Rare Diseases

Rare Disease Product Development Gets Big Boost From FDA

FDA awards 15 grants for clinical trials to stimulate product development for rare diseases.

The U.S. Food and Drug Administration announced that it has awarded 15 new clinical trial research grants totaling more than $22 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and industry across the country.

“Given the often small number of patients facing certain rare diseases, there can be limited resources devoted to researching new drugs and unique challenges with recruiting and conducting the clinical trials needed to develop medicines targeted to rare conditions,” said FDA Commissioner Scott Gottlieb, M.D. “For more than 30 years, the FDA has been committed to investing in trials of potentially life-changing treatments for patients with rare diseases, especially in situations where commercial incentives may not be enough to foster the collection of quality data that can ultimately support efficient development and FDA-approval of treatments for patients who lack effective alternatives.

“By helping to support the cost of development of these potential new drugs, and reduce some of the financial risk, we also hope that these grants will lower the cost of the capital needed to develop these products, boost competition and translate into lower prices for successful medicines,” Gottlieb continued. “This can help increase access to resulting therapies.”

The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program, funded by Congressional appropriations, to encourage clinical development of drugs, biologics, medical devices, or medical foods for use in rare diseases. The grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products targeted to rare diseases.

Approximately 33 percent of the new grant awards fund studies to accelerate cancer research by enrolling patients with rare forms of cancer. Sixty percent of these studies target devastating forms of brain and peripheral nervous system cancers, including glioblastoma and anaplastic astrocytoma. One study recruits children as young as one year old with a particularly aggressive form of neuroblastoma.

Other studies span a broad range of diseases and address unmet needs like treating hyperphagia in Prader-Willi syndrome, a genetic disease that primarily affects children, and idiopathic osteoporosis in premenopausal women. Two studies recruit patients with unmet need in sickle cell disease. In addition, one study evaluates a new combination of existing antibiotics to treat pulmonary tuberculosis (TB), including multidrug-resistant TB. TB is a leading killer of HIV-positive patients, and, though not as common…