ABSTRACT
The goal of any clinical trial is to yield valuable and reliable scientific information that can ultimately guide medical practice. To that end, appropriate attention to study design, including the selection of reasonable and relevant trial endpoints, is essential for success. Trial endpoints are events or measurements that reflect the effects of an intervention on the study participants. Most trials are designed to collect data on many different endpoints, the nature and number of which will depend on the condition or disease of the individuals who are in the study, the anticipated pharmacological and toxic effects of the intervention, and the objective of the clinical investigation. In the initial studies, where safety and dosing information is of prime interest, study endpoints usually are measurements of pharmacokinetics and safety. In later clinical development, there is growing emphasis on evaluation of activity and, ultimately, efficacy. Efficacy is generally evaluated by using adequate and well-controlled clinical trials designed to show that the intervention meaningfully affects the trial’s efficacy endpoints.
