Meeting 1: Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases - 09/20/2024
The FDA's Center for Biologics Evaluation and Research (CBER) held a patient listening meeting focused on gathering perspectives from patients and care partners regarding safety considerations for approved rare disease gene therapies.
This meeting affects manufacturers of approved and investigational gene therapies for rare diseases, as well as functions involved in clinical safety, patient advocacy, and regulatory strategy.
This indicates an increasing focus by CBER on patient-focused drug development (PFDD) within the gene therapy space. The insights gathered likely inform how the FDA evaluates long-term safety data and risk-benefit profiles for rare disease treatments. Regulatory teams may find that patient risk tolerance and safety priorities directly influence the agency's expectations for post-market surveillance and risk mitigation strategies.
Regulatory and clinical teams should monitor subsequent FDA meeting summaries to identify specific safety concerns prioritised by patients. Organizations developing rare disease gene therapies may want to refine their post-market risk communication and patient engagement strategies to align with these emerging perspectives.
FDA CBER Patient Listening Meeting: Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases
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