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  4. Public Meeting: FDA Rare Disease Day 2022 - 03/04/2022
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Meeting | Virtual

Event Title
Public Meeting: FDA Rare Disease Day 2022
March 4, 2022

Scheduled
Date:
March 4, 2022
Organized By:
Organizer
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Rare Disease Day 2022 Main Header

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Meeting materials: 

Summary:

FDA hosted 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 was “Sharing Experiences in Rare Diseases Together.”  During presentations and panel discussions various stakeholders shared their perspectives on and experiences in rare disease product development.  

Topics for discussion:

FDA Building 1 is illuminated in observance of Rare Disease Week, Feb 28 - March 4 2022
FDA Building 1 is illuminated in observance of Rare Disease Week, Feb 28 - March 4 2022

This public meeting included six interactive panel discussions. Panelists, including current and former FDA staff, patients, and caregivers, spoke 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

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
OOPDOrphanEvents@fda.hhs.gov
301-796-7634

Event Materials

Title File Type/Size
Agenda FDA Rare Disease Day March 4, 2022 pdf (407.69 KB)
Speaker Bios FDA Rare Disease Day March 4, 2022 pdf (2.65 MB)
Glossary of Terms for FDA RDD 2022 pdf (247.99 KB)
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