Overview of Cell and Gene Therapy Guidance

Cellular therapy products include cellular immunotherapies, cancer vaccines, and other types of both autologous and allogeneic cells for certain therapeutic indications, including hematopoietic stem cells and adult and embryonic stem cells. Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use.  The National Institutes of Health (NIH) reports that nearly 7,000 rare diseases affect more than twenty-five million Americans. Approximately 80% of rare diseases are caused by a single-gene defect, and about half of all rare diseases affect children. Since most rare diseases have no approved therapies, there is a significant unmet need for effective treatments, and many rare diseases are serious or life-threatening conditions.

Multiple guidances have been published by the Food and Drug Administration as 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. To design clinical development programs for such products, it is apparent that there is a limited study population size and potential feasibility and safety issues, as well as issues relating to the interpretability of bioactivity/efficacy outcomes may be unique to rare diseases or for the specific GT product. GT-related research and development in the area of rare diseases continues to grow at a rapid rate. FDA has presented multiple guidances on pre-approval and post-approval regulatory requirements and expectations for clinical data to inform the benefit-risk (B/R) profile to ensure clear and practicable expectations of safety and efficacy for the development of cellular therapies.

Please see https://www.fda.gov/vaccines-blood-biologics/biologics-guidances/cellular-gene-therapy-guidances for further information of the following guidances: