FDA announces $22m for research into products for patients with rare diseases

Food and Drug Administration

The US Food and Drug Administration (FDA) has announced research grants of more than $22m to develop products for patients with rare diseases over the next four years.

The US agency, which protects public health, stated it would give 15 new clinical trial research grants over the next four years “to enhance the development of products for patients with rare diseases to principal investigators from academia and industry across the country”.

FDA commissioner Scott Gottlieb said: “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.”

Gottlieb added that for more than 30 years, the FDA had 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”.

He said that by helping to support the cost of development of these potential new drugs, and by reducing some of the financial risk, the FDA hoped these grants would “lower the cost of the capital needed to develop these products, boost competition and translate into lower prices for successful medicines”. This could help increase access to resulting therapies, he added.

The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program, to encourage clinical development of drugs, biologics, medical devices, or medical foods for use in rare diseases.

According to the FDA, approximately 33% of the new grant awards fund studies to boost cancer research by enrolling patients with rare forms of cancer, including forms of brain and peripheral nervous system cancers.

Commenting on the funding, Rachel Sherman, the FDA’s principal deputy commissioner, said: “The grants awarded this year will support needed research in a range of rare diseases that have little, or no, treatment options for patients,” she added.

Last updated
The Pharmaceutical Journal, FDA announces $22m for research into products for patients with rare diseases;Online:DOI:10.1211/PJ.2017.20203709

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