Our Work

Our Work Consensus/Advisory Activity Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union

Completed

Topics

Legislation and Policy

Health and Medicine

In response to a Congressional request, an ad hoc committee of the National Academies of Sciences, Engineering, and Medicine will conduct a study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the United States and the European Union.

To Increase Pace and Volume of Drug Approvals for Rare Diseases, Report Recommends FDA Enhance Information Sharing, Improve Collaboration Sep 12, 2024 News Release

About Publications/Resources Past Events

Events

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Report Launch Event

September 27, 2024
1:00 PM - 2:00 PM (EDT)

Webinar
Past

Please join us for a webinar to learn about this new National Academies’ report, Regulatory Processes for Rare Disease Drugs in the United States and European Union, on Friday, September 27, 2024. Mem...

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 3 - Virtual)

February 6 - 7, 2024

Past

This is the third meeting of the committee and will be conducted in a virtual environment. The meeting will include a closed and public session. Written comments can be sent to the committee via email...

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 2 - Hybrid)

December 4 - 5, 2023

Past

The second meeting of the committee will take place on December 4 and 5, 2023 and will be hybrid - including both closed and public sessions. This meeting will include presentations on FDA and EMA dru...

Meetings

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Committee Media Training

August 20, 2024
11:00 AM - 12:00 PM (EDT)

Closed
Meeting
Past

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Committee Media Training

August 16, 2024
11:00 AM - 12:00 PM (EDT)

Closed
Meeting
Past

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Report Review Meeting

August 5, 2024
4:00 PM - 5:00 PM (EDT)

Closed
Meeting
Past

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 5 - Hybrid)

May 23 - 24, 2024

Closed
Past

This is the fifth meeting of the committee and will be hybrid with both in-person and virtual participants. The meeting will be closed and will not include a public session.

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 2 Meeting

May 10, 2024
4:00 PM - 4:30 PM (EDT)

Closed
Past

This is a closed-session writing group meeting of the consensus study committee members.

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Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Report Launch Event

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 3 - Virtual)

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 2 - Hybrid)

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Committee Media Training

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Committee Media Training

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Report Review Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 5 - Hybrid)

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 3 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 1 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 4 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Data Analysis Readout

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 4 - Virtual)

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 1 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 4 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 3 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 1 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 3 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 1 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 1 - Virtual)

My Academies

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union

Dropdown items

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Report Launch Event

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 3 - Virtual)

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 2 - Hybrid)

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Committee Media Training

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Committee Media Training

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Report Review Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 5 - Hybrid)

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 3 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 1 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Writing Group 4 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Data Analysis Readout

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 4 - Virtual)

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 1 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 4 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 3 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 1 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 3 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 2 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union - Working Group 1 Meeting

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union (Meeting 1 - Virtual)