The FDA has awarded 15 grants totaling more than $19 million to boost the development of medical device, drug and biological products for patients with rare diseases, with at least a quarter of the funding going to studies focused solely on pediatrics.
The FDA awards grants for clinical studies on safety and/or effectiveness of products that could either result in, or substantially contribute to, approval of the products.
“The FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, one of which is this grants program,” said Gayatri R. Rao, M.D., director of the FDA’s Office of Orphan Product Development. “The grants awarded this year support much-needed research in difficult-to-treat diseases that have little, or no, available treatment options.”
The program is administered through the FDA’s Orphan Products Grants Program, created by the Orphan Drug Act, passed in 1983, to promote the development of products for rare diseases. Since its inception, the program has given more than $330 million to fund more than 530 new clinical studies on developing treatments for rare diseases and has been used to bring more than 50 products to marketing approval.
The 2014 grant recipients are:
For the grants program therapies, a disease or condition is considered rare if it affects less than 200,000 persons in the U.S. There are about 7,000 rare diseases and conditions, according to the NIH. In total, nearly 30 million Americans suffer from at least one rare disease.