IDF submitted a response to Senator Bill Cassidy's request for information (RFI) on access to gene and cell therapies in ultra-rare disease communities. Cassidy is a ranking member of the Senate Committee on Health, Education, Labor, and Pensions (HELP).
Even though the first successful gene therapy clinical trial treated a primary immunodeficiency (PI), there is not yet a Food and Drug Administration (FDA) approved gene therapy for any form of PI. The letter pointed to a lack of incentives for commercial companies to develop promising gene therapies beyond clinical trials when patient populations are small, using Orchard Therapeutics' decision to abandon gene therapy for adenosine deaminase (ADA) deficient severe combined immune deficiency (SCID) as an example. IDF is committed to evaluating potential policy solutions that address the commercialization of gene and cell therapies for rare diseases.
