Clinical research: overview in France



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Clinical research: overview in France Pr A. Syrota President & CEO Inserm President AVIESAN 1

Clinical research infrastructures : support to investigation and to study management Investigator-driven clinical research Industry-sponsored clinical research F - C R I N Academic sponsors Inserm, Direction de la Recherche Clinique et de l'innovation, Groupement Inter-régional de la Recherche Clinique et de l'innovation, Data centres Support to study management Clinical trial units (Unités de Recherche Clinique) SMEs Later phase : Investigation networks Support to investigation Industry sponsors Industry study management or CROs Interface with industry sponsors CeNGEPS Recruitment, contracting, networks Early phase / experimental medicine : Clinical Investigation Centres (Clinical research facilities)

Clinical research infrastructures : support to investigation and to study management Investigator-driven clinical research Industry-sponsored clinical research F - C R I N Academic sponsors Inserm, Direction de la Recherche Clinique et de l'innovation, Groupement Inter-régional de la Recherche Clinique et de l'innovation, Data centres Support to study management Clinical trial units (Unités de Recherche Clinique) SMEs Later phase : Investigation networks Support to investigation Industry sponsors Industry study management or CROs Interface with industry sponsors CeNGEPS Recruitment, contracting, networks Early phase / experimental medicine : Clinical Investigation Centres (Clinical research facilities)

INSERM Created in 1963, related to both Ministry of Research and Ministry of Health The only French public organisation entirely dedicated to biological, medical and public health research The largest European RPO devoted to biomedical research Excellence and multidisciplinary research programmes on most human diseases, even the rarest ones The link between the research laboratory and the hospital bed Life and health sciences progress for better and affordable health care for patients 4

INSERM PUBLICATIONS Clinical Research NEJM, Lancet, JAMA, Nat. Med, J. Exp. Med., JCI, Lancet Neurol., Lancet Oncol., J. Clin. Oncol., Plos Med., Lancet Infect. Dis., Circulation, Cell Met. Cancer Cell, Hepatology, Gastroenterology, J. Am. Coll. Cardiol., Mol. Psy., 250 200 150 100 50 0 126 pub/year 192 pub/year 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 + 52% Basic Biomedical Research Nature, Science, Nat. Gen., Nat. Neu., Nat Biotech., Nat. Struct. Mol. Biol., Nat. Cell Biol., Nat. Nano., Nat. Immun., Nat Neuro., Cell, Immunity, Neuron, Mol. Cell, Cell Stem Cell, Plos Biol., Gen. Dev., Gen. Res., Am. J. Hum. Gen., Cancer Cell, Prog. Lip. Res. 250 200 150 100 50 0 95 pub/year 135 pub/year 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 + 42% 5

A RESEARCH CONTINUUM Inserm Hospitals Industry Mechanisms Targets Identif. Targets validation Pre-clinical studies Phase I Phase II Phase III Phase IV Research Discovery Pre-clinal development Clinical development Optimisation Transfer Translational research Clinical trials Public Health research Basic research 6

Sponsor : Inserm Coverage : National Type : Oriented towards clinical translational research (clinical studies in relation with results obtained in Inserm units) Missions Regulatory aspects Pharmacovigilance +/- monitoring Budget and contracts (Inserm Transfert) About 150 studies (40 new projects each year) Limited funding to support clinical projects Special department for AIDS and viral hepatitis (Agence Nationale de Recherche sur le SIDA) 7

Inserm sponsor : protocols 90% 80% 70% 60% 80% Medical devices Molecular and strutural bases of living organisms Cell biology, development and evolution Cancer Circulation, metabolism, nutrition Genetics, genomics and bioinformatics Immunology, hematology, pneumology Microbiology and infectious diseases Neurosciences, cognitive sciences, neurology and psychiatry Public Health Health technologies 1% 1% 3% 1% 4% 50% Cell and gene therapy 23% 40% Therapeutical trial 30% Pathophysiology 1% 20% 10% 0% 6% 3% Total 12% 52% 8% 6% 8

Coverage :Local >national Located in hospitals Academic sponsors 41 Directions de la Recherche Clinique et de l'innovation Mostly University hospitals (28) Type : projects with direct impact on patients care and health Missions Regulatory aspects Pharmacovigilance Monitoring Budget and contracts Limited funding to support clinical projects 9

