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Indirect comparisons of treatment-specific outcomes across separate studies often inform decision-making in the absence of head-to-head randomized comparisons. Differences in baseline characteristics between study populations may introduce…

Applications · Statistics 2020-04-09 David Cheng , Rajeev Ayyagari , James Signorovitch

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…

Applications · Statistics 2021-11-05 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

Population adjustment methods such as matching-adjusted indirect comparison (MAIC) are increasingly used to compare marginal treatment effects when there are cross-trial differences in effect modifiers and limited patient-level data. MAIC…

Methodology · Statistics 2022-05-12 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

Anchored covariate-adjusted indirect comparisons inform reimbursement decisions where there are no head-to-head trials between the treatments of interest, there is a common comparator arm shared by the studies, and there are patient-level…

Methodology · Statistics 2022-12-06 Antonio Remiro-Azócar

Population adjustment methods such as matching-adjusted indirect comparison (MAIC) are increasingly used to compare marginal treatment effects when there are cross-trial differences in effect modifiers and limited patient-level data. MAIC…

Methodology · Statistics 2022-12-06 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

Population-Adjusted Indirect Comparisons (PAICs) are used to estimate treatment effects when direct comparisons are infeasible and individual patient data (IPD) are only available for one trial. Among PAIC methods, Matching-Adjusted…

Methodology · Statistics 2025-07-22 Arnaud Serret-Larmande , Jérôme Lambert , Stéphane Gaudry , David Hajage

Indirect comparisons have been increasingly used to compare data from different sources such as clinical trials and observational data in, e.g., a disease registry. To adjust for population differences between data sources,…

Methodology · Statistics 2021-07-27 Jixian Wang

Matching-adjusted indirect comparison (MAIC) has been increasingly employed in health technology assessments (HTA). By reweighting subjects from a trial with individual participant data (IPD) to match the covariate summary statistics of…

Machine Learning · Statistics 2026-05-13 Yixin Fang , Weili He

We discuss how to handle matching-adjusted indirect comparison (MAIC) from a data analyst's perspective. We introduce several multivariate data analysis methods to assess the appropriateness of MAIC for a given data set. These methods focus…

Applications · Statistics 2022-03-18 Ekkehard Glimm , Lillian Yau

This paper studies inference on the average treatment effect in experiments in which treatment status is determined according to "matched pairs" and it is additionally desired to adjust for observed, baseline covariates to gain further…

Econometrics · Economics 2023-10-20 Yuehao Bai , Liang Jiang , Joseph P. Romano , Azeem M. Shaikh , Yichong Zhang

In light of newly developed standardization methods, we evaluate, via simulation study, how propensity score weighting and standardization -based approaches compare for obtaining estimates of the marginal odds ratio and the marginal hazard…

Methodology · Statistics 2023-10-10 Harlan Campbell , Julie E Park , Jeroen P Jansen , Shannon Cope

Effect modification occurs when the effect of the treatment on an outcome varies according to the level of other covariates and often has important implications in decision making. When there are tens or hundreds of covariates, it becomes…

Methodology · Statistics 2021-11-23 Qingyuan Zhao , Dylan S. Small , Ashkan Ertefaie

Externally controlled single-arm trials are critical to assess treatment efficacy across therapeutic indications for which randomized controlled trials are not feasible. A closely-related research design, the unanchored indirect treatment…

Methodology · Statistics 2026-02-13 Harlan Campbell , Antonio Remiro-Azócar

In this commentary, we highlight the importance of: (1) carefully considering and clarifying whether a marginal or conditional treatment effect is of interest in a population-adjusted indirect treatment comparison; and (2) developing…

Methodology · Statistics 2021-11-05 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

Objective: In randomized clinical trials, prediction models can be used to explore the relationships between patients' variables (e.g., clinical, pathological, or lifestyle variables, and also biomarker or genomic data) and treatment effect…

Quantitative Methods · Quantitative Biology 2026-02-03 Elvire Roblin , Paul-Henry Cournède , Stefan Michiels

Effect modification occurs when the effect of the treatment on an outcome differs according to the level of a third variable (the effect modifier, EM). A natural way to assess effect modification is by subgroup analysis or include the…

Methodology · Statistics 2021-12-22 Asma Bahamyirou , Mireille E. Schnitzer , Edward H. Kennedy , Lucie Blais , Yi Yang

We examine four important considerations in the development of covariate adjustment methodologies for indirect treatment comparisons. Firstly, we consider potential advantages of weighting versus outcome modeling, placing focus on…

Methodology · Statistics 2026-05-07 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

In observational studies, potential unobserved confounding is a major barrier in isolating the average causal effect (ACE). In these scenarios, two main approaches are often used: confounder adjustment for causality (CAC) and instrumental…

Methodology · Statistics 2024-11-26 Roy S. Zawadzki , Daniel L. Gillen

This paper combines causal mediation analysis with double machine learning to control for observed confounders in a data-driven way under a selection-on-observables assumption in a high-dimensional setting. We consider the average indirect…

Econometrics · Economics 2021-02-17 Helmut Farbmacher , Martin Huber , Lukáš Lafférs , Henrika Langen , Martin Spindler

Causal variable selection in time-varying treatment settings is challenging due to evolving confounding effects. Existing methods mainly focus on time-fixed exposures and are not directly applicable to time-varying scenarios. We propose a…

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