Determination of the clinical importance of study results

Abstract

Formal statistical methods for analyzing clinical trial data are widely accepted by the medical community. Unfortunately, the interpretation and reporting of trial results from the perspective of clinical importance has not received similar emphasis. This imbalance promotes the historical tendency to consider clinical trial results that are statistically significant as also clinically important, and conversely, those with statistically insignificant results as being clinically unimportant. In this paper, we review the present state of knowledge in the determination of the clinical importance of study results. This work also provides a simple, systematic method for determining the clinical importance of study results. It uses the relationship between the point estimate of the treatment effect (with its associated confidence interval) and the estimate of the smallest treatment effect that would lead to a change in a patient's management. The possible benefits of this approach include enabling clinicians to more easily interpret the results of clinical trials from a clinical perspective, and promoting a more rational approach to the design of prospective clinical trials.

FIGURE 1

Possible combinations for the relationship between clinical importance and statistical significance (see text for the details).