Randomized Clinical Trials

TLDR

Despite controversy, randomized clinical trials remain persuasive, though innovations such as adaptive allocation and sequential stopping are underutilized and difficult to apply in large, complex trials. Ethical and methodological evidence shows that randomized trials are superior to uncontrolled studies and are the most reliable means of evaluating therapeutic efficacy.

Abstract

In spite of the controversy over the role of randomized clinical trials in medical research, the rationale underlying such trials remains persuasive as compared to recent suggestions for alternative non-randomized studies such as those relying on the use of historical controls and adjustment technics. Others have suggested that recent statistical innovations for improving clinical trials, including adaptive allocation of treatment to patients and sequential stopping procedures, are underutilized. These innovations, though theoretically interesting, are not easily adapted to large-scale, complex medical trials in which there may be multiple end points and delayed response times. Ethical considerations suggest that randomized trials are more suitable than uncontrolled experimentation in protecting the interests of patients. Randomized clinical trials remain the most reliable method for evaluating the efficacy of therapies.