1 / 20

Rare Diseases Foundation Céline Hubert Pr Nicolas Lévy www.fondation-maladiesrares.org

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

orrick
Download Presentation

Rare Diseases Foundation Céline Hubert Pr Nicolas Lévy www.fondation-maladiesrares.org

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. Rare DiseasesFoundationCéline 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 diseasesresearchweaknesses 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 DiseaseFoundation • Creation of a national rare diseasesdatabasis • RADICO project (RAreDIseaseCohorts)

  6. Rare diseasesFoundation 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 DiseasesFoundationis a non-profit privateorganization

  11. A unique model of alliance 5 founders

  12. Universities and Hospitalsinvolved

  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 medicalGeneticsdepartment Marseille University-Hospital Marseille University Rare diseasesFoundationDirector Céline Hubert OperationalDirector

  14. On dedicated people per region • Profil: • Scientist, physician, pharmacistwithknowledge of rare diseasesfield • Mission: • Networking of rare diseasesfieldactors • National rare diseasesdatabasis and RADICO projectdeployment • Promotion of Foundation calls to projects • Facilitation of access to technologicalplateforms • Fundraising

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

  16. Expectedbenefits ? • 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 • Foundersfinancial support, • French Ministry of Researchfinancial support, • Grants, • Public-privatepartnerships.

  18. Whereweare… 8th of February 2012 • Rare diseasesFoundationcreation • First management board meeting • Pressconference and launchat Science Academy • Web site launch: www.fondation-maladiesrares.org • Second management board meeting • First scientificcommittee meeting • Team hiring • First calls for projects • Contacts withpotentialpartners 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

More Related