Ethics and Regulatory
Overview
Co-chairs:
The advent of pragmatic clinical trials that leverage electronic health records and use research methods such as cluster randomization promises to answer critical research questions with increased sample sizes at lower costs, and could greatly increase production of the evidence needed to improve healthcare. This research, which relies on data collected as part of routine clinical care and may evaluate similar treatments in comparative effectiveness studies, poses a unique set of regulatory and ethical challenges. The Ethics and Regulatory Core identifies areas of regulatory and ethical uncertainty and works with the NIH Collaboratory Trials to navigate regulatory and ethical complexities associated with pragmatic clinical trials conducted within healthcare systems.
Tools for Data and Safety Monitoring
- Data Monitoring in Pragmatic Clinical Trials: Points to Consider
- Charter Template for Data Monitoring Committees of Pragmatic Clinical Trials
- Data and Safety Monitoring Living Textbook Chapter
- Data Monitoring Committees for Pragmatic Clinical Trials
- Principles and Procedures for Data and Safety Monitoring in Pragmatic Clinical Trials
- Additional Resources
The Core is helping to develop approaches to the ethical design and conduct of pragmatic clinical trials that are in compliance with policies and regulations. Its activities supporting this work include the following:
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Address common areas of concern for pragmatic clinical trials, such as minimal risk criteria and informed consent requirements
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Liaise with regulatory and ethical oversight bodies, including institutional review boards (IRBs), the US Food and Drug Administration, and the Office for Human Research Protections
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Conduct empirical research, when needed, to inform these issues
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Document experiences and lessons learned to serve as a guide for other entities engaging in research within healthcare systems
NIH Collaboratory Trial Ethics/Regulatory Discussions: Minutes and Supplementary Materials
The Core facilitates discussions on ethical and regulatory issues relevant to each of the NIH Collaboratory Trials. These discussions typically include representation from study principal investigators and study teams, members of the Ethics and Regulatory Core, NIH staff, and NIH Collaboratory Coordinating Center personnel, with additional input sometimes provided by representatives from the Office for Human Research Protections.
Areas of Focus
The Core convened groups of stakeholders to produce academic articles on a set of 11 ethical and regulatory issues related to the conduct of pragmatic clinical trials. The papers review the issues, and where possible, identify best practices and provide guidance for future pragmatic clinical trials. The following topics are addressed:
Interview
During the NIH Pragmatic Trials Collaboratory 2024 Steering Committee meeting, Drs. Stephanie Morain and Pearl O'Rourke discussed ethics and regulatory challenges and lessons learned.