David Stein
Senior Director - Clinical Trial Management Regeneron Pharmaceuticals
Seminars
Wednesday 25th March 2026
Case Study: Using Real-World Data from a Compassionate Use Program to Support Regulatory Filing in an Ultra-Rare Disease
2:00 pm
- Examining a practical case study of how RWD was collected and applied support a regulatory submission in an ultra-rare indication
- Highlighting lessons learned from challenges in data collection, validation, and integration across functions
- Exploring how insights from RWD informed patient access, program design, or engagement with stakeholders
- Providing actionable takeaways for translating RWD into strategies that enhance EAP efficiency and impact
