Welcome and Introduction
Presenter Katherine Greene, Forum for Collaborative Research
www.forumresearch.org Time Topic Presenters
2:15 Session I: Project Overview
2:15 Welcome and Introduction Katherine Greene, Forum for Collaborative Research Lara Dimick-Santos, U.S. Food and Drug Administration Elmer Schabel, European Medicines Agency 14:25 Regulatory Perspective Updates and Remarks Irene Tebbs, U.S. Food and Drug Administration Daniel Krainak, U.S. Food and Drug Administration 3:10 Session II: Cirrhosis
3:10 Compensated Cirrhosis & Clinically Meaningful Benefit Naga Chalasani, Indiana University School of Medicine
3:25 Cirrhosis Endpoints: ACLF and MELD Rajiv Jalan, University College London
Decompensated Cirrhosis: Experience from U.S. Pivotal 3:40 Arun Sanyal, Virginia Commonwealth University and Phase 2B Trials
3:55 Group Discussion
4:35 Break
www.forumresearch.org Time Topic Presenters
5:00 Session III: U.S. Payer and Care Delivery Perspectives
5:00 Lessons Learned in HIV and HCV Carl Schmid, The AIDS Institute
5:10 Medicare Coverage: A Review Louis Jacques, ADVI
Louis Jacques, ADVI 5:40 Panel Discussion Heather Patton, Kaiser Permanente Hal Yee, LA County Department of Health Services 6:30 Session IV: Working Groups Sophie Megnien, Genfit 6:30 Case Definitions Working Group Brent Tetri, Saint Louis University 6:40 Pediatric Issues Working Group Miriam Vos, Emory University 6:50 Placebo Arm Data Working Group Eric Lefebvre, Allergan Manal Abdelmalek, Duke University 7:00 Standard of Care Working Group Sven Francque, Antwerp University Hospital 7:10 Session V: Wrap-Up 7:15 Adjourn / Evening Reception
www.forumresearch.org 3 Liver Forum Steering Committee
Arun Sanyal, VCU Scott Friedman, Mount Sinai Co-Chairs Miriam Vos, Emory University David Shapiro, Intercept Vlad Ratziu, Hôpital Pitié Salpêtrière
Lara Dimick, FDA/CDER/DGIEP Academia Detlef Schuppan, Mainz University Stephen Harrison, Pinnacle Clinical Ruby Mehta, FDA/CDER/DGIEP Research Regulatory Laurent Castera, Hôpital Beaujon Chris Leptak, FDA/CDER/OND Tom Hemming Karlsen, Oslo University Elmer Schabel, EMA/BfArM Gary Burgess, Vectura Limited William Baldyga Laurent Fischer, Allergan Patient University Illinois at Chicago Industry Advocates Sophie Megnien, Genfit Donna Cryer, Global Liver Institute Rob Myers, Gilead
LF7/OCT 2017 www.forumresearch.org 4 Liver Forum 6
• Liver Forum 6, Amsterdam, The Netherlands – 174 attendees: 124 in-person, 47 remote Attendance by Organization Attendance by Region 1% 1% 6% 9% 6% Community 14% Australia Government 26% Asia Other Europe Academic 64% North America 73% Industry
LF7/OCT 2017 www.forumresearch.org 5 Participation Over Time
300 250 200 150 Remote In-Person 100 50 0 LF 1 LF 2 LF 3 LF 4 LF 5 LF 6 LF 7
LF7/OCT 2017 6 Reminders
• Attendance Rules: – Meetings are closed/ not public – 2 individuals in-person per company – No marketing/commercial staff in-person – No investors
LF7/OCT 2017 www.forumresearch.org 7 Reminders
• Membership: – We restrict participation to experts with the necessary scientific knowledge from organizations or entities with a clear commitment to advancing the diagnostic and therapeutic field of NASH and liver fibrosis. – We recruit project members meeting the scientific expertise criteria from the various stakeholder groups. – We expect all meeting participants to engage in discussion, and discourage the presence of passive observers.
