Cohortias – FDA grants funds for clinical trials in rare diseases

A contract will study if the physical evaluation of ALS patients in the at-home setting can reduce burden on patients.
rare disease

FDA grants funds for clinical trials in rare diseases

The US Food and Drug Administration (FDA) has awarded 19 new grants and two contracts worth over $38m to support clinical trials, natural history studies and regulatory science tools in rare diseases. 

The Orphan Products Grants Program of the regulatory agency is funding the grants and contracts for the coming four years.

These awards are aimed at progressing medical product development for treating rare diseases. 

Various awards back the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (ACT for ALS), which established the FDA Rare Neurodegenerative Disease Grant Program lately to progress the development of medical products for rare neurodegenerative diseases such as ALS. 

The agency received 33 clinical trial grant applications and granted over $25m over the coming four years to 11 trials that promote the development of products for rare diseases.

Additionally, seven of the grants support studies into rare cancers, mostly focusing on brain and peripheral nerve cancers. 

rare diseases

The FDA also received 43 natural history grant applications and offered eight grants worth over $11m for the following four years for natural history studies that back innovative research to guide the development of medical products. 

The agency also funded two contracts linked to rare neurodegenerative ailments.

A contract is co-financed by the National Institutes of Health (NIH) and the FDA to study if physical evaluation of patients with ALS can be conducted remotely in the at-home setting to reduce the burden on patients. 

This will potentially reduce trial expenses and facilitate decentralised trials, boosting access to trials for individuals in rural regions and lower-resource healthcare settings. 

The other contract is a landscape assessment of patient preference information studies that emphasises on brain-computer interface devices. 

FDA Office of Orphan Products Development director Sandra Retzky said: “These grants provide important funding to researchers who are working to develop better treatments for rare disease patients.

“The contracts aim to advance treatment options for patients, help inform regulatory decision-making, and promote diversity, equity and inclusion in clinical research.”

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