Forum for Collaborative Research: Primary Sclerosing Cholangitis (PSC) Forum

Forum for Collaborative Research Public/private partnership including government agencies, industry, researchers and clinicians, insurers, foundations and patient advocacy community HIV Forum HCV Forum HBV Forum CMV Forum (transplantation Liver Forum (NASH, fibrosis) The PSC Forum forum research.org

Forum Overview Miller_Jun'17 forum research.org

Regulatory Industry Academia Patients Miller_Jun'17 forum research.org

Forum Gov’t Industry Patients Academia Miller_Jun'17 forum research.org

Forum’s Operating Principle “Once new drug candidates and therapeutic strategies are identified, their efficient, safe development is in the best interest of all stakeholders, most of all, the patients” forum research.org

Key Characteristics “Information Democracy” Non-competitive, safe environment Independent Co-ownership Synergy vs. duplication Transparency Allow everyone to contribute what they do best

Regulatory Process It’s all about benefit-risk balance Who defines it? Who decides what is sufficient benefit and what is too much risk?

Regulatory Process Sponsor ↔ Agency Advantages of Forum model Approval Pre-Clinical Phase 1 Phase 2 Phase 3 Sponsor ↔ Agency Single sponsor Single agency Confidential proceedings Advantages of Forum model Involvement of all concerned stakeholders Cross-Atlantic regulatory perspectives Cross-Atlantic academic and patient/community perspective forum research.org

Impact on Drug Development Increase Clarity Efficiency Collaboration Innovation Decrease Uncertainty Redundancy Development time Risk Miller_Jun'17 forum research.org

Win-Win Accelerate drug development not by lowering standards, but by increasing efficiency through collaboration Miller_Jun'17

Improving efficiency in drug development is the only ethical and responsible way to proceed Miller_Jun'17

PSC Members Regulators Informal yet structured FDA DGIEP OND CDRH EMA Societies AASLD Industry Academic US Europe Patient Informal yet structured “Non-player ecosystem orchestrator” “Multi-stakeholder partnerships that enables adaptive management” PSC Partners- Ricky Safer Miller_Jun'17 forum research.org

Strategy Collaborative dialogue and deliberation on urgent gaps Build trust Allow consensus to evolve Always science based Don’t force it if the science does not back it Identify knowledge gaps and mechanisms to address these gaps Build more trust Tackle more challenging issues Miller_Jun'17

What impact are we looking for? New perspectives on disease and regulatory process Regulators are responsive to public health needs Paradigm shifts in regulatory thinking Incremental steps in improving efficiencies forum research.org

HCV Forum HCV DAA’s (Direct Acting Antivirals) Standard-of-care: injectable pegylated interferon and ribavarin How can we compare the benefit of an all-oral regiment to an injectable with many side effects? Allow use of historic controls vs. Standard-of-Care comparator arms Paradigm shift forum research.org

Liver Forum Need to increase knowledge of natural history Lack of standardization in the field First step: Allow cross-comparison among clinical trials Standardize disease case definitions Standardize what/how data is collected in clinical trials Responsible data sharing: Placebo-Arm Cohorts Qualification of non-invasive biomarkers No more liver biopsy!! Incremental Paradigm shift forum research.org

Opportunities in PSC Increase the impact of each stakeholder group’s efforts Increase the size of and number of players in the “sandbox” Standardization of case definitions Responsible data sharing forum research.org

What can PSC Community Contribute? Awareness and promotion of clinical research Clinical research knowledge base Patient registry Patient’s perspective on what is ethical research What level of benefit is sufficient? What level or risk is too much? forum research.org

Thank You! forum research.org