Europe are based in Germany, the UK,
Italy, Switzerland, Spain and France
229
.
The following table shows the top 10 medical technology companies in 2015 in the world.
COMPANY
230
1 Medtronic
2 Johnson & Johnson (medical device
segment)
3 Philips Healthcare (Royal Philips
Electronics)
4 GE Healthcare(General Electric)
5 Fresenius (medical care segment)
6 Siemens Healthineers (Siemens)
7 Cardinal Health (medical segment)
8 Becton, Dickinson (medical segment)
9 Baxter (medical products segment)
10 Stryker
1.2. Products
A distinct characteristic of the medical technology industry is the wide variety of products
covered by it. There are more than 500,000 registered medical devices
–
from syringes,
surgical kits and hip replacements, to pacemakers, in vitro diagnostic devices and
radiotherapy units.
The rather high level of R&D activities in the field results in a constant flow of innovations in
the sector. Accordingly, the highest number of patents filed to the European Patent Office
(EPO) in 2015 came from the medical technology industry (7.8%)
–
more than the ones in
2015 REVENUE
$28,833,000,000
$25,137,000,000
$19,817,952,415
$17,639,000,000
$16,739,425,600
$11,652,847,873
$11,395,000,000
$10,282,000,000
$9,968,000,000
$9,946,000,000
MedTech Europe. The European Medical Technology industry in figures. 2016
The European Medical Technology Industry
–
in figures 2016
229
Ibid.
230
Medical Design & Outsourcing annual look at the world’s 100 largest medical technology companies 2016
-
2016 Top 100 Medical devices companies.pdf
228
227
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digital and computer communication.
231
The number of medical devices receiving CE marking
in 2015 is estimated to be around 4,500.
232
1.3 Classification of Products
The European classification depends on rules that involve the medical device's duration of
body contact, invasive character, use of an energy source, effect on the central circulation or
nervous system, diagnostic impact, or incorporation of a medicinal product.
The initial classification of medical devices in the EU is set out in Article IX of Council
Directive 93/42/EEC. Essentially, they fall under four categories: non-invasive devices,
invasive medical devices, actives devices and, last, but least, special rules, which include
contraceptive, disinfectant, and radiological diagnostic medical devices). Additionally,
medical devices are further segmented into classes. While there is a separate classification
scheme for IVDs and implantable devices do not follow the same classification system as
provided by the Medical Devices Directive, they are subject to similar requirements as Class
III. There are three groups covering from low to high risk devices:
Class I
–
Provided non-sterile or do not have a measuring function (low risk)
Class I
–
Provided sterile and/or have a measuring function (low/medium risk)
Class IIa (medium risk)
Class IIb (medium/high risk)
Class III (high risk)
2. Regulatory Framework
The existing regulatory framework for the medical devices dates back to the 1990s. The three
Directives (Council Directive 90/385/EEC, Council Directive 93/42/EEC and Council
Directive 98/79/EC) have been recently revised, as an array with issues with divergences in
the interpretations and applications of the regulations, the technological progress, along with
some incidents which involved malfunctions of medical devices highlighted the need for a
review.
In April 2017, two legislative proposals on medical and in-vitro diagnostic Regulations were
adopted, replacing the existing Directives. Firstly, it is Regulation (EU) 2017/745 on medical
devices, amending Directive 2001/83/EC, Regulation (EC) No 178/2002 and Regulation (EC)
No 1223/2009 and repealing Council Directives 90/385/EEC and 93/42/EEC. Secondly, it is
Regulation (EU) 2017/746 on in vitro diagnostic medical devices repealing Directive
98/79/EC and Commission Decision 2010/227/EU.
The new regulation was designed to ensure a high level of consistency of health and safety
protection for EU citizens using medical devices. Moreover, the legislation safeguards the
free trade of products throughout the Union, and responds to the significant scientific and
technological developments, which have occurred in the sector over the last two decades.
231
232
MedTech Europe. The European Medical Technology industry in figures. 2016
MedTech Europe. The European Medical Technology industry in figures. 2016
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The new regulations include a number of improvements to modernise the existing regulatory
system. These improvements include:
Provision for a stricter ex-ante control for high-risk devices through a new pre-market
scrutiny mechanism;
A reinforcement of the criteria for designation and processes for oversight of Notified
Bodies;
inclusion of some aesthetic devices which, under the scope of these Regulations,
present the same characteristics and risk profile as analogous medical devices;
introduction of a new risk classification system for in vitro diagnostic medical devices;
improved transparency is assured through the establishment of a comprehensive EU
database on medical devices; along with this a device traceability system is also
introduced based on Unique Device Identification;
an "implant card" containing information about implanted medical devices for a
patient;
reinforcement of the rules on clinical evidence, including an EU-wide coordinated
procedure for authorisation of multi-centre clinical investigations;
strengthening of post-market surveillance requirements for manufacturers;
last, but not least, an improved coordination mechanisms between EU countries in the
fields of vigilance and market surveillance.
These new rules will only enter into force after three years for the Regulation on medical
devices (spring 2020) and after five years for the Regulation on in vitro diagnostic medical
devices (spring 2022).
2.1 Post-market surveillance
The enforcement of the harmonised legislation on medical devices is the responsibility of the
relevant authorities in the Member States. They are expected to gather record and analyse
relevant data on the quality, performance and safety of devices. The necessary conclusions
need to be drawn, and any preventive and corrective actions have to be determined.
