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Register Now

On This Page:

• Meeting Materials
• Summary
• Topics for Discussion
• Public Docket
• Contact
• Request for open public comment period speakers

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Meeting materials: 

• Webcast 
• Agenda (PDF- 399KB)
• Speaker Biographies (PDF- 2.46MB)

Summary:

FDA will host a virtual public meeting on March 4, 2022 to join the global observance of Rare Disease Day. The theme for FDA’s Rare Disease Day is “Sharing Experiences in Rare Diseases Together.” 

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.  

Topics for discussion:  

This public meeting will include six interactive panel discussions. Panelists, including current and former FDA staff, patients, and caregivers, will speak about their experiences in:

• Enhancing product development using a public-private partnership approach for a rare disease
• Reviewing gene therapies for neurocognitive disorders in children 
• Engaging in review processes that led to approval of two drugs to treat rare tumors
• Using patient input and benefit-risk assessments in approval of devices for rare orthopedic conditions
• Interacting with FDA
• Developing initiatives to improve FDA’s work in rare diseases

Panel discussions will be moderated and allow panelists to provide individual perspectives. Panelists will be able to respond to attendee questions using a chat feature. Another way to communicate during the event is for meeting attendees to submit an email to [email protected] that will be monitored during the public meeting.

Public Docket:

Stakeholders are invited to provide their perspectives on the discussion questions through the public docket.  All comments must be identified with the docket number (FDA-2022-N-0116) through April 8, 2022.

Contact:

Eleanor Dixon-Terry
Office of Orphan Products Development
Office of Clinical Policy and Programs
Food and Drug Administration
[email protected]
301-796-7634

Request for open public comment period speakers: 

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 seven days before the public meeting.  We will try to accommodate all who wish to speak, either through the open public comment period or audience participation during the meeting; however, the duration of comments may be limited by time constraints.

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