FDA Grants Support Rare Disease Research

FDA awards grants for new clinical trial research worth more than $23 million over the next 4 years for rare disease drug development.

The FDA has awarded 21 grants for new clinical trial research worth more than $23 million to be spent over the next 4 years on the development of products for patients with rare diseases. The grants were awarded to principal investigators with research spanning domestic and international clinical sites.

Typically, the FDA awards grants through the Orphan Products Clinical Trials Grants Program intended for clinical studies that measure the safety and effectiveness of products. The goal is to increase the development and FDA approval of biologics, drugs, medical devices, and medical foods for treating rare diseases.

“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 FDA’s Office of Orphan Product Development, in a press release. “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 Orphan Products Clinical Trials Grants Program was created in 1983. Since its inception, it has provided $370 million to fund more than 590 new clinical studies and supported the marketing approval of 55 products.

Five of the studies funded by the program supported product approvals in 2015. Forty-three percent of this year’s grants fund studies that enroll pediatric patients. Of these studies, 2 focus on research in transplantation.