The U.S. Food and Drug Administration today announced it has awarded six new research grants for natural history studies in rare diseases. The aim of the research is to inform medical product development by better understanding how specific rare diseases progress over time. One potential application of these studies is the opportunity to eventually use natural history models to augment the need for placebo arms in studies of drugs that target very rare disease, where trial recruitment can be challenging.
This is the first time the FDA is providing funding through its Orphan Products Grants Program to conduct rare disease natural history studies. The FDA is providing a total of $6.3 million over the next five years to fund four natural history studies. In addition, through a partnership with the National Institutes of Health’s (NIH) National Center for Advancing Translational Sciences (NCATS), the FDA received $3.5 million to be combined with FDA funding to fund an additional two studies. NCATS’ support was made possible through its Therapeutics for Rare and Neglected Diseases (TRND) program.
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