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Causal inference and model interpretability are gaining increasing attention, particularly in the biomedical domain. Despite recent advance, decorrelating features in nonlinear environments with human-interpretable representations remains…

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How should researchers analyze randomized experiments in which the main outcome is latent and measured in multiple ways but each measure contains some degree of error? We first identify a critical study-specific noncomparability problem in…

Econometrics · Economics 2026-01-13 Jiawei Fu , Donald P. Green

The estimand framework provides guidance on handling intercurrent events, such as treatment discontinuation, in the analysis of clinical trial responses. Under ICH E9(R1), the treatment policy (TP) strategy incorporates post-discontinuation…

Methodology · Statistics 2026-04-07 Myeongjong Kang , Sangyoon Yi

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

Survival is poor for patients with metastatic cancer, and it is vital to examine new biomarkers that can improve patient prognostication and identify those who would benefit from more aggressive therapy. In metastatic prostate cancer, two…

Methodology · Statistics 2024-08-07 Glenn Heller , Sean M. Devlin

Sequential parallel comparison design (SPCD) clinical trials aim to adjust active treatment effect estimates for placebo response to minimize the impact of placebo responders on the estimates. This is potentially accomplished using a two…

The aim of clinical effectiveness research using repositories of electronic health records is to identify what health interventions 'work best' in real-world settings. Since there are several reasons why the net benefit of intervention may…

Methodology · Statistics 2020-06-19 Jie Zhu , Blanca Gallego

A surrogate endpoint S in a clinical trial is an outcome that may be measured earlier or more easily than the true outcome of interest T. In this work, we extend causal inference approaches to validate such a surrogate using potential…

Methodology · Statistics 2022-02-04 Emily Roberts , Michael Elliott , Jeremy M. G. Taylor

Pre-treatment selection or censoring (`selection on treatment') can occur when two treatment levels are compared ignoring the third option of neither treatment, in `censoring by death' settings where treatment is only defined for those who…

Methodology · Statistics 2017-09-20 Edward H. Kennedy , Steve Harris , Luke J. Keele

Surrogate markers are often employed in clinical trials to replace primary outcomes that may be difficult, expensive, or time-consuming to measure directly. These markers can accelerate the evaluation of new treatments, provided they…

Methodology · Statistics 2026-03-17 Pietro Carlotti , Layla Parast

Estimating causal effects for survival outcomes in the high-dimensional setting is an extremely important topic for many biomedical applications as well as areas of social sciences. We propose a new orthogonal score method for treatment…

Methodology · Statistics 2024-12-04 Jue Hou , Jelena Bradic , Ronghui Xu

Precision medicine promises to transform health care by offering individualised treatments that dramatically improve clinical outcomes. A necessary prerequisite is to identify subgroups of patients who respond differently to different…

Machine Learning · Computer Science 2026-03-03 Adam Marcus , Paul Agapow

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

Randomized Controlled Trials (RCTs) are often considered the gold standard for estimating causal effect, but they may lack external validity when the population eligible to the RCT is substantially different from the target population.…

Methodology · Statistics 2023-01-11 Bénédicte Colnet , Julie Josse , Erwan Scornet , Gaël Varoquaux

We develop a criterion to certify whether causal effects are identifiable in linear structural equation models with latent variables. Linear structural equation models correspond to directed graphs whose nodes represent the random variables…

Statistics Theory · Mathematics 2025-07-25 Nils Sturma , Mathias Drton

Participants in longitudinal studies on the effects of drug treatment and criminal justice system interventions are at high risk for institutionalization (e.g., spending time in an environment where their freedom to use drugs, commit…

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Kaplan-Meier curves stratified by treatment allocation are the most popular way to depict causal effects in studies with right-censored time-to-event endpoints. If the treatment is randomly assigned and the sample size of the study is…

Methodology · Statistics 2024-02-26 Robin Denz , Nina Timmesfeld

Contrasting marginal counterfactual survival curves across treatment arms is an effective and popular approach for inferring the causal effect of an intervention on a right-censored time-to-event outcome. A key challenge to drawing such…

Methodology · Statistics 2022-04-29 Andrew Ying , Yifan Cui , Eric J. Tchetgen Tchetgen

Explanatory studies, such as randomized controlled trials, are targeted to extract the true causal effect of interventions on outcomes and are by design adjusted for covariates through randomization. On the contrary, observational studies…

Methodology · Statistics 2022-05-02 Riddhiman Adib , Sheikh Iqbal Ahamed , Mohammad Adibuzzaman

Considerations regarding clinical effectiveness and cost are essential in comparing the overall value of two treatments. There has been growing interest in methodology to integrate cost and effectiveness measures in order to inform policy…

Methodology · Statistics 2019-12-04 Andrew J. Spieker , Nicholas Illenberger , Jason A. Roy , Nandita Mitra