March 05, 2016
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FDA funds $2 million for natural history studies in rare diseases

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The FDA will provide $2 million in research grants to fund studies assessing how rare diseases progress in patients over time, according to a press release.

The goal of the studies is to gain a better understanding of the natural history of rare diseases for product development support, the release said. It is the first time the FDA is offering funds from its Orphan Products Grants to support studies for rare diseases, defined as diseases or conditions that affect less than 200,000 people in the United States, according to the FDA. Overall, there are approximately 7,000 known rare diseases that affect 30 million patients in the U.S.

Rare diseases are often poorly understood,” Gayatri Rao, MD, JD, director of the FDA’s Office of Orphan Products Development, said in the release. “Not understanding how a rare disease progresses is often a major obstacle in the development of lifesaving medical products. Information about a disease’s natural history can aid in clinical design, identify study end points and lead to faster, better trials — hopefully leading to new and effective diagnostics and treatments.”

With the funds from the new Orphan Products Natural History Grants Program, researchers will be able to characterize the natural progression of rare diseases, identify subpopulations and develop or confirm outcome measures, disease biomarkers and diagnostics.

The FDA is offering a maximum of $400,000 up to 5 years for prospective natural history studies, and $150,000 up to 2 years for retrospective or survey studies, according to the release. Grant applications are due Oct. 14, and will be evaluated by a panel of rare disease and natural history experts. The funds are expected to be available for use by March 2017.

“Despite their importance, it is very difficult for groups, such as patient advocacy organizations, to find funding to conduct natural history studies,” Rao said in the release. “We are excited to announce a program that will bridge some of this funding gap and complement existing efforts to help bring new and important therapies to rare disease patients.”