FDA Awards Multi-Million Dollar Grants for Rare Disease Research

The FDA recently announced it has awarded $23 million worth of grants for 21 clinical research trials to increase the creation of treatments for rare diseases.

These awards were granted through the Orphan Products Clinical Trials Grants Program, which aims to increase the development of drugs, biologics, medical devices, or medical foods for the treatment of patients with rare diseases, according to a press release from the FDA.

The grants are awarded to the principal investigators of clinical trials examining the safety and efficacy of products that could potentially be indicated to treat rare diseases. Typically, rare diseases affect a small population, and there are limited treatment options.

By funding research, and providing incentives for starting clinical trials, the FDA hopes to expand this type of research.

Since 1983, the Orphan Products Clinical Trials Grants Program has provided more than $370 million to fund 590 clinical trials supporting the approval of more than 55 products, the FDA reported. In 2015 alone, 5 of the funded studies resulted in FDA approvals for treatments of neuroblastoma, lymphangioleiomyomatosis, hypoparathyroidism, and hypophosphatasia.

In accordance with Vice President Joe Biden’s National Cancer Moonshot Initiative, 24% of the new grants fund cancer research. Of the funded cancer research, 40% of the studies investigate brain cancers, including 1 that is enrolling children with aggressive brain tumors.

Approximately 42% of the grants are going to clinical studies that are enrolling pediatric patients as young as newborns, while 2 focus on research for transplantation and other issues, according to the FDA.

Another funded study investigates the use of a novel medical device to create an implantable neuroprosthesis for grasp, reach, and trunk function in patients with spinal cord injuries. This device could potentially allow these patients to use their limbs and trunk independently.

A total of 68 grant applications were received this year, and the FDA has announced 21 of the studies will be funded. The funded studies will receive their allotted funds over a period of 4 years.

“We are proud of our 30-year track record of fostering and encouraging the development of safe and effective therapies for rare diseases through our clinical trials grant program,” said Gayatri R. Rao, MD, JD, director of FDA’s Office of Orphan Product Development, within the Office of Special Medical Programs. “The grants awarded this year will support much-needed research in 21 different rare diseases, many of which have little, or no, available treatment options.”