Clin Trials. 2015 Mar 1. pii: 1740774515571140. [Epub ahead of print]

Ethical and regulatory issues of pragmatic cluster randomized trials in contemporary health systems.

Anderson ML1, Califf RM2, Sugarman J3; for the participants in the NIH Health Care Systems Research Collaboratory Cluster Randomized Trial Workshop.

Author information

• 1Division of Cardiology, Department of Medicine, Duke University School of Medicine, Durham, NC, USA Duke Clinical Research Institute, Durham, NC, USA monique.anderson@dm.duke.edu.
• 2Division of Cardiology, Department of Medicine, Duke University School of Medicine, Durham, NC, USA Duke Clinical Research Institute, Durham, NC, USA Duke Translational Medicine Institute, Duke University, Durham, NC, USA.
• 3Berman Institute of Bioethics, Johns Hopkins University, Baltimore, MD, USA Department of Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA.

Abstract

Cluster randomized trials randomly assign groups of individuals to examine research questions or test interventions and measure their effects on individuals. Recent emphasis on quality improvement, comparative effectiveness, and learning health systems has prompted expanded use of pragmatic cluster randomized trials in routine health-care settings, which in turn poses practical and ethical challenges that current oversight frameworks may not adequately address. The 2012 Ottawa Statement provides a basis for considering many issues related to pragmatic cluster randomized trials but challenges remain, including some arising from the current US research and health-care regulations. In order to examine the ethical, regulatory, and practical questions facing pragmatic cluster randomized trials in health-care settings, the National Institutes of Health Health Care Systems Research Collaboratory convened a workshop in Bethesda, Maryland, in July 2013. Attendees included experts in clinical trials, patient advocacy, research ethics, and research regulations from academia, industry, the National Institutes of Health Collaboratory, and other federal agencies. Workshop participants identified substantial barriers to implementing these types of cluster randomized trials, including issues related to research design, gatekeepers and governance in health systems, consent, institutional review boards, data monitoring, privacy, and special populations. We describe these barriers and suggest means for understanding and overcoming them to facilitate pragmatic cluster randomized trials in health-care settings.