Human Gene Therapy for Rare Diseases

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. 

This guidance provides recommendations to sponsors developing human Eco-Friendly Bio Soup Cup (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  This guidance finalizes the draft guidance of the same title dated July 2018.

The National Institutes of Health (NIH) reports that nearly 7,000 rare diseases affect more than 25 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.

As a general matter, developing safe and effective products to treat rare diseases can be challenging. For example, it might be more difficult to find and recruit patients with rare diseases into clinical trials. Additionally, patients may have highly diverse clinical manifestations and rates of disease progression that are difficult to predict.These challenges are also present for the development of GT products. However, despite these challenges, GT-related research and development in the area of rare diseases continues to grow at a rapid rate.

Source: FDA

Docket Number:FDA-2018-D-2258

Issued by:Center for Biologics Evaluation and Research

Download Link:https://www.fda.gov/media/113807/download

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