Rare Daily Staff

The U.S. Food and Drug Administration today said it has awarded $6.3 million over the next five years to fund six new research grants for natural history studies in rare diseases, the first time the agency is providing funding through its Orphan Products Grants Program to conduct such studies.

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.

Through a partnership with the National Institutes of Health’s National Center for Advancing Translational Sciences, 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 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. “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.”

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

The FDA received more than 80 grant applications that were reviewed and evaluated for scientific and technical merit by more than 60 rare disease and natural history experts, which included representatives from academia, patient groups, NIH, and the FDA. The six studies awarded cover a broad spectrum of diseases and address unmet needs.

Grants being funded by the FDA:

Children’s Hospital of Philadelphia, David Lynch, Prospective study in Friedreich’s ataxia, approximately $2 million over 5 years

Columbia University Medical Center, Adi Cohen, Prospective study in pregnancy and lactation-associated osteoporosis, approximately $2 million over 5 years

University of Iowa, Alicia Gerke, Retrospective study in sarcoidosis, approximately $300,000 over 2 years

University of North Carolina at Chapel Hill, Kenneth Ataga, Prospective study in sickle cell anemia to determine biomarkers of endothelial function changes in chronic kidney disease, approximately $2 million over 5 years

The two additional grants primarily being funded by NIH/NCATS include:

Children’s Hospital Corporation, Wen-Hann Tan, Prospective study in Angelman syndrome, approximately $2 million over 5 years

University of Utah, Nicholas Johnson, Prospective study in Myotonic Dystrophy Type 1 to Determine Biomarkers and Clinical Endpoints, approximately $2 million over 5 years.

October 6, 2017