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Evaluation of longitudinal surrogate markers

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  • Denis Agniel
  • Layla Parast

Abstract

The use of surrogate markers to examine the effectiveness of a treatment has the potential to decrease study length and identify effective treatments more quickly. Most available methods to investigate the usefulness of a surrogate marker involve restrictive parametric assumptions and tend to focus on settings where the surrogate is measured at a single point in time. However, in many clinical settings, the potential surrogate marker is often measured repeatedly over time, and thus, the surrogate marker information is a trajectory of measurements. In addition, it is often difficult in practice to correctly specify the relationship between a treatment, primary outcome, and surrogate marker trajectory. In this paper, we propose a model‐free definition for the proportion of the treatment effect on the primary outcome that is explained by the treatment effect on the longitudinal surrogate markers. We propose three novel flexible methods to estimate this proportion, develop the asymptotic properties of our estimators, and investigate the robustness of the estimators under multiple settings via a simulation study. We apply our proposed procedures to an AIDS clinical trial dataset to examine a trajectory of CD4 counts as a potential surrogate.

Suggested Citation

  • Denis Agniel & Layla Parast, 2021. "Evaluation of longitudinal surrogate markers," Biometrics, The International Biometric Society, vol. 77(2), pages 477-489, June.
    • Handle: RePEc:bla:biomet:v:77:y:2021:i:2:p:477-489
    DOI: 10.1111/biom.13310
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    References listed on IDEAS

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    1. Emily K. Roberts & Michael R. Elliott & Jeremy M. G. Taylor, 2023. "Solutions for surrogacy validation with longitudinal outcomes for a gene therapy," Biometrics, The International Biometric Society, vol. 79(3), pages 1840-1852, September.

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