FDA Awards 21 Grants to Stimulate Product Development for Rare Diseases

Funds intended for clinical studies evaluating drugs, biologics, medical devices, or medical foods

The FDA has awarded 21 new clinical trial research grants totaling more than $23 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and industry with research spanning domestic and international clinical sites.

“We are proud of our 30-year track record of fostering and encouraging the development of safe and effective therapies for rare diseases through our clinical trials grant program,” said Gayatri R. Rao, MD, JD, Director of the FDA’s Office of Orphan Product Development. “The grants awarded this year will support much-needed research in 21 different rare diseases, many of which have little, or no, available treatment options.”

The FDA awards 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. The grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products.

Since its creation in 1983, the Orphan Products Clinical Trials Grants Program has provided more than $370 million to fund more than 590 new clinical studies and supported the marketing approval of more than 55 products. Five of the studies funded by this grants program supported product approvals in 2015 alone, including much-needed treatments for neuroblastoma, lymphangioleiomyomatosis, hypoparathyroidism, and hypophosphatasia.

Consistent with the tenor set by Vice President Joe Biden’s National Cancer Moonshot Initiative to accelerate cancer research, 24% of the new grant awards fund studies enrolling patients with cancer; 40% of these studies target devastating forms of brain cancer, one of which recruits children with recurrent or progressive malignant brain tumors.

Forty-three percent of this year’s awards fund studies that enroll pediatric patients as young as newborns. Of these, two focus on research in transplantation and related issues.

In addition, one funded project is a medical device trial to develop a fully implantable neuroprosthesis for grasp, reach, and trunk function in individuals with spinal cord injury with the potential to enable these patients to use their hand, arm, and trunk more independently.

A total of 68 grant applications were received for this fiscal year, with a funding rate of 31% (21/68).

Source: FDA; October 17, 2016.