ANRS CO22 HEPATHER Cohort



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ANRS CO22 HEPATHER Cohort THERAPEUTIC OPTION FOR HEPATITIS B AND C: A FRENCH NATIONWIDE COHORT STUDY

Public partners Private partners Public/Private Partnership management :

Hepatitis context in France : early access program 2013 2014 ATU in October ATU in December ATU in March ATU in November ATU in December Sofosbuvir Sovaldi Simeprevir Olysio Daclatasvir Daklinza Sofosbuvir Ledipasvir Harvoni Paritaprevir Ombitasvir Viekirax Dasabuvir Exviera Restricted to «priority» patients F3F4 -> F2F3F4 Symptomatic cryoglobulinemic vasculitis -> All extrahepatic manifestations Waiting for liver or renal transplantation After liver transplantation -> Genotype 3 infection -> High risk of transmission

26/07/2013 ANRS CO22 HEPATHER COHORT SUMMARY May 31st, 2015 OBJECTIVES To describe progression of chronic HCV and HBV infections on the long term and to identify associated prognosis factors (including biomarkers). To compare the clinical and cost effectiveness and safety of treatments in «real life» To provide resources for researches in tailored HCV or HBV subpopulations Inclusion 2012 2013 2014 Follow up 2015 2016 2017 2018 2019 25 000 (10.000 HBV & 15.000 HCV) Patients 8 years follow-up, 32 clinical centers GENERAL APPROACH TO DATA COLLECTION AND FOLLOW-UP Centralized Biobank (blood, urine) at inclusion Clinical follow-up by interval: 1 to 2 yearly visits according to the status «A la carte» clinical follow-up : Treatment initiation, Clinical event Follow-up by individual matching on medico-administrative database (SNIIRAM, CEPI-DC) May 31st, 2015 17,691 enrolled - 12,418 HCV with 4170 HCV treated after inclusion - 6783 HBV 4

ANRS CO22 HEPATHER Cohort : Challenges in settingup a Public-Private Partnership The scientific value and the public health improvement must be the main incentive (No pure marketing studies). Involve industrial partners in the definition of data / samples collection for align, in a certain extent, the objectives / expectations of partners. Integrate industrial partner(s) from the start and all along the project. Share the relative risk on ongoing project Protect the integrity of professional judgment and preserve public trust

Governance External Scientific Committee Inserm Ethic Committee Advisory Industrial Committee Gold and Platinum partners Plenary session : With industrial partners representative General Council Directed by ANRS Limited session : - ANRS, Inserm, ITM0 Santé Publique - Public Partners - Institution representative Advisory Institutional Committee AFEF, HAS, InVS, ANSM, DGS Scientific Committee (observatory INDUSTRIAL members) S. Pol (Alternate : H. Fontaine) / F. Carrat Executive Committee S. Pol (Alternate : H. Fontaine) / F. Carrat Virology F. Zoulim JM. Pawlotsky Pathology & Physiopathology P. Nahon J.Capeau Therapeutic M. Bourlière D. Larrey P. Marcellin Public Health C. Larsen P. Carrieri Sponsor Inserm- ANRS Partnership Management Inserm Transfert Investigators Database & Biobank UMR S1136/ F Carrat

ANRS CO22 HEPATHER Cohort : partnership philosophy The willing of Hepather s partnership is to follow the drugs life: development and launch onto the market. Two levels of partnership have been defined : GOLD partners Having specific rights in terms of PLATINUM partners 1. Hepather data access (database, biobank) 2. IP and exploitation Rights on results 3. Position in the governance 5 years commitment is request to ensure the viability and durability of Hepather. Two principles for the partnership functioning: The older partners will be privileged. The bigger partners (cumulated funding over the years) will be privileged.

