Liver Forum 5 November 10, 2016 The Westin Copley Place Boston, MA
1:30 PM Welcome Coffee Reception & Check-in Moderators: Veronica Miller, Forum for Collaborative HIV Research 2:00 PM Session I: Introduction & Updates Arun Sanyal, Virginia Commonwealth University Medical Center David Shapiro, Intercept Pharmaceuticals Presenters: 2:00 PM Liver Forum Updates Arun Sanyal, Virginia Commonwealth University Medical Center Veronica Miller, Forum for Collaborative HIV Research Presenters: Lara Dimick-Santos, U.S. Food and Drug Administration 2:15 PM Regulatory Updates Elmer Schabel, Bundesinstitut für Arzneimittel und Medizinprodukte Presenters: Arun Sanyal, Virginia Commonwealth University Medical Center David Shapiro, Intercept Pharmaceuticals 2:45 PM Recognition for Outstanding Service Recipient: Captain Anissa Davis-Williams, U.S. Food and Drug Administration
Moderators: 2:50 PM Session II: Focus on Biomarkers Veronica Miller, Forum for Collaborative HIV Research Quentin Anstee, Newcastle University Medical School FDA Biomarker Qualification in Drug Development Under Presenter: 2:50 PM IND or NDA/BLA Shashi Amur, U.S. Food and Drug Administration Presenter: 3:10 PM FNIH: NASH Biomarker Consortium Roberto Calle, on behalf of FNIH Presenter: 3:20 PM Liver Forum - FNIH Collaborations Arun Sanyal, co-chair FNIH Biomarker Consortium Presenter: 3:30 PM IMI: Accelerated Drug Portal Julia Brosnan, on behalf of IMI Moderators: 3:35 PM Session III: Definitions WG Update David Shapiro, Intercept Pharmaceuticals Stephen Harrison, University of Oxford
Presenter: Sophie Megnien, Genfit Corp
Discussants: Beyond Baseline Definitions: Scientific Agenda Moving Pierre Bedossa, University of Paris Diderot 3:35 PM Forward Vlad Ratziu, Hôpital Pitié Salpêtrière et Université Pierre et Marie Curie Brent Tetri, Saint Louis University School of Medicine Saul Karpen, Emory University School of Medicine Jeff Schwimmer, University of California, San Diego School of Medicine Vincent Wong, The Chinese University of Hong Kong
4:15 PM Coffee Reception
Moderators: 4:45 PM Session IV: Pediatric Updates Joel Lavine, Columbia University Medical Center Richard Torstenson, Novo Nordisk
Presenters: 4:45 PM Regulatory Updates Elmer Schabel, Bundesinstitut für Arzneimittel und Medizinprodukte Ruby Mehta, U.S. Food and Drug Administration
Presenters and Panelists Miriam Vos, Emory University School of Medicine 5:00 PM Status of Pediatric NASH Research Jeff Schwimmer, University of California, San Diego School of Medicine Ruby Mehta, U.S. Food and Drug Administration Rajarshi Banerjee, Perspectum Diagnostics Moderators: Session V: Mechanism of Action and Surrogate 5:40 PM David Shapiro, Intercept Pharmaceuticals Endpoints Arun Sanyal, Virginia Commonwealth University Medical Center
Panel: Laurent Fischer, Tobira Therapeutics, Inc. Rob Myers, Gilead Sciences, Inc. Linking Mechanism of Action to Endpoint Selection in NASH 5:40 PM Scott Friedman, Icahn School of Medicine at Mount Sinai Trial Anna Mae Diehl, Duke University Medical Center Detlef Schuppan, Mainz University Medical Center Eileen Navarro, U.S. Food and Drug Administration
Moderators: 6:20 PM Session VI: New WG Prioritization Veronica Miller, Forum for Collaborative HIV Research Arun Sanyal, Virginia Commonwealth University Medical Center
Discussants: Possible New Working Groups: Claudia Filozof, Covance 1. Efficiency in Trial Recruitment Donna Cryer, Global Liver Institute 2. Framework for Placebo Arm Cohort Peter Traber, Galectin Therapeutics, Inc. 3. Adaptive trial design and new analytic approaches Robert Arch, Takeda Pharmaceuticals International, Inc. 6:20 PM 4. Biomarker Surrogate Endpoints Sudha Shankar, FNIH 5, DILI/NASH Claude Cohen-Bacrie, SuperSonic Imagine 7. HCC and Other Disease States Claude Sirlin, University of California, San Diego 6. Other? Erwin de Buijzer, Humedics GmbH Arie Regev, Eli Lilly and Company Joanne Imperial, FibroGen, Inc.
