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Related papers: Handling Missing Data in Within-Trial Cost-Effecti…

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Practical problems with missing data are common, and statistical methods have been developed concerning the validity and/or efficiency of statistical procedures. On a central focus, there have been longstanding interests on the mechanism…

Methodology · Statistics 2020-03-26 Rui Duan , C. Jason Liang , Pamela Shaw , Cheng Yong Tang , Yong Chen

Citing comprehensively and appropriately has become a challenging task with the explosive growth of scientific publications. Current citation recommendation systems aim to recommend a list of scientific papers for a given text context or a…

Information Retrieval · Computer Science 2024-03-05 Kehan Long , Shasha Li , Pancheng Wang , Chenlong Bao , Jintao Tang , Ting Wang

Estimating conditional average treatment effects (CATE) is challenging, especially when treatment information is missing. Although this is a widespread problem in practice, CATE estimation with missing treatments has received little…

Machine Learning · Statistics 2023-04-19 Milan Kuzmanovic , Tobias Hatt , Stefan Feuerriegel

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

Missing data is a pernicious problem in epidemiologic research. Research on the validity of complete case analysis for missing data has typically focused on estimating the average treatment effect (ATE) in the whole population. However,…

Methodology · Statistics 2024-01-30 Michael Webster-Clark , Rachael K Ross

Cluster randomized trials (CRTs) randomly assign an intervention to groups of individuals (e.g., clinics or communities) and measure outcomes on individuals in those groups. While offering many advantages, this experimental design…

Conditional average treatment effect (CATE) estimation is the de facto gold standard for targeting a treatment to a heterogeneous population. The method estimates treatment effects up to an error $\epsilon > 0$ in each of $M$ different…

Machine Learning · Computer Science 2026-01-12 Sílvia Casacuberta , Moritz Hardt

Randomized Controlled Trials (RCT)s are relied upon to assess new treatments, but suffer from limited power to guide personalized treatment decisions. On the other hand, observational (i.e., non-experimental) studies have large and diverse…

Methodology · Statistics 2023-03-07 Zeshan Hussain , Ming-Chieh Shih , Michael Oberst , Ilker Demirel , David Sontag

Accurately estimating the intra-class correlation coefficient (ICC) is crucial for adequately powering clustered randomized trials (CRTs). Challenges arise due to limited prior data on the specific outcome within the target population,…

Methodology · Statistics 2025-04-23 Chen Yang , Márcio A. Diniz , Deukwoo Kwon , Madhu Mazumdar

Randomized experiments can provide unbiased estimates of sample average treatment effects. However, estimates of population treatment effects can be biased when the experimental sample and the target population differ. In this case, the…

Methodology · Statistics 2022-11-10 Wenqi Shi , Xi Lin

This vision paper demonstrates that it is crucial to consider Return-on-Investment (ROI) when performing Data Analytics. Decisions on "How much analytics is needed"? are hard to answer. ROI could guide for decision support on the What?,…

Machine Learning · Computer Science 2020-09-15 Gouri Deshpande , Guenther Ruhe

Objective: Electronic health records (EHR) data are prone to missingness and errors. Previously, we devised an "enriched" chart review protocol where a "roadmap" of auxiliary diagnoses (anchors) was used to recover missing values in EHR…

Machine Learning · Computer Science 2025-10-07 Sarah C. Lotspeich , Abbey Collins , Brian J. Wells , Ashish K. Khanna , Joseph Rigdon , Lucy D'Agostino McGowan

In studies that rely on data from electronic health records (EHRs), unstructured text data such as clinical progress notes offer a rich source of information about patient characteristics and care that may be missing from structured data.…

Computation and Language · Computer Science 2024-05-22 Reagan Mozer , Aaron R. Kaufman , Leo A. Celi , Luke Miratrix

Missing data can lead to inefficiencies and biases in analyses, in particular when data are missing not at random (MNAR). It is thus vital to understand and correctly identify the missing data mechanism. Recovering missing values through a…

Methodology · Statistics 2022-12-08 Jack Noonan , Adetola Adedamola Adediran , Robin Mitra , Stefanie Biedermann

Existing sequential generalized estimating equation methodology for longitudinal and group-correlated data focuses on narrow hypotheses concerning treatment efficacy and often makes modeling assumptions that impede the desirable robustness…

Methodology · Statistics 2026-03-16 Nathan T. Provost , Abdus S. Wahed

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

A multivariate mixed-effects model seems to be the most appropriate for gene expression data collected in a crossover trial. It is, however, difficult to obtain reliable results using standard statistical inference when some responses are…

Methodology · Statistics 2023-09-12 Savita Pareek , Kalyan Das , Siuli Mukhopadhyay

Causal inference for testing clinical hypotheses from observational data presents many difficulties because the underlying data-generating model and the associated causal graph are not usually available. Furthermore, observational data may…

Efficacy testing is a cornerstone of clinical trials, ensuring that medical interventions achieve their intended therapeutic effects. Over the decades, a wide range of statistical methodologies have been developed to address the…

Other Statistics · Statistics 2026-04-13 Dhrubajyoti Ghosh , Samhita Pal

Randomized controlled trials (RCTs) are often underpowered to detect treatment heterogeneity in subgroups defined by cross-classifications of multiple covariates, due to sparse sample sizes in some strata. External RCT data can help, but…

Methodology · Statistics 2026-04-23 Youqi Yang , Walter Dempsey , Bhramar Mukherjee