ANRS CO22 HEPATHER Cohort : How PPP works? Inclusion of HBV or HCV infected patients (10 000 HBV; 15 000 HCV) 60% naives; 10% healing; 40% under treatment 32 centers. HEPATHER Platform Patient Data: Clinical, biological, treatment, environmental and social data Samples: Blood, serum and urine Specific Research Projects Database Raw data gathering Biobank Collection of the samples in Cell&Co Patient Dashboard characteristics of patients class, treatments Product Dashboard pharmaco-epidemio, undesirable events

ANRS CO22 HEPATHER Cohort : Which modalities of partnership? Platinum Partners Gold Partners Annual report Patients Dashboard (characteristics of patients class, treatments ) Products Dashboard which provides data on patients profiles under platinum partner product, effectiveness and safety outcomes,... 2 specific research projects (SRP) per year Biobank specific access fees (to be added to SRP related costs) + 1 specific research project per year Including Postmarketing studies based on Hepather data No Biobank specific access fees (only additional costs related to SRP) HAS approval of a mock-up protocol

ANRS CO22 HEPATHER Cohort : Industrial partners point of view ORGANIZATION Governance well organized with dedicated bodies to operational, strategic & scientific aspects independent scientific evaluation every 3 years A scientific expert network through Scientific Committee organized in thematic axis allowing to enhance international renown Complex structure slowing cohort exploitation by Industrial Partners METHODOLOGY Numerous patients enrolled in the cohort (25 000) giving access to sub-group analysis Linkage with medico-economic databases (such as SNIIRAM) Real life data generation interesting to study sub-group of patients who are not enrolled in clinical trials Biobank with a high-quality phenotyping level HEALTH AUTHORITIES Allowed post-marketing studies (pre-approved protocol by HAS) PV and/or Drug/Drug interaction studies Scientific guarantee INTERNATIONAL VISIBILITY Through its magnitude, its label (Investissements d avenir), its sponsor (ANRS), its experts network, contributing to French attractiveness Collaborations with French and European cohorts Complex structure not easy to set up (delay in clinical center opening, in enrollment, and in first exploitable data generation) Study cohort representativeness (mainly regarding the bias introduced through an enrollment in an expert center) Enhance Hepather visibility for EMA (especially for conditional marketing authorization)

Acknowledgments Laurent Alric, Toulouse Fabien Zoulim, Lyon François Raffi, Nantes Paul Cales, Angers Patrick Marcellin, Clichy Stanislas Pol, Paris Victor De Ledinghen, Bordeaux Marc Bourlière, Marseille Albert Tran, Nice Yves Benhamou, Paris Xavier Causse, Orléans Jean-Claude Trinchet, Jean Verdier Dominique Guyader, Rennes François Habersetzer, Strasbourg Jean-Pierre Bronowicki, Nancy Dominique Larrey, Montpellier Sophie Métivier, Toulouse Imane Amri Marc Bourlière Patrice Cacoub Jacqueline Capeau Fabrice Carrat Patrizia Carrieri Victor De Ledinghen Céline Dorival-Mouly ALL THE PATIENTS INVESTIGATORS AND COLLABORATORS SCIENTIFIC COMITTEE Jean Dubuisson Hélène Fontaine Dominique Larrey Christine Larsen Marianne L'Henaff Patrick Marcellin Philippe Mathurin Pierre Nahon Jean-Pierre Zarski, Grenoble Ghassan Riachi, Rouen Christophe Hézode, Créteil Olivier Chazouillères, Saint Antoine Didier Samuel, Paul Brousse Armand Abergel, Clermont-Ferrand Brigitte Le Guillou, Metz Isabelle Rosa, Créteil Véronique Loustaud-Ratti, Limoges Philippe Mathurin, Lille Anne Minello, Dijon Louis D'Alteroche, Tours Jérome Gourney, Nantes Isabelle Portal, Marseille Eric Saillard, Pointe à Pître Francesco Negro George-Philippe Pageaux Jean-Michel Pawlotsky Ventzislava Petrov- Sanchez Stanislas Pol Michelle Sizorn Jean-Claude Trinchet Linda Wittkop Yazdan Yazdanpanah Jean-Pierre Zarski Fabien Zoulim The ANRS CO22 Hepather cohort is sponsored by ANRS. It was financial supported by ANRS, ANR, MSD, Janssen Cilag, Gilead, BMS, Roche, Abbvie and the Direction Générale de la Santé and supported by the Biologic center resources of Hôpital St Antoine, and conducted in collaboration with AFEF INSERM UMR-S 1136 Fabrice Carrat Céline Dorival-Mouly Sandy Lucier Georges Haour Frederic Chau Manon Bergier ANRS Jean-François Delfraissy Ventzislava Petrov Sanchez Mélanie Simony INSERM TRANSFERT Mireille Caralp Françoise Crevel Caroline Moutaudouin- Roy Marion Pirot Sarita Ghanem Elodie Munier Claire Vezier Alpha Diallo Imane Amri Vincent Bonnemains