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The proliferation of early diagnostic technologies, including self-monitoring systems and wearables, coupled with the application of these technologies on large segments of healthy populations may significantly aggravate the problem of…

Machine Learning · Computer Science 2021-07-23 Anna Fedyukova , Douglas Pires , Daniel Capurro

Data collected in clinical trials are often composed of multiple types of variables. For example, laboratory measurements and vital signs are longitudinal data of continuous or categorical variables, adverse events may be recurrent events,…

Methodology · Statistics 2023-01-12 Tuo Wang , Rachel Zilinskas , Ying Li , Yongming Qu

In epidemic or pandemic situations, resources for testing the infection status of individuals may be scarce. Although group testing can help to significantly increase testing capabilities, the (repeated) testing of entire populations can…

Populations and Evolution · Quantitative Biology 2021-10-29 Günther Koliander , Georg Pichler

In most nonrandomized observational studies, differences between treatment groups may arise not only due to the treatment but also because of the effect of confounders. Therefore, causal inference regarding the treatment effect is not as…

Methodology · Statistics 2018-07-04 Debashis Ghosh

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

While observational data are routinely used to estimate causal effects of biomedical treatments, doing so requires special methods to adjust for observed confounding. These methods invariably rely on untestable statistical and causal…

Methodology · Statistics 2026-03-02 Arman Oganisian

In this paper, we consider recent progress in estimating the average treatment effect when extreme inverse probability weights are present and focus on methods that account for a possible violation of the positivity assumption. These…

Methodology · Statistics 2022-10-26 Roland A. Matsouaka , Yunji Zhou

Many scientific questions in biomedical, environmental, and psychological research involve understanding the effects of multiple factors on outcomes. While factorial experiments are ideal for this purpose, randomized controlled treatment…

Methodology · Statistics 2025-12-03 Ruoqi Yu , Peng Ding

Researchers addressing post-treatment complications in randomized trials often turn to principal stratification to define relevant assumptions and quantities of interest. One approach for estimating causal effects in this framework is to…

Methodology · Statistics 2016-06-09 Avi Feller , Fabrizia Mealli , Luke Miratrix

In multicenter randomized trials, when effect modifiers have a different distribution across centers, comparisons between treatment groups that average over centers may not apply to any of the populations underlying the individual centers.…

Interference arises when an individual's potential outcome depends on the individual treatment level, but also on the treatment level of others. A common assumption in the causal inference literature in the presence of interference is…

Methodology · Statistics 2018-05-15 Georgia Papadogeorgou , Fabrizia Mealli , Corwin M. Zigler

Missing data is unavoidable in longitudinal clinical trials, and outcomes are not always normally distributed. In the presence of outliers or heavy-tailed distributions, the conventional multiple imputation with the mixed model with…

Methodology · Statistics 2022-03-22 Siyi Liu , Yilong Zhang , Gregory T Golm , Guanghan , Liu , Shu Yang

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured…

Methodology · Statistics 2022-02-18 Liangyuan Hu , Jiayi Ji , Ronald D. Ennis , Joseph W. Hogan

Estimating heterogeneous treatment effects has become increasingly important in many fields and life and death decisions are now based on these estimates: for example, selecting a personalized course of medical treatment. Recently, a…

Methodology · Statistics 2019-04-01 Sören R. Künzel , Simon J. S. Walter , Jasjeet S. Sekhon

Dropout poses a significant challenge to causal inference in longitudinal studies with time-varying treatments. However, existing research does not simultaneously address dropout and time-varying treatments. We examine selective…

Methodology · Statistics 2025-03-18 Zhichao Jiang , Eli Ben-Michael , D. James Greiner , Ryan Halen , Kosuke Imai

Biomarker measurements can be relatively easy and quick to obtain and they are useful to investigate whether a compound works as intended on a mechanistic, pharmacological level. In some situations, it is realistic to assume that patients,…

Methodology · Statistics 2018-06-26 Björn Bornkamp , Georgina Bermann

Clinical prediction models (CPMs) are used to predict clinically relevant outcomes or events. Typically, prognostic CPMs are derived to predict the risk of a single future outcome. However, with rising emphasis on the prediction of…

Methodology · Statistics 2020-10-29 Glen P. Martin , Matthew Sperrin , Kym I. E. Snell , Iain Buchan , Richard D. Riley

For testing the statistical significance of a treatment effect, we usually compare between two parts of a population, one is exposed to the treatment, and the other is not exposed to it. Standard parametric and nonparametric two-sample…

Computation · Statistics 2012-11-02 Bikram Karmakar , Kumaresh Dhara , Kushal Kumar Dey , Analabha Basu , Anil Ghosh

Estimating causal effects with propensity scores relies upon the availability of treated and untreated units observed at each value of the estimated propensity score. In settings with strong confounding, limited so-called "overlap" in…

Methodology · Statistics 2017-10-25 Corwin M Zigler , Matthew Cefalu

In this paper, we study causal inference when the treatment variable is an aggregation of multiple sub-treatment variables. Researchers often report marginal causal effects for the aggregated treatment, implicitly assuming that the target…

Econometrics · Economics 2026-01-08 Carolina Caetano , Gregorio Caetano , Brantly Callaway , Derek Dyal
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