A Primer on Effectiveness and Efficacy Trials

Clin Transl Gastroenterol. 2014 Jan 2;5(1):e45. doi: 10.1038/ctg.2013.13.


Although efficacy and effectiveness studies are both important when evaluating interventions, they serve distinct purposes and have different study designs. Unfortunately, the distinction between these two types of trials is often poorly understood. In this primer, we highlight several differences between these two types of trials including study design, patient populations, intervention design, data analysis, and result reporting.