Dive Brief:
- The FDA on Monday announced it would make $2 million in new grants available for rare disease research, aiming to improve data on how rare diseases progress in individuals over time.
- This is the first time the FDA has set aside money to fund so-called natural history studies, which follow individuals from a disease’s inception through medical treatment. The initial grants will launch a new Orphan Products Natural History Grants Program.
- Grant applications will be due Oct. 14, 2016, and the anticipated start date of funding for grant winners is March 2017.
Dive Insight:
The goal of this new program is to clarify the natural history of rare diseases, information which typically is absent for many of the estimated 7,000 rare diseases affecting Americans.
Natural history studies trace the course of diseases in individuals from when a disease first takes hold, through symptoms and clinical treatment. Since this type of information on rare diseases is often incomplete, there exist "major obstacle[s] in the development of life-saving medical products," according to Gayatri Rao, director of the FDA’s Office of Orphan Products Development. The program will bridge funding gaps and "complement existing efforts" to find treatments to patients, Rao said.
The new grants come in two types. The first type will give a maximum of $400,000 in total costs per year for up to five years for forward looking natural history studies which include clinical examination of individuals afflicted with a rare disease. The second offers a maximum of $150,000 in total costs per year for up to two years for retrospective natural history studies or survey studies on the progression of a rare disease.
The FDA has distributed over $350 million for more than 570 clinical studies through the founding of its Orphan Products Grants Program in 1983.