Alternatives to classic randomized trials

Abstract

Randomized clinical trials are regarded as the most credible way of generating scientific data that compare the benefits of different therapies. However, randomized trials present difficulties in their execution. Often physicians are unwilling to participate in such studies because they do not wish to inform the patient that the treatment program will be chosen by a chance mechanism. They believe that such a discussion may compromise the physician-patient relationship. Alternatives to classic randomized trials have been discussed. Also considered are some aspects of the strategy of clinical experimentation. It has been pointed out that the initiation of definitive Phase III trials made on the basis of little prior expectation of success ("trying something out") tends to generate false-positive results.