european union support to research on rare diseases

Eurordis Membership Meeting 5-6 May 2006 Berlin

EURORDIS

European Union Support to

Research on Rare Diseases

Dr Fabrizia BIGNAMIEurordis Therapeutic Development Officer


EURORDIS

Directorate General for Research (DG Research)=

European public research funding body

• complementation of private sector-funded research• European added value

Framework Programmes

RARE DISEASES

Rare Diseases: is research needed?


EURORDIS

General objectives of FP - Health:

• improve the prevention and management of important causes of mortality and ill health in Europe,

• pool Europe’s research resources for tackling (rare) diseases.

Specificobjectives of FP:

Topics mentioned in the work programmes of the calls for

proposals

Rare Diseases: is research needed?


EURORDIS Previous programmes: Promote cooperation, collaboration and knowledge building

EU historical support to RD research

FP5 (1998- 2002): Total 47 RD projects, 64 million €• R&D: Friedreich's Ataxia; Peroxisomal diseases; Myopathies;

Congenital disorders of glycosylation; Juvenile systemic lupus erythematosus; Cystic fibrosis

• INFRA: EuroBioBank: European network of DNA, cell and tissue banks on rare diseases

• Biotech: Gene therapy for inherited diseases; Genechiptechnology for detection of molecularly heterogeneous genetic diseases

Such

as

ftp://ftp.cordis.lu/pub/lifescihealth/docs/reprint_rec48300_rare_dis_060207.pdf


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Make a reality of the “European Research Area”, with a view to stepping up innovation in Europe, in conjunction with all the efforts made to this end at regional, national and

European level.

RD research in FP6

FP6: Develop scientific and technical excellence


EURORDIS

INTEGRATING EUROPEAN RESEARCH

THEMATIC PRIORITIES

1 2 3 4 5 6 7

SPECIFIC ACTIVITIES COVERING A WIDER FIELD OF RESEARCH

Supporting EU policies

Anticipating scientific and technological needs

Horizontal research activities involving SMEs

Specific measures in support of international cooperation

Life scien

ces, gen

omics an

d

biotec

hnology for he

alth

Inform

ation society tech

nologies

Nan

otec

hnologies an

d na

nosciences,

intellige

nt m

at., ne

w prod

uction

processes

Aeronau

tics &

spa

ce

Food

qua

lity an

d safe

ty

Sustaina

ble de

velopm

ent, globa

l ch

ange

and ec

osystems

Citize

ns &

governa

nce in a knowled

ge-

based society

Non-nuclear activities of the Joint Research Centre

STRUCTURING THE EUROPEAN RESEARCH

AREA (ERA)

STRENGTHENING THE

FOUNDATIONS OF ERA Research & Innovation

Human res., mobility

Research infrastruct.

Science and Society

Coordination of activities

Development of research/innovation policies

RD research in FP6


EURORDIS

Introduction of new instruments:

•Integrated Projects (IP)

•Networks of Excellence (NoE)

To complement STREP/CA/SSA

Priority 1 (overall budget = 2,255 Million €)

RD research in FP6

Objective: Translate genome data into practical applications to improve human health

How to achieve this objective?: Necessity to reach a critical mass and integration of the research capacities


EURORDIS FP6 and Rare Diseases (RD)

• 4 calls for proposals

• 46 RD relevant projects selected for funding(including Priority, 1 and 8 and ERA-Net scheme)

• Expected global budget for RD: around € 180 million(some projects of the 4th call are still in the negotiation phase)

RD research in FP6


EURORDIS

Rare Diseases ≠ necessary keyword to identify relevant topics in the work

programme

RD research in FP6

Advanced genomics and its applications for health• Fundamental knowledge and basic tools• Biotechnology for health

Combating major diseases• Applications-orientated genomic approaches to medical knowledge - Cardiovascular disease, diabetes and rare diseases- Brain and combating diseases of the nervous system• Combating cancer

+ Actions across priority 1http://www.cordis.lu/lifescihealth/major/rare-diseases-projects-1.htm


