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Multiple outcomes, both continuous and discrete, are routinely gathered on subjects in longitudinal studies and during routine clinical follow-up in general. To motivate our work, we consider a longitudinal study on patients with primary…

Applications · Statistics 2013-04-17 Arnošt Komárek , Lenka Komárková

Clustered and longitudinal data are pervasive in scientific studies, from prenatal health programs to clinical trials and public health surveillance. Such data often involve non-Gaussian responses--including binary, categorical, and count…

Methodology · Statistics 2025-09-19 Yibo Wang , Chenlei Leng , Cheng Yong Tang

Investigators often use multi-source data (e.g., multi-center trials, meta-analyses of randomized trials, pooled analyses of observational cohorts) to learn about the effects of interventions in subgroups of some well-defined target…

Methodology · Statistics 2024-02-06 Guanbo Wang , Alexander Levis , Jon Steingrimsson , Issa Dahabreh

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

A new meta-algorithm for estimating the conditional average treatment effects is proposed in the paper. The main idea underlying the algorithm is to consider a new dataset consisting of feature vectors produced by means of concatenation of…

Machine Learning · Statistics 2019-09-10 Lev V. Utkin , Mikhail V. Kots , Viacheslav S. Chukanov

Causal inference is widely used in various fields, such as biology, psychology and economics, etc. In observational studies, we need to balance the covariates before estimating causal effect. This study extends the one-dimensional entropy…

Methodology · Statistics 2022-05-19 Juan Chen , Yingchun Zhou

In Randomised Controlled Trials (RCT) with treatment non-compliance, instrumental variable approaches are used to estimate complier average causal effects. We extend these approaches to cost-effectiveness analyses, where methods need to…

Methodology · Statistics 2016-12-02 Karla DiazOrdaz , Angelo Franchini , Richard Grieve

Most cluster randomized trials (CRTs) randomize fewer than 30-40 clusters in total. When performing inference for such ``small'' CRTs, it is important to use methods that appropriately account for the small sample size. When the generalized…

Methodology · Statistics 2025-12-01 Shifeng Sun , Xueqi Wang , Zhuoran Hou , Elizabeth L. Turner

Statistical power is often a concern for clustered RCTs due to variance inflation from design effects and the high cost of adding study clusters (such as hospitals, schools, or communities). While covariate pre-specification is the…

Methodology · Statistics 2020-05-07 Peter Z. Schochet

Practitioners in diverse fields such as healthcare, economics and education are eager to apply machine learning to improve decision making. The cost and impracticality of performing experiments and a recent monumental increase in electronic…

Machine Learning · Computer Science 2023-08-01 Fredrik D. Johansson , Uri Shalit , Nathan Kallus , David Sontag

In many social, behavioral, and biomedical sciences, treatment effect estimation is a crucial step in understanding the impact of an intervention, policy, or treatment. In recent years, an increasing emphasis has been placed on…

Methodology · Statistics 2024-10-10 Xinhai Zhang , Xingye Qiao

We propose causal effect estimators based on empirical Fr\'{e}chet means and operator-valued kernels, tailored to functional data spaces. These methods address the challenges of high-dimensionality, sequential ordering, and model complexity…

Methodology · Statistics 2025-06-04 Yordan P. Raykov , Hengrui Luo , Justin D. Strait , Wasiur R. KhudaBukhsh

Multistate process data are common in studies of chronic diseases such as cancer. These data are ideal for precision medicine purposes as they can be leveraged to improve more refined health outcomes, compared to standard survival outcomes,…

Methodology · Statistics 2022-11-28 Giorgos Bakoyannis

Recent methods to improve generalizations from nonrandom samples typically invoke assumptions such as the strong ignorability of sample selection that are often controversial in practice to derive point estimates. Rather than focus on the…

Applications · Statistics 2017-01-06 Wendy Chan

Randomized controlled trials (RCTs) are increasingly prevalent in education research, and are often regarded as a gold standard of causal inference. Two main virtues of randomized experiments are that they (1) do not suffer from…

We describe methods that extend (generalize or transport) causal inferences from cluster randomized trials to a target population of clusters, under a general nonparametric model that allows for arbitrary within-cluster dependence. We…

Randomized Controlled Trials (RCTs) represent a gold standard when developing policy guidelines. However, RCTs are often narrow, and lack data on broader populations of interest. Causal effects in these populations are often estimated using…

Machine Learning · Computer Science 2023-03-07 Zeshan Hussain , Michael Oberst , Ming-Chieh Shih , David Sontag

We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials…

Methodology · Statistics 2021-07-08 Laura B. Balzer , Wenjing Zheng , Mark J. van der Laan , Maya L. Petersen

In stepped wedge cluster randomized trials (SW-CRTs), observations collected under the control condition are, on average, from an earlier time than observations collected under the intervention condition. In a cohort design, participants…

Methodology · Statistics 2023-02-23 Jale Basten , Katja Ickstadt , Nina Timmesfeld

When treatments are non-randomly assigned, continuous, and yield heterogeneous effects at the same intensity, causal identification becomes particularly challenging. In such contexts, existing approaches often fail to provide…

Econometrics · Economics 2026-03-05 Augusto Cerqua , Roberta Di Stefano , Raffaele Mattera
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