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In healthcare, predictive models increasingly inform patient-level decisions, yet little attention is paid to the variability in individual risk estimates and its impact on treatment decisions. For overparameterized models, now standard in…

机器学习 · 计算机科学 2026-04-16 Elizabeth W. Miller , Jeffrey D. Blume

The restricted mean survival time (RMST) model has been garnering attention as a way to provide a clinically intuitive measure: the mean survival time. RMST models, which use methods based on pseudo time-to-event values and inverse…

统计方法学 · 统计学 2024-06-11 Keisuke Hanada , Masahiro Kojima

We consider clinical trials with multiple, overlapping patient populations, that test multiple treatment policies specifically tailored to these populations. Such designs may lead to multiplicity issues, as false statements will affect…

统计方法学 · 统计学 2025-11-13 Remi Luschei , Werner Brannath

When treatment effect modifiers influence the decision to participate in a randomized trial, the average treatment effect in the population represented by the randomized individuals will differ from the effect in other populations. In this…

While learning with limited labelled data can improve performance when the labels are lacking, it is also sensitive to the effects of uncontrolled randomness introduced by so-called randomness factors (e.g., varying order of data). We…

计算与语言 · 计算机科学 2024-12-03 Branislav Pecher , Ivan Srba , Maria Bielikova

Symmetry plays a central role in the sciences, machine learning, and statistics. While statistical tests for the presence of distributional invariance with respect to groups have a long history, tests for conditional symmetry in the form of…

统计方法学 · 统计学 2025-12-12 Kenny Chiu , Alex Sharp , Benjamin Bloem-Reddy

Randomized controlled trials (RCTs) frequently utilize covariate-adaptive randomization (CAR) (e.g., stratified block randomization) and commonly suffer from imperfect compliance. This paper studies the identification and inference for the…

计量经济学 · 经济学 2025-05-02 Federico A. Bugni , Mengsi Gao , Filip Obradovic , Amilcar Velez

The literature on cluster-randomized trials typically allows for interference within but not across clusters. This may be implausible when units are irregularly distributed across space without well-separated communities, as clusters in…

统计方法学 · 统计学 2025-10-29 Michael P. Leung

Stratification in both the design and analysis of randomized clinical trials is common. Despite features in automated randomization systems to re-confirm the stratifying variables, incorrect values of these variables may be entered. These…

统计方法学 · 统计学 2023-07-24 Neal Thomas

Randomized controlled trials (RCTs) are the gold standard for evaluating the causal effect of a treatment; however, they often have limited sample sizes and sometimes poor generalizability. On the other hand, non-randomized, observational…

统计方法学 · 统计学 2021-09-23 Shuxiao Chen , Bo Zhang , Ting Ye

Major advances in public health have resulted from disease prevention. However, prevention of a new infectious disease by vaccination or pharmaceuticals is made difficult by the slow process of vaccine and drug development. We propose an…

种群与进化 · 定量生物学 2021-04-15 Jussi Taipale , Ioannis Kontoyiannis , Sten Linnarsson

Clinical trials with a hybrid control arm (a control arm constructed from a combination of randomized patients and real-world data on patients receiving usual care in standard clinical practice) have the potential to decrease the cost of…

统计方法学 · 统计学 2021-08-20 Joanna Harton , Brian Segal , Ronac Mamtani , Nandita Mitra , Rebecca Hubbard

Online marketplace designers frequently run A/B tests to measure the impact of proposed product changes. However, given that marketplaces are inherently connected, total average treatment effect estimates obtained through Bernoulli…

统计方法学 · 统计学 2020-04-28 David Holtz , Ruben Lobel , Inessa Liskovich , Sinan Aral

This paper studies inference in randomized controlled trials with multiple treatments, where treatment status is determined according to a "matched tuples" design. Here, by a matched tuples design, we mean an experimental design where units…

计量经济学 · 经济学 2023-11-06 Yuehao Bai , Jizhou Liu , Max Tabord-Meehan

A/B testing is a standard approach for evaluating the effect of online experiments; the goal is to estimate the `average treatment effect' of a new feature or condition by exposing a sample of the overall population to it. A drawback with…

社会与信息网络 · 计算机科学 2013-05-31 Johan Ugander , Brian Karrer , Lars Backstrom , Jon Kleinberg

Covariate adjustment is desired by both practitioners and regulators of randomized clinical trials because it improves precision for estimating treatment effects. However, covariate adjustment presents a particular challenge in…

统计方法学 · 统计学 2023-07-20 Yunfan Li , Jessica L. Ross , Aaron M. Smith , David P. Miller

Conditional power calculations are frequently used to guide the decision whether or not to stop a trial for futility or to modify planned sample size. These ignore the information in short-term endpoints and baseline covariates, and thereby…

统计方法学 · 统计学 2019-04-11 Kelly Van Lancker , An Vandebosch , Stijn Vansteelandt

Biometric recognition is used across a variety of applications from cyber security to border security. Recent research has focused on ensuring biometric performance (false negatives and false positives) is fair across demographic groups.…

统计方法学 · 统计学 2022-08-24 Michael Schuckers , Sandip Purnapatra , Kaniz Fatima , Daqing Hou , Stephanie Schuckers

Randomized block factorial experiments are widely used in industrial engineering, clinical trials, and social science. Researchers often use a linear model and analysis of covariance to analyze experimental results; however, limited studies…

统计方法学 · 统计学 2022-08-04 Hanzhong Liu , Jiyang Ren , Yuehan Yang

Background: Randomized controlled trials are often used to inform policy and practice for broad populations. The average treatment effect (ATE) for a target population, however, may be different from the ATE observed in a trial if there are…

统计方法学 · 统计学 2023-01-19 Trang Quynh Nguyen , Benjamin Ackerman , Ian Schmid , Stephen R. Cole , Elizabeth A. Stuart
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