Case Study: Building Natural History Data for Rare Disorders to Improve Existing Strategies for Gene Therapy Development
Time: 12:30 pm
day: Day 1- Clinical 1
Details:
- Leveraging patient registries, electronic health records (EHRs), and patient-reported outcomes to gather natural history data quickly and cost-effectively
- Using natural history data to define clinical endpoints, establish baseline variability, and select appropriate patient populations for trials
- Identifying and validating surrogate endpoints that can predict clinical benefit, facilitate regulatory interactions, and enable faster market access
