FDA Providing $2 Million in Research Grants for Rare Disease Studies

FDA Providing $2 Million in Research Grants for Rare Disease Studies
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February 29, 2016

The Food and Drug Administration (FDA) is making available $2 million in research grants to fund studies focusing on rare diseases.

The funding will help researchers collect data on how specific rare diseases progress in patients, which will help in the development of treatments for these diseases. Funding for the grants is being provided through its Orphan Products Grants program.

Natural history is the course the disease takes in affected people from the time a person develops the disease through the final stages without treatment. For many rare diseases, this type of information is often not available or incomplete.

"Rare diseases are often poorly understood," said the FDA's Gayatri Rao. "Not understanding how a rare disease progresses is often a major obstacle in the development of life-saving medical products. Information about a disease's natural history can aid in clinical trial design, identify study end points and lead to faster, better trials – hopefully leading to new and effective diagnostics and treatments."

Rare diseases are those that affect less than 200,000 people in the United States. As a whole, however, 7,000 known rare diseases affect about 30 million Americans.

The increased funding from the FDA should help patient advocacy organizations conduct these studies and close a funding gap. Grants will be a maximum of $400,000 in total costs for five years for future research and a maximum of $150,000 in total costs to cover two years of previous studies. Funding is expected to begin March 2017.