The Food and Drug Administration (FDA, Agency, or we) is announcing the availability of a draft guidance entitled ``Potency
Assurance for Cellular and Gene Therapy Products.'' FDA is issuing this draft guidance to provide recommendations to help assure the potency of human cellular therapy or gene therapy (CGT) products at all stages of the product lifecycle. FDA is recommending a comprehensive approach to potency assurance of CGT products that is grounded in quality risk management. For investigational products, we describe how to progressively implement a strategy for potency assurance during product development and provide additional considerations to help assure the potency of products that are undergoing rapid clinical development. For licensed products, we describe requirements for potency assurance, including testing required for lot release.
FDA is announcing the availability of a draft document entitled ``Potency Assurance for Cellular and Gene Therapy Products.'' FDA is issuing this draft guidance to provide recommendations to help assure the potency of human CGT products that are regulated as biological products under section 351 of the Public Health Service Act (42 U.S.C. 262). In this draft guidance, we provide recommendations for developing a science- and risk-based strategy to help assure the potency of human CGT products. A potency assurance strategy is a multifaceted approach that reduces risks to the potency of a product through: (1) manufacturing process design, (2) manufacturing process control, (3) material control, (4) in-process testing, and (5) potency lot release assays. The goal of a potency assurance strategy is to ensure that every lot of a product released will have the specific ability or capacity to achieve the intended therapeutic effect. In this draft guidance, we emphasize that potency assays and their corresponding acceptance criteria should be designed to make meaningful contributions to potency assurance by reducing risks to product potency. We provide illustrative examples of approaches to potency assay development that are grounded in quality risk management. Due to the diversity of CGT products and the product-specific nature of potency assays, the recommendations in this draft guidance regarding the selection and design of potency assays are necessarily general. This draft guidance, when finalized, is intended to supersede the document entitled ``Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products,'' dated January 2011. This draft guidance is being issued consistent with FDA's good guidance practices regulation (21 CFR 10.115). The draft guidance, when finalized, will represent the current thinking of FDA on potency assurance for cellular and gene therapy products. It does not establish any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations.