2.2 Market surveillance
The new regulations provide clearer rights and obligation of market surveillance authorities,
along with clearer procedures for national provisional measures. Member States are
anticipated to exchange mutual information and control.
2.3 Vigilance
The new regulations provide the establishment of an EU vigilance portal. Additionally,
serious incidents are expected to be centrally reported. Following such cases, field safety
corrective actions are expected to be taken. Moreover, trend reporting and enhanced
coordination between authorities are expected to take place under the new regulations.
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2.4 Market Access
The market access process presents a more heterogeneous picture for medical technologies,
compared to the pharmaceutical products. Health Technology Assessments for medical
technologies are currently carried out less frequently and on a smaller, less centralised scale
(See
Fig. 3).
The pricing and reimbursement judgement is typically taken in a decentralised
manner, through local (hospital level) decisions or procurement, with limited input from HTA
reports developed on a national, or centralised, level. Public procurement (instead of a central
decision on reimbursement) is often the final step of the market access path.
3. Other considerations
For medical technologies, a number of issues should be taken into account when considering
HTA:
Heterogeneity of the products, including multiple uses for the same device.
Incremental innovation and a short lifecycle. The quick, incremental innovations,
which can take place within 18-24 month on the medical technologies, may affect the
efficacy and cost of the device.
Learning curve of the device user (patient or health professional) means that the
outcomes are less favourable during the period of training.
Wider economic and organisational implications, such as required trainings,
operational costs or savings due to shorter hospitalisation.
Availability of evidence; randomised controlled trials can be more challenging to
design, due to the learning curve, difficulties in blinding or randomisation.
For IVDs, there are two further challenges. Firstly, the value of improved diagnosis
cannot be separated from the value of the treatment determined by the diagnosis.
Secondly, technologies may have multiple uses.
233 234 235
Such methodological challenges were pointed out in a number of submissions in the public
consultation (e.g. COCIR, Siemens, etc.)
Rummel, P, Hawlik, K, Wild, C. Health Technology Assessments on Medical Devices in Europe. Final
Report. LBI-HTA Rapid Assessment Nr. 12; 2016. Vienna: Ludwig Boltzmann Institute for Health Technology
Assessment.
234
Drummond, M., Griffin, A. and Tarricone, R. (2009), Economic Evaluation for Devices and Drugs—Same or
Different?, Value in Health, 12: 402–404.
235
MedTech and COCIR contributions to the open public consultation
233
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Figure 3. Schematic market access process of health technologies
140
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Annex VI. European Cooperation on HTA
The EU Cooperation on Health Technology Assessment (HTA) has had a long history, from
the first main steps in the beginning of the 1990's through different networking and
coordination projects to the largest ever Joint Action funded by the Public Health Programme,
the EUnetHTA Joint Action 3. To date, over EUR 50 million will have been invested in the
EU cooperation jointly by Member States and the EU by 2020. This Annex summarises the
main elements of the projects and cooperation conducted so far.
PUBLIC HEALTH PROGRAMME
EUR-ASSESS (1994-1997)
ECHTA-ECHAI (1999-2001)
EUnetHTA project (2006-2008)
EUnetHTA Joint Action 1 (2010-2012)
EUnetHTA Joint Action 2 (2012-2015)
EUnetHTA Joint Action 3 (2016-2020)
FP 7 RESEARCH PROGRAMME
AdhopHTA (7th Framework Programme)
MedtecHTA (7th Framework Programme)
Integrate-HTA (7th Framework Programme)
Advance-HTA (7th Framework Programme)
INNOVATIVE MEDICINES INITIATIVE
ADAPT SMART (Innovative Medicines Initiative)
GET REAL (Innovative Medicines Initiative)
PUBLIC HEALTH PROGRAMME
EUR-ASSESS (1994-1997)
Between 1994 and 1997 the EUR-ASSESS project took place with the following aims: to
improve methods of priority setting, to develop and formulate HTA methodologies, to ensure
that effective dissemination strategies were being used throughout European agencies, and to
improve decision making by stimulating wider use of technology assessments.
All the then 15 Member States and Switzerland took part in the project and it was concluded
that there was a need to promote the field of HTA, improve its use and impact in decision
making and that further EU cooperation was needed. One identified example of the need of
further cooperation was duplication of effort; where it was exemplified that certain
technologies could have up to 10 different assessments with limited coordination between
different HTA agencies.
The European Collaboration for Assessment of Health Interventions-Health Technology
Assessment (ECHTA/ ECAHI) (1999-2001)
Following the EUR-ASSESS, with the ECHTA/ECAHI (1999-2001) project, the ambition
level increased further. The project assembled 6 different working groups on different aspects
of HTA and possible ways of cooperation. The project had the following aims:
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o
To assess health promotion and disease prevention activities in terms of
benefits, risks and economic, social and ethical implications as a complement
to community health indicators.
o
To develop systems for routine exchange of information between programmes
on:
Emerging technology issues
Priorities for future evaluation
Conduct and timing of ongoing evaluations, including findings from
evaluations.
o
To identify possible joint assessments and to co-ordinate findings and existing
resources within the community to support joint assessments.
o
To develop and disseminate best practice in undertaking and reporting
assessments. To identify needs for methodological development.
o
To develop and co-ordinate education and support networks for individuals
and organisations undertaking or using assessment of health interventions. To
identify needs in the field and assist in the establishment of new provisions.
o
To identify and share successful approaches to link findings of assessments,
their contribution to health indicators and health care decision-making.
