FDA Awards Grants to Bolster Rare Disease Research


Fifteen researchers receive more than $22 million for rare disease clinical trials.

Recently, the FDA announced that it awarded 15 new clinical trial research grants for more than $22 million over the next 4 years. These grants were awarded to principal investigators from academia and the pharma industry with the goal of increasing the development of therapies for patients with rare diseases, according to the press release.

“Given the often small number of patients facing certain rare diseases, there can be limited resources devoted to researching new drugs and unique challenges with recruiting and conducting the clinical trials needed to develop medicines targeted to rare conditions,” said FDA Commissioner Scott Gottlieb, MD. “For more than 30 years, the FDA has been committed to investing in trials of potentially life-changing treatments for patients with rare diseases, especially in situations where commercial incentives may not be enough to foster the collection of quality data that can ultimately support efficient development and FDA-approval of treatments for patients who lack effective alternatives.”

The grants were given through the Orphan Products Clinical Trials Grants Program, which stimulates the creation of drugs, biologics, medical devices, or medical foods for patients with rare diseases, according to the release.

Since the program’s creation, it has awarded more than $390 million to fund more than 600 clinical trials. The FDA said that at least 60 grants have supported the approval of more than 55 orphan products.

“By helping to support the cost of development of these potential new drugs, and reduce some of the financial risk, we also hope that these grants will lower the cost of the capital needed to develop these products, boost competition and translate into lower prices for successful medicines,” Dr Gottlieb said. “This can help increase access to resulting therapies.”

The grants were designated to fund clinical studies that evaluate the safety and efficacy of products that may be approved to treat a rare disease, according to the release.

The FDA reports that 33% of new grants were given to studies exploring treatments for rare cancers, including brain and peripheral nervous system cancers. Other studies focus on addressing unmet needs in a number of conditions, including sickle cell disease, idiopathic osteoporosis in premenopausal women, and hyperphagia in Prader-Willi syndrome.

A total of 76 applications were received, with 15 winners being chosen to receive the grants over the next few years, according to the release.

“The clinical trials grant program is an important part of the FDA’s ongoing commitment to encouraging and supporting the development of safe and effective therapies for rare diseases,” said Rachel Sherman, MD, MPH, principal deputy commissioner at the FDA. “The grants awarded this year will support needed research in a range of rare diseases that have little, or no, treatment options for patients.”

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