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 FDA is issuing this draft guidance to provide recommendations to help assure the potency of human CGT products that are regulated as biological products under section 351 of the Public Health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In this draft guidance, the FDA provides recommendations for developing a science- and risk-based strategy to help assure the potency of human CGT products.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In this draft guidance, the FDA emphasizes that potency assays and their corresponding acceptance criteria should be designed to make meaningful contributions to potency assurance by reducing risks to product potency. They 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 Due to the diversity of CGT products and the product-specific nature of potency assays, the recommendations in this draft guidance regarding the selection and design of potency assays are necessarily general.