Resources for Patients and Families

National Institutes of Health Resources

The National Institutes of Health and National Center for Advancing Translational Sciences offer a wide range of information, resources, and services for patients and families. Many of these are dedicated to engaging the patient community throughout the translational science process:

The Genetic and Rare Diseases Information Center (GARD) from NIH’s National Center for Advancing Translational Sciences has easy-to-understand information about rare or genetic diseases. You may also call 1-888-205-2311 (toll-free) to talk to a GARD information specialist.

The NCATS Toolkit for Patient-Focused Therapy Development has a collection of online resources that can help patient groups advance medical research for rare diseases.

The Rare Diseases Registry Program from NIH’s National Center for Advancing Translational Sciences offers online resources for patient groups on setting up and managing a successful registry.

The Undiagnosed Diseases Network (UDN) is a research study funded by NIH that aims to improve the diagnosis and care of patients with undiagnosed diseases. You can contact the UDN Coordinating Center at UDN@hms.harvard.edu or 1-844-746-4836 (toll-free).

ClinicalTrials.gov has information about clinical studies at NIH and other institutions that are currently enrolling patients. For more information on studies conducted at NIH, contact the Patient Recruitment and Public Liaison Office at prpl@mail.cc.nih.gov or 1-800-411-1222 (toll-free).

Rare Diseases Community Resources: NCATS offers free materials and resources to help patients, caregivers, patient support organizations, healthcare providers, and scientists learn about rare diseases and help advance research.

Rare-Diseases-Registry-Program-RaDaR image

The Rare Diseases Registry (RaDaR) Program defines best practices for developing patient registries.

Learn about FDA Patient Engagement: Understand the different opportunities in which patients and caregivers can get involved.

Coalition of Patient Advocacy Groups Webinar Series

From July 2020 through January 2021, the RDCRN-CPAG will host a five-part, bi-monthly webinar series entitled, “Navigating the World of Rare Disease Clinical Trials from the Patient Perspective.” Please check back periodically for new postings.

CPAG Webinar: An Introduction to Clinical Trials, 7-23-20 from RDCRN on Vimeo.

Clinical Trials - Working with your Patient Community, 10-25-20 from RDCRN on Vimeo.

CPAG Webinar: Working with Industry in the Conduct of Clinical Trials, 2-25-2021 from RDCRN on Vimeo.

CPAG Webinar: Working with the FDA in the Conduct of Clinical Trials, 9-29-21 from RDCRN on Vimeo.

The Rare Diseases Clinical Research Network (RDCRN) is funded by the National Institutes of Health (NIH) and led by the National Center for Advancing Translational Sciences (NCATS) through its Office of Rare Diseases Research (ORDR). The RDCRN websites are hosted by the network’s Data Management and Coordinating Center at Cincinnati Children’s Hospital Medical Center, which is funded by NCATS and the National Institute of Neurological Disorders and Stroke (NINDS) under grant number TR002818.