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The probability of benefit is a valuable and important measure of treatment effect, which has advantages over the average treatment effect. Particularly for an ordinal outcome, it has a better interpretation and can make apparent different…

Methodology · Statistics 2023-05-19 Erin E Gabriel , Michael C Sachs , Andreas Kryger Jensen

Treatment effect estimands based on win statistics, including the win ratio, win odds, and win difference are increasingly popular targets for summarizing endpoints in clinical trials. Such win estimands offer an intuitive approach for…

Methodology · Statistics 2026-02-13 Kenneth M. Lee , Xi Fang , Fan Li , Michael O. Harhay

This paper investigates the finite sample performance of a range of parametric, semi-parametric, and non-parametric instrumental variable estimators when controlling for a fixed set of covariates to evaluate the local average treatment…

Econometrics · Economics 2022-12-15 Hugo Bodory , Martin Huber , Michael Lechner

Multi-target regression is useful in a plethora of applications. Although random forest models perform well in these tasks, they are often difficult to interpret. Interpretability is crucial in machine learning, especially when it can…

Machine Learning · Computer Science 2023-03-30 Avraam Bardos , Nikolaos Mylonas , Ioannis Mollas , Grigorios Tsoumakas

Clinical researchers often select among and evaluate risk prediction models using standard machine learning metrics based on confusion matrices. However, if these models are used to allocate interventions to patients, standard metrics…

Machine Learning · Statistics 2020-06-03 Alejandro Schuler , Aashish Bhardwaj , Vincent Liu

Fields like public health, public policy, and social science often want to quantify the degree of dependence between variables whose relationships take on unknown functional forms. Typically, in fact, researchers in these fields are…

Statistics Theory · Mathematics 2019-12-10 Octavio César Mesner , Cosma Rohilla Shalizi

Respondent-driven sampling is a widely-used network sampling technique, designed to sample from hard-to-reach populations. Estimation from the resulting samples is an area of active research, with software available to compute at least four…

Applications · Statistics 2010-12-21 Amber Tomas , Krista J. Gile

In a completely randomized experiment, the variances of treatment effect estimators in the finite population are usually not identifiable and hence not estimable. Although some estimable bounds of the variances have been established in the…

Statistics Theory · Mathematics 2022-09-20 Ruoyu Wang , Qihua Wang , Wang Miao , Xiaohua Zhou

Health disparity research often evaluates health outcomes across demographic subgroups. Multilevel regression and poststratification (MRP) is a popular approach for small subgroup estimation due to its ability to stabilize estimates by…

Methodology · Statistics 2023-06-26 Katherine Li , Yajuan Si

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

Methodology · Statistics 2026-01-22 Xinyuan Chen , Fan Li

Many interventions are both beneficial to initiate and harmful to stop. Traditionally, to determine whether to deploy that intervention in a time-limited way depends on if, on average, the increase in the benefits of starting it outweigh…

The comparison of different medical treatments from observational studies or across different clinical studies is often biased by confounding factors such as systematic differences in patient demographics or in the inclusion criteria for…

Methodology · Statistics 2025-05-15 Ekkehard Glimm , Lillian Yau

Interference occurs when a unit's treatment (or exposure) affects another unit's outcome. In some settings, units may be grouped into clusters such that it is reasonable to assume that interference, if present, only occurs between…

Methodology · Statistics 2023-08-24 Chanhwa Lee , Donglin Zeng , Michael G. Hudgens

Although treatment effects can be estimated from observed outcome distributions obtained from proper randomization in clinical trials, covariate adjustment is recommended to increase precision. For important treatment effects, such as odds…

Methodology · Statistics 2025-07-03 Susanne Dandl , Torsten Hothorn

When extending inferences from a randomized trial to a new target population, the transportability condition for conditional difference effect measures is invoked to identify the marginal causal mean difference in the target population.…

Methodology · Statistics 2024-07-18 Guanbo Wang , Alexander Levis , Jon Steingrimsson , Issa Dahabreh

To take sample biases and skewness in the observations into account, practitioners frequently weight their observations according to some marginal distribution. The present paper demonstrates that such weighting can indeed improve the…

Methodology · Statistics 2018-11-05 Tobias Niebuhr , Mathias Trabs

Researchers are often interested in treatment effects on outcomes that are only defined conditional on a post-treatment event status. For example, in a study of the effect of different cancer treatments on quality of life at end of…

Causal inference concerns not only the average effect of the treatment on the outcome but also the underlying mechanism through an intermediate variable of interest. Principal stratification characterizes such a mechanism by targeting…

Methodology · Statistics 2022-03-29 Zhichao Jiang , Shu Yang , Peng Ding

Although randomized controlled trials (RCTs) are a cornerstone of comparative effectiveness, they typically have much smaller sample size than observational studies because of financial and ethical considerations. Therefore there is…

Methodology · Statistics 2023-11-16 Lauren D. Liao , Emilie Højbjerre-Frandsen , Alan E. Hubbard , Alejandro Schuler

Provider profiling has the goal of identifying healthcare providers with exceptional patient outcomes. When evaluating providers, adjustment is necessary to control for differences in case-mix between different providers. Direct and…

Methodology · Statistics 2024-10-28 Herbert Susmann , Yiting Li , Mara A. McAdams-DeMarco , Iván Díaz , Wenbo Wu
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