It was concluded that a more sustainable network was needed to support the EU cooperation
on HTA and that project based solutions would not be adequate to proceed further. Therefore,
there was a call for a permanent coordination secretariat funded by the European Commission
with a caveat that it should respect the subsidiarity principle.
EUnetHTA project (2006-2008)
The project was led by the National Board of Health of Denmark, Danish Centre for HTA.
The EUnetHTA network collaboration was initiated in 2006 with the EUnetHTA project to
connect public national health technology assessment (HTA) agencies, research institutions
and health ministries, enabling an affective exchange of information and support to policy
decisions by Member States. The concept of the (paper based) HTA Core Model and the
framework for producing and sharing structured HTA information were developed.
EUnetHTA Joint Action 1 (2010-2012)
The EUnetHTA Joint Action 1 (JA1) was led by Danish Health and Medicines Authority
(DHMA) and involved 33 collaborating partners from 26 EU Member States, Norway and
Croatia. The budget of JA1 was 6 million EUR. EUnetHTA JA1 built on the methods and
tools developed by the earlier EUnetHTA project (2006-2008) and EUnetHTA Collaboration
2009. JA1 was a voluntary, time-limited initiative with a defined work plan.
The JA1 developed a background review and a HTA Core Model for rapid Relative
Effectiveness Assessment of pharmaceuticals,. It also developed the POP (Planned and On-
going Projects) database which in 2011 turned into an online tool. The JA also collaborated
with EMA on the improvement of European Public Assessment Reports (EPARs), which are
the full scientific assessment report published by EMA for every medicine granted a central
marketing authorisation. The JA also signed a Memorandum of Understanding with
INAHTA, the International Network of Agencies for Health Technology Assessment, which
links 52 Member agencies working with HTA, in 29 countries.
EUnetHTA Joint Action 2 (2012-2015)
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EUnetHTA Joint Action 2 (JA2) was led by the National Board of Health of Denmark,
Danish Centre for HTA and involved 69 organisations in 31 countries across Europe. A total
number of 49 government-appointed organisations from 28 EU Member States and Norway
participated in the work. The budget of the JA2 was 9,5 million EUR.
The JA2 tested the tools and methodologies developed in the first EUnetHTA JAs through the
cross-border HTA pilots. The aim was to generate evidence on the costs, quality and
feasibility of European cooperation as applied to concrete assessment projects.
Through joint work the partners produced approximately 20 joint reports, including REA
(focusing on the clinical/therapeutic added value) and Full HTA (including assessment of
economic and organisational aspects). The cooperation also facilitated over 20 early dialogues
between technology developers and HTA bodies, which help industry to design the studies in
terms of regulatory and HTA requirements.
EUnetHTA Joint Action 3 (2016-2020)
236
The EUnetHTA Joint Action 3 (JA3) with a budget of 20 million EUR is led by the Dutch
National Healthcare Institute (ZIN). JA3 comprises around 80 HTA bodies from all Member
States, Norway and Switzerland. The challenges have been the setup of a new coordinating
office and the involvement of many new organisations (80 versus 69 in previous Joint
Actions).
Under JA3 further progress is expected from the previous Joint Actions since the
collaboration foresees 80 joint reports and 35 early dialogues by 2020. Increased uptake of the
joint work at national level is also an important aim of the Joint Action. In addition, JA3 will
also perform a revision of current guidelines, models, methodologies and other tools, as well
as the development of new ones, with the aim of facilitating HTA collaboration at EU level
beyond the end of the project in 2020.
FP 7 FRAMEWORK PROGRAMME (2007-2013)
Through the FP7 work programme (2007-2013) managed by DG RTD, several projects were
launched focusing on developing methodologies, tools and guidelines on different topics
related to HTA which started 2013 and were finalised in 2015. These were:
AdHopHTA
–
Adopting hospital-based Health Technology Assessment in Europe
237
The project AdHopHTA aimed to bolster the use and impact of high-quality health
technology assessment (HTA) in hospital settings. The ultimate goal was to facilitate the
adoption of health technologies with proven value in hospitals and to keep costly pseudo-
innovations without proven benefit at bay.
AdHopHTA findings and results have been collected in a “handbook for hospital-based
HTA
(HB-HTA)”
that includes a set of guiding principles for good practices in HB-HTA
units. The
handbook has an accompanying toolkit to put it into practice. The project has developed as
well a database of hospital-based HTA reports produced by the project partners.
236
237
http://www.eunethta.eu/
http://www.adhophta.eu/
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INTEGRATE-HTA
238
The INTEGRATE-HTA project developed conceptual and methodological guidance for a
comprehensive, patient–centered, and integrated assessment of complex technologies. The
assessment comprises effectiveness as well as economic, socio-cultural, ethical, and legal
factors and takes into account patient-specific factors, context, and implementation issues. Its
starting point is the perspective of the stakeholders involved, palliative care has been chosen
as a case study.
MedtecHTA
239
The general objective of MedtecHTA was to identify improvements of HTA methods by
making evaluation of medical devices more comprehensive and by acknowledging
complexities rising from their integration into clinical practice. The project aims at filling the
gap on the current research debate on the challenges to the available methodological
framework for HTA when applied to medical devices.
Advance HTA
240
The objective of ADVANCE-HTA project was to contribute to the advancement of HTA by
addressing areas of intense methodological debate (value for money, value assessment,
quality of life measurement, rare and orphan diseases, HTA for selected medical devices,
HTA in emerging settings). Ultimately, it aims to bring about improvement in HTA methods
which can be taken further by competent authorities at national and supra-national levels
towards a common understanding of choices in healthcare decision-making.
