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A unified evaluation of differential vaccine efficacy

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Listed:
  • Erin E. Gabriel
  • Michael C. Sachs
  • Dean A. Follmann
  • Therese M‐L. Andersson

Abstract

Many infectious diseases are well prevented by proper vaccination. However, when a vaccine is not completely efficacious, there is great interest in determining how the vaccine effect differs in subgroups conditional on measured immune responses postvaccination and also according to the type of infecting agent (eg, strain of a virus). The former is often called correlate of protection (CoP) analysis, while the latter has been called sieve analysis. We propose a unified framework for simultaneously assessing CoP and sieve effects of a vaccine in a large Phase III randomized trial. We use flexible parametric models treating times to infection from different agents as competing risks and estimated maximum likelihood to fit the models. The parametric models under competing risks allow for estimation of both cumulative incidence‐based contrasts and instantaneous rates. We outline the assumptions with which we can link the observable data to the causal contrasts of interest, propose hypothesis testing procedures, and evaluate our proposed methods in an extensive simulation study.

Suggested Citation

  • Erin E. Gabriel & Michael C. Sachs & Dean A. Follmann & Therese M‐L. Andersson, 2020. "A unified evaluation of differential vaccine efficacy," Biometrics, The International Biometric Society, vol. 76(4), pages 1053-1063, December.
    • Handle: RePEc:bla:biomet:v:76:y:2020:i:4:p:1053-1063
    DOI: 10.1111/biom.13211
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    References listed on IDEAS

    as
    1. Ying Huang & Peter B. Gilbert, 2011. "Comparing Biomarkers as Principal Surrogate Endpoints," Biometrics, The International Biometric Society, vol. 67(4), pages 1442-1451, December.
    2. David Benkeser & Peter B. Gilbert & Marco Carone, 2019. "Estimating and Testing Vaccine Sieve Effects Using Machine Learning," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 114(527), pages 1038-1049, July.
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    5. Peter B. Gilbert & Michael G. Hudgens, 2008. "Evaluating Candidate Principal Surrogate Endpoints," Biometrics, The International Biometric Society, vol. 64(4), pages 1146-1154, December.
    6. Paul C. Lambert & Patrick Royston, 2009. "Further development of flexible parametric models for survival analysis," Stata Journal, StataCorp LP, vol. 9(2), pages 265-290, June.
    7. M. Juraska & P. B. Gilbert, 2013. "Mark-Specific Hazard Ratio Model with Multivariate Continuous Marks: An Application to Vaccine Efficacy," Biometrics, The International Biometric Society, vol. 69(2), pages 328-337, June.
    8. Ying Huang & Shibasish Dasgupta, 2019. "Likelihood-Based Methods for Assessing Principal Surrogate Endpoints in Vaccine Trials," Statistics in Biosciences, Springer;International Chinese Statistical Association, vol. 11(3), pages 504-523, December.
    9. Dean Follmann, 2006. "Augmented Designs to Assess Immune Response in Vaccine Trials," Biometrics, The International Biometric Society, vol. 62(4), pages 1161-1169, December.
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