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Prader-Willisyndrome (STREP)

Nuclear disorders(2 STREP)

RD of mitochondria

(IP)

RD of plasma membrane

transporters for amino-acids

(STREP)

Planning for early clinical trials in Wilson disease

(CA) Coordination ofRD research (SSA)

Genetic skin disorders (CA)

Inborn errors of metabolism (CA)

Cystic fibrosis (CA)

Lysosomaldisorder:

α-mannosidosis(STREP)

Neuromuscular disorders (NoE)

Protein folding(STREP)

Autoimmune disorders (3 STREP)

RESEARCH

NETWORKING


EURORDIS

Rare hereditary neurological disorders: ataxias (IP)

Rare monogenic neurological disorders (STREP)

Rare Diseases (Neuro + Cancer):Examples of projects

Connective tissues cancer network (NoE)

Uncommon cancers: bone tumours network(NoE)

Prognosis and therapeutic targets for Ewing tumours family(STREP)


EURORDIS

DNA damage and repair mechanisms in health and disease (IP)

Functional genomics: study of peroxisomes in health and disease (IP)

Low abundance proteomics: cystic fibrosis model (STREP)

Functional Genomics + BiotechExamples of Rare Diseases Projects

Genetic testing in Europe (NoE)

Molecular diagnostics in mitochondrial diseases (STREP)

Retroviral transgenesis in gene therapy of inherited diseases (IP)

Clinical gene transfer and therapy (NoE)

Improving precision of nucleic acid-based therapy of cystic fibrosis (STREP)

Gene transfer for curative therapy of human skin disease (STREP)

Tissue engineering approaches to treat birth defects (IP)


EURORDIS

EURO-POLICY-PID (STREP): Policy-oriented and harmonising research activities in primary immunodeficiency diseases (PIDs)

Underpin the formulation and implementation of Community policies

One action line devoted to Public Health issues, including RD

Priority 8RD research in FP6

Encourage and support initiatives undertaken by several countries, to develop synergy between their existing activities,as well as to define and implement joint activities, in areas such as health, including the RD area.

Coordination of Activities (ERA-Net scheme)

E-Rare (SSA + CA): ERA-Net for research programmes on RD


EURORDIS Research Themes for FP7?Open consultation on thematic domains for future European support under FP7

Domains where support at European level• is most needed• will have the greatest impact

based on: • European added value • Contribution to EU policy objectives • European research potential

Towards FP7


EURORDIS

In particular with respect to:

• types of projects (collaborative research projects,coordination/networking, new facilities, etc.)

• size of projects (limited and/or extended) • potential priorities in fields to be covered.

April 2005: DG RTD workshop to identify the future

needs of the rare diseases scientificcommunity

Towards FP7

(http://www.cordis.lu/lifescihealth/major/rare-diseases.htm)


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What’s new ?

Main new elements compared to FP6:

• Annual budget doubled

(EUR 5 billion ► 10 billion)

• Basic research (~ EUR 1,5 billion per year)

• Simplification of procedures

• Logistical and administrative tasks transferred to external structures


EURORDIS

What’s new ?

Main new elements compared to FP6:

• Annual budget doubled

(EUR 5 billion ► 10 billion)

• Basic research (~ EUR 1,5 billion per year)

• Simplification of procedures

• Logistical and administrative tasks transferred to external structures

??

?


EURORDIS

Cooperation – Collaborative researchCooperation – Collaborative research

People – Human PotentialPeople – Human Potential

JRC (nuclear)JRC (nuclear)

Ideas – Frontier ResearchIdeas – Frontier Research

Capacities – Research CapacityCapacities – Research Capacity

JRC (non-nuclear)JRC (non-nuclear)

