Rare-disease trials face unique challenges – small patient populations, dispersed sites, and limited data. This session explores how AI-first protocol design transforms feasibility and speed in rare-disease research, enabling smarter, patient-centric, and regulator-ready studies from day one. Key Takeaways:
- How AI-first protocols reduce amendments and time-to-first-patient in small-population studies
- Using real-world and registry data to simulate feasibility and optimize site selection
- Building adaptive, patient-centric designs aligned with EMA and FDA expectations
- Integrating AI into cross-functional workflows — from clinical to regulatory teams
- Case examples showing measurable acceleration and cost savings in rare-disease trials