Clinical Trials in Surgery

by

David S. Jones

• LAST MODIFIED: 17 April 2025
• DOI: 10.1093/obo/9780197768723-0021

Introduction

Surgery makes a remarkable demand on patients: they must trust that the surgeon, by cutting into their body, will succeed at relieving their suffering. As the scope and invasiveness of surgical interventions increased in the late nineteenth century, surgeons and their patients had to think seriously about a fundamental question: Were they confident that their operations would indeed help? Debates about evidence and standards of proof soon emerged—and these have persisted ever since. Histories of evidence in medicine generally focus on the rise of clinical trials, especially randomized controlled trials (RCTs). These were developed during the early twentieth century and gained status as the “gold standard” for clinical research by the 1970s. Regulatory agencies now typically require evidence from multiple RCTs before approving new medications. The history of clinical trials in surgery has followed a distinctive path. Surgeons certainly recognized the importance of evidence to demonstrate surgical efficacy, and they have joined other physicians in pursuit of methodological rigor. However, differences between medications and surgical interventions have fueled substantial concerns among surgeons about research methods. For instance, while RCTs of drugs are ideally double-blinded to minimize several forms of bias, this generally cannot be done in surgery: Surgeons have been willing to use “sham” controls for only a small number of minimally invasive procedures. Since it is often not possible to perform a “perfect” surgical RCT, surgeons have argued that the technique is not appropriate for surgery. As Lawrence Bonchek wrote in the New England Journal of Medicine in 1979, “We should resist the almost religious fervor of those who would sanctify randomized studies as the only means of learning the truth.” While historians have written extensively about clinical trials in medicine, the historiography of clinical trials in surgery remains more modest. The work that has been done offers important lessons. Surgeons have pursued careful clinical research for centuries. They have engaged in serious debate about the many difficult, possibly intransigent, methodological and ethical questions raised by surgical research. As a result of these concerns, clinical trials often play a different role in surgery than in medicine. Instead of being used to judge the efficacy of new operations, surgical RCTs are often used to provide clarity and resolve controversies when established surgical procedures become controversial. Surgeons continue to debate the role of RCTs and other clinical trials in surgical research today. Historical research can inform these debates by revealing the practical, intellectual, political, and economic contexts of surgical research, and also by indicating potential areas for future progress.

General Overviews

While there are many excellent book-length studies of clinical trials in medicine, these have largely focused on pharmaceuticals. Historians have not yet written similar studies of the history of clinical trials in surgery. As a result, it is necessary to rely on shorter surveys of the topic or on specific case studies. Fortunately, there are many excellent examples of each, written by authors from several different perspectives. Prompted by recognition of the rapidly rising costs of Medicare in the early 1970s, scholars in health policy examined what could be done to contain these costs. One edited collection, Bunker, et al. 1977, includes fascinating case studies of surgery and surgical decision-making that offer careful analyses of the history of clinical trials for specific operations (several of these are discussed in the section on Historical Case Studies of Clinical Trials in Surgery). Surgeons have written several review articles about the history of trials, especially RCTs, in surgery, including Pollock 1989 and Rahbari, et al. 2007. Historians have now begun to examine the history of surgical trials. Two edited collections of recent scholarship on the history of surgery—Schlich and Crenner 2017 and Schlich 2018—contain many valuable case studies, several of which are focused on clinical trials (and are discussed under Historical Case Studies of Clinical Trials in Surgery). Two recent works, Jones 2018 and Bothwell and Jones 2021, offer the most specific historical discussions of clinical trials in surgery. While all of this work is promising, it only scratches the surface of the topic. A full-length treatment of the history of surgical trials is sorely needed.

• Bothwell, Laura, and David S. Jones. “Innovation and Tribulation in the History of Randomized Controlled Trials in Surgery.” Annals of Surgery 274 (December 2021): e616–e624.

  DOI: 10.1097/sla.0000000000003631

  This historical review essay, written by historians for a surgical audience, traces key events and themes in the history of clinical trials in surgery.

• Bunker, John P., Benjamin A. Barnes, and Frederick Mosteller, eds. Costs, Risks, and Benefits of Surgery. New York: Oxford University Press, 1977.

  An edited collection that contains several important case studies of the rise and fall of specific surgical treatments. These analyses look carefully at the role of evidence and clinical trials in surgery.

• Jones, David S. “Surgery and Clinical Trials: The History and Controversies of Surgical Evidence.” In The Palgrave Handbook of the History of Surgery. Edited by Thomas Schlich, 479–501. London: Palgrave Macmillan, 2018.

  DOI: 10.1057/978-1-349-95260-1_23

  A review of the historical literature on clinical trials in surgery, tracing the origins of the technique, the debates about the efficacy of RCTs in surgery, and the current controversies.

• Pollock, A. V. “The Rise and Fall of the Random Controlled Trial in Surgery.” Theoretical Surgery 4 (1989): 163–170.

  DOI: 10.1136/bmj.324.7351.1448

  A review article by a British surgeon that examines the rise of clinical trials in surgery, as well as their recent decline. Offers several explanations for why surgeons had lost enthusiasm for RCTs in the 1980s.

• Rahbari, N. N., M. K. Diener, M. N. Wente, and C. M. Seiler. “Development and Perspectives of Randomized Controlled Trials.” American Journal of Surgery 194.4, Suppl. (2007): S148–S152.

  DOI: 10.1016/j.amjsurg.2007.05.010

  A review article by German surgeons that seeks to explain the paucity of surgical RCTs. Offers several explanations and makes the case for why surgeons should be more committed to the approach.

• Schlich, Thomas, ed. The Palgrave Handbook of the History of Surgery. London: Palgrave Macmillan, 2018.

  This edited collection provides historiographical review essays on many key topics in the history of surgery (e.g., specialization, anesthesia, infections, instruments, gender, emotion, etc.), many of which discuss clinical trials.

• Schlich, Thomas, and Christopher Crenner, eds. Technological Change in Modern Surgery: Historical Perspectives on Innovation. Rochester, NY: University of Rochester Press, 2017.

  An edited collection by two prominent historians. While the essays focus on questions of innovation and technological change, clinical trials play an important role in many of the narratives.