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Guide to Statistics and Methods
Randomized Clinical Trials in Surgery
October 26, 2022

Practical Guide to Designing a Clinical Trial in Surgery

Author Affiliations
  • 1Department of Surgery, State University of New York, Downstate Health Sciences University, Brooklyn
  • 2Department of Emergency Medicine, Harbor-UCLA Medical Center, David Geffen School of Medicine at UCLA, Torrance, California
  • 3Statistical Editor, JAMA Surgery
  • 4Department of Surgery, Amsterdam UMC location AMC, University of Amsterdam, Amsterdam, the Netherlands
JAMA Surg. 2022;157(12):1152-1153. doi:10.1001/jamasurg.2022.4883

A randomized clinical trial represents a controlled experiment in which the investigator applies an intervention to a group of patients and subsequently observes its effect on 1 or more outcomes over time. In this article, we outline the key elements that need to be considered during the design phase of a surgical clinical trial (Box).

Inception of a clinical trial starts by formulating a well-thought-out research question that is stated in the form of a hypothesis that will be tested. Trials are best reserved for mature questions that are supported by existing literature and can address a well-defined knowledge gap. Issues of patient safety and ethics deserve a special consideration in surgical trials, since the invasive nature of the procedures tested or, inversely, the risk of withholding surgical treatment due to study protocol requirements may both be associated with patient harm. For this reason, assessing patient and clinician equipoise for the proposed research question is important,1 as lack of such equipoise limits trial feasibility and adversely affects the implementation of results.

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