Efficacy as an important facet of "safety" in clinical trials: how can we do our best for our patients?

Ensuring optimal efficacy is an important facet of patient safety during the conduct of a clinical trial, if, by "safety," we mean the anticipation, prevention, and assessment of any event that can have an unfavorable impact on the enrolled patients. This article addresses what could be termed the "nonsafety" aspects of safety during clinical trials, with a focus on situations in which we, as drug developers and clinical investigators, can err and thereby put our study patients at an unanticipated and unintended risk for a poor outcome. Approaches to mitigating the safety risk of efficacy are also discussed.