Le Lézard
Classified in: Health
Subjects: SVY, EXE

FDA awards six grants for natural history studies in rare diseases

SILVER SPRING, Md., Oct. 6, 2017 /PRNewswire-USNewswire/ -- 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. 

U.S. Food and Drug Administration (FDA) logo (PRNewsFoto/FDA)

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.

"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 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:

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

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, as defined by the Orphan Drug Act, are diseases or conditions with a prevalence of less than 200,000 persons in the United States. Though the diseases are individually rare, together, the 7,000 known rare diseases affect approximately 30 million Americans.

For additional information:

FDA Office of Orphan Products Development

NIH NCATS Therapeutics for Rare and Neglected Diseases

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Media Inquiries:
FDA: Sandy Walsh, 301-796-4669, Sandy.Walsh@fda.hhs.gov
NIH NCATS: Steven Benowitz, 301-435-0888, Steven.Benowitz@nih.gov 
Consumer Inquiries: 888-INFO-FDA


SOURCE U.S. Food and Drug Administration

These press releases may also interest you

at 17:00
MELBOURNE, Fla., Aug. 14, 2018 /PRNewswire-PRWeb/ -- Ross A. Clevens, MD, FACS is excited to announce the arrival of CoolSculpting 2.0, the next generation of freezing fat, at Clevens Face and Body Specialists. Dr. Clevens is known for introducing...

at 17:00
The American Association of Stem Cell Physicians (AAOSCP) hosted its weekend conference this Aug. 10-12, 2018 in Miami Florida, at the Mandarin Oriental. The event was sold out. Its huge success was thanks to the participation from some of the most...

at 16:50
U. S. News & World Report released its 2018-2019 Best Hospitals National Ranking and the Department of Orthopaedic Surgery at Rush University Medical Center (RUMC), consisting of Midwest Orthopaedics at Rush (MOR) physicians, moved from the fifth to...

at 16:50
WASHINGTON, D.C., Aug. 14, 2018 /PRNewswire-PRWeb/ -- Children's National Health System and PetSmart Charities today announced the animal welfare funder's commitment of more than $270,000 to expand a popular pet therapy program. The grant will...

at 16:45
Vanda Pharmaceuticals Inc. (Vanda) announced today that the U.S. Court of Appeals for the Federal Circuit denied petitions for panel rehearing and rehearing en banc filed by West-Ward Pharmaceuticals, a subsidiary of Hikma Pharmaceuticals, relating...

at 16:41
Rockwell Medical, Inc. (the "Company") today provided a business update, financial results for the second quarter ended June 30, 2018 and announced the restatement of its financial results for the first quarter ended March 31, 2018. Recent Business...

News published on 6 october 2017 at 10:47 and distributed by: