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We study the design of experiments with multiple treatment levels, a setting common in clinical trials and online A/B/n testing. Unlike single-treatment studies, practical analyses of multi-treatment experiments typically first select a…

Methodology · Statistics 2025-10-07 Jiachen Xu , Jian Qian , Zijun Gao

To draw real-world evidence about the comparative effectiveness of multiple time-varying treatments on patient survival, we develop a joint marginal structural survival model and a novel weighting strategy to account for time-varying…

Methodology · Statistics 2023-08-08 Liangyuan Hu , Jiayi Ji , Himanshu Joshi , Erick Scott , Fan Li

We study the problem of estimation of Individual Treatment Effects (ITE) in the context of multiple treatments and networked observational data. Leveraging the network information, we aim to utilize hidden confounders that may not be…

Machine Learning · Computer Science 2023-12-20 Abhinav Thorat , Ravi Kolla , Niranjan Pedanekar , Naoyuki Onoe

Covariate adjustment is a general method for improving precision when estimating treatment effects in randomized trials and is recommended by the FDA in its 2023 guidance when baseline variables are prognostic for the primary outcome. We…

Estimating varying treatment effects in randomized trials with noncompliance is inherently challenging since variation comes from two separate sources: variation in the impact itself and variation in the compliance rate. In this setting,…

Applications · Statistics 2024-08-28 Jared D. Fisher , David W. Puelz , Sameer K. Deshpande

Crossover designs randomly assign each unit to receive a sequence of treatments. By comparing outcomes within the same unit, these designs can effectively eliminate between-unit variation and facilitate the identification of both…

Methodology · Statistics 2026-04-21 Zhichao Jiang , Peng Ding

New crop varieties are extensively tested in multi-environment trials in order to obtain a solid empirical basis for recommendations to farmers. When the target population of environments is large and heterogeneous, a division into…

Applications · Statistics 2020-04-14 Maryna Prus , Hans-Peter Piepho

This paper studies Difference-in-Differences (DiD) setups with repeated cross-sectional data and potential compositional changes across time periods. We begin our analysis by deriving the efficient influence function and the semiparametric…

Econometrics · Economics 2025-11-17 Pedro H. C. Sant'Anna , Qi Xu

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

When using observational causal models, practitioners often want to disentangle the effects of many related, partially-overlapping treatments. Examples include estimating treatment effects of different marketing touchpoints, ordering…

Econometrics · Economics 2025-07-03 Enes Dilber , Colin Gray

Important questions for impact evaluation require knowledge not only of average effects, but of the distribution of treatment effects. The inability to observe individual counterfactuals makes answering these empirical questions…

Econometrics · Economics 2026-05-25 Bruno Fava

Determining which organizations are more effective in implementing an intervention program is essential for theoretically and empirically characterizing exemplary practice and for intervening to enhance the capacity of ineffective ones. Yet…

Applications · Statistics 2026-03-18 Guanglei Hong , Jonah Deutsch , Peter Kress , Jose Eos Trinidad , Zhengyan Xu

Estimating individualised treatment effect (ITE) -- that is the causal effect of a set of variables (also called exposures, treatments, actions, policies, or interventions), referred to as \textit{composite treatments}, on a set of outcome…

Machine Learning · Computer Science 2025-12-19 Vinod Kumar Chauhan , Lei Clifton , Gaurav Nigam , David A. Clifton

Multi-regional clinical trials (MRCTs) enable efficient global drug development by assessing treatment effects across regions within a single protocol. While powered for overall efficacy, MRCTs are typically not designed to provide…

We consider estimation of the target population average treatment effect (TATE) when outcome information is unavailable. Instead, we observe the outcome in multiple source populations and wish to combine the treatment effects therein to…

Methodology · Statistics 2025-05-16 Zehao Su , Helene Charlotte Rytgaard , Henrik Ravn , Frank Eriksson

Estimating Individual Treatment Effects (ITE) from observational data is challenging due to confounding bias. Most studies tackle this bias by balancing distributions globally, but ignore individual heterogeneity and fail to capture the…

Machine Learning · Computer Science 2025-11-14 Fuyuan Cao , Jiaxuan Zhang , Xiaoli Li

In evidence synthesis, effect modifiers are typically described as variables that induce treatment effect heterogeneity at the individual level, through treatment-covariate interactions in an outcome model parametrized at such level. As…

Methodology · Statistics 2026-05-07 Antonio Remiro-Azócar

Contextual sensing and delivery of digital interventions to improve health outcomes have gained significant traction in behavioral and psychiatric studies. Micro-randomized trials (MRTs) are a common experimental design for obtaining…

Methodology · Statistics 2025-04-01 Jieru Shi , Zhenke Wu , Walter Dempsey

Methods for estimating heterogeneous treatment effect in observational data have largely focused on continuous or binary outcomes, and have been relatively less vetted with survival outcomes. Using flexible machine learning methods in the…

Applications · Statistics 2021-07-09 Liangyuan Hu , Jiayi Ji , Fan Li

Clinical trials traditionally employ blinding as a design mechanism to reduce the influence of placebo effects. In practice, however, it can be difficult or impossible to blind study participants and unblinded trials are common in medical…

Applications · Statistics 2016-06-22 Elias Chaibub Neto