Strategy and Options in Clinical Trials

Abstract

At the present time there are a large number of clinical trials being conducted to find better ways of treating cancer. These trials come in many varieties. Some are based on small numbers of patients, others on large number of patients; some are multi-institutional, others are carried out within a single institution; some are randomized, others are non-randomized. However, regardless of the scientific quality and the care with which a trial is carried out, if the clinical investigator claims that the therapy under investigation is beneficial, it is likely to influence patient treatment everywhere. The problem of choosing the “best” treatment for the cancer patient is difficult and for many cancer sites unresolved. One reason for this is that the outcome depends on many factors; e.g. anatomic stage, pathology, demographic factors, physical status of patient, prior history, extra disease symptoms, as well as special characteristics of the natural history of the disease site. One point of universal agreement is that the treatment outcome with most patients is not well predicted because of the wide fluctuations and complex nature of the natural history of the disease. Poorly conducted trials may ignore the influence of characteristics of the natural history of the disease and report a positive effect of therapy which may be an artifact.

This paper was supported in part by grants from the U.S. Public Health Service, CA-06516 and CA-23415.