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Tie-breaker designs trade off a statistical design objective with short-term gain from preferentially assigning a binary treatment to those with high values of a running variable $x$. The design objective is any continuous function of the…

Methodology · Statistics 2022-10-20 Harrison H. Li , Art B. Owen

Two commonly used methods for improving precision and power in clinical trials are stratified randomization and covariate adjustment. However, many trials do not fully capitalize on the combined precision gains from these two methods, which…

Background: It has long been advised to account for baseline covariates in the analysis of confirmatory randomised trials, with the main statistical justifications being that this increases power and, when a randomisation scheme balanced…

Methodology · Statistics 2021-12-09 Tim P. Morris , A. Sarah Walker , Elizabeth J. Williamson , Ian R. White

Paired cluster-randomized experiments (pCRTs) are common across many disciplines because there is often natural clustering of individuals, and paired randomization can help balance baseline covariates to improve experimental precision.…

Methodology · Statistics 2024-07-03 Charlotte Z. Mann , Adam C. Sales , Johann A. Gagnon-Bartsch

Under a generalised estimating equation analysis approach, approximate design theory is used to determine Bayesian D-optimal designs. For two examples, considering simple exchangeable and exponential decay correlation structures, we compare…

Methodology · Statistics 2024-02-16 Laura Etfer , James M. S. Wason , Michael J. Grayling

To increase statistical efficiency in a randomized experiment, researchers often use stratification (i.e., blocking) in the design stage. However, conventional practices of stratification fail to exploit valuable information about the…

Methodology · Statistics 2025-10-28 Zikai Li

We construct optimal designs for estimating fetal malformation rate, prenatal death rate and an overall toxicity index in a toxicology study under a broad range of model assumptions. We use Weibull distributions to model these rates and…

Methodology · Statistics 2009-02-10 Holger Dette , Andrey Pepelyshev , Weng Kee Wong

Covariate-adaptive randomization is widely employed to balance baseline covariates in interventional studies such as clinical trials and experiments in development economics. Recent years have witnessed substantial progress in inference…

Methodology · Statistics 2024-05-30 Jiahui Xin , Hanzhong Liu , Wei Ma

We consider the optimal experimental design problem for an uncertain Kuramoto model, which consists of N interacting oscillators described by coupled ordinary differential equations. The objective is to design experiments that can…

Optimization and Control · Mathematics 2021-05-04 Youngjoon Hong , Bongsuk Kwon , Byung-Jun Yoon

In a clustered observational study, a treatment is assigned to groups and all units within the group are exposed to the treatment. We develop a new method for statistical adjustment in clustered observational studies using approximate…

Methodology · Statistics 2023-03-06 Luke Keele , Eli Ben-Michael , Lindsay Page

Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

Machine Learning · Statistics 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

This paper studies the use of highly stratified designs for the efficient estimation of a large class of treatment effect parameters that arise in the analysis of experiments. By a "highly stratified" design, we mean experiments in which…

Econometrics · Economics 2026-03-30 Yuehao Bai , Jizhou Liu , Azeem M. Shaikh , Max Tabord-Meehan

We study the design of multi-armed parallel group clinical trials to estimate personalized treatment rules that identify the best treatment for a given patient with given covariates. Assuming that the outcomes in each treatment arm are…

Statistics Theory · Mathematics 2022-07-13 David Azriel , Yosef Rinott , Martin Posch

We develop a unified theory of designs for controlled experiments that balance baseline covariates a priori (before treatment and before randomization) using the framework of minimax variance and a new method called kernel allocation. We…

Statistics Theory · Mathematics 2017-08-02 Nathan Kallus

Blocking is often used to reduce known variability in designed experiments by collecting together homogeneous experimental units. A common modelling assumption for such experiments is that responses from units within a block are dependent.…

Methodology · Statistics 2018-11-07 W. G. Mueller , A. Rappold , D. C. Woods

We develop a mathematical framework to define an optimal individualized treatment rule (ITR) within the context of prioritized outcomes in a randomized controlled trial. Our optimality criterion is based on the framework of generalized…

Methodology · Statistics 2025-06-17 François Petit , Gérard Biau , Raphaël Porcher

In observational studies, propensity scores are commonly estimated by maxi- mum likelihood but may fail to balance high-dimensional pre-treatment covariates even after specification search. We introduce a general framework that unifies and…

Methodology · Statistics 2017-03-22 Qingyuan Zhao

In clinical trials, there is potential to improve precision and reduce the required sample size by appropriately adjusting for baseline variables in the statistical analysis. This is called covariate adjustment. Despite recommendations by…

Methodology · Statistics 2022-06-20 Kelly Van Lancker , Joshua Betz , Michael Rosenblum

This paper studies inference in randomized controlled trials with multiple treatments, where treatment status is determined according to a "matched tuples" design. Here, by a matched tuples design, we mean an experimental design where units…

Econometrics · Economics 2023-11-06 Yuehao Bai , Jizhou Liu , Max Tabord-Meehan

The split-plot design assigns different interventions at the whole-plot and sub-plot levels, respectively, and induces a group structure on the final treatment assignments. A common strategy is to use the OLS fit of the outcome on the…

Methodology · Statistics 2021-10-25 Anqi Zhao , Peng Ding