FDA awards grants and contracts linked to rare diseases, including ALS

The agency said the grants and contracts aim to advance the development of medical products to treat rare diseases such as amyotrophic lateral sclerosis (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. “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.”​

The FDA received 33 clinical trial grant applications and awarded more than US$25m to 11 clinical trials aimed at supporting product development for rare disease treatments. Seven of the awards fund studies of rare cancers, mostly targeting cancers of the brain and peripheral nerves.  

Evaluating disease progression ​

Natural history studies look closely at how specific diseases progress over time. The FDA received 43 natural history grant applications and funded eight new grants totaling more than US$11m, which, it said, will be spread over the next four years for research to inform medical product development.

Several studies seek to characterize certain subgroups within a disease, identify novel clinical outcome measures and biomarkers, which have the potential to improve the current standard of care and inform future drug development, including gene therapies.

Three of the natural history studies awarded by the FDA are related to rare neurodegenerative diseases including for ALS, Myotonic Dystrophy Type 1, and Ataxia-Telangiectasia. The study for ALS, partially funded by the National Institutes of Health (NIH) has the potential to advance existing knowledge of the natural history of ALS, inform drug development and possibly support future regulatory decisions.

Remote monitoring 

Additionally, the agency said it funded two contracts related to rare neurodegenerative diseases. One contract, co-funded by NIH and the FDA, will study whether a physical assessment of ALS patients, typically done in a health care professional’s office, can be done remotely at home to minimize the burden on patients. This can ultimately lead to lower clinical trial costs and enable decentralized trials, where appropriate, improving access to trials for patients in rural areas and lower-resource healthcare settings.

The second contract is a landscape analysis of patient preference information (PPI) studies focused on brain-computer interface (BCI) devices. The FDA said it is specifically interested in BCI devices that communicate with the brain and provide patients, who are no longer able to speak or move, with the ability to interact with their families and health care professionals. The contract will review the literature to determine what is already known about BCI devices and PPI studies in ALS.  

The FDA said it remains committed to supporting rare disease research by providing existing grantees with critical funding and encouraging innovative trial designs, collaborations among stakeholders, early and ongoing patient engagement, use of innovative methods, and use of established infrastructure.