Ethical and Practical Dilemmas in the Design of Clinical Trials for Evidence-Based Medicine

• Activity Number: 204
• Type: Invited
• Date/Time: Monday, August 4, 2014 : 2:00 PM to 3:50 PM
• Sponsor: Section on Statistical Consulting
• Author(s): David Alan Schoenfeld*+
• Companies: Massachusetts General Hospital
• Keywords: Trial Design ; Human Subjects Protection ; Evidence Based Medicine

Abstract

The design of a clinical trial is often determined by the need to treat patients ethically in the context of clinical realities. This is particularly true in the evaluation of treatment strategies when "usual care" covers a wide range of choices. If clinical trials compare specific strategies, then the results will only be useful if they can help us evaluate strategies that weren't tested. However, trials that compare a specific strategy to usual care run the risk that the treatment arms may have substantial overlap. To see that this dilemma has an ethical dimension, we consider a trial that was stopped by the Office of Human Research Protections which compared four alternative methods of fluid management because the office felt that it would be impossible to tell from the data whether any of the treatment arms were worse than the care that was usually given to patients. The discussions that led to the eventual restart of this trial considered the issue about how to develop control groups in trials in patients with acute life threatening illness. The example illustrates many of the dilemmas that clinical trialists face when they try develop evidence based medicine.