The EThe European R Research A Area - NetNetworkon rare diseases: E-Rare E-Rare

GIS-Institut des Maladies Rares / INSERM (Paris, France)GIS-Institut des Maladies Rares / INSERM (Paris, France)E-Rare Coordination UnitE-Rare Coordination Unit

Sophie KOUTOUZOVSophie KOUTOUZOV

Support for the coordination of activities

E-Rare objectivesE-Rare objectivesE-Rare objectivesE-Rare objectives

- EC FP6 - funded project (2 Mi.€)

- Coordination action, 4 years (start date: June 2006)

- Objectives: coordinate national or regional research

programmes on RD and develop joint and strategic

activities to:

Harmonize and develop synergies between national research programmes on rare diseases

Develop common research policy on rare diseases

Implement transnational research funding activities (Joint Calls)

E-Rare: ERA-Net for research programmes on rare diseasesE-Rare: ERA-Net for research programmes on rare diseases

E-Rare : A consortium of 8 EU Countries E-Rare : A consortium of 8 EU Countries

Research Funding Agencies/MinistriesResearch Funding Agencies/Ministries

E-Rare : A consortium of 8 EU Countries E-Rare : A consortium of 8 EU Countries

Research Funding Agencies/MinistriesResearch Funding Agencies/Ministries

Systematic exchange of information and best practices

* Increased mutual knowledge and awareness on national RD programmes Set-up: inventory of, and comparison between, national research programmes

* Systemic information for research project managers Set-up: overview of ongoing RD research in Europe through customized queries to Orphanet

Strategic priorities

* WS “Opening of national programmes to more international cooperation ” Outcome: inform researchers on existing national possibilities

* WS “Towards a working plan for the transnational opening of programmes for Rotation and Mobility for RD and OD research” Outcome: catalogue of national possibilities / working plan paper / recommendations for E-Rare

* WS “ Future themes and needs for RD research funding “ Outcome: strategic analysis / priorities for E-Rare funding

Recommendations

* WS “ Database management systems and biobanks ” Outcome: information and help for researchers to scale-up quality of proposed systems

E-Rare achievements and successes (1)E-Rare achievements and successes (1)E-Rare achievements and successes (1)E-Rare achievements and successes (1)

E-Rare achievements and successes (2)E-Rare achievements and successes (2)E-Rare achievements and successes (2)E-Rare achievements and successes (2)

Joint Transnational Activities: 2 Joint Transnational Calls (JTCs) for research projects on rare

diseases

* JTC participating countries :

- 2007 (Fr, It, Ger, Sp, Turk, Isr) (6 MS)(6 MS)

- 2008-2009 (Fr, It, Ger, Sp, Turk, Isr, Nether, Port, Aust, GrePort, Aust, Gre) (10 MS) (10 MS)

* Launch and completion :

- 2007 March 2007 – Nov 2007

- 2008-2009 Dec 2008 – Dec 2009

Clinical studiesEpidemiology / Natural history of diseases

Registries, databases, biomarkers, diagnosis/prognosis markers

Rare cancers, rare drug effects, clinical trials

E-Rare JTCs for research in RD: E-Rare JTCs for research in RD:

A broad medical and scientific scopeA broad medical and scientific scope

E-Rare JTCs for research in RD: E-Rare JTCs for research in RD:

A broad medical and scientific scopeA broad medical and scientific scope

Human & Social Sciences

Genetics / PhysiopathologyGene/mutation search, epigenetics, molecular mechanisms, new animal

models, omics, biomarkers, development of new therapeutic tools

Pre-clinical Therapeutic ResearchProof-of-concept, therapeutic targets, innovative biotechn.

research, drug toxico/pharmacol.

Nb of research groups / country

Nb of eligible projects

Numbers of research groups in eligible proposals JTC 2007 vs. JTC 2009

Numbers of research groups in eligible proposals JTC 2007 vs. JTC 2009

Total Nb of research groups

0 20 40 60 80

AUSTRIA

FRANCE

GERMANY

GREECE

ISRAEL

ITALY

PORTUGAL

SPAIN

THE NETHERLANDS

TURKEY

OTHERS

% partner in no. of proposals (JTC 2009)

Nb of research groups/proposal

NB OF C

ONSORTIA

(PROPOSED)

NB OF C

ONSORTIA

(FUNDED)

JTC 2007 JTC 2009

NB OF C

ONSORTIA

(PROPOSED)

NB OF C

ONSORTIA

(FUNDED)

Number of funded research teams/country

68 73

Funding of successful research groups JTC 2007 vs JTC 2009

Funding of successful research groups JTC 2007 vs JTC 2009

Eligible proposals Successful proposals

Neurology

Hematol./Immunol.Metabolic dis.

Cardiology

Dermatology

Dysmorphology

Ophtalmology

Pulmonary dis.

GastroenterologyNephrology

Rheumatology

Endocrinology

Otolaryngology

Psychiatry/Psychol.

Sensory dis.

Systemic dis.

Cancer

Musculoskeletal dis.

JTC 2009

JTC 2007

JTC 2009

JTC 2007

Medical specialtiesJTC 2007 vs JTC 2009

Medical specialtiesJTC 2007 vs JTC 2009

• 2 Moi € from the EC for E-Rare coordination activities = « glue money »

• A « virtual » common pot filled by E-Rare JTC partners (MS money)

- each participating country (i.e., National Funding Agencies/Ministries ) puts funds in the « pot » = earmarked budget

- each participating call partner country finances its national research teams within selected joint research projects

Hence, the need to match level of « national » Hence, the need to match level of « national » funding with « national » research strength funding with « national » research strength

Funds in and funding, E-Rare JTCsFunds in and funding, E-Rare JTCs

(M€)

(€)

JTC 2009

Oversubscription in eligible proposals (JTC2009)

Austria

France

Germany

Greece

Israel

Italy

Portugal

Spain

Netherlands

Turkey

Available budget Funded for

E-Rare JTC budgetE-Rare JTC budget

E-Rare JTCs : lessons learntE-Rare JTCs : lessons learnt

Success of the E-Rare Joint Transnational Calls reflects expectations and needs of the transnational RD research community

Satisfactory general process for a call with size of ≈ 120-150 proposals, but unsatisfactory success rate (≈ 10%) (compared to the very high amount of work on part of the applicants and agencies)

Easy inclusion of new partners to a call (AU, GR, PT) due to updated call documents and procedures of high quality that meet international standards

The transnational calls have managed to leverage funds from agencies which do not have a national programme on rare diseases

Continuation/Extension of E-Rare: E-Rare-2 (2010-2014) Continuation/Extension of E-Rare: E-Rare-2 (2010-2014)

E-Rare-2 12 Countries 15 Full Partners 1 Affiliated Partner

     

Aims and objectives of E-Rare-2 (2010-2014)Aims and objectives of E-Rare-2 (2010-2014)

Aim:

- develop a wide transnational RD research programme

Objectives: - widen European cooperation by including additional partners

- establish E-Rare as a European portal for funding joint transnational research on rare diseases

* Increase of internal and external communication

* Set up a series of indicators (for funded projects and quality of E-

Rare)

* launch yearly JTCs (2 x large scope-open and 2 X smaller thematically restricted calls…)

* develop the current funding philosophy of E-Rare 2 towards a strategic research policy (increase of national funds)

* increase and diversify activities of E-Rare 2 (clinical trials?)

* develop plans for sustainability of the E-Rare 2 network

Thank you for your attention!

www.e-rare.euwww.e-rare.eu

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