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

2. 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

3. Forum Overview
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forum research.org

4. Regulatory
Industry
Academia
Patients
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forum research.org

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

6. 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

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

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

9. 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

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

11. Win-Win
Accelerate drug development not by lowering standards, but by increasing efficiency through collaboration
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12. Improving efficiency in drug development is the only ethical
and responsible way to proceed
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13. 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
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forum research.org

14. 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
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15. 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

16. 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

17. 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

18. 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

19. 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?
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20. Thank You!
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