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Some patients benefit from a treatment while others may do so less or do not benefit at all. We have previously developed a two-stage network meta-regression prediction model that synthesized randomized trials and evaluates how treatment…

Individual participant data (IPD) meta-analyses of randomised trials are considered a reliable way to assess participant-level treatment effect modifiers but may not make the best use of the available data. Traditionally, effect modifiers…

Often both Aggregate Data (AD) studies and Individual Patient Data (IPD) studies are available for specific treatments. Combining these two sources of data could improve the overall meta-analytic estimates of treatment effects. Moreover,…

统计方法学 · 统计学 2021-11-15 Neha Agarwala , Junyong Park , Anindya Roy

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

Meta-analysis is a well-established tool used to combine data from several independent studies, each of which usually compares the effect of an experimental treatment with a control group. While meta-analyses are often performed using…

统计方法学 · 统计学 2025-06-02 Phuc Thien Tran , Long-Hao Xu , Christian Röver , Tim Friede

Obtaining causally interpretable meta-analysis results is challenging when there are differences in the distribution of effect modifiers between eligible trials. To overcome this, recent work on transportability methods has considered…

统计方法学 · 统计学 2025-03-10 Tat-Thang Vo , Tran Trong Khoi Le , Sivem Afach , Stijn Vansteelandt

In evidence synthesis, multilevel modeling approaches (MMAs) are commonly employed to combine aggregate data (AD) and individual participant data (IPD). These approaches rely on an aggregate outcome model that is ideally obtained by…

统计方法学 · 统计学 2026-02-18 Tran Trong Khoi Le , Marie-Felicia Béclin , Sivem Afach , Tat-Thang Vo

Comparative binary outcome data are of fundamental interest in statistics and are often pooled in meta-analyses. Here we examine the simplest case where for each study there are two patient groups and a binary event of interest, giving rise…

统计方法学 · 统计学 2018-06-12 Rose Baker , Dan Jackson

Estimating treatment effects conditional on observed covariates can improve the ability to tailor treatments to particular individuals. Doing so effectively requires dealing with potential confounding, and also enough data to adequately…

Random-effects meta-analyses are used to combine evidence of treatment effects from multiple studies. Since treatment effects may vary across trials due to differences in study characteristics, heterogeneity in treatment effects between…

统计方法学 · 统计学 2017-07-10 Tim Friede , Christian Röver , Simon Wandel , Beat Neuenschwander

Meta-analyses are commonly performed based on random-effects models, while in certain cases one might also argue in favour of a common-effect model. One such case may be given by the example of two "study twins" that are performed according…

统计方法学 · 统计学 2024-09-04 Christian Röver , Tim Friede

Background: Pairwise and network meta-analyses using fixed effect and random effects models are commonly applied to synthesise evidence from randomised controlled trials. The models differ in their assumptions and the interpretation of the…

统计方法学 · 统计学 2017-08-04 Shijie Ren , Jeremy E. Oakley , John W. Stevens

It is recommended that measures of between-study effect heterogeneity be reported when conducting individual-participant data meta-analyses (IPD-MA). Methods exist to quantify inconsistency between trials via I^2 (the percentage of…

Meta-analysis is commonly used to combine results from multiple clinical trials, but traditional meta-analysis methods do not refer explicitly to a population of individuals to whom the results apply and it is not clear how to use their…

Quantifying the heterogeneity is an important issue in meta-analysis, and among the existing measures, the $I^2$ statistic is most commonly used. In this paper, we first illustrate with a simple example that the $I^2$ statistic is heavily…

统计方法学 · 统计学 2025-06-10 Ke Yang , Enxuan Lin , Wangli Xu , Liping Zhu , Tiejun Tong

Effect modification occurs while the effect of the treatment is not homogeneous across the different strata of patient characteristics. When the effect of treatment may vary from individual to individual, precision medicine can be improved…

During drug development, evidence can emerge to suggest a treatment is more effective in a specific patient subgroup. Whilst early trials may be conducted in biomarker-mixed populations, later trials are more likely to enrol…

统计方法学 · 统计学 2023-06-07 Lorna Wheaton , Dan Jackson , Sylwia Bujkiewicz

Understanding treatment heterogeneity is essential to the development of precision medicine, which seeks to tailor medical treatments to subgroups of patients with similar characteristics. One of the challenges to achieve this goal is that…

统计方法学 · 统计学 2019-08-21 Shujie Ma , Jian Huang , Zhiwei Zhang , Mingming Liu

An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…

统计方法学 · 统计学 2018-11-27 Marius Thomas , Björn Bornkamp , Katja Ickstadt

In this paper we review recent advances in statistical methods for the evaluation of the heterogeneity of treatment effects (HTE), including subgroup identification and estimation of individualized treatment regimens, from randomized…

统计方法学 · 统计学 2024-10-22 Ilya Lipkovich , David Svensson , Bohdana Ratitch , Alex Dmitrienko
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