BIO Submits Comments on Considerations for Design of Early-Phase Clinical Trials for Cell and Gene Therapy Products

BIO commends FDA on the release of this Draft Guidance, which will help to increase the efficiency of early clinical research for, and ultimately facilitate the development of, cellular and gene therapy (CGT) products. In general, the Draft Guidance is very well written and provides a useful review of the numerous factors to consider during the design of early-phase trials for CGT products.

Because many CGT products currently in development target rare diseases, BIO believes that it would be beneficial for FDA to cross-reference, where possible, appropriate guidance documents that address considerations for developing therapeutic products for indications with limited patient populations. BIO would also welcome in the Draft Guidance a discussion of FDA’s views on any unique considerations for CGT products that target rare diseases.