Fireside Discussion: Expanding Compassionate Use for Rare & Small Patient Populations: Emerging Models & Ethical Imperative
Time: 9:30 am
day: Day 2
Details:
• Adapting compassionate use frameworks for ultra-rare conditions as regulatory and ethical models evolve to support access without robust trial data
• Enabling cross-stakeholder coordination through pilot initiatives like the UK’s Rare Therapies Launchpad to build infrastructure for small population access
• Balancing safety, speed, and evidence generation for regulators, clinicians, and sponsors supporting compassionate use in paediatric rare disease settings
