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Meta-analytic-predictive use of historical variance data for the design and analysis of clinical trials

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  • Schmidli, Heinz
  • Neuenschwander, Beat
  • Friede, Tim

Abstract

Continuous endpoints are common in clinical trials. The design and analysis of such trials is often based on models assuming normally distributed data, possibly after an appropriate transformation. When planning a new trial, information on the variance of the endpoint is usually available from historical trials. Although the idea to use historical data for a new trial is not new, literature on how to formally summarize and use these data on variances is scarce. The meta-analytic-predictive (MAP) approach consists of a random-effects meta-analysis of the historical variance data and a prediction of the variance in the new clinical trial. Two applications that rely on the MAP approach are considered: first, the selection of the sample size in the new trial, guided by the prediction of the variance; and, second, the inclusion of the predicted variance in a Bayesian analysis of the new trial. A clinical trial in patients with wet age-related macular degeneration illustrates the methodology.

Suggested Citation

  • Schmidli, Heinz & Neuenschwander, Beat & Friede, Tim, 2017. "Meta-analytic-predictive use of historical variance data for the design and analysis of clinical trials," Computational Statistics & Data Analysis, Elsevier, vol. 113(C), pages 100-110.
    • Handle: RePEc:eee:csdana:v:113:y:2017:i:c:p:100-110
    DOI: 10.1016/j.csda.2016.08.007
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    References listed on IDEAS

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    1. Julian P. T. Higgins & Simon G. Thompson & David J. Spiegelhalter, 2009. "A re‐evaluation of random‐effects meta‐analysis," Journal of the Royal Statistical Society Series A, Royal Statistical Society, vol. 172(1), pages 137-159, January.
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    5. Heinz Schmidli & Sandro Gsteiger & Satrajit Roychoudhury & Anthony O'Hagan & David Spiegelhalter & Beat Neuenschwander, 2014. "Robust meta-analytic-predictive priors in clinical trials with historical control information," Biometrics, The International Biometric Society, vol. 70(4), pages 1023-1032, December.
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