The FDA awarded Six Grants For Rare Diseases


The FDA announced on Friday it has awarded six new research grants for natural history studies in rare diseases. The focus of the research is to educate medical product development by getting a better understanding on how particular rare diseases progress over a period of time. One potential application of these studies is the opportunity to potentially use natural history models to debate the need for placebo arms in studies of drugs that target rare diseases, where trial recruitment can be difficult.

The will be the first time the FDA is giving funding through its Orphan Products Grants Program to undergo rare disease natural history studies. The FDA is providing a total amount of of $6.3 million over the next five years to fund four natural history studies. Also, through a partnership with the National Institutes of Health’s (NIH) National Center for Advancing Translational Sciences (NCATS), the FDA got $3.5 million to be fused together with FDA funding to fund an additional two studies. NCATS’ endorsement was possible through its Therapeutics for Rare and Neglected Diseases (TRND) program.

“We believe these important studies will provide key information about how these rare diseases develop and progress and can ultimately help in the development of models of disease behavior that can make the development process more efficient,” said FDA Commissioner Scott Gottlieb, M.D. “We’ve been working overtime to develop models that can simulate the behavior of placebo arms in the setting of very rare diseases, where recruiting for clinical trials can be especially hard. We’re grateful to NCATS for its support of this important program, which enabled us to extend our support to two additional studies.”

The natural history of a disease is the course a disease takes from its onset, through the presymptomatic and clinical stages, to a final outcome in the absence of treatment. Because the natural history of many rare diseases remains relatively unknown, efficiently developing diagnostics and therapeutics for these patients poses big challenges.

“One of the challenges we encounter developing therapies for rare diseases is the lack of natural history data to guide the design of successful clinical trials,” said Nora Yang, Ph.D., director of portfolio management and strategic operations in NCATS’ Division of Pre-Clinical Innovation. “We are pleased that we can collaborate with the FDA to fund two natural history studies this year that will help NCATS develop novel treatments for patients who suffer from these devastating diseases.

The FDA got more than 75 grant applications that were administered and evaluated for scientific and technical value by greater than 60 rare diseases and natural history professionals, which entailed representatives from academia, patient groups, NIH and the FDA. The six studies that were awarded cover a wide range of diseases and tackle unmet needs.

Congress appropriates funding to the FDA for the study of rare diseases. The FDA is using these funds for the Orphan Products Grants Program to support natural history studies as well as clinical trials.

Rare diseases, by definition from the Orphan Drug Act, are diseases or conditions with a prevalence of less than 200,000 persons in the U.S. Despite the diseases are separately rare, jointly, the 7,000 known rare diseases affect nearly 30 million Americans.


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