The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program.
Officials with the US FDA this week announced that they have awarded 12 new clinical trial research grants totaling more than $18 million over the next 4 years to enhance the development of medical products for patients with rare diseases. These new 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. The grants are intended for academic and industry investigators on clinical studies that evaluate the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products targeted to the treatment of rare diseases. Grant applications were reviewed and evaluated for scientific and technical merit by more than 100 rare disease experts, which included representatives from academia, the National Institutes of Health and the FDA.
The grant recipients, principal investigators and approximate funding amounts, listed alphabetically, are:
“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, O.D., acting director of the 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.”
One-third (33 percent) of the new awards aim to accelerate cancer research by enrolling patients with rare forms of cancer, including advanced pancreatic cancer, head 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 addresses an unmet need in primary sclerosing cholangitis, a rare, chronic and potentially serious bile duct disease.
About 42% of the grants fund studies that enroll children and adolescents, targeting a variety of rare diseases in children.
To date, the program’s grants have supported research that led to the marketing approval of more than 60 orphan products. Among the recent product approvals which were supported by studies funded by this grants program are a marketing approval for a much-needed treatment of HIV-1 infection in adults with multidrug resistant HIV-1 infection and another approval to reduce the acute complications of sickle cell disease in adult and pediatric patients.
The FDA is also currently supporting 6 natural history studies for rare diseases to further advance the mission of bringing new therapies to market.