Continuous covariate imbalance and conditional power for clinical trial interim analyses

Contemp Clin Trials. 2014 May;38(1):9-18. doi: 10.1016/j.cct.2014.02.007. Epub 2014 Mar 7.

Abstract

Oftentimes valid statistical analyses for clinical trials involve adjustment for known influential covariates, regardless of imbalance observed in these covariates at baseline across treatment groups. Thus, it must be the case that valid interim analyses also properly adjust for these covariates. There are situations, however, in which covariate adjustment is not possible, not planned, or simply carries less merit as it makes inferences less generalizable and less intuitive. In this case, covariate imbalance between treatment groups can have a substantial effect on both interim and final primary outcome analyses. This paper illustrates the effect of influential continuous baseline covariate imbalance on unadjusted conditional power (CP), and thus, on trial decisions based on futility stopping bounds. The robustness of the relationship is illustrated for normal, skewed, and bimodal continuous baseline covariates that are related to a normally distributed primary outcome. Results suggest that unadjusted CP calculations in the presence of influential covariate imbalance require careful interpretation and evaluation.

Keywords: Conditional power; Covariate adjusted analysis; Covariate imbalance.

MeSH terms

  • Clinical Trials as Topic / methods*
  • Computer Simulation
  • Data Interpretation, Statistical*
  • Humans
  • Research Design*