INNOVATIVE MEDICINES INITIATIVE (IMI)
The Innovative Medicines Initiative (IMI) is Europe's largest public-private partnership with a
€2 billion budget funded equally between the European Commission and industry, aiming to
improve the drug development process by supporting a more efficient discovery and
development of better and safer medicines for patients. IMI is since 2015 undertaking two
projects relevant to the EU cooperation on HTA.
GetReal
241
GetReal aims to show how robust new methods of Real World Evidence (RWE) collection
and synthesis could be developed and considered for adoption earlier in pharmaceutical R&D
and the healthcare decision making process. This will require companies, healthcare decision
makers and other stakeholders to work together to generate a consensus on best practice in the
use of RWE in regulatory and reimbursement decision-making.
It is expected that alternative evidence generating strategies will deliver more focused
research in pharmaceutical research and development , and allow healthcare decision makers
to be more certain when providing patients with access to new treatments. GetReal is carrying
out work to develop intelligence, evidence, tools, techniques and training to realise the full
potential of RWE.
238
239
http://www.integrate-hta.eu/
http://www.medtechta.eu/wps/wcm/connect/site/medtechta/home
240
http://www.advance-hta.eu/
241
http://www.imi-getreal.eu/
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Accelerated Development of Appropriate Patient Therapies (AdaptSMART)
242
AdaptSMART aims to establish a platform for coordinating activities related to 'Medicines
Adaptive Pathways to Patients' (MAPPs) and enable stakeholder dialogue in this field. The
main aims of the project are the following:
Identify the scientific challenges and opportunities related to the implementation of
MAPPs and foster the aligned understanding of consortium members and their
constituents.
Support new IMI2 research and innovation actions by facilitating the inclusion of
MAPPs enablers, tools and methodologies to address its challenges and opportunities.
Conduct horizon scanning and research on key topics to produce actionable advice and
recommendations for IMI and other stakeholders to further the broad implementation
and adoption of MAPPs.
Distribute findings, key discoveries and case studies from ongoing or completed
MAPPs pilot projects, creating a MAPPs repository of knowledge and opportunities.
242
http://adaptsmart.eu/
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Annex VII: International Outlook
While Europe is generally considered a leading example in applying HTA in decision-
making, it is interesting to note developments in other parts of the world.
Most developed and many developing countries are implementing HTA systems. Most OECD
countries have national agencies responsible for HTA, although they have varying capacities,
institutional settings, scope and mandates. In the majority of cases, HTA is used to inform
coverage and pricing decisions. In this regard, when it comes to pharmaceuticals, HTA
agencies only provide scientific assessment, while the particular government, third-party
payers or joint associations of medical bodies make the final coverage and pricing
decisions
243
.
HTA is often, yet not always used for medical technologies in OECD states. Depending on
Structural Policy Country Notes: Medium-term Policy Challenges; Southeast Asian Economic Outlook 2013,
OECD
https://www.oecd.org/dev/49954247.pdf
243
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the type of medical devices, HTA is used to inform coverage decisions relative to either the
medical device itself (e.g. implantable devices) or to diagnostic and therapeutic procedures
using the device (e.g. imaging or surgery). According to a recent study, carried out by the
OECD
244
, two-thirds of its member countries use HTA to make decisions on devices or
interventions, whether systematically or in "some circumstances".
Looking at particular countries,
Canada
is one to offer a positive international example in the
realm of HTA. The country, which has federal structure, consists of three territories and 10
provinces. Before the 2000s the HTA processes in Canada were decentralised
–
each
jurisdiction was performing its own HTA. This led to significant discrepancies between the
sheer numbers and types of drugs assessed in the different parts of the country. In an attempt
to reduce the differences between the healthcare services Canadians were receiving based on
where they lived, Canadian Agency for Drugs and Technologies in Health (CADTH) was set
up
245246
. It is a national organisation, where federal, provincial and territorial healthcare
assessments are made. Quebec is the only territory which is does not participate in CADTH
due to constitutional provisions. Consequently, Canada managed to reduce the divergences
between the different provinces. In brief, the move towards harmonisation in Canada has met
its objective to a large degree thanks to the establishment of the Canadian Agency for Drugs
and Technologies in Health and by establishing common HTAs.
Australia
has also a well-established HTA system comparable to the ones in place in several
Member States, i.e. based on independent body providing recommendations to the pricing and
reimbursement authority. The assessment of pharmaceutical and other technologies is
conducted by a specialised agency, the Medical Services Advisory Committee, and,
increasingly by insurers.
247
It is interesting to note that also Australia regularly undertakes a
review of its HTA system to reduce duplication and fragmentation of the processes which
have been identified as having cost and time implications for economic stakeholders, patients
and administrations.
248
In
USA,
due to the very different nature of healthcare system, based mainly on many private
payers providing care to its members, HTA is equally fragmented. Providers of care may
perform directly of via academic institutions, evidence reviews or comparative analysis,
Structural Policy Country Notes: Medium-term Policy Challenges; Southeast Asian Economic Outlook 2013,
OECD
https://www.oecd.org/dev/49954247.pdf
245
CADTH website accessed 27 July 2017:
https://www.cadth.ca/about-cadth/who-we-are/history
246
Science-Matrix, Evalution of CADTH, December 2016 :
https://www.cadth.ca/sites/default/files/pdf/2016%20CADTH%20Evaluation.pdf
247
Healy J, Sharman E, Lokuge B. Australia: Health system review.
