Related papers: Rank-based methods for estimating landmark win pro…
Cluster randomization trials commonly employ multiple endpoints. When a single summary of treatment effects across endpoints is of primary interest, global hypothesis testing/effect estimation methods represent a common analysis strategy.…
The win ratio (WR) is a widely used metric to compare treatments in randomized clinical trials with hierarchically ordered endpoints. Counting-based approaches, such as Pocock's algorithm, are the standard for WR estimation. However, this…
We propose a random-effects approach to missing values for generalized linear mixed model (GLMM) analysis. The method converts a GLMM with missing covariates to another GLMM without missing covariates. The standard GLMM analysis tools for…
Recent advances have shown that statistical tests for the rank of cross-covariance matrices play an important role in causal discovery. These rank tests include partial correlation tests as special cases and provide further graphical…
Stepped-wedge cluster randomised trials (SW-CRTs) increasingly evaluate complex interventions, yet methodological guidance for analysing composite endpoints using generalized pairwise comparisons (GPC)remains limited. This work investigates…
The primary outcome of Randomized clinical Trials (RCTs) are typically dichotomous, continuous, multivariate continuous, or time-to-event. However, what if this outcome is unstructured, e.g., a list of variables of mixed types, longitudinal…
Analyses of cluster randomized trials (CRTs) can be complicated by informative missing outcome data. Methods such as inverse probability weighted generalized estimating equations have been proposed to account for informative missingness by…
We propose a restricted win probability estimand for comparing treatments in a randomized trial with a time-to-event outcome. We also propose Bayesian estimators for this summary measure as well as the unrestricted win probability. Bayesian…
Clinical trials often involve the assessment of multiple endpoints to comprehensively evaluate the efficacy and safety of interventions. In the work, we consider a global nonparametric testing procedure based on multivariate rank for the…
In clinical trials, an experimental treatment is sometimes added on to a standard of care or control therapy in multiple treatment phases (e.g., concomitant and maintenance phases) to improve patient outcomes. When the new regimen provides…
Win statistics have become increasingly popular for analyzing hierarchical composite endpoints in clinical trials, because they summarize treatment benefit through pairwise comparisons that respect the clinical importance order among…
In randomized trials, repeated measures of the outcome are routinely collected. The mixed model for repeated measures (MMRM) leverages the information from these repeated outcome measures, and is often used for the primary analysis to…
What can be considered an appropriate statistical method for the primary analysis of a randomized clinical trial (RCT) with a time-to-event endpoint when we anticipate non-proportional hazards owing to a delayed effect? This question has…
The analysis of randomized trials is often complicated by the occurrence of intercurrent events and missing values. Even though there are different strategies to address missing values it is still common to require missing values…
Commonly used methods to analyze incomplete longitudinal clinical trial data include complete case analysis (CC) and last observation carried forward (LOCF). However, such methods rest on strong assumptions, including missing completely at…
In clinical trials, mixed effects models for repeated measures (MMRM) and pattern mixture models (PMM) are often used to analyze longitudinal continuous outcomes. We describe a simple missing data imputation algorithm for the MMRM that can…
Observed events in recommendation are consequence of the decisions made by a policy, thus they are usually selectively labeled, namely the data are Missing Not At Random (MNAR), which often causes large bias to the estimate of true outcomes…
The restricted mean survival time (RMST) difference offers an interpretable causal contrast to estimate the treatment effect for time-to-event outcomes, yet a wide range of available estimators leaves limited guidance for practice. We…
Background: For RCTs with time-to-event endpoints, proportional hazard (PH) models are typically used to estimate treatment effects and logrank tests are commonly used for hypothesis testing. There is growing support for replacing this…
Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…