On this page:
- FDA's Rare Disease Day Activities
- FDA Voices
- FDA Rare Disease Photo Video Project
- Artfully Rare - Beyond the Diagnosis
- Other FDA Activities Relevant to Rare Diseases
- Contact Information
On March 5, 2021, FDA will join the global observance of Rare Disease Day, which was created to raise awareness about the 7,000 known rare diseases, many of which have no treatment. Although each rare disease affects fewer than 200,000 Americans, in total, these disease affect an estimated 30 million people in the United States. This year’s FDA Rare Disease Day will be virtual and we are looking forward to having you join!
FDA Public Meeting: FDA will be holding a virtual public meeting on Friday, March 5, 2021, from 9:00 a.m. to 4:00 p.m. EST. The purpose of this meeting is to highlight strategies to support rare disease product development. Patients, patient advocates, researchers, and medical product developers may benefit from attending this public meeting on rare disease product development. During presentations and panel discussions various stakeholders will share their perspectives on and experiences in rare disease product development.
FDA's Acting Commissioner, Dr. Janet Woodcock, will be making the introductory remarks for the afternoon session. You don't want to miss it!
View the links below for more information and to sign up:
- Public Meeting - FDA Rare Disease Day 2021
- Meeting Packet with Agenda and Speaker Bios
- Meeting Webcast
Acting FDA Commissioner, Janet Woodcock, and the Director of the Office of Orphan Products Development, Janet Maynard, reflect on the progress that has been made, and the work that needs to be done, to advance rare disease treatment in their FDA Voice Piece, FDA Shows Sustained Support of Rare Disease Product Development During the Public Health Emergency.
The FDA captured brief stories from patients with rare diseases and FDA staff who work on rare disease product development across the Agency. The opinions and viewpoints expressed in these videos are personal opinions and viewpoints.
Of note, population estimates may change over time and there are different definitions of rare diseases. If you have questions regarding the criteria for specific orphan product designations, please see FDA's webpage on Developing Products for Rare Diseases and Conditions.
Stories from the Rare Disease Community
Rare Disease Work Across the FDA
FDA’s Rare Disease Day 2021 public meeting will include portraits from the Beyond the Diagnosis Art Exhibit to showcase portraits of diverse children with rare diseases. This exhibit has traveled to medical schools, research institutes and hospitals around the globe to encourage the medical community to look "beyond the diagnoses" of patients. This year, Beyond the Diagnosis is featuring the Artfully Rare - Beyond the Diagnosis 3D Art Exhibit. "Beyond the Diagnosis unites art and science to inspire research and innovations of treatments for people living with rare and neglected diseases."
SHIP-MD 2021 (Virtual Workshop): The Critical Path Institute (C-Path) in collaboration with the U.S. Food and Drug Administration’s (FDA) Center for Devices and Radiological Health (CDRH), AdvaMed, the American Academy of Pediatrics (AAP) and multiple pediatric health system leaders, is proud to announce the System of Hospitals for Innovation in Pediatrics – Medical Devices (SHIP-MD) Virtual Workshop to be held on February 9-11, 2021. The workshop will focus on the acceleration of medical device development for the unique needs of children. You may register for the workshop here.
Evaluating RWE from Observational Studies in Regulatory Decision Making (Virtual Workshop): The U.S. Food and Drug Administration and the Duke-Margolis Center for Health Policy will hold a two-day virtual workshop on February 16-17, 2021, focused on considerations about observational (non-interventional) study design and conduct when using evidence from observational studies to inform regulatory decisions. Individuals can learn more and register for the workshop at FDA’s Evaluating RWE from Observational Studies in Regulatory Decision-Making webpage.
PAS Listening Sessions: Listening Sessions are one of many way patients, caregivers, and their advocates can share their experience with a disease or condition by talking directly with FDA staff. Learn more about the Patient Listening Sessions here.
*Check back for updates on FDA's Rare Disease Day activities*
Email: OOPDOrphanEvents@fda.hhs.gov Call: 301-796-7634