Statistics in Medicine

Group sequential two-stage preference designs

The two-stage preference design (TSPD) enables inference for treatment efficacy while allowing for incorporation of patient preference to treatment. It can provide unbiased estimates for selection and preference effects, where a selection effect …

Maximin optimal cluster randomized designs for assessing treatment effect heterogeneity

Cluster randomized trials (CRTs) are studies where treatment is randomized at the cluster level but outcomes are typically collected at the individual level. When CRTs are employed in pragmatic settings, baseline population characteristics may …

Variance estimation for the Kappa statistic in the presence of clustered data and heterogeneous observations

We present a methodology motivated by a controlled trial designed to validate SPOT GRADE, a novel surgical bleeding severity scale. Briefly, the study was designed to quantify inter‐ and intra‐surgeon agreement for characterizing the severity of …