The National Institutes of Health (NIH) is providing $29 million in funding to study more than 200 rare diseases. The initiative aims to expand the Rare Diseases Clinical Research Network (RDCRN), which is led by NIH’s National Center for Advancing Translational Sciences (NCATS). Physician scientists at 22 consortia will collaborate with representatives of 98 patient advocacy groups to advance clinical research and investigate new treatments.
NIH said researchers at the 22 RDCRN consortia will conduct a minimum of two multisite clinical studies, including one longitudinal natural history study for a group of at least three related rare diseases. Support will be provided by the RDCRN Data Management and Coordinating Center with technologies and tools to collect and analyze standardized clinical research data as well as supporting study design.
Six new RDCRN consortia have been established including bone diseases, lung diseases, food allergy disorders, and three separate neurological diseases concentration areas— amyotrophic lateral sclerosis and related disorders, autism and intellectual disabilities, and frontotemporal lobar degeneration.
Each consortium will partner with relevant patient advocacy groups. Network consortia also establish training programs for clinical investigators interested in rare diseases research, provide information to the public about the rare diseases that they study, and enable proof of concept in clinical research studies, according to NIH.
NCAT said it seeks to tackle challenges to developing rare disease treatments such as difficulties in diagnosis, widely dispersed patients and scientific experts, a perception of high risk, and a lack of data from natural history studies.
“NCATS seeks to tackle these challenges in an integrated way by working to identify common elements among rare diseases,” said NCATS Director Christopher P. Austin, M.D. “The RDCRN consortia provide a robust data source that enables scientists to better understand and share these commonalities, ultimately allowing us to accelerate the development of new approaches for diagnosing and treating rare diseases.”
Recently, NIH has been on a funding spree. Last week, DecImmune was awarded $3 million to support studies for vascular inflammatory diseases treatment. In September, the BRAIN initiative was granted $46 million and the Centers for Collaborative Research in Fragile X program was provided with $35 million in funding over the next five years.