The 12 new grants will support clinical trials aimed at improving treatments for patients with rare diseases.
The FDA announced that it has awarded 12 new clinical trial research grants totaling $15 million over the next four years to help accelerate the development of medical products for patients with rare diseases.
The agency awarded the grants through the Orphan Products Clinical Trials Grants Program (OOPD), a program funded by Congress to encourage the development of drugs, biologics, medical devices, and medical foods to treat rare diseases.
According to OOPD, rare diseases are those that affect fewer than 200,000 people in the US. Approximately 30 million Americans are affected by 7,000 known rare diseases, but only a few hundred have approved treatments.
The FDA received 89 clinical trial grant applications that were reviewed by more than 100 rare disease experts. The awards focus on supporting product development to meet the needs of patients impacted by a variety of rare diseases, specifically those affecting children and certain cancers.
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Recipients of the grants include Cincinnati Children’s Hospital Medical Center, Columbia University Health Sciences, Massachusetts General Hospital, and University of Texas MD Anderson Cancer Center.
“For more than 35 years, the FDA has been providing much-needed financial support for clinical trials of potentially life-changing treatments for patients with rare diseases. To date, the Orphan Products Clinical Trials Grants Program’s grants have supported research that led to the marketing approval of more than 60 treatments for rare diseases,” said FDA Principal Deputy Commissioner Amy Abernethy, MD, PhD.
“We are encouraged by the amount of interest we continue to have in the grants program and are committed to working with researchers and industry to facilitate and support the study and development of treatments for patients with rare diseases.”
Seventy-five percent of the new awards will fund clinical trials that enroll children, some as young as one month. These trials will focus on a number of rare diseases, including Duchenne Muscular Dystrophy, a genetic disorder that causes progressive muscle loss and weakness, as well as sickle cell disease, a group of inherited blood diseases that can cause anemia, infections, and stroke.
Sixty-seven percent of the new awards will fund clinical trials that develop products for various rare cancers. One of the new awards will focus on advancing research on brain cancers that affect children, enrolling children as young as 36 months and testing an innovative treatment to kill tumor cells. Another study will focus on developing novel therapies for retinoblastoma, a rare eye cancer that commonly affects young children.
The agency’s new awards will advance research for rare diseases and improve treatment for patients affected by these conditions.
“The majority of rare diseases still do not have approved therapies and the FDA is committed to fostering product development in areas of unmet need. The Orphan Products Grants Program is one of several ways that the FDA supports the development of products for rare diseases. Since its creation in 1983, the program has provided more than $400 million to fund more than 600 new clinical studies,” said Janet Maynard, MD, director of the FDA’s Office of Orphan Products Development.
“We are pleased to continue to support research for a variety of rare diseases that have little, or no, treatment options for patients. By helping to spark research, we hope to speed the development of products for rare diseases, and ultimately, make needed treatments available to those patients who need them most.”
Date: October 16, 2019
Source: Health IT Analytics
