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Conditional effect estimation has great scientific and policy importance because interventions may impact subjects differently depending on their characteristics. Most research has focused on estimating the conditional average treatment…

Methodology · Statistics 2023-04-25 Alec McClean , Zach Branson , Edward H. Kennedy

In learning-phase clinical trials in drug development, adaptive designs can be efficient and highly informative when used appropriately. In this article, we extend the multiple comparison procedures with modeling techniques (MCP-Mod)…

Methodology · Statistics 2021-08-02 Shiyang Ma , Michael P. McDermott

In cluster-randomized crossover (CRXO) trials, groups of individuals are randomly assigned to two or more sequences of alternating treatments. Since clusters serve as their own control, the CRXO design is typically more statistically…

Methodology · Statistics 2026-01-30 Dane Isenberg , Michael O. Harhay , Andrew B. Forbes , Paul J. Young , Fan Li , Nandita Mitra

Many research questions in public health and medicine concern sustained interventions in populations defined by substantive priorities. Existing methods to answer such questions typically require a measured covariate set sufficient to…

Methodology · Statistics 2023-06-30 Audrey Renson , Michael Hudgens , Alexander Keil , Paul Zivich , Allison Aiello

Doubly robust estimators of causal effects are a popular means of estimating causal effects. Such estimators combine an estimate of the conditional mean of the outcome given treatment and confounders (the so-called outcome regression) with…

Methodology · Statistics 2019-01-17 David Benkeser , Weixin Cai , Mark J van der Laan

When making treatment selection decisions, it is essential to include a causal effect estimation analysis to compare potential outcomes under different treatments or controls, assisting in optimal selection. However, merely estimating…

Machine Learning · Statistics 2024-10-08 Sherly Alfonso-Sánchez , Kristina P. Sendova , Cristián Bravo

Randomized controlled trials (RCTs) can be used to generate guarantees on treatment effects. However, RCTs often spend unnecessary resources exploring sub-optimal treatments, which can reduce the power of treatment guarantees. To address…

Computers and Society · Computer Science 2024-10-16 Santiago Cortes-Gomez , Naveen Raman , Aarti Singh , Bryan Wilder

In cluster-randomized trials (CRTs), there is emerging interest in exploring the causal mechanism in which a cluster-level treatment affects the outcome through an intermediate outcome. The majority of existing causal mediation methods are…

Methodology · Statistics 2026-01-12 Chao Cheng , Fan Li

Oversubscribed treatments are often allocated using randomized waiting lists. Applicants are ranked randomly, and treatment offers are made following that ranking until all seats are filled. To estimate causal effects, researchers often…

Methodology · Statistics 2018-10-24 Clement de Chaisemartin , Luc Behaghel

This paper studies inference in two-stage randomized experiments under covariate-adaptive randomization. In the initial stage of this experimental design, clusters (e.g., households, schools, or graph partitions) are stratified and randomly…

Econometrics · Economics 2026-01-16 Jizhou Liu

In this paper, we consider an experimental setting where units enter the experiment sequentially. Our goal is to form stopping rules which lead to estimators of treatment effects with a given precision. We propose a fixed-width confidence…

Statistics Theory · Mathematics 2024-05-07 Mattias Nordin , Mårten Schultzberg

The difference-in-differences (DID) research design is a key identification strategy which allows researchers to estimate causal effects under the parallel trends assumption. While the parallel trends assumption is counterfactual and cannot…

Methodology · Statistics 2026-05-12 Jonas M. Mikhaeil , Christopher Harshaw

In a unified framework, we provide estimators and confidence bands for a variety of treatment effects when the outcome of interest, typically a duration, is subjected to right censoring. Our methodology accommodates average, distributional,…

Methodology · Statistics 2017-10-04 Pedro H. C. Sant'Anna

While palliative care is increasingly commonly delivered to hospitalized patients with serious illnesses, few studies have estimated its causal effects. Courtright et al. (2016) adopted a cluster-randomized stepped-wedge design to assess…

Randomized clinical trials are considered the gold standard for estimating causal effects. Nevertheless, in studies that are aimed at examining adverse effects of interventions, such trials are often impractical because of ethical and…

Methodology · Statistics 2020-01-20 Anthony D. Scotina , Andrew R. Zullo , Robert J. Smith , Roee Gutman

This article studies the benefits of using spatially randomized experimental designs which partition the experimental area into distinct, non-overlapping units with treatments assigned randomly. Such designs offer improved policy evaluation…

Statistics Theory · Mathematics 2025-11-18 Ying Yang , Chengchun Shi , Fang Yao , Shouyang Wang , Hongtu Zhu

A treatment may be appropriate for some group (the ``sick" group) on whom it has a positive effect, but it can also have a detrimental effect on subjects from another group (the ``healthy" group). In a non-targeted trial both sick and…

Machine Learning · Computer Science 2025-04-23 Georgios Mavroudeas , Malik Magdon-Ismail , Kristin P. Bennett , Jason Kuruzovich

Causal identification of treatment effects for infectious disease outcomes in interconnected populations is challenging because infection outcomes may be transmissible to others, and treatment given to one individual may affect others'…

Methodology · Statistics 2021-05-11 Xiaoxuan Cai , Eben Kenah , Forrest W. Crawford

Estimating the effect of medical treatments on subject responses is one of the crucial problems in medical research. Matched-pairs designs are commonly implemented in the field of medical research to eliminate confounding and improve…

Methodology · Statistics 2017-11-08 Jun Wang , Wei Gao , Man-Lai Tang

Background: Children are frequently prescribed medication off-label, meaning there has not been sufficient testing of the medication to determine its safety or effectiveness. The main reason this safety knowledge is lacking is due to…

Machine Learning · Computer Science 2014-09-03 Jenna M. Reps , Jonathan M. Garibaldi , Uwe Aickelin , Daniele Soria , Jack E. Gibson , Richard B. Hubbard