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Much evidence in comparative effectiveness research is based on observational studies. Researchers who conduct observational studies typically assume that there are no unobservable differences between the treated and control groups.…

应用统计 · 统计学 2020-01-23 Luke Keele , Stephen O'Neill , Richard Grieve

The propensity score is a common tool for estimating the causal effect of a binary treatment in observational data. In this setting, matching, subclassification, imputation, or inverse probability weighting on the propensity score can…

统计方法学 · 统计学 2018-01-03 Michael J Lopez , Roee Gutman

We consider a randomized controlled trial between two groups. The objective is to identify a population with characteristics such that the test therapy is more effective than the control therapy. Such a population is called a subgroup. This…

统计方法学 · 统计学 2021-12-06 Shintaro Yuki , Kensuke Tanioka , Hiroshi Yadohisa

Propensity Score Matching (PSM) is an useful method to reduce the impact ofTreatment - Selection Bias in the estimation of causal effects in observational studies. After matching, the PSM significantly reduces the sample under…

统计方法学 · 统计学 2019-02-01 Daniel García Iglesias

A common goal in comparative effectiveness research is to estimate treatment effects on pre-specified subpopulations of patients. Though widely used in medical research, causal inference methods for such subgroup analysis remain…

In classical study designs, the aim is often to learn about the effects of a treatment or intervention on a single outcome; in many modern studies, however, data on multiple outcomes are collected and it is of interest to explore effects on…

统计方法学 · 统计学 2017-06-15 Edward H. Kennedy , Shreya Kangovi , Nandita Mitra

Commonly, clinical trials report effects not only for the full study population but also for patient subgroups. Meta-analyses of subgroup-specific effects and treatment-by-subgroup interactions may be inconsistent, especially when trials…

统计方法学 · 统计学 2025-12-23 Renato Panaro , Christian Röver , Tim Friede

A fundamental limitation of causal inference in observational studies is that perceived evidence for an effect might instead be explained by factors not accounted for in the primary analysis. Methods for assessing the sensitivity of a…

统计方法学 · 统计学 2018-09-14 Colin B. Fogarty

We propose a multi-threshold change plane regression model which naturally partitions the observed subjects into subgroups with different covariate effects. The underlying grouping variable is a linear function of covariates and thus…

统计方法学 · 统计学 2018-08-03 Jialiang Li , Yaguang Li , Baisuo Jin

Background: Subgroup analyses are frequently conducted in randomized clinical trials to assess evidence of heterogeneous treatment effect across patient subpopulations. Although randomization balances covariates within subgroups in…

统计方法学 · 统计学 2021-05-27 Siyun Yang , Fan Li , Laine E. Thomas , Fan Li

Population-adjusted indirect comparisons estimate treatment effects when access to individual patient data is limited and there are cross-trial differences in effect modifiers. Popular methods include matching-adjusted indirect comparison…

应用统计 · 统计学 2021-11-05 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

Subgroup analysis is a frequently used tool for evaluating heterogeneity of treatment effect and heterogeneity in treatment harm across observed baseline patient characteristics. While treatment efficacy and adverse event measures are often…

应用统计 · 统计学 2018-08-14 Nicholas C. Henderson , Ravi Varadhan

For testing the statistical significance of a treatment effect, we usually compare between two parts of a population, one is exposed to the treatment, and the other is not exposed to it. Standard parametric and nonparametric two-sample…

统计计算 · 统计学 2012-11-02 Bikram Karmakar , Kumaresh Dhara , Kushal Kumar Dey , Analabha Basu , Anil Ghosh

The growing availability of large health databases has expanded the use of observational studies for comparative effectiveness research. Unlike randomized trials, observational studies must adjust for systematic differences in patient…

统计方法学 · 统计学 2026-01-21 Haidong Lu , Fan Li , Laine E. Thomas , Fan Li

Methods for extending -- generalizing or transporting -- inferences from a randomized trial to a target population involve conditioning on a large set of covariates that is sufficient for rendering the randomized and non-randomized groups…

We propose a novel regression adjustment method designed for estimating distributional treatment effect parameters in randomized experiments. Randomized experiments have been extensively used to estimate treatment effects in various…

计量经济学 · 经济学 2024-07-24 Undral Byambadalai , Tatsushi Oka , Shota Yasui

Assessing population-level effects of vaccines and other infectious disease prevention measures is important to the field of public health. In infectious disease studies, one person's treatment may affect another individual's outcome, i.e.,…

统计方法学 · 统计学 2024-03-26 Kayla W. Kilpatrick , Chanhwa Lee , Michael G. Hudgens

The paper presents an investigation of estimating treatment effect using different matching methods. The study proposed a new method which is computationally efficient and convenient in implication-'largest caliper matching' and compared…

应用统计 · 统计学 2018-06-07 Sharif Mahmood

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…

统计方法学 · 统计学 2024-02-06 Guanbo Wang , Alexander Levis , Jon Steingrimsson , Issa Dahabreh

Many scientific questions in biomedical, environmental, and psychological research involve understanding the effects of multiple factors on outcomes. While factorial experiments are ideal for this purpose, randomized controlled treatment…

统计方法学 · 统计学 2025-12-03 Ruoqi Yu , Peng Ding