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Predictive biomarkers are playing an essential role in precision medicine. Identifying an optimal cutoff to select patient subsets with greater benefit from treatment is critical and more challenging for predictive biomarkers measured with…

应用统计 · 统计学 2025-04-29 Chi Zhang , Wei Shi , Spencer Woody , Qing Liu

We develop a framework for quantifying omitted variable bias (OVB) in nonlinear instrumental variable (IV) estimators, including the local average treatment effect (LATE), the LATE for the treated (LATT), and the partially linear IV model…

计量经济学 · 经济学 2026-04-07 Yu-Min Yen

With medical tests becoming increasingly available, concerns about over-testing and over-treatment dramatically increase. Hence, it is important to understand the influence of testing on treatment selection in general practice. Most…

统计方法学 · 统计学 2020-08-11 Yun Li , Irina Bondarenko , Michael R. Elliott , Timothy P. Hofer , Jeremy M. G. Taylor

We used survival analysis to quantify the impact of postdischarge evaluation and management (E/M) services in preventing hospital readmission or death. Our approach avoids a specific pitfall of applying machine learning to this problem,…

统计方法学 · 统计学 2024-02-14 Hongjing Xia , Joshua C. Chang , Sarah Nowak , Sonya Mahajan , Rohit Mahajan , Ted L. Chang , Carson C. Chow

Machine learning (ML) methods offer a wide range of configurable hyperparameters that have a significant influence on their performance. While accuracy is a commonly used performance objective, in many settings, it is not sufficient.…

机器学习 · 计算机科学 2023-09-27 Romain Egele , Tyler Chang , Yixuan Sun , Venkatram Vishwanath , Prasanna Balaprakash

Random-effects models are frequently used to synthesise information from different studies in meta-analysis. While likelihood-based inference is attractive both in terms of limiting properties and of implementation, its application in…

应用统计 · 统计学 2018-05-25 Sophia Kyriakou , Ioannis Kosmidis , Nicola Sartori

Estimating the average treatment effect (ATE) from observational data is challenging due to selection bias. Existing works mainly tackle this challenge in two ways. Some researchers propose constructing a score function that satisfies the…

机器学习 · 计算机科学 2022-09-07 Yiyan Huang , Cheuk Hang Leung , Shumin Ma , Qi Wu , Dongdong Wang , Zhixiang Huang

Decision-makers often deploy the best-performing treatment from a randomized experiment, creating a winner's curse: selection favors treatments whose observed outcomes are high partly because of statistical noise, so the na\"ive estimate of…

计量经济学 · 经济学 2026-05-20 Ron Berman , Walter W. Zhang , Hangcheng Zhao

The estimation of heterogeneous treatment effects in the potential outcome setting is biased when there exists model misspecification or unobserved confounding. As these biases are unobservable, what model to use when remains a critical…

统计方法学 · 统计学 2024-05-09 Shonosuke Sugasawa , Kosaku Takanashi , Kenichiro McAlinn , Edoardo M. Airoldi

Randomized clinical trials are considered the gold standard for estimating causal effects. Nevertheless, in studies that are aimed at examining adverse effects of interventions, such trials are often impractical because of ethical and…

统计方法学 · 统计学 2020-01-20 Anthony D. Scotina , Andrew R. Zullo , Robert J. Smith , Roee Gutman

In observational studies, adjusting for confounders is required if a treatment comparison is planned. A crude comparison of the primary endpoint without covariate adjustment will suffer from biases, and the addition of regression models…

统计方法学 · 统计学 2026-02-11 Shiyu Shu , Toshimitsu Hamasaki , Scott Evans , Lauren Komarow , David van Duin , Guoqing Diao

Meta-analyses of clinical trials targeting rare events face particular challenges when the data lack adequate numbers of events for all treatment arms. Especially when the number of studies is low, standard meta-analysis methods can lead to…

应用统计 · 统计学 2020-01-20 Burak Kürsad Günhan , Christian Röver , Tim Friede

Bayesian optimization (BO) is a sample-efficient approach for tuning design parameters to optimize expensive-to-evaluate, black-box performance metrics. In many manufacturing processes, the design parameters are subject to random input…

机器学习 · 计算机科学 2022-06-06 Samuel Daulton , Sait Cakmak , Maximilian Balandat , Michael A. Osborne , Enlu Zhou , Eytan Bakshy

In matched observational studies with continuous treatments, individuals with different treatment doses but the same or similar covariate values are paired for causal inference. While inexact covariate matching (i.e., covariate imbalance…

统计方法学 · 统计学 2025-02-13 Anthony Frazier , Siyu Heng , Wen Zhou

Covariate balance is crucial in obtaining unbiased estimates of treatment effects in observational studies. Methods based on inverse probability weights have been widely used to estimate treatment effects with observational data. Machine…

统计方法学 · 统计学 2021-04-08 Michele Santacatterina

Our approach to Mendelian Randomization (MR) analysis is designed to increase reproducibility of causal effect "discoveries" by: (i) using a Bayesian approach to inference; (ii) replacing the point null hypothesis with a region of practical…

统计方法学 · 统计学 2022-08-11 Linyi Zou , Teresa Fazia , Hui Guo , Carlo Berzuini

When an optimal treatment regime (OTR) is considered, we need to evaluate the OTR in a valid and efficient way. The classical inference applied to the mean outcome under OTR, assuming the OTR is the same as the estimated OTR, might be…

统计方法学 · 统计学 2026-04-21 Shuoxun Xu , Xinzhou Guo

Approving and assessing new drugs is complex because multiple criteria must be considered simultaneously. A common approach is benefit-risk analysis, often conducted within a Bayesian framework to account for uncertainty and combine data…

统计方法学 · 统计学 2026-05-05 Konstantinos Vamvourellis , Konstantinos Kalogeropoulos , Lawrence Phillips

Precision cancer medicine aims to determine the optimal treatment for each patient. In-vitro cancer drug sensitivity screens combined with multi-omics characterization of the cancer cells have become an important tool to achieve this aim.…

统计方法学 · 统计学 2024-03-14 Zhi Zhao , Marco Banterle , Alex Lewin , Manuela Zucknick

Joint misclassification of exposure and outcome variables can lead to considerable bias in epidemiological studies of causal exposure-outcome effects. In this paper, we present a new maximum likelihood based estimator for the marginal…

统计方法学 · 统计学 2019-01-16 Bas B. L. Penning de Vries , Maarten van Smeden , Rolf H. H. Groenwold