Source: EURLEX
Language: en
Format: md

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# 52013SC0254

**COMMISSION STAFF WORKING DOCUMENT EXECUTIVE SUMMARY OF THE IMPACT ASSESSMENT Accompanying the document Proposal for a DECISION OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL on the participation by the European Union in a second European and Developing Countries Clinical Trials Partnership programme (EDCTP2) undertaken by several Member States /\* SWD/2013/0254 final \*/**

  

Disclaimer: This executive summary
commits only the Commission’s services involved in the preparation and does not
prejudge the final form of any decision to be taken by the Commission.

This
Executive Summary outlines the main findings of the Impact Assessment (IA)
report accompanying the Commission’s proposal for a decision on the continued
participation of the European Union (EU) in a second European and Developing
Countries Clinical Trials Partnership programme (EDCTP2), as requested by the
participating European states and recommended in the independent evaluation of
the first EDCTP programme (EDCTP1). It falls under Article 185 of the Treaty
on the Functioning of the EU, which is the basis for the EU to participate in
the joint implementation of national programmes for research and development.
The proposal is put forward in the context of the Multiannual Financial
Framework MFF 2014-20 as part of the implementation of the EU Framework
Programme for Research and Innovation, Horizon 2020. The budget allocation
for EDCTP2 is subject to the outcome of the EU’s decision on the MFF 2014-20
and Horizon 2020.

1.           Objectives

The European and
Developing Countries Clinical Trials Partnership (EDCTP) was established in
2003 in response to the global health crisis caused by the three main poverty-related
diseases — HIV/AIDS, malaria and tuberculosis — and to the EU’s commitment to
achieving the United Nation’s Millennium Development Goals by 2015. The active
funding period for the first EDCTP programme (EDCTP1) is now over. Despite the
achievements of the EDCTP so far, the socio-economic burden of poverty-related
diseases persists and hinders the sustainable development of developing
countries, particularly in sub-Saharan Africa.

1.1.        Effective medical
interventions for poverty-related diseases are lacking

Poverty-related
diseases have huge negative impacts on health, society and the economy. They
particularly affect the world’s poorest and most marginalised communities. More
than 1 billion people, including 400 million children, are suffering from one
or more of the three major poverty-related diseases — HIV/AIDS, malaria and
tuberculosis — or from neglected infectious diseases, such as Buruli ulcer,
trachoma, lymphatic filariasis and sleeping sickness. Malaria and tuberculosis
alone kill an estimated 2.1 million people annually. These diseases undermine
productivity and increase insecurity and infirmity, thus perpetuating the cycle
of poverty. Sub-Saharan Africa is disproportionately affected by such diseases,
with approximately 90 % of all malaria-related deaths occurring in Africa in 2010. This region also accounted for over two thirds (68 %) of all people
living with HIV and for nearly three quarters (72 %) of AIDS-related
deaths in 2008.

General improvements of
nutrition, sanitation and health infrastructures are important, but long-term
control requires the development of new or improved medical interventions. Medical
interventions of these diseases are either lacking or no longer effective. Most
of the new medical interventions — drugs, vaccines, microbicides — under
development are stuck at the stage of early clinical development.

1.2.        These problems persist,
mainly due to insufficient investment, weak local capacity and fragmented
public support

The lack of effective
medical interventions is due to five key drivers: (i)
insufficient investment (market failures), (ii) weak clinical research capacity
in sub-Saharan African countries, (iii) fragmented public support, (iv) limited
scope of the first EDCTP programme, and (v) insufficient links to other EU
initiatives.

–
Firstly, medical interventions are not developed
due to insufficient investment from both the private and public sector. This is
linked to market failures: the research required is risky and expensive, especially
late-stage clinical trials in humans. Moreover, research costs cannot be fully
recovered because neither the people affected by these diseases nor the health
system in developing countries can afford to pay the full market price to secure
the return on private investment.

–
Secondly, most developing countries, in particular in sub-Saharan Africa, lack the basic infrastructure, human resources and know-how to tackle these
problems on their own and conduct clinical trials in compliance with
international standards of good clinical practice.

–
Thirdly, EU Member States acting through
uncoordinated national research policies, programmes and projects compromise
the critical mass and effectiveness of European public action. This is
exacerbated by the fact that aid budgets (which account for about 60 % of
research funding for poverty-related diseases) are shrinking as a result of the
European economic and financial crisis.

–
Fourthly, the limited scope of the first EDCTP
programme made it difficult to tackle poverty-related diseases in a
comprehensive manner. It prevented support for other poverty-related diseases
such as neglected infectious diseases. Extending its scope would also allow the
EDCTP programme to support all stages of clinical development.

–
Fifthly, stepping up cooperation between EDCTP
and EU development assistance could unlock significant synergies, and promote
introduction of newly available medical interventions that are more effective
and safe, and their delivery.

