How to improve Clinical Trials in Rare Diseases? Cécile COLOMBAN OrphanDev, CIC CPCET Timone, Marseille

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1 How to improve Clinical Trials in Rare Diseases? Cécile COLOMBAN OrphanDev, CIC CPCET Timone, Marseille Franco-British symposium on clinical research, 17th October 2012

2 National context Specificities of Rare Diseases Clinical Trials OrphanDev s structuration Our experience in Rare Diseases Clinical Trials

3 National context Specificities of Rare Diseases Clinical Trials OrphanDev s structuration Our experience in Rare Diseases Clinical Trials

4 FRANCE: A LEADING COUNTRY IN EU FOR RARE DISEASES HEALTH POLICIES AND INITIATIVES Strong lobbying of patients organizations AFM (French organization for Muscular Dystrophies) and Téléthon (TV program which collects funds dedicated to research on Rare Diseases) Rare diseases Alliance (more than 200 organizations) European database on Rare Diseases : ORPHANET (INSERM) website which provides information on Rare Diseases (RD) dedicated to clinicians, researchers, patients and general population

5 FRANCE: A LEADING COUNTRY IN EU FOR RARE DISEASES HEALTH POLICIES AND INITIATIVES National Plans for Rare Diseases To improve quality of care to patients suffering from RD : creation of Reference Centers (131) and Competence Centers (501) for RD : develop research thanks to a National Foundation

6 FRANCE: A LEADING COUNTRY IN EU FOR RARE DISEASES HEALTH POLICIES AND INITIATIVES An area of excellence in France (LEEM survey 2010 and 2012 about the place of France in international clinical research) Mean number of patients recruted by active center (Preliminary results, Soizic Courcier, oct 2012) Oncology/Onco-hematology Cardiovascular/Metabolism Diabetes Neurology/Psychiatry/Alzheimer 4,9 Scandinavie 11,4 Am. Latine 4,7 Europe Est 10,7 Australasie 4,1 Am. Latine 10,3 Asie 3,8 Asie 9,7 Europe Est 3,8 Australasie 9,5 Allemagne 3,5 Autres Eur. Ouest 9,0 Canada 3,4 Afr. Moyen Or. 8,8 Espagne 3,4 Royaume Uni 8,7 Scandinavie 3,4 Espagne 7,5 Autres Eur. Ouest 3,3 Allemagne 7,4 Etats-Unis 3,2 Etats-Unis 7,0 Royaume Uni 3,2 Canada 7,0 Afr. Moyen Or. Etats-Unis 3,2 Italie 6,1 Italie 2,8 France 4,6 France 4,0 Asie Europe Est Am. Latine Australasie France Scandinavie Canada Autres Eur. Ouest Allemagne Royaume Uni Afr. Moyen Or. Espagne Italie 7,9 7,8 7,7 7,6 6,9 6,9 5,8 5,6 5,5 5,3 4,9 4,2 12,0 Europe Est Afr. Moyen Or. Allemagne Canada Autres Eur. Ouest Espagne Asie Etats-Unis Australasie Am. Latine France Scandinavie Italie Royaume Uni 6,2 5,3 5,2 5,1 5,1 5,1 4,8 4,7 4,6 4,4 4,3 4,2 4,1 4,1 Anti Infectious/Virology/Vaccines Rares Diseases Inflammation/Rheumatology Respiratory diseases Asie Am. Latine Scandinavie Allemagne Europe Est Espagne France Australasie Royaume Uni Canada Etats-Unis Afr. Moyen Or. Autres Eur. Ouest 45,5 Asie 17,8 Autres Eur. Ouest 15,0 Royaume Uni 10,4 France 8,5 Am. Latine 8,2 Canada 7,9 Europe Est 7,7 Afr. Moyen Or. 6,8 Allemagne 6,6 Italie 6,3 Australasie Scandinavie 5,9 Etats-Unis 4,7 Italie Espagne 4,1 2,2 4,4 4,2 4,2 4,2 4,0 4,0 3,4 3,1 2,7 2,6 2,3 2,2 6,2 Am. Latine Europe Est Asie Canada Etats- Unis Australasie Allemagne Espagne France Scandinavie Italie Autres Eur. Ouest Royaume Uni Afr. Moyen Or. 5,4 5,2 4,8 4,5 4,3 4,2 3,6 3,3 3,3 3,2 3,0 2,9 6,5 7,5 Scandinavie Asie Afr. Moyen Or. Europe Est Australasie Allemagne Espagne Royaume Uni Etats-Unis Autres Eur. Ouest Am. Latine France Canada Italie 7,9 7,7 6,3 6,3 6,2 6,2 6,1 5,4 5,3 5,1 4,6 10,7 10,3 10,2

7 National context Specificities of Rare Diseases Clinical Trials OrphanDev s structuration Our experience in Rare Diseases Clinical Trials

8 SPECIFICITIES OF CLINICAL TRIALS IN RARE DISEASES Usual quality and regulatory requirements for all clinical trials (CT) Difficulties specific to rare diseases are added: Translational research

