11/8/26 Learning More from Less: Smarter Design and Execution in Rare Disease Trials Previous Designing Neuromuscular Trials for Successful Execution: The Role of Specialized CROs, Sites and Clinical Research Vendors Next Pediatric Rare Disease Development: Evidence, Flexibility, and Regulatory Pathways You Might Also Like Who Really Decides Which Rare Disease Therapies Survive: Regulators, Investors, Payers, or Patients? Modeling Value Across the Rare Disease Patient Journey OPENING KEYNOTE - From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients. Write Your Own Endpoint Justification From Data to Discovery: Finding the Right Patients Across the Therapy Lifecycle
11/8/26 Learning More from Less: Smarter Design and Execution in Rare Disease Trials Previous Designing Neuromuscular Trials for Successful Execution: The Role of Specialized CROs, Sites and Clinical Research Vendors Next Pediatric Rare Disease Development: Evidence, Flexibility, and Regulatory Pathways You Might Also Like Who Really Decides Which Rare Disease Therapies Survive: Regulators, Investors, Payers, or Patients? Modeling Value Across the Rare Disease Patient Journey OPENING KEYNOTE - From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients. Write Your Own Endpoint Justification From Data to Discovery: Finding the Right Patients Across the Therapy Lifecycle
