FDA Rare Disease Day 2021: Virtual Public Meeting
Virtual Public Meeting: FDA Rare Disease Day 2021
March 5, 2021 from 9:00 AM - 4:00 PM ET
Hosted by the Office of Orphan Products Development, US Food & Drug Administration
To register for the free public meeting, please visit the following website: https://www.surveymonkey.com/r/FDARDD2021
Rare diseases, often referred to as orphan diseases, are diseases that affect less than 200,000 persons in the United States. While these diseases are individually rare, collectively they are not rare. There are more than 7,000 rare diseases affecting an estimated 30 million people in the United States. Many of these rare diseases are serious or life-threatening and many affect children.
The combination of government incentives and scientific advances has fueled extraordinary development in treatments for rare diseases. Despite this progress, the vast majority of rare diseases still do not have approved treatments. Developing treatments for rare disease presents unique challenges.
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. The meeting will include examples of rare disease product development programs, such as studies funded by the Orphan Products Grants Program, to illustrate the types of challenges faced and strategies used to address them. Additional discussion topics include the importance of patient engagement and natural history studies in rare disease product development, strategies to support rare disease product development during the COVID-19 pandemic, and current perspectives on rare disease product development from leadership across FDA’s medical product centers.
For Further Information, Contact:
Eleanor Dixon-Terry, Office of Orphan Products Development, Food and Drug Administration
301-796-7634, [email protected]