Health Systems in Transition
2006; 8(5): 1–
158.
248
Australian Government-Productivity Commission (2015) Efficiency in Health. Available at:
http://www.pc.gov.au/research/completed/efficiency-health/efficiency-health.pdf
244
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however the systems is not really comparable with the ones in place in most of European
countries
249
.
In
South Korea,
HTAs were first introduced in the 1990s within the National Health
Insurance (NHI). Swiftly increasing expenditures for healthcare have been a challenge of the
NHI, which considered health technology management as a cost controlling measure.
Currently, the HTA process is governed by a governmental committee within the Ministry of
Health, Welfare and Family Affairs (MIHWFA). It comprises of twenty members who
technically supported by the HTA centre created within the National Health Insurance
structure. The institutionalisation of HTA in South Korea has been driven mainly by the
requirements of the NHI. It has manifested both strengths as well as weaknesses
250
. Most
recently the South Korean government is establishing a new organization for HTA,
independent from the NHI.
Another proof for the worldwide interest in HTA has been established by
international
scientific societies,
such as ISPOR (International Society for Pharmaceutical and Outcomes
Research) or HTAi (Health Technology
Assessment international). They work to improve the
quality of HTAs and their role in decision-making.
249 European Obervatory on Health Systems and Policy (2013) United States of America. Health system review.
Vol. 15 No. 3 2013. Available at:
http://www.euro.who.int/__data/assets/pdf_file/0019/215155/HiT-United-
States-of-America.pdf
250 Health Technology Assessment in South Korea, Chang-yup Kim (2009) International Journal of Technology
Assessment in Health Care, 25:Supplement 1, 219-223 https://www.cambridge.org/core/services/aop-cambridge-
core/content/view/S0266462309090667
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Annex VIII. Earlier Market Access Calculations
One of the proposed policy options (PO 4) in the Inception Impact Assessment on Strengthening the
EU cooperation on Health Technology Assessment foresees a EU 'joint REA' for all innovative
pharmaceuticals, which is available at the time of the marketing authorisation by EMA. This joint
REA has the potential to speed up the market access process for innovative pharmaceuticals. The
extent of this time gains have been estimated at 2-6 weeks by the CRA/EFPIA study
251
. The following
calculation aims to estimate the monetary gains from the industry's perspective of this earlier access.
Methods:
The calculation was done for a cohort of pharmaceuticals that were launched in quarter 2 2008
The
total turnover at manufacturer price newly launched non-generic products
(13
pharmaceuticals) was extracted from IMS aggregated across all available countries (covering most of
the EEA) and retail/hospital sectors for Q1 2008- Q3 2016 (column 'current revenues'). The cohort
was defined as non-generic medicinal products for which no sales data were reported in the first
quarter of 2008 and sales data were reported for all subsequent quarters. This cohort concerns 13
medicinal products (as distinguished in the data-set by their international product name). All 13
products are originator products (no biological products were present in this cohort).
The hypothetical revenues of a 1 month quicker market access was calculated (column `1 month
quicker market access revenues') for the first 12 years following market launch. It can be assumed
after 12 years the concerned product markets would be genericised. Revenues for the first 8.5 years
(34 quarters) were directly taken from the IMS database. For the remaining 3.5 years (14 quarters) it
was assumed revenues had plateaued at the observed level of the 34
th
quarter following market launch
(available data confirm that revenues had stabilized after 7 years at an aggregate turnover of around
EUR 400 million). Revenue flows were discounted to their 2008 quarter 1 value assuming an assumed
(annual) 2% inflation rate. A sensitivity analysis with 1% discount rate gave similar results (0.97 %
instead of 1.06%).
It was assumed that such an earlier HTA report does not affect the length of the subsequent steps
(appraisal, pricing negotiations, etc.).
Results:
For the selected products launched in one quarter, the one month earlier access means
EUR
130.315.010
additional revenue over the 12 years; which is a
1 % increase.
A similar gain can be
expected for all innovative products launched.
Calculations:
EFPIA/ Charles River Associates, 2017, Assessing the wider benefits of the EU’s proposal on strengthening
cooperation on health technology assessment from the industry perspective
251
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1 month
quicker
market access
Year
Quarter Current revenues revenues
0
0
0
2.508.758
1
1
7.489.107
13.688.977
1
2
25.959.881
33.207.704
1
3
47.467.771
51.566.109
1
4
59.467.651
64.219.113
2
5
73.357.966
78.720.897
2
6
89.005.032
94.878.120
2
7
106.097.741
111.107.492
2
8
120.528.816
125.103.809
3
9
133.590.794
149.952.111
3
10
181.772.621
193.075.711
3
11
214.616.761
226.704.152
3
12
249.639.987
251.761.445
4
13
254.740.105
255.008.146
4
14
254.282.245
258.785.786
4
15
266.470.396
247.667.150
4
16
209.023.290
215.210.439
5
17
226.460.826
234.655.215
5
18
249.804.230
274.252.199
5
19
321.552.295
318.990.281
5
20
312.316.250
314.258.464
6
21
316.571.768
317.637.486
6
22
318.189.769
325.051.358
6
23
337.101.525
338.531.931
6
24
339.706.802
334.925.638
7
25
323.756.529
329.688.632
7
26
339.866.107
335.953.804
7
27
326.508.758
327.923.923
7
28
329.120.849
333.386.936
8
29
340.230.571
340.792.139
8
30
340.226.754
344.143.985
8
31
350.240.229
343.587.787
8
32
328.651.826
336.365.295
9
33
350.054.936
350.874.201
9
34
350.771.875
350.771.875
9
35
349.039.615
349.039.615
9
36
347.315.910
347.315.910
10
37
345.600.717
345.600.717
10
38
343.893.995
343.893.995
10
39
342.195.701
342.195.701
10
40
340.505.794
340.505.794
11
41
338.824.233
338.824.233
11
42
337.150.976
337.150.976
11
43
335.485.982
335.485.982
11
44
333.829.210
333.829.210
12
45
332.180.620
332.180.620
12
46
330.540.172
330.540.172
12
47
328.907.825
328.907.825
12
48
327.283.539
327.283.539
total
12.827.396.352 12.957.711.362
difference between current and earlier revenues
130.315.010
percentage
101,016%
150
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1852047_0152.png
Annex IX. Implementation Mechanisms and Policy Options
For the five policy options, the following five implementation mechanisms were considered.
