Cincinnati Children’s to serve as NIH rare disease data center

The National Institutes of Health has awarded a five-year, $28 million grant to Cincinnati Children’s to serve as a data management and coordinating center for the Rare Diseases Clinical Research Network.

The RDCRN program, overseen by NIH’s National Center for Advancing Translational Sciences, is meant to advance medical research on rare diseases by providing support for clinical studies and fostering collaboration, study enrollment and data sharing.

RDCRN’s Data Management and Coordinating Center at Cincinnati Children’s is designed to enable high-quality data collection and analysis as well as information sharing across the network of 20 individual clinical research consortia, supporting studies involving thousands of scientists.

According to Cincinnati Children’s, the center will collect, organize, analyze and share the growing volume of big data on rare diseases. Developing methods for sharing “these evolving information has become a specialty” for the pediatric research and teaching institution.

“For scientists, this project will provide access to a wide range of more-standardized clinical, molecular and genomic data along with clinical images and other forms of data, all of which can be used to conduct more robust studies,” says Eileen King, professor in the Division of Biostatistics and Epidemiology at Cincinnati Children's, who will co-lead the center.


In addition to serving as the centralized source for data storage and sharing among the 20 consortia, Cincinnati Children’s will function as a learning center to help scientists share best practices about conducting rare disease research.

To achieve this, center will offer “state-of-the-art informatics, statistical and epidemiological expertise in clinical research study design and data management technology and processes, in order to guarantee the production of evidence that can support the progression of clinical and translational research from Phase I through Phase III trials to adoption within standard clinical practice,” contends Cincinnati Children’s.

RDCRN research has been conducted at more than 350 U.S. sites and more than 50 foreign sites. According to NIH, rare diseases impact more than 25 million Americans—about half of those impacted are children—and many rare diseases are life-threatening.

“Some of the RDCRN research groups have been working together for 10 or 15 years and have gathered important data and developed a good understanding of the diseases they study, in addition to new potential therapies,” says Anne Pariser, MD, director of the NCATS Office of Rare Diseases Research. “We’re emphasizing the need to be prepared to conduct clinical trials.”