1.3.        EDCTP1 has made substantial
achievements

EDCTP1 has produced a
number of important results:

–
It has funded 55 clinical trials projects,
involving 88 individual clinical trials, with so far 8 of which have resulted
in recommendations for improved patient treatment;

–
A new anti-retroviral formulation for HIV-infected
children in Africa, which was tested in an EDCTP project, was approved by the
US Food and Drug Administration (FDA);

–
National regulatory authorities and ethics
review capacities have been strengthened in many African countries;

–
The Pan-African Clinical Trials Registry (PACTR)
was created with the support of EDCTP1 and is now officially recognised as a
WHO Primary Registry;

–
EDCTP1 has helped to structure the research
landscape in Africa by creating African Networks of Excellence for clinical
trials.

EDCTP is also an
excellent example of a principle that we advocate in Europe: to open our
research programmes to worldwide collaboration. The EDCTP is spectacularly good
at this, with projects that involve institutions from Europe and Africa, with 75 % of funding going to African institutions and with 73 % of
projects led by African researchers. In addition to boosting clinical
development and capacity in sub-Saharan Africa, the programme has succeeded in
triggering structural changes in terms of better coordination of the
participating European states’ national programmes. The level of integrating
national programmes in EDCTP has now reached around 30 % of overall national
research investment in clinical trials for medical interventions against the
three big poverty-related diseases.

1.4.        The lessons learned from
EDCTP1 have fed into the design of EDCTP2

Despite its
achievements, implementation of EDCTP also revealed a number of shortcomings:

i)            The current scope of EDCTP is
too limited to provide a comprehensive response to poverty-related diseases:
more diseases and all stages of clinical development should be included.

ii)            The potential for coordinating
and integrating European national programmes in the scope of EDCTP has not yet
been fully exploited: aligned and concerted activities among participating
European states are now being implemented (so-called Participating States
Initiated Activities) and procedures are being simplified.

iii)           The monitoring and evaluation
of specific targets need to be stepped up: systematic performance and impact
indicators have therefore been developed upfront for EDCTP2;

iv)           Stable working relations with
major research funders and with pharmaceutical industry have not been
established yet: strategic discussions with other funders, such as the Bill and
Melinda Gates Foundation and pharmaceutical industry, are ongoing.

v)           Coordination with EU external
policy and development assistance has not been
developed sufficiently: to tackle this, work is ongoing to coordinate with
other EU initiatives of relevance to EDCTP.

1.5.        The initiative would have
a significant impact on people and stakeholders

Tackling the problem and its drivers would
have a high positive impact on health, well-being and the economic development
of millions of people living in sub-Saharan Africa, in particular on children
and women in the region who are disproportionately affected by these diseases.
Supporting the fight against poverty-related diseases
would also help to safeguard Europe’s citizens from these diseases as
increasing global mobility (including tourism) and migratory movements mean
that Europe will be facing new or returning challenges from infectious
diseases. Global warming may amplify these risks in Europe as it may lead to a higher
prevalence and shift in geographic distribution of these diseases. European
and African researchers would also benefit from better
coordinated and structured research programmes and activities on
poverty-related diseases at European and international level.

1.6.        Public intervention at EU
level is fully justified

The market failures and
resulting investment gap outlined above provide strong justification for public
intervention. Intervention at EU level is necessary to
bring together compartmentalised national research programmes, help design
common research and funding strategies across national borders, and achieve the
critical mass of actors and investments that is needed to tackle major global
health challenges, which couldn't be tackled by single countries alone. It
would also increase the cost-effectiveness and impact of European activities
and investments in this field.

EU intervention is in
line with the Treaty on the Functioning of the EU and related EU
policies. It contributes to delivering on the EU’s commitments to promote aid
effectiveness, inclusive growth and progress towards the achievement of the
Millennium Development Goals.

The aim of this
initiative is to better align EU and national research programmes on
poverty-related diseases. It is embedded into the Treaty’s objectives to
strengthen the EU’s scientific and technology bases (Article 179.1 TFEU), and
to create a European research area based on cooperation between researchers
across borders (Article 179.2 TFEU), such as through the EU’s participation in
research and development programmes undertaken by several Member States (Article
185 TFEU). It also contributes to the EU’s new and extended competences brought
in by the Lisbon Treaty on the EU (TEU) on pursuing common actions in
the field of international relations and cooperation (Article 21 TEU) and thus
to a Global Europe.

2.           Objectives

2.1.        General objectives

In line with the Europe
2020 strategy, the Innovation Union flagship initiative, Horizon
2020, the EU-Africa strategic partnership and the EU’s commitment to the
2012 Rio+20 conference conclusions on the development and achievement of
internationally agreed Sustainable Development Goals, including the Millennium
Development Goals, the general objective of this initiative is to contribute
to the reduction of the social and economic burden of poverty-related diseases
in developing countries, particularly in sub-Saharan Africa, by accelerating
the clinical development of effective, safe and affordable medical interventions
for poverty-related diseases.

