Progress toward a more ethical method for clinical trials

Abstract

Methodology for conducting clinical trials of new drugs and treatments on people need not be regarded as fixed. After reviewing the currently most popular method (randomization) and its ethical problems, this paper explores the possibilities of a new method for conducting such trials. It relies on new Bayesian technology for eliciting the opinions of medical experts. These opinions are conditioned on specific predictor variables, and are held in a computer. At any stage in a trial, these opinions can be updated in the computer using the information collected in the trial up to that point. Consider as an admissible treatment for a patient having specific values of predictor variables only those treatments that at least one expert regards as best (in the computer model) for this patient. It is proposed that only admissible treatments, so defined, be allowed to be assigned to the patient. The ethical and statistical consequences of this principle are explored. Experience to date with a trial at Johns Hopkins designed on this principle is reported.

KIE: Kadane explores the possibilities of using new methods of conducting clinical trials which assign patients to a treatment that at least one expert judges best for that patient. Bayesian methods are used for eliciting the opinions of medical experts which can be changed as information on treatments is collected. The author believes that, while most patients in clinical trials receive reasonable care, they may not receive the best possible care, and that alternatives to standard randomization techniques must be devised. He describes experience with a trial at Johns Hopkins using the optimal treatment method.