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FDA’s Comprehensive Advice for Cell and Gene Therapy Product Development
(Thursday, November 21, 2024) The number of cell and gene therapy (CGT) products has increased steadily over the last decade using traditional product development guidance documents. Although the FDA has released about 37 guidance documents governing the requirements for these products, developers frequently need additional help to define the specific non-clinical, clinical, and manufacturing requirements. A new FDA Guidance aims to connect about 25 years of regulatory experience into a comprehensive roadmap to developing CGTs. CGTs are a class of their own, being the most complex and challenging of all products regulated by the FDA. CGTs include both products directly regulated by the FDA via the IND and BLA routes, as well as those that do not require formal approval under the PHS 361 regulation. The new guidance describes the requirements only for products requiring formal approval. The Q&A- format guidance contain four sections with a total of 36 questions and their answers. The sections outline processes for meeting with the FDA to discuss development programs, non-clinical studies, clinical trials, and manufacturing requirements. The guidance is designed as a standalone document, referring primarily to regulations (CFR references) rather than previous guidance. This reflects a cautious approach to link requirements directly to laws rather than past guidance or FDA opinions. Referring to the CFR chapters makes these recommendations more impactful and legally enforceable. Readers will find most of the advice logical, albeit burdensome. Let’s assume that while the FDA has presented seemingly burdensome advice in this guidance document, it will remain open to its often touted support for the “least burdensome approach” when manufacturers present their development plans to the FDA. 
AUTHOR
Dr. Mukesh Kumar Founder & CEO, FDAMap Email: [email protected] Linkedin: Mukesh Kumar, PhD, RAC Instagram: mukeshkumarrac Twitter: @FDA_MAP Youtube: MukeshKumarFDAMap |
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