🧬FDA Guidance on Postapproval Safety and Efficacy for CGT Products
The Food and Drug Administration (FDA, Agency, or we) is announcing the availability of a draft document entitled "Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products; Draft Guidance for Industry." The draft guidance document discusses methods and approaches for capturing postapproval safety and efficacy data for cell and gene therapy (CGT) products.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, the collection of postapproval study data for CGT products is important for gathering data on product safety and effectiveness over time.
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The Food and Drug Administration (FDA, the Agency, or we) is announcing the availability of a draft document entitled "Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations." The draft guidance document provides recommendations to sponsors who are planning clinical trials of cell and gene therapy (CGT) products intended for use in a disease or condition that affects a small population, generally one that meets the definition of a rare disease or condition under section 526(a)(2) of the FD&C Act (21 U.S.C. 360bb(a)(2)). It describes FDA requirements and provides considerations for the use of various clinical trial designs and endpoints to generate clinical evidence to support product licensure. The recommendations are intended for sponsors developing CGTs intended for use in small populations to leverage the use of innovative trial designs to simultaneously expedite drug development and generate data necessary to demonstrate substantial evidence of effectiveness.
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