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The program is intended to provide support to ongoing efforts in rare disease product development.
FDA launched the Natural History Grant Program, a new grants program to fund natural history studies of rare diseases. The agency made the announcement in a blog post on Feb. 29, 2016, officially known as Rare Disease Day 2016.
Katherine Needleman, PhD, the director of orphan products grants program of FDA’s Office of Orphan Products Development, published the blog on FDA’s website, saying, “a lack of understanding of the natural history is often a major obstacle to developing life-saving products for patients with rare disease.”
The grants program will provide two funding levels for those interested in conducting natural history studies:
According to FDA, patient advocacy groups play a “critical role” in collecting natural history data, but they are often prevented from conducting studies because of a lack of funding. Needleman says in the blog post that FDA hopes the program will assist in accelerating the development of rare disease treatments.
A grants program like the National History Grant Program could provide a viable solution for drug developers conducting clinical trials of rare diseases and could help manufacturers provide supplemental data for trials with small numbers of participants. In January 2015 FDA released a 73-page document expressing concerns about clinical trial data from the drug eteplirsen, STAT reported. Eteplirsen is intended to treat children suffering from Duchenne muscular dystrophy, and was developed by Sarepta Therapeutics. Sarepta’s clinical data on the drug came from a small trial with a sample size of 12 boys. FDA expressed concern that such a small sample may not demonstrate the drugs effectiveness.