Estimation of expectedness: predictive accuracy of standard therapy outcomes in randomized phase 3 studies in epithelial ovarian cancer.

摘要: BACKGROUND The anticipated clinical outcome of the standard/control arm is an important parameter in design randomized phase 3 (RP3) trials to properly calculate sample size, power, and study duration. Changing patterns care or variation population enrolled may lead a deviation from initially outcome. The authors hypothesized that recent changes epithelial ovarian cancer (EOC) have led challenges correctly estimating control groups. METHODS A systematic review literature was conducted for RP3 EOC published between January 2000 December 2010. expected as well actual achieved by this cohort collected ratio (actual-over-expected ratio) calculated. estimation deemed accurate if 0.75 1.25 times outcome. RESULTS A total 35 were eligible analysis. Fifteen had survival primary endpoint whereas 20 progression-based endpoint. In total, 12 15 with survival-based significantly underestimated arm, only 4 did. Studies more frequently than those progression (P<.001). CONCLUSIONS Survival has been trials. This underestimation means initial statistical assumptions these inaccurate. Underestimating result being underpowered demonstrate absolute benefit they designed show. Cancer 2015;121:413–422. © 2014 American Society.