Advocacy Invite: FDA to Host Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases on Friday, September 20

As part of our commitment to raising patient voices in the regulatory process, we wanted to share an invite from the FDA’s Center for Biologics Evaluation and Research (CBER), which is hosting a patient and care partner listening meeting – Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases on Friday, September 20 from 11am – 4:30pm ET.

Per the FDA, 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 of participating in post-market long-term follow up studies, and to help inform a patient-centered protocols for long-term follow up of gene therapy products. 

The meeting will be divided into two sessions and patients, advocates, and caregivers are encouraged to request to speak during the registration process. The deadline to request to speak is Friday, August 30, 2024 at 11:59 pm ET.