Case Study: Building Natural History Data for Rare Disorders to Improve Existing Strategies for Gene Therapy Development
Time: 12:00 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
