Human Gene Therapy for Rare Diseases; Draft Guidance for Industry; Availability

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled ``Human Gene Therapy for Rare Diseases; Draft Guidance for Industry.'' The draft guidance document provides recommendations to stakeholders developing a human gene therapy (GT) product intended to treat a rare disease in adult and/or pediatric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.