Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases
September 20, 202411:00 AMVirtualThe FDA Center for Biologics Evaluation and Research (CBER) is hosting a public patient listening meeting and opening a docket 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 are 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. The meeting will be divided into two sessions.
Meeting Logistics and Registration
Date: September 20, 2024
Time: 11:00 a.m.– 4:30 p.m. ET
Location: This is a virtual public listening meeting and will be held via Zoom.
Registration: This meeting is free and open to the public; however, registration is required. Early registration is recommended.
Registration will close on Thursday, September 19, 2024, at 11:59 pm ET. A recording and other relevant meeting materials will be posted online following the event.