2.2.        Specific
objectives

In order to meet the above
general objective, the specific objectives of the EDCTP2 programme are to
result in:

·
An increased number of new or improved
medical interventions for HIV/AIDS, tuberculosis, malaria and other
poverty-related diseases, and by the end of the
programme to have delivered at least one new medical product, such as a new
drug or a new vaccine against TB or any other poverty-related disease; to have
issued at least 30 guidelines for improved or extended use of existing drugs;
and to have progressed the clinical development of at least 20 candidate
products.

·
Strengthened cooperation with sub-Saharan
African countries, in particular on building their capacity for conducting
clinical trials in full compliance with fundamental
ethical principles and relevant national, Union and international legislation,
including the Charter of Fundamental Rights of the European Union, the European
Convention on Human Rights and its Supplementary Protocols, the 2008 version of
the World Medical Association’s Declaration of Helsinki and the ICH standards
on good clinical practice.

·
Enhanced coordination, alignment and
integration of relevant national programmes and
thus increased cost-effectiveness of European public investments.

·
Extended international cooperation and
increased leverage of investments from other public
and private funders.

·
An increased impact of the partnership due to
effective cooperation with relevant EU initiatives,
such as EU development assistance.

3.           Policy options

The impact assessment considered a number of options and sub-options
with different legal bases, scope, duration, budget and EU contribution.

Under Option 1 (‘no
EU action’), there would be no EDCTP2, and no provision
in EU policies, programmes or funded actions to support EDCTP objectives,
either in terms of clinical trials or to integrate Member States’ national
research programmes to combat poverty-related diseases. European support for
clinical trials and related capacity-building would be based solely on Member
States’ national programmes.

Under Option 2 (‘programme-based’),
there would be no EDCTP2 but provision would be made in EU policies, programmes
or funded actions to support EDCTP objectives. Support for
clinical trials and related capacity-building would thus rely on Member States’
national programmes and EU programmes.

Under Option 3 (‘business-as-usual’
— baseline scenario), EDCTP1 would essentially be extended as it is: same
thematic focus, same funding strategy and activities, same budget and duration,
i.e. € 500 million over five years.

Under Option 4 (‘extended
scope’), EDCTP1 would be extended with the same geographical focus (sub-Saharan Africa) but with an extended duration and thematic scope
consisting of (i) doubling the lifetime of the programme to 10 years, (ii) addressing
other poverty-related diseases (in addition to the big three diseases HIV/AIDS,
malaria and tuberculosis), and (iii) supporting all stages of clinical
development.

Regarding the overall
budget and the EU’s contribution, three sub-scenarios were considered: Under sub-option 4A, the total budget of EDCTP2 would be € 0.85
billion with an EU contribution of up to € 350 million to match the participating
European states' contribution of at least € 500 million. Under sub-option
4B, the total budget of EDCTP2 would be € 1 billion with an EU
contribution of up to € 500 million to match the participating European
states' contribution of at least € 500 million. Under sub-option 4C,
the total budget would be € 2 billion with an EU contribution of up to € 1
billion to match the participating European states ' contribution of at least € 1
billion.

4.           Assessment of impacts and
comparison of options

The impacts
of each of the policy options were compared in terms of their effectiveness,
efficiency and consistency in reaching the general and specific objectives.

Option 4C, which would maintain EDCTP's geographical scope but extend its
duration, thematic scope and budget, is the preferred option.

Option 4C would
be the most effective, efficient and consistent option. It requires the
largest EU budget, but it has the potential to transform EDCTP into a major
global player in product development for global health. It would have
sufficient financial volume to provide leadership in developing new effective and
safe medical interventions against the three major and other poverty-related and
neglected diseases, for instance in developing a tuberculosis vaccine. This
option would transform EDCTP from a pure collaborative research programme
between Europe and sub-Saharan Africa into a programme that would contribute to
the long-term sustainable development of sub-Saharan Africa.

This option would also:

–
allow EDCTP to launch expensive late-stage
trials, which cost € 50-400 million;

–
increase the leverage effect of EU public
spending on poverty-related diseases;

–
maintain the EU’s leadership in poverty-related
diseases research and innovation.

5.           Monitoring and evaluation

It is important to
devise a monitoring and evaluation system at programme and project level to
make sound assessments of whether EDCTP2 is on track and successfully achieving
its objectives. The evaluation framework should be composed of the following:

–
updates on EDCTP2 indicators published annually;

–
annual reports on the implementation,
performance and progress of EDCTP2 towards meeting its objectives and targets;

–
an independent mid-term evaluation on the
performance and quality of the EDCTP2 implementation and funded activities
carried out no later than 31 December 2017, and at the end of the EDCTP2 programme,
no later than 31 December 2023; and

–
a final independent ex-post evaluation conducted
no later than 31 December 2026.

The Commission will
ensure that all action taken in the context of EDCTP2 respects the Charter of
Fundamental Rights of the EU and is in line with international standards on
good clinical practice.

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