Pediatric Gene Therapy Access: Communicating with Families, Managing Expectations & Supporting Early Expanded Access Requests
- Educating patients and families about the risks and benefits of gene therapy and the expanded access process
- Communicating transparently when programs are early and pediatric data is limited
- Working compliantly with patient advocacy groups, who are monitoring clinical trials and other potential pathways to treatment
- Managing sensitive conversations around eligibility, timelines, and uncertainty in lifelimiting pediatric conditions