The U.S. Food and Drug Administration (FDA) will allocate over $38 million in funding for 19 grants and two contracts aimed at studying and treating rare diseases. According to an announcement made by the agency on Oct. 17, these funds will be disbursed over the next four years to back clinical trials, natural history studies, and regulatory science tools that will give the FDA a greater understanding of the ways that rare diseases, including amyotrophic lateral sclerosis (ALS), can be treated.

The approval of these initiatives is part of the implementation of a five-year plan to treat rare neurodegenerative conditions. The funding of the grants and contracts came from the FDA’s Orphan Products Grants Program, which was started in 1983 to approve products for treating rare diseases. To date, the program has facilitated approvals for 80 rare disease products. 

The agency also awarded grants under the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (ACT for ALS) which recently established a new source of funding by creating the FDA Rare Neurodegenerative Disease Grant Program.

Out of 33 applications for clinical trial grants, only 11 were selected with a combined disbursement of $25 million over four years. The 11 selected trials will fund treatments for rare diseases, including seven studies of rare cancers of the brain and peripheral nervous system. 

The FDA also received 43 applications for natural history grants. According to the agency, natural history studies examine how specific diseases progress over time. Of the 43 applications received, the FDA granted 8 applications to receive over $11 million in the next four years to research subgroups within larger disease categories, identify new clinical measures and biomarkers for diseases, and inform medical product development. The FDA expects the approved studies to improve the standard of care for these conditions and inform future treatments including drug development and gene therapies.

The two contracts approved by the FDA each help tackle an issue faced by patients with neurodegenerative diseases. The first contract is co-funded by the National Institute of Health (NIH) and FDA and will study whether physical assessments of ALS patients can be performed remotely in order to ease the burden placed on patients. If successful, it could lead to lower costs for clinical trials and enable decentralized trials with increased access for patients in rural or poorer areas.

The second contract awarded by the FDA focused on brain-computer interface (BCI) devices. The agency has awarded nearly $6 million in research and science grants to explore the potential of BCI devices that may allow patients who are no longer able to move or speak to interact with their families or healthcare providers.

FDA Commissioner Dr. Robert M. Califf, M.D. stated,  “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.”