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Semiparametric inference for a two-stage outcome-dependent sampling design with interval-censored failure time data

Author

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  • Qingning Zhou

    (University of North Carolina at Charlotte)

  • Jianwen Cai

    (University of North Carolina at Chapel Hill)

  • Haibo Zhou

    (University of North Carolina at Chapel Hill)

Abstract

We propose a two-stage outcome-dependent sampling design and inference procedure for studies that concern interval-censored failure time outcomes. This design enhances the study efficiency by allowing the selection probabilities of the second-stage sample, for which the expensive exposure variable is ascertained, to depend on the first-stage observed interval-censored failure time outcomes. In particular, the second-stage sample is enriched by selectively including subjects who are known or observed to experience the failure at an early or late time. We develop a sieve semiparametric maximum pseudo likelihood procedure that makes use of all available data from the proposed two-stage design. The resulting regression parameter estimator is shown to be consistent and asymptotically normal, and a consistent estimator for its asymptotic variance is derived. Simulation results demonstrate that the proposed design and inference procedure performs well in practical situations and is more efficient than the existing designs and methods. An application to a phase 3 HIV vaccine trial is provided.

Suggested Citation

  • Qingning Zhou & Jianwen Cai & Haibo Zhou, 2020. "Semiparametric inference for a two-stage outcome-dependent sampling design with interval-censored failure time data," Lifetime Data Analysis: An International Journal Devoted to Statistical Methods and Applications for Time-to-Event Data, Springer, vol. 26(1), pages 85-108, January.
    • Handle: RePEc:spr:lifeda:v:26:y:2020:i:1:d:10.1007_s10985-019-09461-5
    DOI: 10.1007/s10985-019-09461-5
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    References listed on IDEAS

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