Impact, Challenges and Differences in Establishing Pre- Approval Patient Registries and Natural History Studies for Rare Disease Drug Development Clinical Trials
Time: 2:15 pm
day: Clinical Operations Discussion Day
Details:
- Registries vs natural history studies – the differences and how each contributes to the drug development process
- Understanding the associated benefits in better describing the natural history of a rare disease to inform the drug development process
- How to effectively incorporate multiple stakeholder perspectives when planning and designing natural history data collection efforts
- Lessons learned from the approval of a gene therapy where a retrospective natural history study was the basis of the approval