On Oct. 8, 2020, The U.S. Food and Drug Administration (FDA) announced that it has awarded 12 new clinical trial research grants totaling more than $15 million over the next four years to enhance the development of medical products for patients with rare diseases. The grants were awarded to principal investigators from academia and industry across the country.

The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program, funded by Congress to encourage clinical development of drugs, biologics, medical devices and medical foods for the treatment of rare diseases. The grants are intended to substantially contribute to marketing approval of products to treat rare diseases or provide essential data needed for development of such products.

The FDA received 89 clinical trial grant applications that were reviewed and evaluated for scientific and technical merit by more than 100 rare disease experts, including members of academia.

Three-quarters (75%) of the new awards fund studies enrolling children, including children as young as one month. These studies target a variety of rare diseases affecting children and have the potential to advance treatments for these diseases. Some of these diseases include Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle loss and weakness; sickle cell disease, a group of inherited red blood disorders which can cause anemia, infections and stroke; and Fanconi Anemia, a rare inherited condition that can result in bone marrow failure and has a high risk for squamous cell cancers.

Two-thirds (67%) of the new awards fund clinical studies of products for use in various rare cancers. For example, one of the new awards aims to advance research in brain cancers affecting children. This study enrolls affected children as young as 36 months and tests an innovative treatment approach to treat brain cancers using an engineered virus therapy designed to target and kill tumor cells while sparing normal brain cells. Another newly funded study evaluates a novel drug delivery system that delivers chemotherapy on a sustained basis directly to the eye to treat retinoblastoma, a rare cancer in the eye most commonly affecting young children. This delivery system may help address current barriers to drug delivery that patients face.

Past studies funded by this program that have resulted in or contributed to recent product approvals, include: fish oil triglycerides, a first FDA-approved lipid emulsion made from fish oil, approved as a source of calories and fatty acids in children with parenteral nutrition-associated cholestasis; and the first treatment (tafamidis meglumine and tafamidis) for heart disease caused by transthyretin mediated amyloidosis.

In addition to the FDA Orphan Products Clinical Trials Grants Program, the agency also supports natural history studies for rare diseases to further advance the FDA’s mission of bringing new therapies to approval and patients.