Overview

Sponsored by the U.S. Food and Drug Administration (FDA) and the National Center for Advancing Translational Sciences (NCATS), FDA-NIH Rare Disease Day 2025 will be held both in-person at the NIH main campus (Natcher Conference Center) and virtually on Thursday, Feb. 27 and Friday, Feb. 28, 2025, from 10 a.m. to 4 p.m. EST. The event aims to provide the rare disease community—patients, caregivers, families, patient advocate groups, researchers, clinicians, health care providers, and trainees/students—with information relevant to their lived experiences, including activities by FDA and NIH that promote research and product development for this underserved population. The goals are to: 

• Demonstrate the NIH and FDA commitment to advancing research and regulatory support for people affected by rare diseases. 
• Highlight NIH and FDA 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 caregivers, and their communities. 

FDA-NIH Rare Disease Day 2025 seeks to bring together a broad audience including patients, patient advocates, caregivers, health care providers, researchers, trainees, students, industry representatives, and government staff. The event is free and open to the public. Attendees are expected to exercise professionalism, consideration, and respect when speaking, posting, and communicating with others. This year’s event will feature panel discussions, rare diseases stories, in-person exhibits and scientific posters, and an art exhibition. 

Attending In-Person?

Check-in will open at 9 a.m. EST. Before arriving at NIH, please review our tips for accessing the NIH main campus and other visitor information. Be sure to allow at least 45 minutes to pass through security at the NIH Gateway Center. 

In the days leading to the event, attendees should self-monitor for symptoms of any illness. If you are experiencing symptoms, please do not attend the event in person. Masks and hand sanitizer will be available at check-in/registration. 

If you do not want your photo taken during the event, please notify staff at the registration desk. 

Exhibits & Scientific Posters

Exhibitors and poster-presenters may request physical space in Natcher Conference Center and be listed in the event app. Based on previous attendee feedback, virtual exhibit and poster profiles are not planned this year. 

To sign up for an exhibit or poster, please complete an exhibit request form or poster request form, by 5pm ET on February 19th, 2025. Exhibit and poster capacity in Natcher Conference Center is limited, so requests for in-person space will be assigned on a first-come, first-served basis. 

If you previously applied and now are unable to participate, please let Ashley Cornett know. 

Event App

The FDA-NIH Rare Disease Day 2025 event app will allow participants to connect with anyone who has created and shared a profile on the app. Available for both computers and mobile devices, attendees may use the app to: 

• Personalize schedules and access the event's livestream. 
• Submit questions and chat. 
• View a list of in-person exhibitors and poster presenters. 
• Directly message or virtually meet with others. 

Partners in Planning

Planning committee members include representatives from: 

• NIH: National Center for Advancing Translational Sciences (NCATS), Clinical Center, National Cancer Institute (NCI), National Eye Institute (NEI), National Institute on Alcohol Abuse and Alcoholism (NIAAA), NIH National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), National Institute of Neurological Disorders and Stroke (NINDS), Office of Research on Women’s Health (ORWH) 
• Rare Diseases Clinical Research Network’s Coalition of Patient Advocacy Groups (RDCRN-CPAG) 
• The Children’s Inn at NIH 
• FDA: Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH), Center for Drug Evaluation and Research (CDER), Office of the Commissioner (OC), Oncology Center for Excellence (OCE) 
• EveryLife Foundation for Rare Diseases 
• National Organization for Rare Disorders (NORD) 
• argenx