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:

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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.

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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.

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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.

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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).

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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).

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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.

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

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

U.S. Food and Drug Administration Resources

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FDA Drug Development Resources for the Rare Disease Community and More

Academic investigators, patient groups, and small or emerging pharmaceutical and biotechnology companies play a critical role in rare disease drug development and are often the sponsors for rare disease drug development, but they may lack regulatory experience. These resources will help bridge the gap between academic investigation and the regulatory aspects of drug development.


 

Webinar Series: Navigating the World of Rare Disease Clinical Trials from the Patient Perspective

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.

Webinar Series: Rare Advocacy Resource Exchange

The RDCRN Coalition of Patient Advocacy Groups is hosting a webinar series entitled, “Rare Advocacy Resource Exchange.” This webinar series spotlights resources available to help patient advocacy groups accomplish their work. Please view the archived recordings below and check back periodically for new postings:

CPAG Webinar: Research, Rare Disease Day & More: Catching Up with NORD Feb. 17, 2022 from RDCRN on YouTube.

 


CPAG Webinar: Genetic Alliance "Don't Accept a Seat at the Table, Own the Table!" March 24, 2022 from RDCRN on YouTube.

 


CPAG Webinar: Getting to Know the EveryLife Foundation April 12, 2022 from RDCRN on YouTube.

 


CPAG Webinar: Get to Know Global Genes May 12, 2022 from RDCRN on YouTube.

 


CPAG Webinar: The Chan Zuckerberg Initiative's Rare As One Project June 16, 2022 from RDCRN on YouTube.