Regulatory Considerations in Leveraging Data in Rare Disease Development
8:30 am - Thursday 16th July 2026
- Providing expanded and managed access pathways that balance patient access with the collection of data that may be informative for U.S. regulatory purposes
- Designing to leverage data to help address development gaps in rare diseases, such as underrepresented geographies (e.g., U.S. patients)
- Streamlining managed access processes to ensure programs are feasible and accessible for regulators and treating physicians, while maintaining strategic alignment across internal company stakeholders
