Co-Chairs: Jeremy Sugarman, Judith Carrithers, Kevin Weinfurt
NIH Representative: David Wendler
Members: Laura Bankowski, Elaine Bergman, Jenn Boggs, Sheana Bull, Diane Christiansen, Denise Cifelli, Gregory Clarke, Laura Dember, Susan Ellenberg, Sheila Fireman, Beverly Green, Corita Grudzen, Adrian Hernandez, Adeola Jaiyeola, Lindsay Kindler, Margaret Kuklinski, Julie Lima, Rosemary Madigan, David Magnus, Carmit McMullen, Susan Mitchell, Michael Paasche-Orlow, Mehul Patel, Michelle Roberts, Stacy Sterling, Miguel Vazquez, Ben Wilfond, Doug Zatzick
Project Manager: 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:
Address common areas of concern for pragmatic clinical trials, such as regulatory issues, minimal risk criteria, and informed consent
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, Sentinel, the Patient‑Centered Outcomes Research Institute, and the Health Care Systems 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
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.
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:
Kevin Weinfurt, PhD, Duke Clinical Research Institute, and Jeremy Sugarman, MD, MPH, MA, Johns Hopkins Berman Institute of Bioethics, discuss a study comparing alternative approaches to notification and authorization of participants in pragmatic trials evaluating common medical practices.
Products and Publications
9/25/2015: Grand Rounds Presentation: Ethical and Regulatory Issues in Pragmatic Clinical Trials: Introducing a Special Series in Clinical Trials (Slides)