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Rare Diseases Clinical Research Network (RDCRN)

The Rare Diseases Clinical Research Network (RDCRN) program is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing.

About the RDCRN

Cells under a microscope.

Human eosinophils, a type of white blood cell, are shown isolated from blood. In patients with eosinophilic esophagitis, these cells store and release packages of inflammatory proteins (red) that can damage the throat and esophagus. (Cincinnati Children’s Hospital Medical Center Photo/Julie Caldwell)

The Rare Diseases Clinical Research Network (RDCRN) program advances medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through a network of consortia, scientists from multiple disciplines at hundreds of clinical sites around the world work in partnership with patients and patient advocacy groups to study more than 280 rare diseases. The program is federally mandated by Congress: The Rare Diseases Act of 2002 directed the Division of Rare Diseases Research Innovation to support regional "RDCRCs of Excellence" for clinical research, career enhancement, and demonstration of diagnostic, prevention, control and treatment methods for rare diseases.

The RDCRN is designed to promote highly collaborative, multi-site, patient-centric, translational and clinical research. The Rare Diseases Clinical Research Consortia (RDCRCs) focus on unmet clinical trial readiness needs that will move the field of research forward from its current state to one closer to successfully developing treatments for patients.

The RDCRN facilitates clinical research in rare diseases through support for consortia via:

  • Collaborative activities, including multisite longitudinal studies of individuals with rare diseases and/or clinical trials
  • Career enhancement — encouraging the next generation of rare disease researchers
  • Pilot and feasibility projects

The consortia within the RDCRN are supported by a Data Management and Coordinating Center that provides:

  • Data management to facilitate high quality data standards, collection, storage and sharing
  • Clinical research support
  • Access to information about rare diseases for basic and clinical researchers, academic and practicing physicians, patients and the public

RDCRN Clinical Research

RDCRN consortia support a broad range of clinical research, including clinical trial readiness, natural history studies, identification of biomarkers and outcome measures, and early phase clinical trials.

A Collaborative Environment

Funding and scientific oversight for the RDCRN are provided by NCATS and nine other NIH entities: the Eunice Kennedy Shriver National Institute of Child Health and Human Development; 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 Dietary Supplements. In addition, patient advocacy organizations may contribute funding.


RDCRN News

Breaking Down Prednisone Too Quickly May Short-Circuit Its Benefit for Rare Neuromuscular Disorder

February 15, 2024 - NCATS News

  • Rare Diseases Clinical Research Network (RDCRN)

Chemicals produced when the body breaks down the drug prednisone could indicate which patients with a rare muscle disorder were helped by the drug.

Propelling Cures With Genetics Research: Interview With Joni L. Rutter, PhD, Director of the National Center for Advancing Translational Sciences at NIH

December 14, 2023 - Grantee/Partner News

  • 3-D Tissue Bioprinting
  • Bespoke Gene Therapy Consortium (BGTC)
  • Clinical and Translational Science Awards (CTSA) Program
  • New Therapeutic Uses (NTU)
  • Platform Vector Gene Therapy (PaVe-GT)
  • Rare Diseases Clinical Research Network (RDCRN)
  • Somatic Cell Genome Editing (SCGE)
  • Therapeutics for Rare and Neglected Diseases (TRND)
  • Tissue Chip for Drug Screening

Newborn Screening for Rare Immune Disorders Led to Earlier Treatment, Improved Survival

November 20, 2023 - NCATS News

  • Rare Diseases Clinical Research Network (RDCRN)

An NCATS-led Rare Diseases Clinical Research Network consortium changed what is known about severe combined immunodeficiency.


I Am Translational Science Video

Child neurologist Mustafa Sahin, M.D., Ph.D., shares how advances in translation helped his team provide new treatments to young patients with rare neurodevelopmental disorders.

RDCRN Open Funding Opportunities

Find open funding opportunities for RDCRN.  


Related Research

Rare Diseases Registry Program (RaDaR)

We provide easily accessible guidance to the rare diseases community on how to set up and maintain high-quality registries.

Bespoke Gene Therapy Consortium (BGTC)

The Bespoke Gene Therapy Consortium focuses on developing platforms and standards to speed the development and delivery of gene therapies for rare diseases.

Toolkit

Our Toolkit for Patient-Focused Therapy Development is a collection of online resources to assist patient groups in the research and development of new treatments.

Last updated on March 8, 2024