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

DOI10.1214/13-AOAS655MaRDI QIDQ386771zbMATH OpenOpenAlexFDO

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)

Full work available at URL https://arxiv.org/abs/1311.7485, https://projecteuclid.org/euclid.aoas/1380804817

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)

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.

Recommendations

• Dynamic borrowing through empirical power priors that control type I error
• New methods for treatment effect calibration, with applications to non-inferiority trials
• Bayesian design of noninferiority trials for medical devices using historical data
• Elastic priors to dynamically borrow information from historical data in clinical trials
• Design and analysis of non-inferiority trials

Cites work

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

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

This page was built for publication: Likelihood reweighting methods to reduce potential bias in noninferiority trials which rely on historical data to make inference

Report a bug (only for logged in users!)Click here to report a bug for this page (MaRDI item Q386771)