Clinical research infrastructures : support to investigation and to study management Investigator-driven clinical research Industry-sponsored clinical research F - C R I N Academic sponsors Inserm, Direction de la Recherche Clinique et de l'innovation, Groupement Inter-régional de la Recherche Clinique et de l'innovation, Data centres Support to study management Clinical trial units (Unités de Recherche Clinique) SMEs Later phase : Investigation networks Support to investigation Industry sponsors Industry study management or CROs Interface with industry sponsors CeNGEPS Recruitment, contracting, networks Early phase / experimental medicine : Clinical Investigation Centres (Clinical research facilities)

Study management Unités de Recherche Clinique University Hospitals : Methodology and biostatistics Inserm Inserm units Methodology and biostatistics +/- data centres Service Commun 10 : Infectious diseases Methodology Data centre 11

Clinical research infrastructures : support to investigation and to study management Investigator-driven clinical research Industry-sponsored clinical research F - C R I N Academic sponsors Inserm, Direction de la Recherche Clinique et de l'innovation, Groupement Inter-régional de la Recherche Clinique et de l'innovation, Data centres Support to study management Clinical trial units (Unités de Recherche Clinique) SMEs Later phase : Investigation networks Support to investigation Industry sponsors Industry study management or CROs Interface with industry sponsors CeNGEPS Recruitment, contracting, networks Early phase / experimental medicine : Clinical Investigation Centres (Clinical research facilities)

Clinical Investigation Centres Joint programme : Inserm and Ministry of Health Located in a University hospital Both a platform and a Clinical research Unit To provide investigators with clinical research facilities in a working environment that complies with European clinical and ethical directives Design Management Analysis - For academic and industry-sponsored studies - Investigators and personnel training Scientific evaluation (similar to the Inserm research units) Quality audits 13

Clinical Investigation Centres 1992 27 CIC General 2004 9 CIC Epidemiology 2005 10 CIC Biotherapies 2007 8 CIC Medical Devices 14

Map of the 41 Clinical Investigation Bichat Centres Robert Debré Garches Saint-Louis Lille Les 15-20 Necker Enfants Malades HEGP HEGP Cochin Pasteur Paris Est Pitié- Salpêtrière Henri Mondor (94 Créteil) Brest Rennes Rouen Nancy Besançon Strasbourg IGR/Curie (Villejuif/Paris) Nantes Tours Dijon Pointe-à-Pitre Fort-de-France Type of CICs Poitiers Limoges Bordeaux Clermont- Ferrand Lyon Saint-Etienne Grenoble Cayenne CIC-P (27) CIC-EC (9) Toulouse Montpellier Marseille Ile de la Réunion Saint-Pierre CIC-BT (10) CIC-IT (8) 11 multi-module CIC 15

Types of protocols Pathophysiology 25% Proof of concept- Phase I-II 29% Epidemiology 17% Phase IV 4% Phase III 25% 16

Bibliometry Year Publications IF > 5 Top 1 % Top 10 % 2005-08/2011 4033 1338 (33 %) 198 (4.9 %) 719 (17.8 %) 2009 750 222 35 (4.7 %) 128 (17.1 %) 2010 958 290 61 (6.4 %) 163 (17 %) 17

Support to investigation INVESTIGATORS NETWORKS 18

Clinical research infrastructures : support to investigation and to study management Investigator-driven clinical research Academic sponsors Inserm, Direction de la Recherche Clinique et de l'innovation, Groupement Inter-régional de la Recherche Clinique et de l'innovation, Data centres Support to study management Clinical trial units (Unités de Recherche Clinique) Industry-sponsored clinical research SMEs Industry sponsors Industry study management or CROs Interface with industry sponsors CeNGEPS Recruitment, contracting, networks Later phase : Investigation networks Support to investigation Early phase / experimental medicine : Clinical Investigation Centres (Clinical research facilities)

