11/7/26 Modeling Value Across the Rare Disease Patient Journey Previous Who Really Decides Which Rare Disease Therapies Survive: Regulators, Investors, Payers, or Patients? Next From Parent Advocacy to IND: Building a Treatment Pathway for an Ultra-Rare Disease You Might Also Like Learning More from Less: Smarter Design and Execution in Rare Disease Trials Leveraging Natural History and RWE for Rare Approvals From Data to Discovery: Finding the Right Patients Across the Therapy Lifecycle Myth-Busting Hybrid & Home-Based Trials: What’s Real, What’s Not and What Scales OPENING KEYNOTE - From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients.
11/7/26 Modeling Value Across the Rare Disease Patient Journey Previous Who Really Decides Which Rare Disease Therapies Survive: Regulators, Investors, Payers, or Patients? Next From Parent Advocacy to IND: Building a Treatment Pathway for an Ultra-Rare Disease You Might Also Like Learning More from Less: Smarter Design and Execution in Rare Disease Trials Leveraging Natural History and RWE for Rare Approvals From Data to Discovery: Finding the Right Patients Across the Therapy Lifecycle Myth-Busting Hybrid & Home-Based Trials: What’s Real, What’s Not and What Scales OPENING KEYNOTE - From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients.
