[ Draft Guidance for Industry ]

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. 

세포·유전자치료제(CGT)는 환자에게 장기간 영향을 미칠 수 있고, 승인을 위해 수행된 임상시험 참여자가 제한적이므로, 승인 후의 모니터링을 통해 제품의 안전성과 효과에 대한 데이터 수집은 중요하다. 또한 새로운 적응증 추가를 위한 데이터는 본 지침이 적용될 수 없다.

FDA_2025.09.

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/postapproval-methods-capture-safety-and-efficacy-data-cell-and-gene-therapy-productshttps://www.fda.gov/regulatory-information/search-fda-guidance-documents/postapproval-methods-capture-safety-and-efficacy-data-cell-and-gene-therapy-products