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In randomized trials, repeated measures of the outcome are routinely collected. The mixed model for repeated measures (MMRM) leverages the information from these repeated outcome measures, and is often used for the primary analysis to…

Methodology · Statistics 2023-07-20 Bingkai Wang , Yu Du

In causal mediation analysis, the natural direct and indirect effects (natural effects) are nonparametrically unidentifiable in the presence of treatment-induced confounding, which motivated the development of randomized interventional…

Methodology · Statistics 2025-07-22 Ang Yu , Li Ge , Felix Elwert

While randomized trials may be the gold standard for evaluating the effectiveness of the treatment intervention, in some special circumstances, single-arm clinical trials utilizing external control may be considered. The causal treatment…

Methodology · Statistics 2025-05-26 Huan Wang , Fei Wu , Yeh-Fong Chen

In high-stakes settings where machine learning models are used to automate decision-making about individuals, the presence of algorithmic bias can exacerbate systemic harm to certain subgroups of people. These biases often stem from the…

Machine Learning · Computer Science 2026-04-07 Erin Tan , Judy Hanwen Shen , Irene Y. Chen

We examine four important considerations in the development of covariate adjustment methodologies for indirect treatment comparisons. Firstly, we consider potential advantages of weighting versus outcome modeling, placing focus on…

Methodology · Statistics 2026-05-07 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

Case-mix heterogeneity across studies complicates meta-analyses. As a result of this, treatments that are equally effective on patient subgroups may appear to have different effectiveness on patient populations with different case mix. It…

Applications · Statistics 2021-08-25 Tat-Thang Vo , Raphael Porcher , Anna Chaimani , Stijn Vansteelandt

Disagreement remains on what the target estimand should be for population-adjusted indirect treatment comparisons. This debate is of central importance for policy-makers and applied practitioners in health technology assessment.…

Methodology · Statistics 2022-12-06 Antonio Remiro-Azócar

It has been shown that AIC-type criteria are asymptotically efficient selectors of the tuning parameter in non-concave penalized regression methods under the assumption that the population variance is known or that a consistent estimator is…

Machine Learning · Statistics 2017-03-02 Cheryl J. Flynn , Clifford M. Hurvich , Jeffrey S. Simonoff

We consider the problem of indirect comparison, where a treatment arm of interest is absent by design in one randomized controlled trial but available in the other. The former is the target trial, and the latter is the source trial. The…

Methodology · Statistics 2025-06-06 Zehao Su , Helene C. W. Rytgaard , Henrik Ravn , Frank Eriksson

Difference-in-differences (DID) is one of the most popular tools used to evaluate causal effects of policy interventions. This paper extends the DID methodology to accommodate interval outcomes, which are often encountered in empirical…

Econometrics · Economics 2025-12-10 Daisuke Kurisu , Yuta Okamoto , Taisuke Otsu

Many probabilistic models that have an intractable normalizing constant may be extended to contain covariates. Since the evaluation of the exact likelihood is difficult or even impossible for these models, score matching was proposed to…

Statistics Theory · Mathematics 2022-03-21 Jiazhen Xu , Janice L. Scealy , Andrew T. A. Wood , Tao Zou

This paper studies inference for the local average treatment effect in randomized controlled trials with imperfect compliance where treatment status is determined according to "matched pairs." By "matched pairs," we mean that units are…

Econometrics · Economics 2024-06-28 Yuehao Bai , Hongchang Guo , Azeem M. Shaikh , Max Tabord-Meehan

Heterogeneous treatment effects, which vary according to individual covariates, are crucial in fields such as personalized medicine and tailored treatment strategies. In many applications, rather than considering the heterogeneity induced…

Methodology · Statistics 2025-08-26 Peng Wu , Pengtao Zeng , Zhaoqing Tian , Shaojie Wei

The paper presents an investigation of estimating treatment effect using different matching methods. The study proposed a new method which is computationally efficient and convenient in implication-'largest caliper matching' and compared…

Applications · Statistics 2018-06-07 Sharif Mahmood

Imbalances in covariates between treatment groups are frequent in observational studies and can lead to biased comparisons. Various adjustment methods can be employed to correct these biases in the context of multi-level treatments ($>$ 2).…

Applications · Statistics 2021-06-04 Diop S. Arona , Duchesne Thierry , Cumming Steven , Diop Awa , Talbot Denis

Recently, there has been a surge in methodological development for the difference-in-differences (DiD) approach to evaluate causal effects. Standard methods in the literature rely on the parallel trends assumption to identify the average…

Methodology · Statistics 2023-10-17 Pan Zhao , Yifan Cui

In observational causal inference, exact covariate matching plays two statistical roles: (i) it effectively controls for bias due to measured confounding; (ii) it justifies assumption-free inference based on randomization tests. This paper…

Methodology · Statistics 2022-12-02 Kevin Guo , Dominik Rothenhäusler

Diagnostic accuracy studies assess sensitivity and specificity of a new index test in relation to an established comparator or the reference standard. The development and selection of the index test is usually assumed to be conducted prior…

Methodology · Statistics 2022-08-30 Max Westphal , Antonia Zapf

The method of difference-in-differences (DID) is widely used to study the causal effect of policy interventions in observational studies. DID employs a before and after comparison of the treated and control units to remove bias due to…

Methodology · Statistics 2022-06-15 Ting Ye , Luke Keele , Raiden Hasegawa , Dylan S. Small

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of…

Methodology · Statistics 2023-06-12 Kelly Van Lancker , Frank Bretz , Oliver Dukes