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Rare Diseases Foundation Céline Hubert Pr Nicolas Lévy fondation-maladiesrares

Rare Diseases Foundation Céline Hubert Pr Nicolas Lévy www.fondation-maladiesrares.org. Rare diseases in France. 3 millions of people concerned More than 200 patients organizations 1260 diagnostic tests available compared to 7000 rare diseases

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Rare Diseases Foundation Céline Hubert Pr Nicolas Lévy fondation-maladiesrares

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  1. Rare Diseases FoundationCéline HubertPr Nicolas Lévy www.fondation-maladiesrares.org

  2. Rare diseases in France • 3 millions of people concerned • More than 200patients organizations • 1260diagnostic tests availablecompared to 7000 rare diseases • Two national Healthgovernmental planslaunched to improve rare diseases care • 2005-2008 • 2011-2014

  3. 1st National Rare Diseases Plan 10 main stakes Staken° 1 : Conductepidemiologicalresearch Stake n° 2 : Caracterize rare diseasesspecificity Stake n° 3 : Improve patients, healthworkers and general public knowledges Stake n° 4 : Traine physicians to rare diseasesdiagnosis Stake n° 5 : Organize screening and improveaccess to diagnostic testing Stake n° 6 : Improveaccess to specializedunitsfor optimal quality of care Stake n° 7 : Stimulateorphandrugsdevelopment Stake n° 8 : Give optimal support to patients Stake n° 9 : Promoteresearch on rare diseases Stake n° 10 : Developeuropean and international partnerships 134 excellence centerswereapproved all over France

  4. Rare diseases research weaknesses Are due to : • Large number of diseases • Rareness of diseases and weaknumber of patients • Scattering of patients all over France • Clinicalheterogeneity • Collection difficulties of clinical information and biologicalsamples • Lack of interest of pharmaceuticalcompanies

  5. 2ndNational Rare Diseases Plan French Ministry of Health and French Minstry of researchlaunched the 2nd plan for rare diseases on the 28th of February 2011:  47 measuresfor 3 keystakes • Stake A : Reinforcequality of care • Stake B : Developresearch on rare diseases • Stake C : Improveeuropean and international partnerships Main actions stake B: • Creation of the rare Disease Foundation • Creation of a national rare diseases databasis • RADICO project (RAre DIsease Cohorts)

  6. Rare diseases Foundation mission

  7. Mission • To improveknowledge of rare diseases • To structureand to harmonize • To coordinateand to federate • To developand to finance Research initiatives on rare diseases in France

  8. Six main fields of action

  9. Rare diseasesFoundationManagement Board

  10. Organization Management board The Rare Diseases Foundation is a non-profit private organization

  11. A unique model of alliance 5 founders

  12. Universities and Hospitals involved

  13. 8 qualified people, advisors of the Foundation JM Belorgey ENA alumnus Council of State Pr JP Grünfeld Nephrologist Necker Hospital Dr MG Mattei Geneticist Marseille University Pr FN Gilly Surgeon oncologist Lyon University Pr J Kristeva Psychanalist Philosopher Paris VII University Pr G Tchernia Hematologist 2nd national plan coordinator Direction team Pr Nicolas Lévy Head of medical Genetics department Marseille University-Hospital Marseille University Rare diseases Foundation Director Céline Hubert Operational Director

  14. On dedicated people per region • Profil: • Scientist, physician, pharmacist with knowledge of rare diseases field • Mission: • Networking of rare diseases field actors • National rare diseases databasis and RADICO project deployment • Promotion of Foundation calls to projects • Facilitation of access to technological plateforms • Fund raising

  15. Scientific committees • In charge of the Foundationscientificpolicy • Researchers and physicians • All medicalspecialtiesinvolved Scientific committee 25-30 french and international experts 2 meetings/year Operational scientificcommittee 10-12 french experts of rare disease 4 meetings/year Experts committees for projects evaluation, (national & international experts)

  16. Expected benefits ? • Improvement of patients care, • Improvement of ourknowledge, • More clinical trials conducted, • More projectsfinanciallysupported, • Best practices sharing, • Facilitatedaccess to innovative technologies, • More linkedbetweenacademic and privateresearch, • …

  17. Financial resources • Founders financial support, • French Ministry of Research financial support, • Grants, • Public-private partnerships.

  18. Where we are… 8th of February 2012 • Rare diseases Foundation creation • First management board meeting • Press conference and launch at Science Academy • Web site launch: www.fondation-maladiesrares.org • Second management board meeting • First scientific committee meeting • Team hiring • First calls for projects • Contacts with potential partners 22nd of February 2012 29th of February 2012 29th of February 2012 28th of March 2012 May 2012 May – June 2012 May 2012 On going

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