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Imbalances in covariates between treatment groups are frequent in observational studies and can lead to biased comparisons. Various adjustment methods can be employed to correct these biases in the context of multi-level treatments ($>$ 2).…

应用统计 · 统计学 2021-06-04 Diop S. Arona , Duchesne Thierry , Cumming Steven , Diop Awa , Talbot Denis

To estimate the causal effect of an intervention, researchers need to identify a control group that represents what might have happened to the treatment group in the absence of that intervention. This is challenging without a randomized…

统计方法学 · 统计学 2026-03-20 Robert Pickett , Jennifer Hill , Sarah Cowan

Permutation testing in linear models, where the number of nuisance coefficients is smaller than the sample size, is a well-studied topic. The common approach of such tests is to permute residuals after regressing on the nuisance covariates.…

统计方法学 · 统计学 2020-10-09 Jesse Hemerik , Magne Thoresen , Livio Finos

Difference in proportions is frequently used to measure treatment effect for binary outcomes in randomized clinical trials. The estimation of difference in proportions can be assisted by adjusting for prognostic baseline covariates to…

统计方法学 · 统计学 2023-08-31 Jialuo Liu , Dong Xi

This paper introduces a kernel discrepancy-based framework for rerandomization to enhance the precision of causal inference in controlled experiments. We demonstrate that the kernel discrepancy is the key part of the variance upper bound…

统计方法学 · 统计学 2025-11-05 Yiou Li , Lulu Kang

Variational inference is increasingly being addressed with stochastic optimization. In this setting, the gradient's variance plays a crucial role in the optimization procedure, since high variance gradients lead to poor convergence. A…

机器学习 · 计算机科学 2020-10-23 Tomas Geffner , Justin Domke

When randomized controlled trials are impractical or unethical to simultaneously compare multiple treatments, indirect treatment comparisons using single-arm trials offer valuable evidence for health technology assessments, especially for…

统计方法学 · 统计学 2025-09-30 Yuru Zhu , Huiyuan Wang , Haitao Chu , Yumou Qiu , Yong Chen

We consider a setting in which we have a treatment and a large number of covariates for a set of observations, and wish to model their relationship with an outcome of interest. We propose a simple method for modeling interactions between…

统计方法学 · 统计学 2012-12-14 Lu Tian , Ash Alizadeh , Andrew Gentles , Robert Tibshirani

Randomized Controlled Trials (RCTs) may suffer from limited scope. In particular, samples may be unrepresentative: some RCTs over- or under- sample individuals with certain characteristics compared to the target population, for which one…

统计方法学 · 统计学 2024-03-15 Bénédicte Colnet , Julie Josse , Gaël Varoquaux , Erwan Scornet

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

We investigate the optimal design of experimental studies that have pre-treatment outcome data available. The average treatment effect is estimated as the difference between the weighted average outcomes of the treated and control units. A…

Observational studies can play a useful role in assessing the comparative effectiveness of competing treatments. In a clinical trial the randomization of participants to treatment and control groups generally results in well-balanced groups…

Evaluating blocked randomized experiments from a potential outcomes perspective has two primary branches of work. The first focuses on larger blocks, with multiple treatment and control units in each block. The second focuses on matched…

统计方法学 · 统计学 2024-05-31 Nicole E. Pashley , Luke W. Miratrix

In randomized experiments, treatment and control groups should be roughly the same--balanced--in their distributions of pretreatment variables. But how nearly so? Can descriptive comparisons meaningfully be paired with significance tests?…

统计方法学 · 统计学 2008-08-29 Ben B. Hansen , Jake Bowers

Hybrid type 2 studies are gaining popularity for their ability to assess both implementation and health outcomes as co-primary endpoints. Often conducted as cluster-randomized trials (CRTs), five design methods can validly power these…

统计方法学 · 统计学 2026-05-18 Melody Owen , Fan Li , Ruyi Liu , Donna Spiegelman

Standard ANOVA is among the most widely used tests in the life sciences and beyond. Several alternatives are proposed to provide simultaneous confidence intervals, ensure tight control of FWER, be robust to variance heterogeneity, avoid…

统计方法学 · 统计学 2022-06-01 Ludwig A. Hothorn

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of…

统计方法学 · 统计学 2023-06-12 Kelly Van Lancker , Frank Bretz , Oliver Dukes

Randomized controlled trials are the gold standard for measuring causal effects. However, they are often not always feasible, and causal treatment effects must be estimated from observational data. Observational studies do not allow robust…

Background: Experimental treatments pass through various stages of development. If a treatment passes through early phase experiments, the investigators may want to assess it in a late phase randomised controlled trial. An efficient way to…

In this article, we develop methods for sample size and power calculations in four-level intervention studies when intervention assignment is carried out at any level, with a particular focus on cluster randomized trials (CRTs). CRTs…

统计方法学 · 统计学 2022-09-07 Xueqi Wang , Elizabeth L. Turner , John S. Preisser , Fan Li