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Eliciting prior information from clinical trials via calibrated Bayes factor

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  • Macrì Demartino, Roberto
  • Egidi, Leonardo
  • Torelli, Nicola
  • Ntzoufras, Ioannis

Abstract

In the Bayesian framework power prior distributions are increasingly adopted in clinical trials and similar studies to incorporate external and past information, typically to inform the parameter associated with a treatment effect. Their use is particularly effective in scenarios with small sample sizes and where robust prior information is available. A crucial component of this methodology is represented by its weight parameter, which controls the volume of historical information incorporated into the current analysis. Although this parameter can be modeled as either fixed or random, eliciting its prior distribution via a full Bayesian approach remains challenging. In general, this parameter should be carefully selected to accurately reflect the available historical information without dominating the posterior inferential conclusions. A novel simulation-based calibrated Bayes factor procedure is proposed to elicit the prior distribution of the weight parameter, allowing it to be updated according to the strength of the evidence in the data. The goal is to facilitate the integration of historical data when there is agreement with current information and to limit it when discrepancies arise in terms, for instance, of prior-data conflicts. The performance of the proposed method is tested through simulation studies and applied to real data from clinical trials.

Suggested Citation

  • Macrì Demartino, Roberto & Egidi, Leonardo & Torelli, Nicola & Ntzoufras, Ioannis, 2025. "Eliciting prior information from clinical trials via calibrated Bayes factor," Computational Statistics & Data Analysis, Elsevier, vol. 209(C).
  • Handle: RePEc:eee:csdana:v:209:y:2025:i:c:s0167947325000568
    DOI: 10.1016/j.csda.2025.108180
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    1. Carpenter, Bob & Gelman, Andrew & Hoffman, Matthew D. & Lee, Daniel & Goodrich, Ben & Betancourt, Michael & Brubaker, Marcus & Guo, Jiqiang & Li, Peter & Riddell, Allen, 2017. "Stan: A Probabilistic Programming Language," Journal of Statistical Software, Foundation for Open Access Statistics, vol. 76(i01).
    2. John Geweke, 2004. "Getting It Right: Joint Distribution Tests of Posterior Simulators," Journal of the American Statistical Association, American Statistical Association, vol. 99, pages 799-804, January.
    3. Brian P. Hobbs & Bradley P. Carlin & Sumithra J. Mandrekar & Daniel J. Sargent, 2011. "Hierarchical Commensurate and Power Prior Models for Adaptive Incorporation of Historical Information in Clinical Trials," Biometrics, The International Biometric Society, vol. 67(3), pages 1047-1056, September.
    4. Stavros Nikolakopoulos & Ingeborg van der Tweel & Kit C. B. Roes, 2018. "Dynamic borrowing through empirical power priors that control type I error," Biometrics, The International Biometric Society, vol. 74(3), pages 874-880, September.
    5. Peng Yang & Yuansong Zhao & Lei Nie & Jonathon Vallejo & Ying Yuan, 2023. "SAM: Self‐adapting mixture prior to dynamically borrow information from historical data in clinical trials," Biometrics, The International Biometric Society, vol. 79(4), pages 2857-2868, December.
    6. De Santis, Fulvio, 2006. "Power Priors and Their Use in Clinical Trials," The American Statistician, American Statistical Association, vol. 60, pages 122-129, May.
    7. 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.
    8. Samuel Pawel & Frederik Aust & Leonhard Held & Eric-Jan Wagenmakers, 2024. "Power priors for replication studies," TEST: An Official Journal of the Spanish Society of Statistics and Operations Research, Springer;Sociedad de Estadística e Investigación Operativa, vol. 33(1), pages 127-154, March.
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