FDA Awards 19 Grants and Two Contracts Related to Rare Diseases

The U.S. Food and Drug Administration awarded 19 grants and two contracts totaling more than $38 million in funding over the next four years to support clinical trials, natural history studies, and regulatory science tools related to rare diseases.

Photo: Sandra Retzky, director of the FDA’s Office of Orphan Products Development

The FDA’s Orphan Products Grants Program funded the grants and contracts, which aim to advance the development of medical products to treat rare diseases. Several awards support the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (ACT for ALS), which recently established the FDA Rare Neurodegenerative Disease Grant Program to promote medical product development for rare neurodegenerative diseases such as ALS.

“These grants provide important funding to researchers who are working to develop better treatments for rare disease patients,” said Sandra Retzky, director of the FDA’s Office of Orphan Products Development. “The contracts aim to advance treatment options for patients, help inform regulatory decision making, and promote diversity, equity and inclusion in clinical research.”

The FDA received 33 clinical trial grant applications and awarded more than $25 million spread over the next four years to 11 clinical trials that support product development for rare disease treatments. Seven of the awards fund studies of rare cancers, mostly targeting cancers of the brain and peripheral nerves.

The agency received 43 natural history grant applications and funded eight new grants totaling more than $11 million spread over the next four years for natural history studies. The FDA said 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.

The Rare Neurodegenerative Disease Grant Program, which was established specifically for ALS and neurodegenerative conditions, requires that the FDA award grants and contracts to public and private entities to cover costs of research on, and development of interventions intended to prevent, diagnose, mitigate, treat, or cure ALS and other rare neurodegenerative diseases in adults and children. 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.

Additionally, the FDA 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.

The second contract is a landscape analysis of patient preference information (PPI) studies focused on brain-computer interface (BCI) devices. The FDA 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. In total, through collaborative efforts, the FDA and its partners were able to support nearly $6 million in research and science to advance the mission of the ACT for ALS Act.

Author: Rare Daily Staff