Clinical trials for ultra-rare diseases can be particularly challenging to mount. Typically, the patient population is small and geographically diverse. The FDA may allow the use of credible real-world data (RWD) and real-word evidence (RWE) in lieu of data collected in a Phase 3 trial – but the acceptance of that data depends on a sponsor’s understanding the FDA criteria for historical control groups, study data standards, and clinical meaningfulness. As we see in this case, being able to confidently navigate one or more pre-IND meetings is also a critical asset.
This case study will answer questions like:
- What types of real-world trials can sponsors use to support regulatory approval?
- What challenges can sponsors expect when using RWE as part of orphan drug development?
- How can a sponsor work with the FDA to gain agreement on the use of RWE in its development program?
To learn more about how to leverage RWD and RWE in rare disease programs, read our blog post titled Real-World Data in Orphan and Ultra-Rare Diseases: Making the Case to the FDA.