FDA Awards 19 Grants for Rare Disease Research

On October 17, 2022, the FDA announced that its Orphan Products Grants Program has awarded 19 grants and 2 contracts, amounting to over $38 million, for rare disease research. This action will hopefully facilitate the development of rare disease treatments and therapies. Many of the awards are for treating ALS.

“One of the greatest obstacles facing individuals who suffer from rare diseases is the limited treatment options currently available,” said FDA Commissioner Robert M. Califf, MD, in the press release. “Since the beginning of the Orphan Products Grants Program in 1983, it has facilitated the approval of more than 80 rare disease products. Through this and other efforts focused on rare diseases, the FDA continues to advance the development and evaluation of safe and effective medical products that help address patients’ unmet needs.”

Of the 33 clinical trial grant applications, 11 were accepted. Approximately $25 million will be dispersed over the next four years, and 7 of the 11 accepted grants will focus on research for treating rare cancers, specifically cancers impacting the brain and peripheral nerves.

In addition to the grants distributed, the FDA will also fund two contracts for neurodegenerative disease research. For one contract, the FDA and the NIH will co-fund a contract focused on the physical symptoms of ALS patients. The team designated to work on this contract will determine whether physical tests for ALS patients can be done at home to minimize patient burdens.

The second contract will be focused on brain–computer interface devices. The FDA hopes these devices will allow people unable to speak to communicate with providers and loved ones.

“These grants provide important funding to researchers who are working to develop better treatments for rare disease patients,” said Sandra Retzky, DO, JD, MPH, director of the FDA’s Office of Orphan Products Development (OOPD), in the press statement. “The contracts aim to advance treatment options for patients, help inform regulatory decision making, and promote diversity, equity and inclusion in clinical research. OOPD will continue to make investments in progressive studies to advance medical product development.”