"Rare is many. Rare is Strong. Rare is Proud."
On February 29, 2020, 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 illnesses affect an estimated 30 million people in the United States. This year’s Rare Disease Day theme is Reframe Rare for Rare Disease Day!
FDA's Rare Disease Day Activities
FDA Public Meeting: FDA will be holding a public meeting on Monday, February 24, 2020. The goal of this meeting is to obtain stakeholder's perspectives on challenges and solutions in rare disease product development and identify commonalities that can support product development across a variety of rare disease. View the links below for more information and to sign up:
FDA's Commissioner, Dr. Stephen M. Hahn, will be making the introductory remarks for the afternoon session. You don't want to miss it!
- Public Meeting - FDA Rare Disease Day 2020: Supporting the Future of Rare Disease Product Development
- Meeting Webcast
- Register through Eventbrite
- FR Notice
- Announcing the FDA's technology modernization efforts for orphan drug designation, Principal Deputy Commissioner and Principal Deputy Commissioner and Acting Chief Information Officer, Dr. Amy Abernethy, and the Director of the Office of Orphan Products Development, Dr. Janet Maynard discuss in their Voices piece how FDA Harnesses Technology and Collaboration to Support Rare Disease Product Development.
- FDA Commissioner, Dr. Stephen Hahn, and Principal Deputy Commissioner and Acting Chief Information Officer, Dr. Amy Abernethy, highlight the important rare disease efforts across the Agency in their Voice piece, FDA Continues Important Work on Treatments for Rare Diseases.
Beyond the Diagnosis Art Exhibit: FDA is hosting 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.
FDA employees may find the exhibit at White Oak Campus on the rear wall of Building 32 by the badging office. We also anticipate having some of the portraits displayed during FDA's Rare Disease Day public meeting. Due to campus security restrictions, access to this exhibit in Building 32 is limited to FDA employees and scheduled visitors.
Center for Biologics Evaluation Research Activity
CBER Public Workshop: CBER will be hosting a public workshop entitled "Facilitating End-to-End Development of Individualized Therapeutics" on Tuesday, March 3, 2020. The purpose of the public workshop is to foster development of individualized therapeutic products for the treatment of one individual or a very small number of patients, based on engineering a product aimed at the specific molecular mechanism underlying a patient’s (or small group of patients’) illness. For more information, click here.
*Check back often for updates on FDA's Rare Disease Day activities*
For questions about Rare Disease Day and/or the Rare Disease Day Public Meeting: