Resources for Patients and Families

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A variety of government agencies and other organizations offer a wide range of information, resources, and services for patients, families, and patient advocacy groups. Many of these are dedicated to engaging the patient community throughout the translational science process.

Genetic and Rare Diseases Information Center (GARD)

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.

Toolkit for Patient-Focused Therapy Development

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.

Rare Diseases Registry Program

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.

Undiagnosed Diseases Network (UDN)

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

ClinicalTrials.gov offers information about clinical research 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.

Learn About FDA Patient Engagement

Understand the different opportunities in which patients and caregivers can get involved.

Coalition of Patient Advocacy Groups Webinar Series

The RDCRN Coalition of Patient Advocacy Groups hosts webinar series that spotlight resources available to help patients with rare diseases and support patient advocacy groups in their work.

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.