Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products Draft Guidance for Industry September 2025

GUIDANCE DOCUMENT

• Docket Number: FDA-2025-D-3049
• Issued by: Center for Biologics Evaluation and Research, Office of Therapeutic Products

The purpose of this guidance is to discuss methods and approaches for capturing postapproval safety and efficacy data for cell and gene therapy (CGT) products. Given the potential for long-lasting effects of CGT products, and the generally limited number of participants treated in clinical trials conducted to support approval of CGT products, postapproval monitoring is important for gathering data on product safety and effectiveness over time. This guidance does not address data collected for the purpose of expanding clinical indications.

In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidances means that something is suggested or recommended, but not required. 

 

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