LF7/OCT 2017 www.forumresearch.org 8 Evaluation Highlights
Overall Value of the Liver Forum Strongly Agree
I would recommend joining the Liver Agree Forum to a peer/colleague Neutral Participating in the Liver Forum is Disagree valuable to my work Strongly Disagree 0% 20% 40% 60% 80% 100%
Impact on Collaboration Strongly Agree Agree The presentations at Liver Forum 6 will help guide the work of my organization Neutral
Liver Forum 6 facilitated new Disagree collaborations or interactions Strongly Disagree
0% 20% 40% 60% 80% 100% N/A
LF7/OCT 2017 www.forumresearch.org 9 Evaluation Highlights
Primary Motivation to Attend Liver Forum 6 3% 2% 3% Latest developments 5%
33% Regulatory updates Networking opportunities
25% Liver Forum updates Interactive discussions
Timing/ location of event
Positive previous experience
29%
LF7/OCT 2017 www.forumresearch.org 10 Liver Forum 67
• Placebo Arm Data Working Group • Standard of Care Working Group • 2017 NASH Biomarkers Workshop • Statistics Workshops – Adaptive Enrichment (link) – Causal Inference (link)
LF7/OCT 2017 www.forumresearch.org 11 Liver Forum 67
• Outreach to Regulatory Agencies • Launch of PSC Forum – Separate Forum – Jessica Weber: [email protected] • Manuscript Submissions & Publications
LF7/OCT 2017 www.forumresearch.org 12 Manuscript Updates
• Accepted: – Baseline parameters in clinical trials for nonalcoholic steatohepatitis: Recommendations from the Liver Forum – Case definitions for inclusion and analysis of endpoints in clinical trials for NASH • In Preparation / Under Review: – Defining improvement in NAFLD for treatment trial endpoints: Recommendations from the Liver Forum – Regulatory considerations for clinical trials in pediatric nonalcoholic fatty liver disease
LF7/OCT 2017 www.forumresearch.org 13 Baseline Parameters
Baseline Parameters in Clinical Trials for Nonalcoholic Steatohepatitis: Recommendations From the Liver Forum Yuval Patel, Joanne Imperial, Andrew Muir, Quentin Anstee, David DeBrota, Lara Dimick-Santos, Claudia Filozof, Ruby Mehta, Arun Sanyal, Elmer Schabel, Brent Neuschwander- Tetri, and Veronica Miller, on behalf of the Liver Forum’s Data Standardization Working Group
Gastroenterology. 2017;153(3), 621-625.e7 https://doi.org/10.1053/j.gastro.2017.07.024
LF7/OCT 2017 www.forumresearch.org 14 Working Group Members
• Quentin Anstee • Goran Gannedahl • Dan Peres • Sander Bangma • Richard Herrmann, • Stephen Rossi • Melanie Baxter • Dean Hum • Arun Sanyal • Sherif Boulos • Stuart Kendrick • Elmer Schabel • Gary Burgess • Leigh MacConell • David Shapiro • Manu Chakravarthy • Sophie Megnien • Tim St Pierre • Rose Christian • Ruby Mehta • Michael Zemel • Anthony Coombs • Veronica Miller • David DeBrota • Brent Neuschwander- Working Group Chairs: • Lara Dimick-Santos, Tetri • Joanne Imperial • Claudia Filozof • Yuval Patel • Andrew Muir
LF7/OCT 2017 www.forumresearch.org 15 Case Definitions
Case definitions for inclusion and analysis of endpoints in clinical trials for NASH through the lens of regulatory science M. Shadab Siddiqui, Stephen Harrison, Manal Abdelmalek, Quentin Anstee, Pierre Bedossa, Laurent Castera, Lara Dimick-Santos, Scott Friedman, Katherine Greene, David Kleiner, Sophie Megnien, Brent Neuschwander-Tetri, Vlad Ratziu, Elmer Schabel, Veronica Miller, Arun Sanyal, on behalf of the Liver Forum Case Definitions Working Group
Hepatology. 2017;xx(x), xx-xx
LF7/OCT 2017 www.forumresearch.org 16 Working Group Members
• Manal Abdelmalek • Anthony Coombs • David Kleiner • Corinna Schoelch • Quentin Anstee • Michael Cooreman • Joel Lavine • Detlef Schuppan • William Baldyga • Marcelo Costa • Robert Lumsden • David Shapiro • Rajarshi Banerjee • Lara Dimick-Santos • Ruby Mehta • M. Shadab Siddiqui • Sander Bangma • Gregory Everson • Veronica Miller • Tim St. Pierre • Mustafa Bashir • Sven Francque • Nikolai Naoumov • Brent Tetri • Melanie Baxter • Scott Friedman • Melissa Palmer • Chinweike Ukomadu • Pierre Bedossa • Goran Gannedahl • Wenjie Pang • Viktor Voros • Mark Berner Hansen • Michael Hambleton • Markus Peck- • Miriam Vos • Sherif Boulos • Eric Hughes Radosavljevic • Jessica Williams • Helena Brett-Smith • Dean Hum • Dan Peres • Teresa Wright • Gary Burgess • Joanne Imperial • Vlad Ratziu • Bryan Burkey • Tim Jenkins • Arie Regev Working Group Chairs • Laurent Castera • Catherine Kelly • Arun Sanyal • Stephen Harrison • Manu Chakravarthy • Stuart Kendrick • Elmer Schabel • Sophie Megnien LF7/OCT 2017 Member & Sponsor Update
• Total Industry Members: 120 • New Industry Members Since LF6: 9 • Total Current Sponsors: 50 • New Sponsors Since LF6: 3 – GSK – Janssen – Perspectum Diagnostics Ltd.
LF7/OCT 2017 www.forumresearch.org 18 Financial Sponsors
LF7/OCT 2017 19 Financial Sponsors
LF7/OCT 2017 20 Financial Sponsors
LF7/OCT 2017 21 Financial Sponsors
LF7/OCT 2017 22 Financial Sponsors
LF7/OCT 2017 23 Many Thanks
• Forum Staff • Prism Event Management Malene Cobourne Paula Blay Terry Daniels Rachel Heitkamp Katherine Greene Graham Hill Pedro Goicochea Mairead O’Reilly Luis Javier Hernandez Sarah Sanderson Jeffrey Kaminski George Seaton Victoria Mason Veronica Miller Brenda Rodriguez Ken Taymor Jessica Weber LF7/OCT 2017 24
Thank You! Contact Information: Katherine Greene [email protected]
www.forumresearch.org 25