FDA Awards Grants to Stimulate Rare Diseases Trial Development

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The Food and Drug Administration (FDA) just doled out $22 million over the next four years in the form of 15 new clinical trial research grants designed to spur development of new produces for patients with rare diseases.

“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. M.D. announcing the grants.

FDA received 76 grant applications for fiscal year 2017, with a funding rate of 20 percent. Grant recipients include Alkeus Pharmaceuticals, CereNova, Columbia Unversity Medical Center, Dana-Farber Cancer Institute, Duke University, and Johns Hopkins University.

Since its creation in 1983, the Orphan Products Clinical Trial Program has awarded more than $390 million to fund more than 600 clinical studies. At least 60 grants have supported the marketing approval of more than 55 orphan products. Three of the studies funded by the program supported subsequent product approvals in 2016.

Approximately 33 percent of the new grant awards fund studies designed to accelerate cancer research by enrolling patients with rare forms of cancer. Sixty percent of these studies target forms of brain and peripheral nervous system cancers, including glioblastoma and anaplastic astrocytoma.

Other studies receiving grants target a broad range of diseases and address unmet needs like treating hyperphagia in Prader-Willi syndrome, a genetic disease that primarily affects children, or idiopathic osteoporosis in premenopausal women.

“The grants awarded this year will support needed research in a range of rare diseases that have little, or no, treatment options for patients,” added Rachel Sherman, M.D., M.P.H., and FDA’s principal deputy commissioner.

In related news, FDA has awarded six new research grants for natural history studies in rare diseases. The aim of the research is to inform medical product development by better understanding how specific rare diseases progress over time.

The FDA is providing a total of $6.3 million over the next five years to fund four natural history studies. In addition, through a partnership with the National Institutes of Health’s (NIH) National Center for Advancing Translational Sciences (NCATS), the FDA received $3.5 million to be combined with FDA funding to fund an additional two studies. NCATS’ support was made possible through its Therapeutics for Rare and Neglected Diseases (TRND) program.

Author: Michael Causey