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Identifying subgroups, which respond differently to a treatment, both in terms of efficacy and safety, is an important part of drug development. A well-known challenge in exploratory subgroup analyses is the small sample size in the…

Computation · Statistics 2016-06-28 Marius Thomas , Björn Bornkamp

Effect modification occurs when the effect of the treatment on an outcome differs according to the level of a third variable (the effect modifier, EM). A natural way to assess effect modification is by subgroup analysis or include the…

Methodology · Statistics 2021-12-22 Asma Bahamyirou , Mireille E. Schnitzer , Edward H. Kennedy , Lucie Blais , Yi Yang

Beyond conditional average treatment effects, treatments may impact the entire outcome distribution in covariate-dependent ways, for example, by altering the variance or tail risks for specific subpopulations. We propose a novel estimand to…

Machine Learning · Statistics 2026-03-18 Saksham Jain , Alex Luedtke

Cohort studies employ pairwise measures of association to quantify dependencies among conditions and exposures. To reliably use these measures to draw conclusions about the underlying association strengths requires that the measures be…

Quantitative Methods · Quantitative Biology 2017-05-30 Venkateshan Kannan , Kristina Alexandersson , Jesper Tegner

Batch effects are pervasive in biomedical studies. One approach to address the batch effects is repeatedly measuring a subset of samples in each batch. These remeasured samples are used to estimate and correct the batch effects. However,…

Methodology · Statistics 2023-11-07 Hanxuan Ye , Xianyang Zhang , Chen Wang , Ellen L. Goode , Jun Chen

Understanding treatment effect heterogeneity has become increasingly important in many fields. In this paper we study distributions and quantiles of individual treatment effects to provide a more comprehensive and robust understanding of…

Methodology · Statistics 2026-03-31 Zhe Chen , Xinran Li

A sensitivity analysis in an observational study assesses the robustness of significant findings to unmeasured confounding. While sensitivity analyses in matched observational studies have been well addressed when there is a single outcome…

Methodology · Statistics 2015-11-05 Colin B. Fogarty , Dylan S. Small

We provide a principled way for investigators to analyze randomized experiments when the number of covariates is large. Investigators often use linear multivariate regression to analyze randomized experiments instead of simply reporting the…

Statistics Theory · Mathematics 2022-06-08 Adam Bloniarz , Hanzhong Liu , Cun-Hui Zhang , Jasjeet Sekhon , Bin Yu

In observational studies, identification of ATEs is generally achieved by assuming that the correct set of confounders has been measured and properly included in the relevant models. Because this assumption is both strong and untestable, a…

Methodology · Statistics 2020-12-18 Matteo Bonvini , Edward H Kennedy

I introduce a simple permutation procedure to test conventional (non-sharp) hypotheses about the effect of a binary treatment in the presence of a finite number of large, heterogeneous clusters when the treatment effect is identified by…

Econometrics · Economics 2023-02-08 Andreas Hagemann

We propose a novel approach for causal mediation analysis based on changes-in-changes assumptions restricting unobserved heterogeneity over time. This allows disentangling the causal effect of a binary treatment on a continuous outcome into…

Econometrics · Economics 2020-10-13 Martin Huber , Mark Schelker , Anthony Strittmatter

This commentary regards a recent simulation study conducted by Aouni, Gaudel-Dedieu and Sebastien, evaluating the performance of different versions of matching-adjusted indirect comparison (MAIC) in an anchored scenario with a common…

Methodology · Statistics 2022-03-29 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

In this paper, we develop new methods for estimating average treatment effects in observational studies, focusing on settings with more than two treatment levels under unconfoundedness given pre-treatment variables. We emphasize…

Methodology · Statistics 2017-10-11 Shu Yang , Guido W. Imbens , Zhanglin Cui , Douglas Faries , Zbigniew Kadziola

Observational studies are valuable tools for inferring causal effects in the absence of controlled experiments. However, these studies may be biased due to the presence of some relevant, unmeasured set of covariates. One approach to…

Methodology · Statistics 2026-02-17 William Bekerman , Abhinandan Dalal , Carlo del Ninno , Dylan S. Small

In the recent literature on estimating heterogeneous treatment effects, each proposed method makes its own set of restrictive assumptions about the intervention's effects and which subpopulations to explicitly estimate. Moreover, the…

Methodology · Statistics 2023-05-12 Edward McFowland , Sriram Somanchi , Daniel B. Neill

The Mann-Whitney effect is an effect measure for the order of two sample-specific outcome variables. It has the interpretation of a probability and also a connection to the area under the ROC curve. In the literature it has been considered…

Methodology · Statistics 2026-04-02 Dennis Dobler , Alina Schenk , Matthias Schmid

To ensure reliable causal conclusions from observational (i.e., non-randomized) studies, researchers routinely conduct sensitivity analysis to assess robustness to hidden bias due to unmeasured confounding. In matched observational studies…

Methodology · Statistics 2025-11-11 Siyu Heng , Elaine K. Chiu , Hyunseung Kang

Effect modification occurs when a covariate alters the relative effectiveness of treatment compared to control. It is widely understood that, when effect modification is present, treatment recommendations may vary by population and by…

We consider the problem of estimating the effects of a binary treatment on a continuous outcome of interest from observational data in the absence of confounding by unmeasured factors. We provide a new estimator of the population average…

Methodology · Statistics 2020-08-04 James Robins , Mariela Sued , Quanhong Lei-Gomez , Andrea Rotnitzky

Population-adjusted indirect comparisons estimate treatment effects when access to individual patient data is limited and there are cross-trial differences in effect modifiers. Popular methods include matching-adjusted indirect comparison…

Applications · Statistics 2021-11-05 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio