Who We Are

We are the Rare Diseases Clinical Research Network (RDCRN), an initiative of the Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS). We are funded by ORDR, NCATS and collaborating institutes and centers.

Our mission is to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN rare diseases research groups (consortia), researchers and their teams work together with patient organizations (patient advocacy groups) to study nearly 200 rare diseases at clinical centers across the nation and worldwide.

RDCRN Logo

Our History

The RDCRN was established by the ORDR, which is now a part of the NCATS. The RDCRN began in 2003. In total, nearly 200 diseases are studied by the 20 rare disease research groups of the RDCRN.

Press Releases

October 8, 2014: NIH funds research consortia to study more than 200 rare diseases
On October 8, 2014, the NIH announced that physician scientists led 22 consortia consisting of multidisciplinary teams will collaborate with representatives of 98 patient advocacy groups to advance clinical research and investigate new treatments for patients with rare diseases. Funding and scientific oversight for the RDCRN are provided by NCATS and 10 NIH components: the Eunice Kennedy Shriver National Institute of Child Health and Human Development; the National Cancer Institute; the National Heart, Lung and Blood Institute; the National Institute of Allergy and Infectious Diseases; the National Institute of Arthritis and Musculoskeletal and Skin Diseases; the National Institute of Dental and Craniofacial Research; the National Institute of Diabetes and Digestive and Kidney Diseases; the National Institute of Mental Health; the National Institute of Neurological Disorders and Stroke and the Office of the Director. In addition, patient advocacy groups provide funds for many of the projects.
October 5, 2009: NIH Announces Expansion of Rare Diseases Clinical Research Network
May 5, 2006: NIH Launches Clinical Studies Nationwide to Investigate Rare Diseases
February 27, 2003: NIH Establishes Rare Diseases Clinical Research Network

Collaborating Institutes and Centers

Funding and/or programmatic support for the RDCRN is provided by:

Data Management and Coordinating Center (DMCC)

The DMCC at Cincinnati Children’s Hospital Medical Center and the University of Cincinnati is funded by the ORDR at NCATS to facilitate Network operations, research, participant engagement, and data sharing. Specifically, the DMCC:

  • Advances the methods and the practice of rare disease research across the Network and to the broader rare disease community
  • Develops and maintains a leading-edge, shared Commons encompassing data and best practices generated by RDCRN sites, Consortia, and community stakeholders
  • Establishes the RDCRN as a globally connected resource for improving rare disease research across the entire rare disease ecosystem
  • Promotes collaboration and research study readiness, engages patients and families, and creates an “Esprit de Corps” for the RDCRN, through the adoption of Learning System principles

The DMCC is comprised of four Cores, each with distinct attributes and services that it provides to Network participants:

Data Management Core

  • Management system for collection, storage, and quality control of Network-derived research data
  • A web-based platform that allows for real-time tracking of data quality and completeness and that facilitates remote monitoring
  • A portal and tools for research scientists and clinicians to access and manage their own data
  • A portal and tools to share information both within and outside of the RDCRN in a manner that meets all local, federal, and international human subjects protection, data safety and data sharing requirements
  • Cloud computing services and engineering support for facilitating computing and data services across the Network and to the broader research community

Clinical Research Core

  • Protocol development and management support and consultation
  • Biostatistics and study design support and consultation
  • Support in establishing single IRBs
  • Information and guidance for RDCRN members on trans-RDCRN rare disease research issues such as working with industry or navigating the regulatory process
  • Coordination of training issues that cut across topics relevant to multiple RDCRC sites for RDCRN trainees

Engagement and Dissemination Core

  • A broad RDCRN outreach plan for the Network that extends to basic and clinical researchers, academic and practicing physicians, patients, and the general public
  • An internet-based web-portal to serve as a central access point to information generated by the RDCRCs

Administrative Core

  • Overall coordination for the RDCRN and management of RDCRN activities, including steering committee meetings
  • Oversight and coordination of all DMCC Cores
  • Support for Coalition of Patient Advocacy Group meetings
  • Preparation of the annual report for the External Scientific Panel

Key Personnel

Eileen King, PhD contact Principal Investigator

Professor, Division of Biostatistics and Epidemiology

Cincinnati Children’s Hospital Medical Center

Professor, Department of Pediatrics

University of Cincinnati College of Medicine

 

Maurizio Macaluso, MD, DrPH, FACE co-Principal Investigator

Director, Division of Biostatistics and Epidemiology

Cincinnati Children’s Hospital Medical Center

Professor, Department of Pediatrics

University of Cincinnati College of Medicine

 

Peter White, PhD co-Principal Investigator

Rieveschl Professor and Director

Division of Biomedical Informatics

Cincinnati Children’s Hospital Medical Center

Professor and Chair, Department of Biomedical Informatics

University of Cincinnati College of Medicine

Contact email: rd.dmcc@cchmc.org