Project-based coordination
Member States (MS) secretariat
European Commission (EC) secretariat
Existing EU agency
New EU Agency
In one instance, for Policy Option 2 Project-based cooperation on HTA activities, only one
implementation mechanism was suitable, the project-based cooperation.
For the policy options foreseeing a permanent mechanism (PO 3-5), more than one
implementation mechanism is conceivable. Still even for these options, the implementation
mechanisms are not freely interchangeable. For instance, more centralised production of the
joint output requires more centralised structure. Also, policy options requiring the work 15-35
full time staff cannot be hosted by an independent agency. The following table shows the
conceivable pairing of the implementation mechanism per policy option, based on input from
Member States experts, stakeholder consultation, LSE-GOG study and experience with
EUnetHTA Joint Actions.
Scoring table
–
Selection of governance mechanisms per policy option
(based on input
from MS experts, stakeholder consultation, LSE-GÖG study and experience with EUnetHTA
Joint Actions)
Project-
based
coordination
PO2
PO3
PO4
PO5
Observations
Suitable
implementation
mechanism for
voluntary
cooperation
Suitable for EU
cooperation
limited to some
joint outputs,
however with
challenges
related to the
selection of the
MS hosting the
secretariat and
sustainability of
financing
Suitable for EU
cooperation
encompassing
most types of
joint outputs (i.e.
common tools
and
methodologies,
horizon
scanning, ED,
REA) especially
in case of a
phase-in
approach
Suitable for EU
cooperation
encompassing
most types of
joint outputs (i.e.
common tools
and
methodologies,
horizon
scanning, ED,
REA)
Suitable for EU
cooperation
encompassing
all type of joint
outputs,
including Full
HTA, for which
no existing EU
agency has
appropriate
expertise
MS
secretariat
EC
secretariat
Existing
EU agency
New EU
Agency
151
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Legend
Most adequate
Not adequate
For the sake of calculations and presentation of the findings, the study paired each policy
option with one implementation mechanism.
152
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1852047_0154.png
Annex X. Costs of Joint Outputs and Overall Costs per Policy Option and
Implementation Mechanisms
Table 1. Costs of joint outputs per POs and implementation mechanism (Amount in 1000 EUR)
(Adapted from tables 20, 53 and 56 in the GÖG-LSE Study)
Type of Costs
Costs of joint outputs per POs and implementation mechanism (Amount x1000
EUR)
PO2
PO3
MS
secretariat
(cat 1 MS)
EC
secretaria
t
PO4
Implementation
mechanisms
Project-
based
coordinati
on
15 FTEs
13 ED
11-15 REA
EC
secretaria
t
Existing
EU
agency
35.5 FTEs
40 ED
65 REA
New
EU Agency
Staff
Estimated Joint outputs
14 FTEs
40 ED
Uptake of joint outputs
Costs for Common tools,
templates and
methodologies
(Maintenance)
Costs for Common tools,
templates and
methodologies
(Development)
Costs for Joint Early
Dialogues
Costs for Joint REA
Costs for Joint full HTA
Total costs of joint
outputs
voluntary
mandatory
mandatory
210.0
Included in implementation mechanism
300.0
300.0
300.0
596.3
1,598.3
N/R
1,834.8
N/R
N/R
1,834.8
6,821.6
N/R
2,704.6
2,134.8
8,956.4
153
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1852047_0155.png
Table 2. Overall costs per POs and implementation mechanism (Amount x1000 EUR) (Adapted from
tables 20, 52, 53 and 56 in the GÖG-LSE Study)
Overall costs per POs and implementation mechanism (Amount x1000 EUR)
PO2
PO3
PO4
Implementation
mechanisms
Project-
based
coordinati
on
15 FTEs
13 ED
11-15 REA
MS
secretariat
(cat 1 MS)
EC
secretaria
t
EC
secretariat
Existing
EU agency
35.5 FTEs
40 ED
65 REA
New
EU Agency
Staff
Estimated Joint outputs
14 FTEs
40 ED
Uptake of joint outputs
Type of costs
voluntary
mandatory
mandatory
N/R
Start-up costs
Costs running secretariat
Costs joint outputs
Total costs
(Running + joint outputs)
Fees foreseen (100% of
costs of EDs, to be paid by
industry )
Total costs by EU
2,614.5
2,704.6
5,319.10
-596.3
4,722.80
N/R
N/R
N/R
N/R
3,683
2,134.8
5817.8
0
5817.8
3,101
2,134.8
5235.8
0
5235.80
6,926.9
8,956.4
15,883.3
0
15,883.3
8,210
8,956.4
17,166.4
-1,834.80
15,331.60
+ 5-10% of
running costs
in first years
8,210
8,956.4
17,166.4
-1,834.80
15,331.60
154
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1852047_0156.png
Annex XI. Implementation Tables for Preferred Policy Option
Table 1. ESTIMATED HR IMPACTS AND COSTS PER YEAR OF THE
PREFERRED POLICY OPTION (EUR x1000)
–
Comparison of the preferred
governance arrangement (EC Secretariat) with a secretariat run by an EU Agency
Preferred option
–
FULLY OPERATIONAL
Staff
Estimated Joint outputs
65 Joint clinical assessments/REA per year
EC
secretariat
Start-up costs
Costs running secretariat
(staff, premises, IT,
meetings + overheads)
Costs joint outputs*
(joint early dialogues,
clinical
assessments,
horizon scanning)
Total costs
16,000
(Running + joint outputs)
Fees foreseen (100% of costs of
0
EDs, to be paid by industry )
Total costs by EU
16,000
0
17,200
17,200
joint
joint
expert
9,000
9,000
NA
7,000
NA
8,200
Existing
EU agency
35 FTEs
40 Joint early dialogues (ED) per year
Most feasible in an initial phase
*The costs of joint outputs refers mainly to the remuneration of an author and a co-author
HTA body from the Committees dedicated to carry out joint work (e.g. Committee for joint
REA, Committee for joint early dialogues). In special situations more than 2 authors could be
envisaged.
