Fireside Discussion: Expanding Compassionate Use for Rare & Small Patient Populations: Emerging Models & Ethical Imperatives
- 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