Rare Disease Day® takes place worldwide, typically on or near the last day of February each year, to raise awareness among policymakers and the public about rare diseases and their impact on patients’ lives. Each year, NCATS and the NIH Clinical Center (CC) sponsor Rare Disease Day at NIH as part of this global observance. This year’s global theme is “bridging health and social care.”
2019 Event Information
Thursday, Feb. 28, 2019
8:30 a.m.-4:00 p.m. ET
Main Auditorium, Natcher Conference Center, Building 45*
National Institutes of Health
*Note new, bigger location for 2019!
Sponsored by NCATS and the CC, this year’s event will feature interactive panel discussions on collective research models for rare diseases, patient registries, rare cancer research initiatives, and "no disease left behind, no patient left behind." New this year will be a presentation of the first ever Zebbie award for the NCATS Rare Diseases are Not Rare! Challenge. Other highlights include posters and exhibits by rare disease groups and researchers as well as artwork, videos and CC tours. Admission is free and open to the public. In association with Global Genes®, participants are encouraged to wear their favorite pair of jeans. Be sure to follow the event on social media using #RDDNIH.
Partners in Planning
Planning committee members include representatives from NCATS, the CC, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Cancer Institute, the National Institute of Neurological Disorders and Stroke, the National Organization for Rare Disorders, the Rare Diseases Clinical Research Network’s Coalition of Patient Advocacy Groups, the Food and Drug Administration, and The Children’s Inn at NIH.
Event participants and partners include representatives from the Alpha-1 Foundation; the American Partnership for Eosinophilic Disorders; the Barth Syndrome Foundation; Beyond the Diagnosis; the Campaign Urging Research for Eosinophilic Disease; the Consortium of Eosinophilic Gastrointestinal Disease Researchers; Count Me In; the Developmental Synaptopathies Consortium; the Eosinophilic Family Coalition; the EveryLife Foundation for Rare Diseases; Genetic Alliance; Global Genes®; the Inherited Neuropathies Consortium; the International Rare Diseases Research Consortium; Massachusetts General Hospital; My Pediatric and Adult Rare Tumor Network; the National Heart, Lung, and Blood Institute; Rare Disease Legislative Advocates; Sarcoma Alliance for Research through Collaboration; TargetCancer Foundation; the Tuberous Sclerosis Alliance; and the Vasculitis Foundation.
About Rare Disease Day
EURORDIS sponsored the first Rare Disease Day in Europe on Feb. 29, 2008. The United States joined the first global observance the following year along with 23 other countries. Visit Rare Disease Day for more information.
About Rare Disease Day at NIH
Each year, the slogan for NIH’s event has been “Patients & Researchers — Partners for Life.” This slogan aligns with NCATS’ philosophy that researchers must work closely with patients, families, caregivers and advocacy groups to maximize the chances for success in advancing rare diseases research. This philosophy has been put into practice in NCATS’ Toolkit for Patient-Focused Therapy Development, Rare Diseases Clinical Research Network, Therapeutics for Rare and Neglected Diseases program, and Genetic and Rare Diseases Information Center, among other efforts.
The goals of Rare Disease Day at NIH are to:
- Demonstrate the NIH commitment to helping people with rare diseases through research;
- Highlight NIH-supported rare diseases research and the development of diagnostics and treatments;
- Initiate a mutually beneficial dialogue among public and private researchers, patients, patient advocates and policymakers;
- Exchange the latest rare diseases information with stakeholders to advance research and therapeutic efforts; and
- Put a face on rare diseases by sharing stories of patients, their families and their communities.
NCATS and Rare Diseases Research
About 7,000 rare diseases affect humans, of which only a few hundred have any treatment. Although each rare disease affects fewer than 200,000 Americans, in total, these illnesses affect an estimated 30 million people in the United States. Since rare diseases often are difficult to diagnose, it can take years to obtain an accurate diagnosis. Even after a proper diagnosis, treatment often is unavailable, because only about 5 percent of rare diseases have a treatment approved by the Food and Drug Administration.
As a result, rare diseases are devastating and costly for patients, their families and the nation as a whole. Most rare diseases are serious or life-threatening, chronic and progressive disorders that place substantial medical and financial burdens on patients and their families.
Through its work to improve health through smarter science, NCATS supports collaborative and innovative approaches to research on rare diseases. These efforts have the potential to speed development of treatments for multiple rare diseases and ultimately help more patients more quickly.