155
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1852047_0157.png
Table 2. Estimated staffing, workload and costs/year for the progressive
implementation of the preferred policy option
Type of costs
EC Secretariat - YEARS 1-5
% of costs from a fully operational system
YEARS
FTEs (details in Table
3)
YEAR 1
12
4
ADMIN/AST
(EC)
8
SCIENTISTS/
SPECIALIST
S (Seconded
national
experts/EC
staff with
scientific
profile)
YEAR 2
15
4 ADMIN/AST
(EC)
11
SCIENTISTS/
SPECIALISTS
(Seconded
national
experts/EC
staff with
scientific
profile)
YEAR 3
22
5 ADMIN/AST
(EC)
17
SCIENTISTS/
SPECIALISTS
(Seconded
national
experts/EC
staff with
scientific
profile)
YEAR 4
30
8 ADMIN/AST
(EC)
22
SCIENTISTS/
SPECIALISTS
(Seconded
national
experts/EC
staff with
scientific
profile)
YEAR 5
35
10
ADMIN/AST
(EC)
25
SCIENTISTS/
SPECIALIST
S (Seconded
national
experts/EC
staff with
scientific
profile)
Costs
running
secretariat
(% from
total running costs of a
fully
operational
secretariat,
million
EUR)
Staff + premises
Expert
Committee
- meetings
CG meetings
(nr meetings,
million
EUR)
- Joint Committee
meetings
(nr meetings,
million
EUR)
- WG meetings
(nr meetings,
million
EUR)
30%
2.10
40%
2.80
60%
4.20
80%
5.60
100%
7.00
1.60
0.50
2
0.12
10
0.30
8
0.08
2.00
0.80
3
0.18
15
0.45
17
0.17
3.00
1.20
4
0.24
25
0.75
21
0.21
4.10
1.50
4
0.24
35
1.05
21
0.21
4.80
2.20
4
0.24
52
1.56
40
0.40
Costs joint outputs*
#
(% from total costs of
joint outputs of a fully
operational secretariat,
million EUR)
Joint
early
dialogues/ED
(number, costs in
million EUR)
Joint
clinical
assessments/REA
(number, costs in
million EUR)
20%
1.80
30%
2.70
50%
4.50
70%
6.30
100%
9.00
10 ED
0.46
14 ED
0.64
20 ED
0.92
30 ED
1.38
40 ED
1.84
10 REA
1.10
16 REA
1.82
30 REA
3.30
43 REA
4.73
65 REA
7.00
156
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1852047_0158.png
Joint
horizon
scanning,
SOPs,
guidelines (number,
costs in
million EUR)
0.24
0.24
0.24
0.19
0.16
Total costs by EU
(million EUR)
3.90
5.50
8.70
11.90
16.00
*Costs of joint outputs include the remuneration for the Member States bodies carrying out the joint work as authors and co-
authors.
1 Coordination Group meeting = EUR 60,000
1 Joint Committee meeting = EUR 30,000
1 Working Group meeting = EUR 10,000
# As regards scope, health technologies to be assessed will be prioritised from the already limited scope (i.e.
centrally
authorised pharmaceuticals
–
new active substances and extension of indication;
medical technologies
- technologies which
have undergone the scrutiny mechanism in the context of their conformity assessment) according to agreed criteria (e.g.
unmet medical needs; potential impact on patients, public health, or healthcare systems; significant cross-border dimension;
major Union-wide added value).
