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The aim of this paper is twofold. First, three theoretical principles are formalized: randomization, overrepresentation and restriction. We develop these principles and give a rationale for their use in choosing the sampling design in a…

Methodology · Statistics 2016-12-16 Yves Tillé , Matthieu Wilhelm

This paper develops power and sample size formulas for causal inference with time-to-event outcomes. The target estimand is the marginal hazard ratio: the coefficient of a marginal structural Cox proportional hazard model with treatment as…

Methodology · Statistics 2026-05-19 Chengxin Yang , Bo Liu , Fan Li

Bayesian experimental design involves the optimal allocation of resources in an experiment, with the aim of optimising cost and performance. For implicit models, where the likelihood is intractable but sampling from the model is possible,…

Machine Learning · Statistics 2019-02-26 Steven Kleinegesse , Michael Gutmann

Group sequential designs drive innovation in clinical, industrial, and corporate settings. Early stopping for failure in sequential designs conserves experimental resources, whereas early stopping for success accelerates access to improved…

Methodology · Statistics 2025-11-27 Luke Hagar , Shirin Golchi , Marina B. Klein

This paper outlines a framework for quantifying the prior's contribution to posterior inference in the presence of prior-likelihood discordance, a broader concept than the usual notion of prior-likelihood conflict. We achieve this dual…

Methodology · Statistics 2021-01-08 Matthew Reimherr , Xiao-Li Meng , Dan L. Nicolae

As the COVID-19 pandemic progresses, researchers are reporting findings of randomized trials comparing standard care with care augmented by experimental drugs. The trials have small sample sizes, so estimates of treatment effects are…

Econometrics · Economics 2020-06-02 Charles F. Manski , Aleksey Tetenov

After rejecting the null hypothesis in the analysis of variance, the next step is to make the pairwise comparisons to find out differences in means. The purpose of this paper is threefold. The foremost aim is to suggest expression for…

Methodology · Statistics 2023-06-22 Elsayed A. H. Elamir

Online controlled experiments (A/B tests) are fundamental to data-driven decision-making in the digital economy. However, their real-world application is frequently compromised by two critical shortcomings: the use of statistically flawed…

Applications · Statistics 2025-09-30 Srijesh Pillai , Rajesh Kumar Chandrawat

We consider the problem of sequential multiple hypothesis testing with nontrivial data collection costs. This problem appears, for example, when conducting biological experiments to identify differentially expressed genes of a disease…

Machine Learning · Computer Science 2023-11-06 Thomas Cook , Harsh Vardhan Dubey , Ji Ah Lee , Guangyu Zhu , Tingting Zhao , Patrick Flaherty

I propose Nonparametric Bayesian Policy Learning (NBPL) as a framework for uncertainty-aware treatment choice. I consider a decision-maker (DM) seeking to select an expected welfare-maximizing treatment rule using observable…

Econometrics · Economics 2026-05-19 Haonan Ye

Power and sample size analysis comprises a critical component of clinical trial study design. There is an extensive collection of methods addressing this problem from diverse perspectives. The Bayesian paradigm, in particular, has attracted…

Methodology · Statistics 2021-12-08 Jane Pan , Sudipto Banerjee

We study the problem of a decision maker who must provide the best possible treatment recommendation based on an experiment. The desirability of the outcome distribution resulting from the policy recommendation is measured through a…

Econometrics · Economics 2022-04-06 Anders Bredahl Kock , David Preinerstorfer , Bezirgen Veliyev

In clinical trials and other applications, we often see regions of the feature space that appear to exhibit interesting behaviour, but it is unclear whether these observed phenomena are reflected at the population level. Focusing on a…

Statistics Theory · Mathematics 2023-09-21 Henry W. J. Reeve , Timothy I. Cannings , Richard J. Samworth

We develop an empirical Bayes framework for experimental design that leverages information from prior related studies. When a researcher has access to estimates from previous studies on similar parameters, they can use empirical Bayes to…

Econometrics · Economics 2026-02-25 Zhiheng You

The design of experiments involves a compromise between covariate balance and robustness. This paper provides a formalization of this trade-off and describes an experimental design that allows experimenters to navigate it. The design is…

Methodology · Statistics 2023-11-16 Christopher Harshaw , Fredrik Sävje , Daniel Spielman , Peng Zhang

There is a movement in design of experiments away from the classic randomization put forward by Fisher, Cochran and others to one based on optimization. In fixed-sample trials comparing two groups, measurements of subjects are known in…

Methodology · Statistics 2018-10-22 Adam Kapelner , Abba M. Krieger , Uri Shalit , David Azriel

A/B testing, or controlled experiments, is the gold standard approach to causally compare the performance of algorithms on online platforms. However, conventional Bernoulli randomization in A/B testing faces many challenges such as…

Machine Learning · Computer Science 2023-02-13 Yongkang Guo , Yuan Yuan , Jinshan Zhang , Yuqing Kong , Zhihua Zhu , Zheng Cai

Composite binary endpoints are increasingly used as primary endpoints in clinical trials. When designing a trial, it is crucial to determine the appropriate sample size for testing the statistical differences between treatment groups for…

Applications · Statistics 2019-01-15 Marta Bofill Roig , Guadalupe Gómez Melis

Randomized controlled trials are often run in settings with many subpopulations that may have differential benefits from the treatment being evaluated. We consider the problem of sample selection, i.e., whom to enroll in a randomized trial,…

Methodology · Statistics 2024-06-26 Yuchen Hu , Henry Zhu , Emma Brunskill , Stefan Wager

Technological advancements in the field of mobile devices and wearable sensors have helped overcome obstacles in the delivery of care, making it possible to deliver behavioral treatments anytime and anywhere. Increasingly the delivery of…

Applications · Statistics 2017-11-13 Walter Dempsey , Peng Liao , Santosh Kumar , Susan A. Murphy
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