FDA https://www.fda.gov has awarded 19 new grants and new contracts totaling more than $38 million over the next four years to support clinical trials, natural history studies, and regulatory science tools related to rare diseases.
These grants and contracts funded by FDA’s Orphan Products Grants Program aim to advance the development of medical products to treat rare diseases such as ALS, since individuals suffering from rare diseases often have limited treatment options available.
To support rare disease treatments, FDA received 33 clinical trial grant applications and awarded more than $25 million to be awarded over the next four year for eleven clinical trials to support product development for rare disease treatments. Seven of the awards will fund studies of rare cancers, mostly targeting cancers of the brain and peripheral nerves.
In addition, natural history studies look closely at how specific diseases progress over time. FDA received 43 natural history grant applications and has funded eight new grants totaling more than $11 million spread over the next four years for natural history studies that support innovative research to inform medical product development.
Several studies seek to characterize certain subgroups within a disease and identify novel clinical outcome measures and biomarkers, which may be able to improve the current standard of care and inform future drug development, including gene therapies.
In addition, a contract, co-funded by NIH and FDA, will study whether a physical assessment of ALS patients, typically done in a healthcare professional’s office, can be done remotely at home. This could ultimately lead to lower clinical trial costs and enable decentralized trials when appropriate. This may improve access to trials for patients in rural areas and lower resource healthcare settings.