Data and Safety Monitoring

Section 1



Susan Ellenberg, PhD

Jeremy Sugarman, MD, MPH, MA

Doug Zatzick, MD


Contributing Editor

Gina Uhlenbrauck

Liz Wing, MA

There is an ethical obligation to monitor for changes to the risk-benefit balance and data integrity during the course of a clinical trial. The purpose is three-fold: to protect the welfare of participants in the trial, to protect those patients with the same clinical condition outside the trial, and to ensure that the trial results will be informative. Data monitoring committees (DMCs), sponsors, investigators, and other stakeholders are likely to be familiar with practices for monitoring traditional trials, but some special considerations may apply to pragmatic trials that are conducted in the setting of routine healthcare delivery. For example, data quality and timeliness of reporting can be a concern with trial data collected using electronic health records (EHRs). In addition, it may be difficult to collect follow-up data in ways that would deviate from standard clinical workflows.

In this chapter, we discuss issues related to data monitoring that may pose particular challenges in the context of embedded pragmatic clinical trials (ePCTs). These issues are important to consider before study initiation to ensure that an appropriate data monitoring plan is in place—one that balances the pragmatic nature of a trial with the need to maintain trial safety, validity, and integrity. To illustrate these concepts, we will discuss case studies involving planning for data monitoring from the NIH Collaboratory’s Demonstration Projects.



Version History

December 13, 2018: Updated text as part of annual content update (changes made by L. Wing).

Published August 25, 2017


Ellenberg S, Sugarman J, Zatzick D. Data and Safety Monitoring: Introduction. In: Rethinking Clinical Trials: A Living Textbook of Pragmatic Clinical Trials. Bethesda, MD: NIH Health Care Systems Research Collaboratory. Available at: Updated September 12, 2019. DOI: 10.28929/039.