New Funding Opportunities Available for Collaborative Rare Diseases Research

GRDR boy playing on swing

A child with WAGR syndrome.

On June 11, 2018, NCATS announced two new Rare Diseases Clinical Research Network (RDCRN) program funding opportunities to support collaborative clinical research on rare diseases. The Center is accepting applications for Rare Diseases Clinical Research Consortia and a Data Management and Coordinating Center (DMCC); both funding opportunities have a deadline of Oct. 9, 2018.

The RDCRN is intended to advance the diagnosis, management and treatment of rare diseases with a focus on clinical trial readiness. Each consortium will promote highly collaborative, multi-site, patient-centric, translational and clinical research with the intent of addressing unmet clinical trial readiness needs.

Approximately 7,000 rare diseases affect an estimated 25 million — approximately one in 10 — Americans, but only a few hundred of these diseases have an approved treatment. Through the RDCRN, physicians, scientists, and their multidisciplinary teams work together with patients and patient advocacy groups to study more than 200 rare diseases at sites across the nation.

“The RDCRN is uniquely positioned to foster important clinical research advances and bridge translational gaps in the understanding and treatment of rare diseases,” said Anne Pariser, M.D., director of the NCATS Office of Rare Diseases Research, which oversees the RDCRN.

NIH launched the RDCRN program in 2003. RDCRN-supported research includes natural history studies that chart the normal progression of a disease over time, as well as studies focused on the effects of treatments or ways to detect and diagnose disease. Such information is necessary to conduct and evaluate clinical trials for new therapies.

The RDCRN consortia have a long history of accomplishment. For example, in 2014, researchers studying rare immune system disorders through the RDCRN’s Primary Immune Deficiency Treatment Consortium published a report in the New England Journal of Medicine showing the importance of evaluating newborns and early treatment in improving the lives of individuals with a rare immune disease. More recently, in ongoing studies, clinicians and researchers with the RDCRN’s Lysosomal Disease Network have collaborated with scientists at a biotechnology company to examine the use of gene editing in rare disease patients.

The DMCC manages the collection, storage and quality control of clinical research data for the network. The RDCRN will emphasize data standards, which will facilitate data sharing among its members and others outside the network also studying rare diseases. NCATS will provide “cloud” computing services through the DMCC, enabling greater data accessibility and use. The DMCC will provide expertise and consulting to research consortia in several areas, such as the development and management of research study protocols, biostatistics, and study design.

The NIH Institutes, Centers and Offices that partner with NCATS on the RDCRN program include the National Cancer Institute, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the Office of Dietary Supplements, the National Institute of Allergy and Infectious Diseases, the National Institute of Diabetes and Digestive and Kidney Diseases, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institute of Neurological Disorders and Stroke, the National Institute of Mental Health, the National Institute of Dental and Craniofacial Research, and the National Heart, Lung and Blood Institute.

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Posted June 2018