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Statistical Methodology

Leo Laporte1Eric Schmidt1Bert de Jong1David Friedberg1
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Two-Phase Sampling Designs Extended to Ordinal Outcomes Enable Cost-Efficient Biomarker Studies

Two-phase sampling designs, which reduce costs by restricting expensive measurements (e.g., biomarkers, genomic data) to an informative subset of participants, have been extended for the first time to studies with ordinal outcomes. The authors propose three outcome-informed phase 2 selection strateg…

Anytime-Valid Sequential Tests Enable Real-Time Monitoring of Stochastic Dominance

The paper introduces nonparametric e-processes for sequential, anytime-valid tests of stochastic dominance (SD), distinguishing from mean dominance by comparing full distributions. For first-order SD, these e-processes mix asymptotically growth-rate optimal e-variables, achieving power one. The fram…

Exact Study-Based Decomposition of Direct and Indirect Evidence in Network Meta-Analysis via Contrast-Space Projection

The method reformulates network meta-analysis (NMA) in contrast space as an orthogonal projection of observed pairwise treatment contrasts onto the consistency-constrained subspace, yielding an explicit linear mapping that reproduces NMA estimates. It defines direct and indirect evidence through a c…

Two GPC-Based Methods Reliably Control Type I Error in Stepped-Wedge Cluster Trials

Stepped-wedge cluster randomised trials (SW-CRTs) pose analytical challenges when composite endpoints are evaluated using generalized pairwise comparisons (GPC), as most estimators fail to adequately account for clustering and temporal trends. A comprehensive simulation study across varying ICCs, cl…