New Clinical Trial Grants for Rare Diseases | Pediatric Cancer | JAMA | JAMA Network
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News From the Food and Drug Administration
November 23/30, 2021

New Clinical Trial Grants for Rare Diseases

JAMA. 2021;326(20):2003. doi:10.1001/jama.2021.20256

A painful, potentially life-threatening skin disease, an inherited disease that can cause long-term brain development issues, and pediatric brain cancer are among the rare diseases for which the US Food and Drug Administration has awarded 11 new clinical trial research grants.

The grants, totaling more than $25 million over the next 4 years, support clinical trials of products that address unmet needs in rare diseases or significantly improve their treatment or diagnosis. The funds also support a demonstration project to evaluate a tool that could improve data accuracy for multisite clinical trials.

Many of the studies involve children, some as young as newborns. One trial is evaluating the treatment of recessive dystrophic epidermolysis bullosa, the inherited skin disorder that can lead to painful, life-threatening blisters and wounds. Another study is evaluating early treatment of tuberous sclerosis complex before the onset of seizures in infants. And a third will test a novel peptide vaccine to treat pediatric brain cancers. Other grant recipients will focus on treatments for such conditions as Becker muscular dystrophy, short bowel syndrome, and multiple system atrophy.