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:
- A maximum of $400,000 in total costs per year, up to five years for clinical examination of affected individuals
- A maximum of $150,000 in total costs per year up to two years for retrospective natural history studies or survey 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.
Posted by Dr. Tim Sandle
