Controlled Studies in Clinical Cancer Research

Abstract A review of abstracts and papers reporting clinical trials of new anticancer agents reveals that only a small percentage have been controlled. Arguments against employing randomized controls in such trials are understandable but hardly valid at the present stage in cancer research. Randomization of drugs tested or dosage regimens at the earliest stages of clinical use is needed to evaluate relative therapeutic efficacy and toxicity. Potent ethical arguments also require that when a new drug is tried, patients should be given a 50–50 chance of receiving the conceivably better standard therapy. An important and subtle consideration, that of how long to continue a controlled trial, requires continued development of the study-design process and new uses of peer review.