The U.S. Food and Drug Administration announced that it has awarded six new clinical trial research grants to principal investigators from academia and industry totaling over $16 million over the next four years. These trial research grants, awarded through the Congressionally-funded Orphan Products Grants Program, enhance the development of medical products for patients with rare diseases.
“Now, more than ever, we see the important role of these FDA grants to support clinical trials of potentially life-changing treatments for patients with rare diseases,” said FDA Commissioner Stephen M. Hahn, M.D. “As interest in the program has grown, so has the hope for promising approved therapies for patients with rare diseases that currently have no treatment options. These important clinical trials are at a critical time where additional resources are needed to support rare disease research during the COVID-19 pandemic.”
The FDA received 47 clinical trial grant applications that were reviewed and evaluated for scientific and technical merit by more than 90 rare disease and clinical trial experts, including members of academia. The grants awarded support clinical studies of products that address unmet needs in rare diseases or conditions, or provide highly significant improvements in treatment or diagnosis. Below is a complete list in alphabetical order:
“Since its inception over 35 years ago, the Office of Orphan Products Development has successfully continued to support necessary research for rare diseases to inform product development, support scientific advancements, contribute to the development of treatment guidelines, and result in marketing approvals especially for rare diseases without prior-approved therapies. This grants program continues to fill important gaps in rare disease product development, such as supporting recent FDA approvals that help address unmet needs in the treatment of rare diseases,” said Janet Maynard, M.D., M.H.S., director of OOPD.
Examples of recent approvals supported by the grants program include teprotumumab, for the treatment of a rare thyroid eye disease, and triheptanoin, a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders.
This year, as there are new challenges and increased costs for clinical trials due to the COVID-19 pandemic, the FDA remains committed to supporting rare disease research by providing existing grantees with additional funding. These new resources allow ongoing studies to implement necessary steps to allow their research to continue and assure the safety of study participants, to maintain compliance with good clinical practice, and to minimize risks to trial integrity.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.