This guidance provides recommendations to sponsors developing human gene therapy (GT)  products intended to treat a rare disease  in adult and/or pediatric patients regarding the manufacturing, preclinical, and clinical trial design issues for all phases of the clinical development program.  Such information is intended to assist sponsors in designing clinical development programs for such products, where there may be limited study population size and potential feasibility and safety issues, as well as issues relating to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the GT product itself.  This guidance finalizes the draft guidance of the same title dated July 2018.


Posted on the FDA website on 28 January 2020