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There has been significant attention given to developing data-driven methods for tailoring patient care based on individual patient characteristics. Dynamic treatment regimes formalize this through a sequence of decision rules that map…

Methodology · Statistics 2022-02-22 Eric J. Rose , Erica E. M. Moodie , Susan Shortreed

Experimentation is widely utilized for causal inference and data-driven decision-making across disciplines. In an A/B experiment, for example, an online business randomizes two different treatments (e.g., website designs) to their customers…

Methodology · Statistics 2025-01-15 Wenxuan Guo , JungHo Lee , Panos Toulis

The I-SPY2 phase 2 clinical trial is a long-running platform trial that evaluates neoadjuvant treatments for locally advanced breast cancer, assigning subjects to novel agents using response-adaptive randomization. Recently, I-SPY2 was…

Pre-experiment stratification, or blocking, is a well-established technique for designing more efficient experiments and increasing the precision of the experimental estimates. However, when researchers have access to many covariates at the…

Econometrics · Economics 2025-10-01 George Gui , Seungwoo Kim

Two-sample tests with censored outcomes are a classical topic in statistics with wide use even in cutting edge applications. There are at least two modes of inference used to justify two-sample tests. One is usual superpopulation inference…

Statistics Theory · Mathematics 2022-10-06 Xinran Li , Dylan S. Small

The one-sample log-rank test is the method of choice for single-arm Phase II trials with time-to-event endpoint. It allows to compare the survival of the patients to a reference survival curve that typically represents the expected survival…

Methodology · Statistics 2026-03-02 Jannik Feld , Moritz Fabian Danzer , Andreas Faldum , Rene Schmidt

We propose a dynamic allocation procedure that increases power and efficiency when measuring an average treatment effect in sequential randomized trials. Subjects arrive iteratively and are either randomized or paired via a matching…

Methodology · Statistics 2013-05-23 Adam Kapelner , Abba Krieger

Background: Missing data poses an acute threat to sequential multiple assignment randomized trial (SMART) analyses because of the sequential treatment structure and response-dependent re-randomization. Objectives: This study aimed to (1)…

Researchers are increasingly turning to machine learning (ML) algorithms to investigate causal heterogeneity in randomized experiments. Despite their promise, ML algorithms may fail to accurately ascertain heterogeneous treatment effects…

Methodology · Statistics 2024-04-23 Kosuke Imai , Michael Lingzhi Li

Neurodegenerative diseases such as Alzheimer's and Parkinson's often exhibit complex, multivariate longitudinal outcomes that require advanced statistical methods to comprehensively evaluate treatment efficacy. The Longitudinal Rank Sum…

Methodology · Statistics 2025-02-12 Dhrubajyoti Ghosh , Xiaoming Xu , Sheng Luo

Both cluster randomized trials and quasi-experimental designs are used to evaluate the impact of health and social policies and interventions. Stepped-wedge cluster randomized trials randomize a staggered adoption approach, while recent…

Methodology · Statistics 2026-04-15 Haidong Lu , Gregg S. Gonsalves , Fan Li , Guanyu Tong , Lee Kennedy-Shaffer

Rerandomization is an experimental design technique that repeatedly randomizes treatment assignments until covariates are balanced between treatment groups. Rerandomization in the design stage of an experiment can lead to many asymptotic…

Methodology · Statistics 2026-04-10 Antônio Carlos Herling Ribeiro Junior

Modern high-throughput biomedical devices routinely produce data on a large scale, and the analysis of high-dimensional datasets has become commonplace in biomedical studies. However, given thousands or tens of thousands of measured…

Methodology · Statistics 2022-02-28 Vladimir Vutov , Thorsten Dickhaus

Randomized experiments ensure robust causal inference that are critical to effective learning analytics research and practice. However, traditional randomized experiments, like A/B tests, are limiting in large scale digital learning…

Applications · Statistics 2019-02-04 Timothy NeCamp , Josh Gardner , Christopher Brooks

This paper proposes a sequential test procedure for determining the number of regimes in nonlinear multivariate autoregressive models. The procedure relies on linearity and no additional nonlinearity tests for both multivariate smooth…

Econometrics · Economics 2024-06-05 Andrea Bucci

Major advances have been made regarding the utilization of artificial intelligence in health care. In particular, deep learning approaches have been successfully applied for automated and assisted disease diagnosis and prognosis based on…

Methodology · Statistics 2020-03-24 Max Westphal , Antonia Zapf , Werner Brannath

A new approach to adaptive design of clinical trials is proposed in a general multiparameter exponential family setting, based on generalized likelihood ratio statistics and optimal sequential testing theory. These designs are easy to…

Statistics Theory · Mathematics 2011-05-25 Jay Bartroff , Tze Leung Lai

Proportional hazards are a common assumption when designing confirmatory clinical trials in oncology. With the emergence of immunotherapy and novel targeted therapies, departure from the proportional hazard assumption is not rare in…

Methodology · Statistics 2020-08-27 José L. Jiménez

Gene-based testing is a commonly employed strategy in many genetic association studies. Gene-trait associations can be complex due to underlying population heterogeneity, gene-environment interactions, and various other reasons. Existing…

Methodology · Statistics 2020-12-15 Tianying Wang , Iuliana Ionita-Laza , Ying Wei

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