EuratomEuratom

+

FP7 2007 - 2013

44,735 M€ / ND44,735 M€ / ND

Specific ProgrammesSpecific Programmes

11,942 M€ / ND11,942 M€ / ND

7,178 M€ / ND7,178 M€ / ND

7,536 M€ / ND7,536 M€ / ND

EC requested budget /Final budget


Health 8373 M€ / ND


EURORDIS

Total FP7 (2006-20013)=

73,215 M€ / 47,781 M €

Total FP7 (2006-20013)=

73,215 M€ / 47,781 M €

FP7 2007 - 2013


EURORDIS

Collaborative research(Collaborative projects; Networks of Excellence; Coordination/support actions)

Collaborative research(Collaborative projects; Networks of Excellence; Coordination/support actions)

Joint Technology InitiativesJoint Technology Initiatives

Coordination of non-Community research programmes(ERA-NET; ERA-NET+; Article 169)

Coordination of non-Community research programmes(ERA-NET; ERA-NET+; Article 169)

International CooperationInternational Cooperation



EURORDIS

1. Health

2. Food, agriculture and biotechnology

3. Information and communication technologies

4. Nanosciences, nanotechnologies, materials and new production technologies

5. Energy

6. Environment (including climate change)

7. Transport (including aeronautics)

8. Socio-economic sciences and the humanities

9. Security and space

+ Euratom: Fusion energy research, nuclear fission andradiation protection

Cooperation – Collaborative researchCooperation – Collaborative researchFP7 2007 - 2013

Collaborative researchCollaborative research


EURORDIS

From FP6 & FP7:

• Continuity in the research activities

• Focus on genomics has disappeared

• Emphasis put on translational research

• Biomedical technology & engineering(rich in SMEs) is re-introduced

• Health policy-driven research (public health) is strongly reinforced

Collaborative researchCollaborative research

1. Health 1. Health


EURORDIS

Biotechnology, generic tools and technologies for human healthBiotechnology, generic tools and technologies for human health

Translating research for human healthTranslating research for human health

Optimising the delivery of healthcare to European citizensOptimising the delivery of healthcare to European citizens

1. Health1. Health


EURORDIS

• High-throughput research: Tools, Technologies for enhancing data generation, improving standardisation, acquisition, analysis of data & specimen.

• Detection, diagnosis and monitoring.

• Predicting suitability, safety and efficacy of therapies (incl. alternatives to animal testing)

• Innovative therapeutic approaches and intervention


1. Health 1. Health


EURORDIS




1. Health1. Health


EURORDIS

• Integrating biological data and processes

• Research on the brain and related diseases, human

development and ageing

• Translational research in infectious diseases (incl.

HIV/AIDS, malaria, tuberculosis, SARS, etc.)

• Translational research in major diseases: cancer,

cardiovascular disease, diabetes/obesity; rare diseases;and other chronic diseases (e.g. osteoarthritis)

1. Health 1. Health



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• Rare diseases: – focus on Eu-wide studies of natural history,– pathophysiology and on – development of preventive, diagnostic and

therapeutic interventions. – It will include rare Mendelian phenotypes of

common disease

1. Health 1. Health



EURORDIS




1. Health1. Health

Among the strategic issues: child and old people health


EURORDIS

• Translating clinical outcome into clinical practice

• Quality, efficiency and solidarity of health systems

including transitional health systems (i.e. health

systems that are currently under a reform process)

• Enhanced disease prevention and better use of

medicines

• Appropriate use of new health therapies and

technologies


1. Health 1. Health


EURORDIS



JRC (nuclear)JRC (nuclear)


Capacities – Research CapacityCapacities – Research Capacity

JRC (non-nuclear)JRC (non-nuclear)

EuratomEuratom

+

FP7 2007 - 2013

44,735 M€ / ND44,735 M€ / ND

Specific ProgrammesSpecific Programmes

11,942 M€ / ND11,942 M€ / ND

7,178 M€ / ND7,178 M€ / ND

7,536 M€ / ND7,536 M€ / ND



Health 8373 M€ / ND


EURORDIS


Commission

ERC – European Research Council

* Created by Commission decision * * Under the responsibility of the Commission

• Preparation of work programme

• Set up of peer review: pool of reviewers, nomination of review panels, evaluation guidelines