Session I: Welcome, Updates and Award
VM/LF5/NOV 2016 VM/LF5/NOV
Facilitating collaborative research in drug development and health policy most of all, the patients the all, of most stakeholders, all of interest best the efficient, in their development is safe identified, are strategies therapeutic and candidates drug new “Once
Forum’sOperating Principle www.hivforum.org ” VM/LF5/NOV 2016 VM/LF5/NOV
Facilitating collaborative research in drug development and health policy •
Increase • • • • Impact onDrug Development Innovation Collaboration Efficiency Clarity
www.hivforum.org • Decrease • • • • Risk time Development Redundancy Uncertainty
VM/LF5/NOV 2016 VM/LF5/NOV
Facilitating collaborative research in drug development and health policy
• efficiencycollaboration through loweringstandards, but by increasing Accelerate drugdevelopmentnot by www.hivforum.org Win -Win
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Facilitating collaborative research in drug development and health policy • • • • •
What’s said at the LF stays at the LF staysLF atWhat’s theat the said Open discussion anddeliberation Co Equal voice Industry participation: – – – Scientists and clinical researchers clinical and Scientists 2 max/company LF assemblies: support on financial contingent Not - ownership of process ownership Not for marketing and commercial branches Working More mayparticipateGroups in Operating Principles (see handouts) (see www.hivforum.org
9
VM/LF5/NOV 2016 VM/LF5/NOV
Facilitating collaborative research in drug development and health policy • • • • •
What’s said at the LF stays at the LF staysLF atWhat’s theat the said Open discussion anddeliberation Co Equal voice Industry participation: – – – Scientists and clinical researchers clinical and Scientists 2 max/company LF assemblies: support on financial contingent Not - ownership of process ownership Not for marketing and commercial branches Working More mayparticipateGroups in Operating Principles (see handouts) (see www.hivforum.org
10 VM/LF5/NOV 2016 VM/LF5/NOV
Facilitating collaborative research in drug development and health policy
• • • among Liver Forum membersLiver Forumamong New collaborations and networks regulatory perspectives Space for informalof exchange NASH of Increasing regulatorythe clarity field in •
EU, US EU, • (other agencies?)
Achievements www.hivforum.org
VM/LF5/NOV 2016 VM/LF5/NOV
Facilitating collaborative research in drug development and health policy
• • development and regulatory science Principles of collaborative drug Foster professional development • • • • • Worldwide Trans Interns Fellows faculty Junior Tomorrow’s Leadership - Atlantic
www.hivforum.org
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• • Manuscripts: LF1:2014 November
– – – agency review review agency Issue Pediatric review agency Final of Standardization Baseline Parameters ready Submission BaselineDisease Case Definition, Living documents Transatlantic consensus LF4 → LF5 → LF4
–
Preparing for final final for Preparing
–
–
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Facilitating collaborative research in drug development and health policy
• • • Yuval Patel Mohammad Amanda Cheung • • • University Brent Muir, Andrew Duke University University Commonwealth Virginia Sanyal, Arun Tetri Forum “Fellows” www.hivforum.org
Health Saint Louis Saint Health SSM , Hospital Shadab
Siddiqui
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Facilitating collaborative research in drug development and health policy •
NASH BiomarkerWorkshop
– – – workshop driven Abstract 5- May andEME Consortium Biomarker NASH FNIH with In collaboration
Washington DC 6, 2017 6, New Initiatives www.hivforum.org
-1
15 VM/LF5/NOV 2016 VM/LF5/NOV
