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. Since 2011, the NIH Clinical Center and the National Center for Advancing Translational Sciences (NCATS) have sponsored Rare Disease Day at NIH as part of this global observance. Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect, and NIH collaborations that address scientific challenges and advance research for new treatments. See the event agenda here. You may register for free here. New NIH Director Monica M. Bertagnolli, MD, is scheduled to speak at 10:55 EST. Sen. Amy J. Klobuchar (D-MN) will speak via video at 9:20 am EST. Families affected by rare diseases will be sharing their experiences throughout the day. Please refer to the full agenda to see the wealth of experience, concern, and effort that are taking place in the Rare Disease arena and will be on display during the full day of moving sessions.
Event Details—Free to Public and Will Also Be Webcast
Rare Disease Day at NIH will be held at the NIH Main Campus (Natcher Conference Center) on Thursday, Feb. 29, 2024, from 9 a.m. to 5 p.m. EST. There will also be a virtual livestream via NIH VideoCast with the event archived for replay afterward. The event agenda will feature panel discussions, rare diseases stories, in-person exhibitors and scientific posters, and an art exhibition. The event is free and open to the public.
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 the rare diseases community.
- Exchange the latest rare diseases information with stakeholders to advance research and therapeutic efforts.
- Shine a spotlight on stories told by patients living with a rare disease, their families, and their communities.
Rare Disease Day at NIH seeks to bring together a broad audience including patients, patient advocates, caregivers, health care providers, researchers, trainees, students, industry representatives, and government staff. Attendees are expected to exercise professionalism, consideration, and respect when speaking, posting, and communicating with others.
NCATS and Rare Diseases Research
Progress in data science and an increased understanding of disease genetics lead experts to agree that more than an estimated 10,000 rare diseases are affecting about 30 million people in the United States. Most of these people are children. In all, nearly 10% of the U.S. population has a rare disease. Rare diseases often are difficult to diagnose — it can take years. Even after an accurate diagnosis, treatment often is not available because fewer than 500 rare diseases have FDA-approved treatments. Research led by NCATS suggests that nationwide medical costs for individuals with rare diseases are likely as high as those faced by people with common diseases, such as cancer and heart failure.
NCATS is committed to using research to address the public health crisis presented by rare diseases. NCATS’ Division of Rare Diseases Research Innovation (DRDRI) facilitates and coordinates NIH-wide research activities, which have the potential to speed development of treatments for multiple rare diseases and ultimately help more patients more quickly. Learn more about DRDRI and NCATS’ rare diseases research programs and the center’s impact on rare diseases.
Partners in Planning
Rare Disease Day at NIH planning committee members include representatives from the following organizations:
- NCATS
- NIH Clinical Center
- National Cancer Institute (NCI)
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institute on Alcohol Abuse and Alcoholism (NIAAA)
- National Institute of Neurological Disorders and Stroke (NINDS)
- Rare Diseases Clinical Research Network’s Coalition of Patient Advocacy Groups (RDCRN-CPAG)
- U.S. Food and Drug Administration (FDA)
- The Children’s Inn at NIH
- EveryLife Foundation for Rare Diseases
- National Organization for Rare Disorders (NORD)
- Undiagnosed Diseases Network Participant Engagement and Empowerment Resource (UDN PEER)
[News release] [Registration] [Agenda]