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FDA To Provide $2 Million In Rare Disease Study Funding


Natural History Study

Compared to clinical trials, natural history studies cover a broader period of time from diagnosis of a disease all the way through to end stages in the absence of treatment.

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March 2, 2016 | by Sarah Massey, M.Sc.

On Monday, the US Food and Drug Administration (FDA) announced that they will be providing $2 million in research funding for rare disease natural history studies. This is the first time the FDA has made Orphan Products Grants available to researchers conducting this type of study.

Compared to clinical trials, natural history studies cover a broader period of time from diagnosis of a disease all the way through to end stages in the absence of treatment. The goal of natural history studies of rare diseases is to gain a better understanding of rare disease progression – information that is often incomplete or fully unavailable – which could help in the development of new treatments.

“Rare diseases are often poorly understood,” said Dr. Gayatri Rao director of the FDA’s Office of Orphan Products Development, within the Office of Special Medical Programs. “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.”

There are an estimated 7,000 different rare diseases that affect patients in the US. The funding will be made available to identify a number aspects related to the natural history of rare diseases including identifying subpopulations, developing clinical outcomes measures, and identifying disease biomarkers.

“Despite their importance, it is very difficult for groups, such as patient advocacy organizations, to find funding to conduct natural history studies,” said Rao. “As we observe Rare Disease Day today, 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.”

The funding will be administered through the agency’s new Orphan Products Natural History Grants Program. The FDA’s Orphan Products Grants Program has provided over 570 clinical trials for rare diseases with more than $350 million in finding since the program was launched in 1983.

The FDA will offer more – to a maximum of $400,000 – to studies to be conducted prospectively based on recruitment and clinical examination of individuals with a rare disease. A maximum of $150,000 will be awarded to natural history studies using a retrospective form of data collection, such as surveys.

Grant applications – which are due by October of this year – will be evaluated based on scientific and technical measures, before being approved to receive funding. The FDA expects that successful applicants will receive their grant in March of 2017.

Keywords: FDA, Rare Disease, Natural History Study


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