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October 6, 2017

FDA Awards $10M in Grants for Rare Disease Natural History Studies

  • The FDA said today it has awarded six research grants totaling more than $10 million for natural history studies in rare diseases.

    The $10.3 million includes $6.3 million over five years from the FDA’s Orphan Products Grants Program—the first time the FDA is providing funding through the program—and $3.5 million received through a partnership with the NIH’s National Center for Advancing Translational Sciences (NCATS).

    The $3.5 million accounted for most of the $4 million awarded to two of the six grant awardees, with the rest coming from the FDA:

    • Children’s Hospital Corporation, Wen-Hann Tan, MBBS, prospective study in Angelman syndrome, approximately $2 million over 5 years;
    • University of Utah, Nicholas Johnson, M.D., prospective study in myotonic dystrophy type 1 to determine biomarkers and clinical endpoints, approximately $2 million over 5 years.

    The remaining grant awardees, who received the combined $6.3 million from the FDA Orphan Products Grants Program, included:

    • Children's Hospital of Philadelphia, David Lynch, M.D., Ph.D., prospective study in Friedreich’s ataxia, approximately $2 million over 5 years;
    • Columbia University Medical Center, Adi Cohen, M.D., prospective study in pregnancy and lactation-associated osteoporosis, approximately $2 million over 5 years;
    • University of Iowa, Alicia Gerke, M.D., MBA, retrospective study in sarcoidosis, approximately $300,000 over 2 years;
    • University of North Carolina at Chapel Hill, Kenneth Ataga, MBBS, prospective study in sickle cell anemia to determine biomarkers of endothelial function changes in chronic kidney disease, approximately $2 million over 5 years.

    “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,” FDA Commissioner Scott Gottlieb, M.D., said in a statement.

  • Developing Models for Trials

    The FDA said the research is intended to support medical product development by better understanding how rare diseases progress over time. A potential application of these studies, the agency said, 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.

    Since becoming FDA commissioner this year, Dr. Gottlieb has cited the agency’s interest in natural history models.

    Addressing the Regulatory Affairs Professsionals Society (RAPS) 2017 Regulatory Conference on September 11, Dr. Gottlieb said the agency was collaborating with researchers to use computational tools to develop natural history models, based on placebo arms in Parkinson’s disease, Huntington’s disease, Alzheimer’s disease, and muscular dystrophy.

    “If we’re able to make better use of rigorous, reliable natural history models, especially for rare diseases, it can help make clinical trial recruitment more efficient,” Dr. Gottlieb told RAPS conference attendees, according to a transcript posted by on the FDA website.

    The FDA said it 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. Those experts included representatives from academia, patient groups, NIH, and the FDA.

    NCATS’ support was made possible through its Therapeutics for Rare and Neglected Diseases (TRND) program.  

    “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,” added Nora Yang, Ph.D., director of portfolio management and strategic operations in NCATS’ Division of Pre-Clinical Innovation.

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