Grants Awarded to Fund New Clinical Trials for Products to Treat Rare Diseases

The U.S. Food and Drug Administration (FDA) has awarded 12 new clinical trial research grants to principal investigators from academia and industry totaling more than $18 million over the next four years to enhance the development of medical products for patients with rare diseases. The grants were awarded through the Orphan Products Clinical Trials Grants Program, which are intended for clinical studies evaluating the safety and effectiveness of products that could either result in or substantially contribute to FDA approval of products targeted to the treatment of rare diseases. Grant applicants were reviewed and evaluated for scientific and technical merit by more than 100 rare disease experts.

One-third of the new awards aim to accelerate cancer research by enrolling patients with rare forms of cancer, including advanced pancreatic cancer and neck squamous cell carcinoma, myelodysplastic syndrome and acute myeloid leukemia. Another 25 percent of the new awards fund studies evaluating drug products for rare endocrine disorders, including Cushing disease, dopamine agonist intolerant hyperprolactinemia and congenital adrenal hyperplasia. Another study addressed an unmet need in primary sclerosing cholangitis, a rare, chronic and potentially serious bile duct disease. Approximately 42 percent of the grants fund studies that enroll children and adolescents, targeting a variety of rare diseases in children such as Stargardt disease, dystrophic epidermolysis bullosa and bronchopulmonary dysplasia.

To date, the program’s grants have supported research that led to the marketing approval of more than 60 orphan products. “Since its creation in 1983, the Orphan Products Grants Program has provided more than $400 million to fund more than 600 new clinical studies,” said Debra Lewis, OD, acting director of FDA’s Office of Orphan Products Development. “We are encouraged to see so much interest in our grants program and are pleased to support research for a variety of rare diseases that have little or no treatment options for patients.”