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Focused (LF5workshops→ LF6) – – challenges, strategies and innovation and innovation strategies challenges, cohort history 2. natural Pediatric Approaches and New1. Analytic Design Trial Adaptive New Initiatives www.hivforum.org
- 2 –
16 VM/LF5/NOV 2016 VM/LF5/NOV
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FDA Co- Community/Patients EMA Committee Steering Forum Liver • • • • • • • • chairs Institute Donna Cryer, Baldyga. William BfArM Schabel, Elmer Leptak, Chris Ruby CDER/DGIEP Mehta, Lara Dimick, Arun Pharmaceuticals Shapiro, David
Sanyal , VCU CDER/DGIEP CDER/OND Global Liver Liver Global www.hivforum.org Intercept Intercept
Patient Rep Patient
• • • Societies Industry Academia • • • • • • • • • • • Hemming Tom Laurent Harrison, Stephen Rob Myers Sophie Therapeutics, Inc. Fischer Laurent Gary Burgess Center/BIDMC Detlef Vlad Miriam Emory Vos, Friedman, Scott Salpetriere Marie Curie
Ratziu
, Schuppan Megnien , Castera , Gilead Sciences, Sciences, Inc Gilead ,
, Université Pierre et et Pierre Université , Hopital ,
Vectura , Karlsen , , Mount Tobira EASL Genfit Mainz
AASLD
University Pitie Pitie
Sinai Limited
EASL Uni
Med
.
VM/LF5/NOV 2016 VM/LF5/NOV
Facilitating collaborative research in drug development and health policy • • • •
FDA Co- Community/Patients EMA Committee Steering Forum Liver • • • • • • • • chairs Institute Donna Cryer, Baldyga. William BfArM Schabel, Elmer Leptak, Chris Ruby CDER/DGIEP Mehta, Lara Dimick, Arun Pharmaceuticals Shapiro, David
Sanyal , VCU CDER/DGIEP CDER/OND Global Liver Liver Global www.hivforum.org Intercept Intercept
Patient Rep Patient
• • • Societies Industry Academia • • • • • • • • • • • Hemming Tom Laurent Harrison, Stephen Rob Myers Sophie Inc. Therapeutics, Fischer Laurent Gary Burgess Center/BIDMC Detlef Salpetriere Marie Curie Vlad Miriam Emory Vos, Friedman, Scott
Ratziu
, Schuppan , Megnien , Castera
, Gilead Sciences, Inc Sciences, Gilead , , Université, et Pierre Hopital ,
Vectura , Karlsen Mount , EASL Mainz Tobira Genfit AASLD
University
Pitie Pitie Sinai Limited EASL Uni
Med
.
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• • • • Thanks to outgoing SC members SC outgoing to Thanks London) MassimoPinzani (University College Joel Lavine (Columbia) BrosgartCarol (UCSF) Helena Brett www.hivforum.org - Smith (BMS) Smith
19 Industry Membership and Sponsorship Update
Veronica Miller, PhD [email protected]
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TotalIndustry Members Inclusive of 8 total start total 8 of Inclusive • • • • 6 17 Diagnostic 36 Biopharmaceuticals 37 Pharma Clinical Research Research Clinical Industry Members www.hivforum.org
Organizations (CRO) Organizations - ups
-
96 organizations
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Participating Organizations Organizations Participating www.hivforum.org
by Year by
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Industry Sponsorships www.hivforum.org
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Financial Sponsors www.hivforum.org
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Facilitating collaborative research in drug development and health policy
Financial Sponsors www.hivforum.org
VM/LF5/NOV 2016 VM/LF5/NOV
Facilitating collaborative research in drug development and health policy
Financial Sponsors www.hivforum.org
VM/LF5/NOV 2016 VM/LF5/NOV
Facilitating collaborative research in drug development and health policy
Financial Sponsors www.hivforum.org
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Facilitating collaborative research in drug development and health policy •
Forum StaffForum • • • • • • • Ken Taymor Brenda Jeffrey Kaminski Katie Greene Pedro Goicochea Ben Malene Hauschild Rodriguez Cobourne
Many Thanks www.hivforum.org
• Prism Event Management • • • • Mairead Ash Lawson Graham Hill Paula Blay
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