Likelihood reweighting methods to reduce potential bias in noninferiority trials which rely on historical data to make inference

DOI10.1214/13-AOAS655zbMATH Open1454.62370arXiv1311.7485OpenAlexW3102750578MaRDI QIDQ386771FDOQ386771

Authors: Lei Nie, Zhiwei Zhang, Daniel B. Rubin, Jianxiong Chu 

Publication date: 10 December 2013

Published in: The Annals of Applied Statistics (Search for Journal in Brave)

Abstract: It is generally believed that bias is minimized in well-controlled randomized clinical trials. However, bias can arise in active controlled noninferiority trials because the inference relies on a previously estimated effect size obtained from a historical trial that may have been conducted for a different population. By implementing a likelihood reweighting method through propensity scoring, a study designed to estimate a treatment effect in one trial population can be used to estimate the treatment effect size in a different target population. We illustrate this method in active controlled noninferiority trials, although it can also be used in other types of studies, such as historically controlled trials, meta-analyses, and comparative effectiveness analyses.

Full work available at URL: https://arxiv.org/abs/1311.7485

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zbMATH Keywords

generalized linear modelbiaspropensity scoreinverse probability weightingnoninferiority

Mathematics Subject Classification ID

Applications of statistics to biology and medical sciences; meta analysis (62P10) Generalized linear models (logistic models) (62J12)

Cites Work

• Maximum Likelihood Estimation of Misspecified Models
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• Comment: Demystifying double robustness: a comparison of alternative strategies for estimating a population mean from incomplete data
• Nonparametric estimates of standard error: The jackknife, the bootstrap and other methods
• Estimating the current treatment effect with historical control data
• Likelihood reweighting methods to reduce potential bias in noninferiority trials which rely on historical data to make inference

Cited In (4)

• Estimating the population average treatment effect in observational studies with choice-based sampling
• The statistical performance of matching-adjusted indirect comparisons: estimating treatment effects with aggregate external control data
• New methods for treatment effect calibration, with applications to non-inferiority trials
• Likelihood reweighting methods to reduce potential bias in noninferiority trials which rely on historical data to make inference

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