• Oversight of the evaluation procedure

• Annual scientific report

• Information and support to applicants

• Reception / eligibility of proposals

• Organisation and execution of evaluation

• Selection decision

• Scientific and financial follow-up of contracts

• Annual implementation report

• Approval of work programme, as defined by the Scientific Council

• Instruction to implement work programme

• Approval of annual implementation report

• Information to programme committee

Scientific Council*

Externalised tasks**


EURORDIS

Initial training of researchersMarie Curie Networks

Initial training of researchersMarie Curie Networks

Life-long training and career developmentIndividual Fellowships

Co-financing of regional/national/international programmes

Life-long training and career developmentIndividual Fellowships

Co-financing of regional/national/international programmes

Industry-academia pathways and partnershipsIndustry-Academia Scheme

Industry-academia pathways and partnershipsIndustry-Academia Scheme

International dimensionOutgoing International Fellowships; Incoming International Fellowships

International Cooperation Scheme; Reintegration grants

International dimensionOutgoing International Fellowships; Incoming International Fellowships

International Cooperation Scheme; Reintegration grants

Specific actionsExcellence awards

Specific actionsExcellence awards


More on Human Potential


EURORDIS Capacities – Research CapacityCapacities – Research Capacity

Research InfrastructuresResearch Infrastructures

Research for the benefit of SMEsResearch for the benefit of SMEs

Regions of KnowledgeRegions of Knowledge

Research PotentialResearch Potential

Science in SocietyScience in Society

Activities of International CooperationActivities of International Cooperation


EURORDIS

Towards FP7Indicative roadmap for FP7

December 2006Launch Conference

end 2006/begin 2007Publication of first calls for proposals

foreseen September

2006

Adoption of Specific Programmes by theEuropean Council

foreseen June/July

2006

Adoption of FP by European Council andEuropean Parliament

21 September 2005Commission proposals for SpecificProgrammes:

6 April 2005Commission proposal for FP7

foreseen May 2006Decision on Financial Perspectives:


EURORDIS

Information

• EU research: http://europa.eu.int/comm/research

• Seventh Framework Programme: http://europa.eu.int/comm/research/fp6/index_en.html

• Information on research programmes and projects:http://www.cordis.lu

• RTD info magazine:http://europa.eu.int/comm/research/rtdinfo

• Information requests:[email protected]

[email protected]@[email protected]


EURORDIS

THANK YOU!


EURORDIS

1. Research Infrastructures 1. Research Infrastructures

Transnational AccessTransnational Access

Integrating activitiesIntegrating activities

Research e-infrastructuresResearch e-infrastructures

Construction of new research infrastructures and major updates of existing ones

Construction of new research infrastructures and major updates of existing ones

Design studiesDesign studies

Support to existing research infrastructures:

Support to new research infrastructures:

More on Infrastructures


EURORDISInfrastructures - Objectives

• Optimise use and development of best research infrastructures in Europe

• Help create necessary new infrastructures of pan-European interest

• Help industry strengthen knowledge base and technological know-how


EURORDIS

Activities

Support for:

• Existing research infrastructures

– optimizing access utilisation

• New research infrastructures

– (incl. major upgrades)

– through implementation of ‘strategic’ approach (ESFRI: European Strategy Forum on Research Infrastructure)

•European network of bio-banks and genomic resources are included


EURORDIS

CALLS FOR PROPOSALS• Collaborative projects • Networks of excellence• Coordination and support actions• Individual projects• Support for training and career development• Research for the benefit of specific groups (in

particular SMEs and civil society organisations)

DECISIONS BY COUNCIL AND EP• Art. 169• Art. 171

FP7 funding schemes

Towards FP7


EURORDIS

Flexibility – Rationalisation - CoherenceE.g. Rationalisation of requests for information

addressed to participants, reduction of a-prioricontrol, increased autonomy of consortia

Simplification of procedures

Towards FP7

• New structures needed to manage a higher budget, while not increasing the number of Commission staff

• Part of the FP management to be externalised

Management structures

european union support to research on rare diseases

Documents