Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions – Third Meeting

This is the third meeting of the committee of experts and will be conducted virtually. The meeting will include a closed and public session. There will be an opportunity for interested stakeholders to share public comments with the committee on February 7, 2024 at 12:00 p.m.

Public comments will provide the committee with additional insight into key issues related to the study’s statement of task. These include, but are not limited to:

• The use of regulatory flexibilities and supplementary data (e.g., natural history studies and patient registries) when evaluating the safety and efficacy of drugs for rare diseases and conditions
• FDA and EMA engagement of people with lived experience when developing guidance, policies, and programs.

Public comments, alongside other materials stakeholders have shared, will be reviewed by the committee, and may help inform committee deliberations on the statement of task. All comments and materials shared with the committee will be made publicly available in accordance with institutional policies.

Please visit NASEM’s Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union event page for more information on public comments for this meeting.

OPEN SESSION AGENDA HIGHLIGHTS

• Longitudinal trial design
• Master protocols for drug development trials
• External controls for drug development trials
• Considerations for pediatric trials: caregiver perspective
• Considerations for pediatric trials: regulatory science perspective
• Considerations for pediatric trials: industry perspective
• Expanded access programs
• Patient registries and natural history data
• Patient experience data
• Model-informed drug development
• Analysis methods – causal inference
• Flexibilities applied at FD

ABOUT THE COMMITTEE

In response to a Congressional request, an ad hoc committee of the National Academies of Sciences, Engineering, and Medicine is conducting 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.

The committee will evaluate:

• Flexibilities, authorities, or mechanisms available to regulators in the United States and the European Union applicable to rare diseases or conditions;
• The consideration and use of supplemental data submitted during review processes in the United States and the European Union, including data associated with open label extension studies and expanded access programs specific to rare diseases or conditions.
• An assessment of collaborative efforts between United States and European Union regulators related to: product development programs under review; policies under development and those recently issued; and scientific information related to product development or regulation.

Based on its information gathering and internal deliberations, the committee will develop a report with its findings, conclusions, and recommendations for actions that Congress, federal agencies, the pharmaceutical industry, and nongovernmental organizations can take to support collaborative efforts.

Please visit the National Academies’ Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union event page for more information.

The Fourth Meeting of the committee will be held 3-4 April 2024.

The Fifth Meeting of the committee will be held 23-24 May 2024.