Co-Chairs: Jeremy Sugarman, Kevin Weinfurt
NIH Representative: David Wendler
Members: Monique Anderson, Elaine Bergman, Laura Beskow, Denise Cifelli, Susan Ellenberg, Sheila Fireman, Catherine Hammack, Adrian Hernandez, Adeola Jaiyeola, Katie James, Rebecca Kaganov, Lindsay Kindler, Julie Lima, Rosemary Madigan, Carmit McMullen, Susan Mitchell, Jim Sabin, Miguel Vazquez, Doug Zatzick
Project Managers: Tammy Reece
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 Regulatory/Ethics Core identifies areas of regulatory and ethical uncertainty and works with the Demonstration Projects to navigate regulatory and ethical complexities associated with pragmatic clinical trials conducted within healthcare systems.
The Core is developing policies and practices to provide a framework for the conduct of pragmatic clinical trials in an ethical manner and in compliance with federal and state regulations. Its activities supporting this work include the following:
Demonstration Project Regulatory/Ethics Discussions: Minutes and Supplementary Materials
The Core facilitated a series of discussions on ethical and regulatory issues relevant to the Demonstration Projects. These discussions, which took place as teleconferences, included representation from study principal investigators and study teams, members of the Collaboratory Regulatory/Ethics Core, NIH staff, and Collaboratory Coordinating Center personnel, with additional input provided by representatives from the Office for Human Research Protections.
Address common areas of concern for pragmatic clinical trials, such as regulatory issues, minimal risk criteria, and informed consent
Liaise with regulatory and ethical oversight bodies, including institutional review boards (IRBs), the U.S. Food and Drug Administration, and the Office for Human Research Protections
Conduct empirical research, when needed, to inform these issues
Examine approaches to streamline regulatory pathways
Collaborate with other groups, including the Clinical Trials Transformation Initiative, Mini-Sentinel, the Patient‑Centered Outcomes Research Institute, and the HMO Research Network on initiatives such as quality by design and centralized IRB review
Document experiences and lessons learned to serve as a guide for other entities engaging in research within healthcare systems
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: