Listening Meeting Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases

On September 20, 2024, CBER hosted a public patient listening meeting to better understand patient and care partner perspectives on safety considerations and long-term follow-up for approved gene therapy treatments for rare diseases. The objectives of this listening meeting were to hear from patients and care partners on their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations and experience with participating in long-term studies after receiving a gene therapy, and to help inform patient-centered protocols for long-term studies that include gene therapy outcomes. Find more information: https://www.fda.gov/vaccines-blood-bi...