AVIESAN Institutes NEUROSCIENCES PSYCHIATRY GENETICS DEVELOPMENT CANCER INFECTIOUS DISEASES CIRCULATION METABOLISM NUTRITION IMMUNOLOGY HEMATOLOGY PNEUMOLOGY PUBLIC HEALTH HEALTH TECHOLOGY Parkinson Pediatrics Vaccines Pregnacy Sepsis Heart Failure Thrombosis Clinical epidemiology Auto-immune diseases Diabetes Metabolism Medical devices CIC disease-specific networks

CEntre National de Gestion des Essais de Produits de Santé Public/Private partnership Objective to reinforce recruitment in industry-sponsored trials 21 Disease-oriented networks of investigators Clinical research assistants in University Hospitals in all regions 21

EUROPEAN INFRASTRUCTURES 22

Clinical research infrastructures : support to investigation and to study management Investigator-driven clinical research Industry-sponsored clinical research F - C R I N Academic sponsors Inserm, Direction de la Recherche Clinique et de l'innovation, Groupement Inter-régional de la Recherche Clinique et de l'innovation, Data centres Support to study management Clinical trial units (Unités de Recherche Clinique) SMEs Later phase : Investigation networks Support to investigation Industry sponsors Industry study management or CROs Interface with industry sponsors CeNGEPS Recruitment, contracting, networks Early phase / experimental medicine : Clinical Investigation Centres (Clinical research facilities)

IA-Integrating Activity ECRIN National Hub Institution Iceland LANSPITALI New national hub The Netherlands UMCU Norway ST OLAVS Sweden KI Finland FINNMEDI Denmark RH Ireland MMI UK UNIVLEEDS EORTC VSOP UnitransferKlinik Germany KKSN Poland MUW POlAND Czech Republic MU Romania UMFCV Luxembourg CRP Sante INRA EURORDIS Qualissima France Inserm UDUS Austria MUW Hungary HECRIN Portugal FCM-UNL Spain CAIBER Switzerland SCTO Italy ISS Turkey DEU FCRB Mario Negri Institute CIRM Tel Aviv University Serbia SMS

French Clinical Research Infrastructure Network Prof. Olivier RASCOL (Toulouse) Investments for the future (Presidential Plan 2010)

Rationale Gathers the major stakeholders involved in clinical research in France Fills the gaps in clinical research tools and not duplicates - in order to maintain the attractiveness of France in the European competition French hub to ECRIN 26

Objectives Platform and single hub open to both sponsors and investigators for improving the French competitiveness in three major fields : 1. Large and complex multicentre trials 2. Early phases industrial trials (big pharma, start-up, biotech) 3. European projects, coordinated by France or extended to France Infrastructures 1. Disease-specific research networks Highly focused Methodology, investigation, recruitment Linked to translational research Able to apply to european calls 2. Distributed services platforms Approached by both sponsors and investigators Services "à la carte" 27

51 research institutes 285 biobanks 31countries Coordination: Austria France: 50 biobanks + 2 WP BBMRI-ERIC Preliminary phase: 2008-2010 ERIC : 2013

BIOBANQUES encompasses 72 Centres with collections 600 research teams Investments for the future (Presidential Plan 2010)

ACADEMIC RESEARCH FUNDING Major academic calls for clinical research 30

Projects Source Programme M /yr Ministry of Health Ministry of Research Programme hospitalier de Recherche Clinique 90 Soutien aux Technologies Innovantes et Coûteuses 10 Agence Nationale de Sécurité du Médicament 6 Clinical Translational Research 1.5 Institut national du cancer 18 Agence Nationale de la Recherche 25 Inserm/Agence Nationale de Recherche contre le SIDA Inserm/Clinical Translational Research 1.5 Inserm transfert 1.5 Charities Association Française contre les Myopathies 25 20 Cancer league 6 31

Infrastructures Activity Source M /yr Sponsoring Ministry of Health 85 Inserm 1.5 Study management Inserm 6 CeNGEPS 10 Investigation Specific Infrastructures Inserm 3 Ministry of Health 40 Ministry of research (ECRIN) 0.5 Ministry of research (ECRIN) 18 / 10yr Ministry of research (Biobanques) 17 / 10yr 32

Conclusions Experienced investigators Sophisticated infrastructures Numerous resources But Multiple layers Numerous lines of financial support Fragmented, separated, dispersed, sometimes redundant resources 33

END 34