📄💉 FDA Releases Draft Guidance on Cell and Gene Therapy Manufacturing and Postmarketing Studies
The FDA has published two new draft guidance documents concerning cell and gene therapy (CGT) manufacturing and postmarketing studies. The first draft advises manufacturers to conduct thorough risk assessments before implementing any changes in manufacturing practices. It acknowledges the unique challenges faced by CGT manufacturing, such as limited knowledge of product quality attributes and short shelf life. The FDA aims to provide recommendations that account for these challenges and ensure comparability assessments for both investigational and licensed human CGT products.
The second draft focuses on noncompliance with FDA-mandated postmarketing studies following the approval of new drugs or biologics. It outlines the requirements for conducting such studies and the information sponsors must provide to the FDA, including trial completion timetables and periodic reports on study status. The draft guidance specifies circumstances that may constitute good cause for noncompliance and provides procedures for communicating with the FDA on postmarketing studies.
Manufacturers are encouraged to engage in proactive discussions with the FDA regarding proposed changes, either through formal meeting requests or by seeking FDA comments on amendments or product correspondences. Compliance with postmarketing studies is emphasized, and applicants are expected to notify the FDA if they anticipate delays or issues in meeting milestones.
FDA review of explanations for missed milestones will determine appropriate actions to correct or mitigate the problems. Noncompliance may be considered reasonable if the circumstance was directly related to the missed milestone, beyond the applicant’s control, and unforeseeable. Applicants should promptly develop plans to address issues, inform the FDA proactively about any delays, and propose reasonable revised timetables.
The draft guidance does not cover certain pediatric studies, trials conducted as a condition of accelerated approval, or trials required as a condition of approval based on evidence from animal studies. The FDA is accepting public comments on both draft guidance documents until September 12th. 📝🔬💼 #FDA #CGTManufacturing #PostmarketingStudies #DraftGuidance FDA Issues Draft Guidance FDA Issues Draft Guidance
