Last updated: 03-15-2023

Rare Diseases Community Resources

 

NCATS offers free materials and resources to help patients, caregivers, patient support organizations, health care providers and scientists learn about rare diseases and help advance research on them. You can use the resources below on social media, on web pages, or at meetings, clinics and other places to raise awareness and to connect with the rare diseases community.

NCATS Director on Rare Diseases

NCATS Director Joni L. Rutter, Ph.D., guest authored two blog posts about rare diseases. Dr. Rutter’s post on the NIH Director’s Blog discusses the public health challenge of rare diseases. Her post on the National Library of Medicine Director’s blog highlights how NCATS uses data-driven approaches to speed solutions for rare diseases.

Fact Sheet

Get the facts about rare diseases — what they are, whom they affect, and how NCATS supports innovative approaches for understanding and treating rare diseases.

The first page of a fact sheet about rare diseases with a banner image at the top followed by text.

Click the image above to view/download the fact sheet.

Infographic

Did you know that more than 30 million people in the United States have a rare disease? Learn more and spread the word with our infographic about rare diseases.

A graphic image that has stylized depictions of facts and statistics about rare diseases and includes information such as: GARD provides high-quality, widely used resources to educate, engage and empower the rare diseases community; GARD offers disease information pages for more than 6,500 rare diseases; GARD staff have answered more than 90k questions; RDCRN is funded to accelerate medical research across rare diseases; the RDCRN teams study approximately 200 diseases; RDCRN includes 140 patient advocacy groups and partners; 1 out of every 10 people in the U.S. are affected by a rare disease; 1 in 2 patients diagnosed with a rare disease are children; it takes 3 or more years to receive an accurate diagnosis of a rare disease; there are less than 500 approved treatments across 7k rare diseases.

Credit: National Center for Advancing Translational Sciences

Click the image above to download the full-size image.

Resources

For Patients

Stylized text of the Genetic and Rare Diseases Information Center name

Access resources through NCATS’ Genetic and Rare Diseases (GARD) Information Center:

  • Call the GARD Information Center at 1-888-205-2331 to speak to an information specialist about rare or genetic diseases in English or Spanish.
  • Browse GARD’s list of rare diseases and related terms. The list includes the main name for each condition, a summary, symptoms, statistics, research, advocacy organizations and patient registries.
  • Download the GARD handout (PDF - 638 KB).
Stylized text of the Genetic and Rare Diseases Information Center name

The Rare Diseases Clinical Research Network (RDCRN) — coordinated by NCATS in collaboration with nine NIH Institutes and Centers — brings scientists at hundreds of clinical sites from around the world together with their patient support organization partners to study more than 200 rare diseases. The network consists of 20 individual consortia and a Data Management and Coordinating Center that work together with representatives of nearly 130 patient advocacy groups to advance rare diseases clinical research and investigate new treatments for patients. Learn more about currently funded rare diseases research groups and collaborating patient organizations. Download the RDCRN fact sheet.

For Patient Support Organizations

Stylized text of the Toolkit for Patient-Focused Therapy Development name

Whether your patient group is new to supporting rare diseases research or already involved in research initiatives, the NCATS Toolkit for Patient-Focused Therapy Development can provide reliable, relevant information about how to engage in the therapy development process, from discovery through post-approval safety and effectiveness monitoring. Learn about the NCATS Toolkit for Patient-Focused Therapy Development in this fact sheet (PDF - 645KB) or download the NCATS Toolkit handout (PDF - 265 KB).

Stylized text of the Rare Diseases Registry Program name

Looking for advice on setting up and maintaining quality registries for rare diseases to stimulate research? The Rare Diseases Registry Program (RaDaR) explains the different types of patient registries, provides step-by-step guidance on setting up and maintaining registries, and offers resources to promote best practices and data sharing. Download the RaDaR handout (PDF - 423 KB).

Challenges

The Rare Diseases Are Not Rare! Challenge, led by NCATS’ Division of Rare Diseases Research Innovation, seeks creative ways to raise awareness for all rare diseases, encourage collaborations across the community and highlight the importance of rare diseases research and the need for new treatments. The Challenge originally ran in 2018 and returned in 2020. Browse the gallery of creative works from 2020’s winning and honorable mention submissions, including poems, spoken-word performances and personal stories.

Social Media

Stay connected and up to date on rare diseases research! By including the hashtag #RareDiseases in your social media posts, you can help us all be part of the same conversation. Follow @ncats_nih_gov and @DRDRI_NCATS on Twitter for the latest news from NCATS and the NCATS Division of Rare Diseases Research Innovation.

You can use the messages and graphics below to share NCATS’ rare diseases resources on your own social media pages.

Click the images below to download them to use on your social media pages.

People walk down a street facing away from the camera. In the center there is a white rectangle with text reading, “An estimated 30 million people in the United States are affected by rare diseases.” The NCATS logo is in the lower left.
People walk down a street facing away from the camera on the right. On the left, text overlays white space reading “An estimated 30 million people in the United States are affected by rare diseases.” The NCATS logo is in the lower left.
A rectangle with a color gradient behind white text, which says “Only 5% of rare diseases have approved treatments.” To the right of the text box is a stylized image of a stethoscope. The NCATS logo is in the lower left.
Against a color gradient background, three stylized hands appear and touch. To the right, colored text in a white rectangle reads, “Working together to advance rare diseases research.” The NCATS logo appears in the lower right.