Materials
Session I: Ethics and Equipoise in PBC Clinical Trials
Introductory Remarks
Veronica Miller, Forum
Robert-Mitchell Thain, PBC Foundation
Evidence, Equipoise and Methodological Absolutism
Søren Holm, Manchester UniversityEthics and Equipoise in PBC Clinical Trials
Keith Berelowitz, patientRxEvidence and Reasons in Clinical Trial Design and Regulatory Approval
Mark Sheehan, Oxford University
Panel Discussion
Marco Carbone, University of Milano-Bicocca
Mo Christie, PBC Foundation
Session II: Scientific Validity and Statistical Challenges
When the Going Gets Tough: Emulating a Control Arm in PBC When a Randomized Trial is No Longer Feasible
Bettina Hansen, Erasmus UniversityAchieving Robust Evidence while Minimising Expected Control Group Size - An Bayesian Adaptive Trials Perspective
Sofia Villar, MRC Biostatistics Unit at University of CambridgeEfficient Bayesian Model-based Approaches to Reduce Placebo Burden in Rare Disease Trials
Satrajit Roychoudhury, Pfizer*
Panel Discussion
Ingo van Thiel, Deutsche Leberhilfe eV
Bettina Hansen, Erasmus University
Sofia Villar, MRC Biostatistics Unit at University of Cambridge
Alejandra Villamil, Hospital Italiano de Buenos Aires
Satrajit Roychoudhury, Pfizer*
Session III: Risk Stratification in PBC Clinical Trials
Biliary Cirrhosis in PBC is Irreversible: Mandate for Early Stage Detection and Treatment
John Vierling, Baylor College of Medicine
What is Risk to the Patient?
Carol Roberts, PBCers*
Risk Stratification - Industry Panel
Steve Gao, Gilead
Barry Crittenden, Gilead*
Benjamin Miller, Ipsen
Paul Nitschmann, Intercept Pharmaceuticals
Pam Vig, Mirum
Discussion
Danielle Alstat, Life with PBC: Primary Biliary Cholangitis Support Group
David Jones, Newcastle University
Session IV: 21st Century Solutions to 21st Century Problems
Changing the Future: Moving Towards Solutions
Robert-Mitchell Thain, PBC FoundationEASL and the PBC journey
Ana Lleo, Humanitas University
*Remote participation