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Regulatory News | 29 February 2016 | By Michael Mezher
The US Food and Drug Administration (FDA) will award $2 million in grants to fund natural history studies for rare diseases.
Natural history studies are studies that follow the progression of a disease from before it begins through its outcome, which may include a resolution of symptoms, lifelong chronic condition or a patient's death.
For example, natural history studies have identified the six-minute walk distance as an endpoint in clinical trials for potential treatments for Duchenne muscular dystrophy.
For natural history studies, FDA is offering two different grant levels depending on the type of study proposed. For prospective studies involving clinical examinations of patients, the agency will award up to $400,000 per year, for up to five years. For retrospective studies or survey studies FDA is offering up to $150,000 per year for up to two years.
FDA says it will accept applications until 14 October 2016 and will rely on a panel of rare disease and natural history experts to review the proposals. The agency plans to begin funding the winning grants in March 2017.
"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," said Gayatri Rao, director of FDA's Office of Orphan Drug Products.
For many rare diseases, FDA says that natural history studies can help identify clinical endpoints and biomarkers that could be used to measure a treatment's effectiveness.
In August, FDA also released a new draft guidance, Rare Diseases: Common Issues in Drug Development, encouraging drugmakers to conduct natural history studies to inform clinical development programs. However, industry groups have called on FDA to clarify its guidance and give examples of how natural history studies can be used to support a product's approval.
The announcement of the grant program coincides with Rare Disease Day, which began in 2008 to raise awareness about rare diseases. According to FDA there are more than 7,000 rare diseases that affect nearly 10% of Americans.
FDA, FDA Voice