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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

In group sequential designs, where several data looks are conducted for early stopping, we generally assume the vector of test statistics from the sequential analyses follows (at least approximately or asymptotially) a multivariate normal…

统计理论 · 数学 2024-04-22 Long-Hao Xu , Tobias Mütze , Frank Konietschke , Tim Friede

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

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

When considering the effect a treatment will cause in a population of interest, we often look to evidence from randomized controlled trials. In settings where multiple trials on a treatment are available, we may wish to synthesize the…

统计方法学 · 统计学 2023-09-08 Nicole Schnitzler , Eloise Kaizar

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…

The synthetic control method is often applied to problems with one treated unit and a small number of control units. A common inferential task in this setting is to test null hypotheses regarding the average treatment effect on the treated.…

统计方法学 · 统计学 2025-04-22 Lihua Lei , Timothy Sudijono

Recently, Saeb et al (2017) showed that, in diagnostic machine learning applications, having data of each subject randomly assigned to both training and test sets (record-wise data split) can lead to massive underestimation of the…

Network meta-analysis (NMA) allows the combination of direct and indirect evidence from a set of randomized clinical trials. Performing NMA using individual patient data (IPD) is considered as a "gold standard" approach as it provides…

统计方法学 · 统计学 2021-10-22 Edouard Ollier , Pierre Blanchard , Gwénaël Le Teuff , Stefan Michiels

The primary goal of randomized trials is to compare the effects of different interventions on some outcome of interest. In addition to the treatment assignment and outcome, data on baseline covariates, such as demographic characteristics or…

应用统计 · 统计学 2014-01-09 Alisa J. Stephens , Eric J. Tchetgen Tchetgen , Victor De Gruttola

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…

Evaluating the treatment effects has become an important topic for many applications. However, most existing literature focuses mainly on the average treatment effects. When the individual effects are heavy-tailed or have outlier values,…

统计方法学 · 统计学 2023-05-11 Yongchang Su , Xinran Li

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

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…

I introduce a simple permutation procedure to test conventional (non-sharp) hypotheses about the effect of a binary treatment in the presence of a finite number of large, heterogeneous clusters when the treatment effect is identified by…

计量经济学 · 经济学 2023-02-08 Andreas Hagemann

When the individual studies assembled for a meta-analysis report means ($\mu_C$, $\mu_T$) for their treatment (T) and control (C) arms, but those data are on different scales or come from different instruments, the customary measure of…

统计方法学 · 统计学 2023-04-18 Elena Kulinskaya , David C. Hoaglin

Individualized treatment effect lies at the heart of precision medicine. Interpretable individualized treatment rules (ITRs) are desirable for clinicians or policymakers due to their intuitive appeal and transparency. The gold-standard…

统计方法学 · 统计学 2021-08-20 Lili Wu , Shu Yang

This paper introduces an overidentification test of two alternative assumptions to identify the average treatment effect on the treated in a two-period panel data setting: unconfoundedness and common trends. Under the unconfoundedness…

计量经济学 · 经济学 2024-06-25 Martin Huber , Eva-Maria Oeß

Treatment-covariate interaction tests are commonly applied by researchers to examine whether the treatment effect varies across patient subgroups defined by baseline characteristics. The objective of this study is to explore…

统计方法学 · 统计学 2024-03-12 Likun Zhang , Wei Ma

Single subject or n-of-1 research designs have been widely used to evaluate treatment interventions. Many statistical procedures such as split-middle trend lines, regression trend line, Shewart-chart trend line, binomial tests,…

应用统计 · 统计学 2015-06-18 A. Savenkov , S. Wu , D. Neal