Assessing the Impact of Covariate Distribution and Positivity Violation on Weighting-Based Indirect Comparisons: a Simulation Study

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Hauptverfasser: Serret-Larmande, Arnaud, Lambert, Jérôme, Gaudry, Stéphane, Hajage, David
Format: Preprint
Veröffentlicht: 2025
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author Serret-Larmande, Arnaud
Lambert, Jérôme
Gaudry, Stéphane
Hajage, David
author_facet Serret-Larmande, Arnaud
Lambert, Jérôme
Gaudry, Stéphane
Hajage, David
contents 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 Indirect Comparison (MAIC) is the most widely used. However, little is known about how MAIC performs under challenging conditions such as limited covariate overlap or markedly non-normal covariate distributions. We conducted a Monte Carlo simulation study comparing three estimators: (i) MAIC matching first moment (MAIC-1), (ii) MAIC matching first and second moments (MAIC-2), and (iii) a benchmark method leveraging full IPD -- Propensity Score Weighting (PSW). We examined eight scenarios ranging from ideal conditions to situations with positivity violations and non-normal (including bimodal) covariate distributions. We assessed both anchored and unanchored estimators and examined the impact of adjustment model misspecification. We also applied these estimators to real-world data from the AKIKI and AKIKI-2 trials, comparing renal replacement therapy strategies in critically ill patients. MAIC-1 demonstrated robust performance, remaining unbiased in the presence of moderate positivity violations and non-normal covariates, while MAIC-2 and PSW appeared more sensitive to positivity violations. All methods showed substantial bias when key confounders were omitted, emphasizing the importance of correct model specification. In real-world data, a consistent trend was found with MAIC-1 showing narrower confidence intervals with positivity violation. Our findings support the cautious use of unanchored MAICs and highlight MAIC-1's resilience across moderate violations of assumptions. However, the method's limited flexibility underscores the need for careful use in real-world settings.
format Preprint
id arxiv_https___arxiv_org_abs_2507_12241
institution arXiv
publishDate 2025
record_format arxiv
spellingShingle Assessing the Impact of Covariate Distribution and Positivity Violation on Weighting-Based Indirect Comparisons: a Simulation Study
Serret-Larmande, Arnaud
Lambert, Jérôme
Gaudry, Stéphane
Hajage, David
Methodology
Applications
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 Indirect Comparison (MAIC) is the most widely used. However, little is known about how MAIC performs under challenging conditions such as limited covariate overlap or markedly non-normal covariate distributions. We conducted a Monte Carlo simulation study comparing three estimators: (i) MAIC matching first moment (MAIC-1), (ii) MAIC matching first and second moments (MAIC-2), and (iii) a benchmark method leveraging full IPD -- Propensity Score Weighting (PSW). We examined eight scenarios ranging from ideal conditions to situations with positivity violations and non-normal (including bimodal) covariate distributions. We assessed both anchored and unanchored estimators and examined the impact of adjustment model misspecification. We also applied these estimators to real-world data from the AKIKI and AKIKI-2 trials, comparing renal replacement therapy strategies in critically ill patients. MAIC-1 demonstrated robust performance, remaining unbiased in the presence of moderate positivity violations and non-normal covariates, while MAIC-2 and PSW appeared more sensitive to positivity violations. All methods showed substantial bias when key confounders were omitted, emphasizing the importance of correct model specification. In real-world data, a consistent trend was found with MAIC-1 showing narrower confidence intervals with positivity violation. Our findings support the cautious use of unanchored MAICs and highlight MAIC-1's resilience across moderate violations of assumptions. However, the method's limited flexibility underscores the need for careful use in real-world settings.
title Assessing the Impact of Covariate Distribution and Positivity Violation on Weighting-Based Indirect Comparisons: a Simulation Study
topic Methodology
Applications
url https://arxiv.org/abs/2507.12241