- March 5, 2021
- Organized By:
On this page:
- Meeting Materials
- Topics for Discussion at the Public Meeting
- Request for Open Public Comment Period
- Contact Information
To register for the public meeting, please visit the following website: https://www.surveymonkey.com/r/FDARDD2021.
Registration is free.
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.
The meeting will focus on several related topics regarding rare disease product development. Specifically, the meeting will focus on rare disease partnerships and collaborations, scientific advancements, patient involvement, and strategies to support rare disease product development during COVID-19.
This public meeting will consist of presentations and interactive panel discussions. The presentations will provide information to outline different perspectives and experiences in rare disease product development. The panel discussions will be moderated and allow additional panelists to provide individual perspectives. There will be an opportunity for the panelists to respond to attendee questions submitted using a chat feature. Another way to communicate during the event is for meeting attendees to submit an email to OOPDOrphanEvents@fda.hhs.gov that will be monitored during the public meeting.
FDA will hold an open public comment period to give the public an opportunity to comment on the meeting topics. Registration for open public comment will occur in the meeting registration and is on a first-come, first-served basis.
Open public comment period speakers will be notified of their selection approximately 7 days before the public meeting. We will try to accommodate all who wish to speak, either through the open public comment period or audience questions during the meeting; however, the duration of comments may be limited by time constraints.
Eleanor Dixon-Terry, Office of Orphan Products Development, Food and Drug Administration, 301-796-7634, OOPDOrphanEvents@fda.hhs.gov.