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 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. One Shot, No Slack: The Clinical Decisions That Make or Break a Rare Disease Program Myth-Busting Hybrid & Home-Based Trials: What’s Real, What’s Not and What Scales The Plausible Mechanism Pathway: A New Evidence Playbook for Rare Disease Development
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 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. One Shot, No Slack: The Clinical Decisions That Make or Break a Rare Disease Program Myth-Busting Hybrid & Home-Based Trials: What’s Real, What’s Not and What Scales The Plausible Mechanism Pathway: A New Evidence Playbook for Rare Disease Development
