11/8/26 Patient Organizations as Evidence Partners: Shaping the Science Behind Rare Disease Approval Previous Inside the FDA Mindset - Negotiating Endpoints and Surrogates Next Write Your Own Endpoint Justification You Might Also Like Ultra-Rare, Ultra-Lean: Designing for a Viable ROI Under 1,000 Patients OPENING KEYNOTE - From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients. Patient Input and Digital Health Technologies: A Regulatory Perspective Across the Product Lifecycle Leveraging Natural History and RWE for Rare Approvals Modeling Value Across the Rare Disease Patient Journey
11/8/26 Patient Organizations as Evidence Partners: Shaping the Science Behind Rare Disease Approval Previous Inside the FDA Mindset - Negotiating Endpoints and Surrogates Next Write Your Own Endpoint Justification You Might Also Like Ultra-Rare, Ultra-Lean: Designing for a Viable ROI Under 1,000 Patients OPENING KEYNOTE - From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients. Patient Input and Digital Health Technologies: A Regulatory Perspective Across the Product Lifecycle Leveraging Natural History and RWE for Rare Approvals Modeling Value Across the Rare Disease Patient Journey
