Meta-analysis of two studies in the presence of heterogeneity with applications in rare diseases

DOI10.1002/BIMJ.201500236zbMATH Open1369.62294arXiv1606.04969OpenAlexW2438033049WikidataQ41052414 ScholiaQ41052414MaRDI QIDQ5280181FDOQ5280181

Authors: Tim Friede, Simon Wandel, Beat E. Neuenschwander, Christian Röver 

Publication date: 20 July 2017

Published in: Biometrical Journal (Search for Journal in Brave)

Abstract: Random-effects meta-analyses are used to combine evidence of treatment effects from multiple studies. Since treatment effects may vary across trials due to differences in study characteristics, heterogeneity in treatment effects between studies must be accounted for to achieve valid inference. The standard model for random-effects meta-analysis assumes approximately normal effect estimates and a normal random-effects model. However, standard methods based on this model ignore the uncertainty in estimating the between-trial heterogeneity. In the special setting of only two studies and in the presence of heterogeneity we investigate here alternatives such as the Hartung-Knapp-Sidik-Jonkman method (HKSJ), the modified Knapp-Hartung method (mKH, a variation of the HKSJ method) and Bayesian random-effects meta-analyses with priors covering plausible heterogeneity values. The properties of these methods are assessed by applying them to five examples from various rare diseases and by a simulation study. Whereas the standard method based on normal quantiles has poor coverage, the HKSJ and mKH generally lead to very long, and therefore inconclusive, confidence intervals. The Bayesian intervals on the whole show satisfying properties and offer a reasonable compromise between these two extremes.

Full work available at URL: https://arxiv.org/abs/1606.04969

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zbMATH Keywords

Bayesian statisticscoverage probabilitybetween-study heterogeneityrandom-effects meta-analysisorphan disease

Mathematics Subject Classification ID

Bayesian inference (62F15) Applications of statistics to biology and medical sciences; meta analysis (62P10) Parametric tolerance and confidence regions (62F25) Analysis of variance and covariance (ANOVA) (62J10)

Cites Work

• Estimating common mean and heterogeneity variance in two study case meta-analysis
• Valid Inference in Random Effects Meta‐Analysis
• On Constructing Confidence Intervals for a Standardized Mean Difference in Meta-analysis
• Robust meta‐analytic‐predictive priors in clinical trials with historical control information

Cited In (16)

• Random‐effects meta‐analysis models for the odds ratio in the case of rare events under different data‐generating models: A simulation study
• Model averaging for robust extrapolation in evidence synthesis
• Random-effects meta-analysis of phase I dose-finding studies using stochastic process priors
• Exact inference on the random-effects model for meta-analyses with few studies
• How vague is vague? How informative is informative? Reference analysis for Bayesian meta-analysis
• On the exact null-distribution of a test for homogeneity of the risk ratio in meta-analysis of studies with rare events
• Title not available (Why is that?)
• Sensitivity and identification quantification by a relative latent model complexity perturbation in Bayesian meta‐analysis
• Summarizing empirical information on between-study heterogeneity for Bayesian random-effects meta-analysis
• A straightforward meta-analysis approach for oncology phase I dose-finding studies
• Meta‐analysis of clinical trials with rare events
• Bounds for the weight of external data in shrinkage estimation
• Investigating the heterogeneity of ``study twins
• Quantification of empirical determinacy: The impact of likelihood weighting on posterior location and spread in Bayesian meta-analysis estimated with JAGS and INLA
• Subgroup identification in individual participant data meta-analysis using model-based recursive partitioning
• Bayesian heterogeneity in a meta–analysis with two studies and binary data

Uses Software

• R

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