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FDA awards over $38m for advancement of rare disease treatments

Grants will support clinical trials, natural history studies and regulatory science tools


The US Food and Drug Administration (FDA) has awarded 19 new grants and two new contracts, worth over $38m in funding, to advance the development of medical products to treat rare diseases, including amyotrophic lateral sclerosis (ALS).

The grants and contracts, which were funded by the FDA’s Orphan Products Grants Program, will support clinical trials, natural history studies and regulatory science tools related to rare diseases.

More than $25m, spread over the next four years, was awarded to 11 clinical trials, seven of which are studies of rare cancers, mostly targeting cancers of the brain and peripheral nerves.

An additional eight grants totalling over $11m were awarded to natural history studies, which look closely at how specific diseases progress over time.

Several of the funded studies seek to characterise 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.

“One of the greatest obstacles facing individuals who suffer from rare diseases is the limited treatment options currently available,” said FDA commissioner Robert Califf.

In line with the FDA Rare Neurodegenerative Disease Grant Program, which was recently established specifically for ALS and other rare neurodegenerative diseases, three of the natural history studies awarded by the FDA are related to ALS, myotonic dystrophy type 1 and ataxia-telangiectasia.

Additionally, the two FDA funded contracts are also related to rare neurodegenerative diseases. One contract, co-funded by the National Institutes for Health and the FDA, will study whether a physical assessment of ALS patients, typically carried out in a health care professional’s office, can be carried out remotely at home to minimise the burden on patients.

The second contract is a landscape analysis of patient preference information studies focused on brain-computer interface devices, an area the FDA is specifically interested in.

“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 (OOPD).

She continued: “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.”

Article by
Emily Kimber

19th October 2022

From: Research, Regulatory



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