Table 3. EC secretariat - Staff numbers and profiles for the progressive implementation
of the preferred policy option
YEAR
S
FTEs
YEAR 1
12
4 ADMIN/AST
(EC*)
8 SCIENTISTS/
SPECIALISTS
(Seconded national
experts**/EC staff
with scientific
profile*)
Head (1 FTE)
Administrative
support (3 FTE)
o
Head
of
administration
(1 FTE)
o
Project
Manager
(1
FTE)
o
Administrativ
e staff (1 FTE)
Scientific/technic
al support (5
FTE)
o
Head (1 FTE)
o
Scientific
officers
(2
FTE)
o
Methodology,
guidelines,
templates (1
FTE)
o
Administrativ
e staff (1 FTE)
IT support (3
FTE)
o
Internal
support l (1
YEAR 2
15
4 ADMIN/AST
(EC*)
11 SCIENTISTS/
SPECIALISTS
(Seconded national
experts**/EC staff
with scientific
profile*)
Head (1 FTE)
Administrative
support (4 FTE)
o
Head
of
administration
(1 FTE)
o
Project
Manager
(1
FTE)
o
Administrativ
e staff (2 FTE)
Scientific/technic
al support (7
FTE)
o
Head (1 FTE)
o
Scientific
officers
(3
FTE)
o
Methodology,
guidelines,
templates
(2FTE)
o
Administrativ
e staff (1 FTE)
IT support (3
FTE)
o
Internal
support l (1
YEAR 3
22
5 ADMIN/AST
(EC*)
17 SCIENTISTS/
SPECIALISTS
(Seconded national
experts**/EC staff
with scientific
profile*)
Head (1 FTE)
Administrative
support (6 FTE)
o
Head
of
administration
(1 FTE)
o
Project
Manager
(2
FTE)
o
Administrative
staff (3 FTE)
Scientific/technical
support (12 FTE)
o
Head (1 FTE)
o
Scientific
officers (5 FTE)
o
Methodology,
guidelines,
templates
(3
FTE)
o
Administrative
staff (3 FTE)
IT support (3
FTE)
o
Internal support
l (1 FTE)
o
Maintenance of
tools
and
YEAR 4
30
8 ADMIN/AST
(EC*)
22 SCIENTISTS/
SPECIALISTS
(Seconded national
experts**/EC staff
with scientific
profile*)
Head (1 FTE)
Administrative
support (8 FTE)
o
Head
of
administration
(1 FTE)
o
Project
Manager
(3
FTE)
o
Administrativ
e staff (4 FTE)
Scientific/technic
al support ( 17
FTE)
o
Head (1 FTE)
o
Scientific
officers
(8
FTE)
o
Methodology,
guidelines,
templates (3
FTE)
o
Administrativ
e staff (5 FTE)
IT support (4
FTE)
o
Internal
support l (2
YEAR 5
35
10 ADMIN/AST
(EC)
25 SCIENTISTS/
SPECIALISTS
(Seconded national
experts**/EC staff
with scientific
profile*)
Head (1 FTE)
Administrative
support (11 FTE)
o
Head
of
administration
(1 FTE)
o
Project
Manager
(5
FTE)
o
Administrative
staff (5 FTE)
Scientific/technica
l support (19
FTE)
o
Head (1 FTE)
o
Scientific
officers
(10
FTE)
o
Methodology,
guidelines,
templates
(3
FTE)
o
Administrative
staff (5 FTE)
IT support (4
FTE)
o
Internal
support l (2
Profil
es
157
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1852047_0159.png
FTE)
o
Maintenance
of tools and
databases (2
FTE)
FTE)
o
Maintenance
of tools and
databases (2
FTE)
databases
FTE)
(2
FTE)
o
Maintenance
of tools and
databases (2
FTE)
FTE)
o
Maintenance of
tools
and
databases
(2
FTE)
1FTE
EUR 138,000 (EUR 115,000 staff related costs + EUR 23,000 costs related to premises)
*Scientific profiles for both European Commission staff and national experts include:
pharmacists, pharmacologists, biologists, medical doctors, experts in biotechnology, engineers
with expertise in the development of medical devices and in vitro diagnostics, statisticians,
legal experts, researchers in the field of health technologies.
As regards the European Commission, DG JRC, DG SANTE, DG RTD, DG CNECT have
staff with appropriate scientific profiles.
**Seconded national experts are national civil servants or persons employed in the public
sector who are working temporarily for an EU Institution. They remain in the service of that
employer throughout the period of secondment and receive a daily allowance from the
European Commission in line with the provisions in the Staff Regulation.
ESTIMATIONS
Data for the estimation of the running costs: Study on Impact Analysis of Policy Options for
Strengthened EU Cooperation on HTA. 2017. Sogeti, Austrian Public Health Institute,
London School of Economics. (CHAFEA/2016/Health/16)
The running costs of an EU Agency are based on data from the European Medicines Agency
(the only EU Agency in the field of health with experience in working with MS expert
Committees and collecting industry fees). The running costs of a project based secretariat and
Member States secretariat are based on the data from EUnetHTA Joint Action 2 and 3.
Data for the estimation of the outputs costs: Study on Impact Analysis of Policy Options for
Strengthened EU Cooperation on HTA. 2017. Sogeti, Austrian Public Health Institute,
London School of Economics. (CHAFEA/2016/Health/16).
Estimated number of joint outputs: Study on Impact Analysis of Policy Options for
Strengthened EU Cooperation on HTA. 2017. Sogeti, Austrian Public Health Institute,
London School of Economics. (CHAFEA/2016/Health/16). Based on needs identified by
HTA bodies and the volume of ongoing activities carried out of EUnetHTA Joint Action 3.
Estimated progress of work in the first 5 years
–
estimation by DG SANTE based on the
ongoing work of EUnetHTA and opinions expressed during bilateral meeting and public
consultation by HTA bodies that a phased-in approach would allow a smooth transition for
incorporating EU joint activities into the national processes.
158