A Message from the Program Director
Tiina K. Urv, PhD
Rare Diseases Clinical Research Network (RDCRN)
Program Director
Division of Rare Diseases Research Innovation (DRDRI)
National Center for Advancing Translational Sciences (NCATS)
National Institutes of Health (NIH)
There are an estimated 10,000 rare diseases that collectively impact millions of people in the United States. These disorders frequently cause substantial morbidity and mortality. Although our understanding of the causes and mechanisms of rare diseases has advanced considerably, effective treatments are available for fewer than 5% of these conditions.
Researchers encounter numerous challenges when developing therapies for rare diseases. These include limited patient populations, a scarcity of disease experts, and often an incomplete understanding of the variability within each disorder and how it progresses over time. Collaborative partnerships are essential to address these obstacles and reduce the impact of rare diseases.
To facilitate progress in addressing these challenges, the Rare Diseases Act of 2002 (Public Law 107-280) directed the National Institutes of Health (NIH) to support "Rare Disease Regional Centers of Excellence" for clinical research, career enhancement, and demonstration of diagnostic, prevention, control, and treatment methods for rare diseases.
Since the inception of this legislation, numerous NIH Institutes, Centers, and Offices (ICOs) —led by the Division of Rare Diseases Research Innovation (DRDRI) at the National Center for Advancing Translational Sciences (NCATS)—have partnered to support the RDCRN:
- National Center for Research Resources (NCRR)
- Office of the Director (OD)
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- National Institute of Neurological Disorders and Stroke (NINDS)
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- National Institute of Dental and Craniofacial Research (NIDCR)
- National Human Genome Research Institute (NHGRI)
- National Institute on Aging (NIA)
- National Institute of Mental Health (NIMH)
- National Cancer Institute (NCI)
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Eye Institute (NEI)
The RDCRN was established to be a collaborative and coordinated network of investigators and patient groups committed to the investigation of rare diseases working in partnership with leaders in technology to enhance communication and sharing of resources through a multidisciplinary approach.
To date, this program has successfully supported 43 individual Rare Diseases Clinical Research Consortia (RDCRCs) that have conducted research on over 300 individual disorders, as well as a Data Management and Coordinating Center (DMCC). The research conducted within the RDCRN has contributed to the Food and Drug Administration (FDA) approving 13 treatments for rare diseases.
In this, the first year of the fifth cycle of the RDCRN, we are proud to support 10 brand-new consortia. We look forward to the many accomplishments of this community.
