FDA Observes Rare Disease Day

Patient Affairs Staff (PAS)

Food and Drug Administration

U.S. Department of Health and Human Services

FDA Observes Rare Disease Day

On February 28, 2019, FDA joins 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 Bridging Health and Social Care in recognition that rare diseases deeply impact patients, caregivers and their families.

In observance of this day, the Patient Affairs Staff (PAS) wants to highlight several announcements, including the announcement of a public meeting on patient perspectives in rare diseases.

• Public Meeting on 4/29: FDA will host a public meeting - “Patient Perspectives of the Impact of Rare Diseases: Bridging the Commonalities”. The purpose of the meeting is to obtain patients’ and caregivers’ perspectives on the impacts of rare diseases on daily life and to assess commonalities that may help the Agency and medical product developers further understand and advance the development of treatments for rare diseases. The meeting will be held on April 29, 2019 from 1-5 pm at FDA’s White Oak campus. Can’t attend in person? Join by webcast or submit comments to the docket.
• Register here. Please register by April 15th
• View the Federal Register notice
• FDA Voices post: In the latest FDA Voices post, Office of Orphan Products Development (OOPD) Director, Janet Maynard, M.D., M.H.S, and Patient Affairs Staff (PAS) Director, Andrea Furia-Helms, M.P.H., reflect on the progress we have made within the rare disease space, discuss our continued patient engagement efforts, and how we advance, inform, and support medical product development.
• New Patients Matter video: In our latest video - Patients Matter: Giving Patients A Seat at the Table- we highlight five different kinds of meetings, forums, and committees at which patients, caregivers, and advocates are invited to sit at the table with FDA and share their unique perspectives, experiences, and concerns.

To learn more, visit FDA’s Rare Disease Day web page and connect with us on Twitter @FDAPatientInfo