9 RARE DISEASES CLINICAL TRIALS: A NECESSARY ORGANISATIONAL MODEL The need: To overcome these organizational and methodological difficulties to: optimize the design and management of RD CT speed up the access to innovative therapeutics The answer: A network dedicated to the clinical DEVelopment of ORPHAN therapeutics : OrphanDev Aim to increase the number and the quality of RD CT

10 National context Specificities of Rare Diseases Clinical Trials OrphanDev s structuration Our experience in Rare Diseases Clinical Trials

11 RARE DISEASES CLINICAL TRIALS: A NECESSARY ORGANISATIONAL MODEL 2009: creation of OrphanDev In the Clinical Pharmacology Unit of the Timone Hospital, Marseille (CIC-CPCET), in a team with more than 25 years experience in CT Coordinated by Dr Joëlle MICALLEF Regional network Supported by CeNGEPS (National center of health products trials management)

12 RARE DISEASES CLINICAL TRIALS: A NECESSARY ORGANISATIONAL MODEL OrphanDev since 2011: National network Renewal of the CeNGEPS support Member of the French National Foundation for RD, directed by Pr Nicolas LEVY

13 RARE DISEASES CLINICAL TRIALS: A NECESSARY ORGANISATIONAL MODEL Involved in ECRIN-IA European Clinical Research Infrastructure Network Integrating Activity Coordinated by Jacques DEMOTES To structure pan-european investigating networks, focused on rare diseases, medical devices and nutrition. Involved in F-CRIN French part of ECRIN Directed by Vincent DIEBOLT, coordinated by Olivier RASCOL To reinforce the French Clinical Research in Europe and worldwide

14 ORPHANDEV: DEDICATED SKILLS FOR RARE DISEASES CLINICAL TRIALS REALISATION OrphanDev s customized solutions Adapted to each stakeholder s need (researcher, clinician, phamaceutical company, start-up ) Adapted to the specificity of each rare disease Few examples: Support in the translational process Scientific support in the study design Identification and choice of investigation centers Help in research of funding (call for projects ) Access to an expert platform in therapeutic evaluation Optimization and acceleration of the patients recruitment And many other solutions

15 National context Specificities of Rare Diseases Clinical Trials OrphanDev s structuration Our experience in Rare Diseases Clinical Trials

16 OUR EXPERIENCE IN RARE DISEASES: TRANSLATIONAL RESEARCH Clinical Trial in Charcot-Marie-Tooth 1A (CMT 1A) Clinical Trial in Rett Syndrome Clinical Trial in Progeria

17 OUR EXPERIENCE IN RARE DISEASES: FOCUS ON CMT 1A, A SUCCESS STORY 2005: First CT on CMT 1A phase II randomised placebo-controlled multicenter CT Dr Joëlle MICALLEF (PI), Clinical Pharmacology Unit, Marseille Collaboration of researchers, clinicians, pharmacologist, patients organizations A public-private partnership An orphan designation

18 OUR EXPERIENCE IN RARE DISEASES: FOCUS ON CMT 1A, A SUCCESS STORY The recruitment Multicenter study: 3 French centers Specific disposal to improve recruitment : a phone number in Marseille Involvement of the patients organizations 180 CMT 1A patients included in 28 months Highest number of patients recruited in a RD CT at this time

19 OUR EXPERIENCE IN RARE DISEASES: FOCUS ON CMT 1A, A SUCCESS STORY 2010: First industrial promoted CT on CMT 1A (ongoing) SAS Pharnext, EudraCT N phase II randomised placebo-controlled multicenter CT Pr Shahram ATTARIAN (PI), reference center for neuromuscular disorders, Marseille The recruitment Multicenter study: 6 French centers Involvement of the patients organizations before the CT A phone number in Marseille

20 OUR EXPERIENCE IN RARE DISEASES: FOCUS ON CMT 1A, A SUCCESS STORY The recruitment 80 CMT 1A patients included in 6 French centers in 8 months Respect of the inclusion period Role of a unique national phone number: Information for patients (single contact) time saving for investigators

21 OUR EXPERIENCE IN RARE DISEASES: A STRATEGY TO ACHIEVE THE RECRUITMENT The need for a strategy of recruitment set up before the trial adapted to each case Number of subjects required (choice of the good design alternative methodologies) Reference and competence centers Others care units (trajectory of patients in the healthcare system) Registries of patients Early involvement of patients organizations Centralized phone number

22 OUR EXPERIENCE IN RARE DISEASES: COMMUNICATION / TRAINING Training of investigators, students, patients organizations Workshops in congresses

23 CONCLUSION High standards required for CT Need for professional dedicated infrastructures All the more true for RD CT, due to: methodological and logistical difficulties existing public-private partnership model ORPHANDEV network dedicated to RD CT aims to meet the specific needs of support and synergy expressed by clinicians and researchers, whether academic or industrial acts in true partnership with existing structures

24 Thank you for your attention

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