What should a physician know about clinical trials, and why?

First, knowledge of the basics of a clinical trial is important since this type of research highly affects the options that are available to physicians to treat an individual patient. A physician who reviews journal articles to learn about new options also needs to have some understanding of the designs and methodology used in the research described by the journal article, and also to have some fundamental knowledge in order to be able to evaluate the appropriateness of the study that was used. The basic terms and definitions included here will aid in that understanding.

What is a clinical trial?

A clinical trial is defined as a prospective study in human beings that evaluates the safety or effectiveness of a new intervention or drug regimen, or that compares the effectiveness of the new intervention to that of current best practice (or other control group). The details of the particular intervention used (a specific dosage schedule given in a specific manner to a specific type of patient) is summarized in a written document called a research protocol.

Why use clinical trials?

A clinical trial is an effective tool in determining whether or not an intervention actually does have the beneficial results that have been hypothesized. Given the variability of human subjects and the uncertain knowledge of the course of most diseases, it would be extremely difficult or impossible to determine the specific effect of an intervention on the outcome of the disease course if an uncontrolled trial was used.