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Inference in Targeted Group-Sequential Covariate-Adjusted Randomized Clinical Trials

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  • Antoine Chambaz
  • Mark J. Laan

Abstract

type="main" xml:id="sjos12013-abs-0001"> This article is devoted to the construction and asymptotic study of adaptive, group-sequential, covariate-adjusted randomized clinical trials analysed through the prism of the semiparametric methodology of targeted maximum likelihood estimation. We show how to build, as the data accrue group-sequentially, a sampling design that targets a user-supplied optimal covariate-adjusted design. We also show how to carry out sound statistical inference based on such an adaptive sampling scheme (therefore extending some results known in the independent and identically distributed setting only so far), and how group-sequential testing applies on top of it. The procedure is robust (i.e. consistent even if the working model is mis-specified). A simulation study confirms the theoretical results and validates the conjecture that the procedure may also be efficient.

Suggested Citation

  • Antoine Chambaz & Mark J. Laan, 2014. "Inference in Targeted Group-Sequential Covariate-Adjusted Randomized Clinical Trials," Scandinavian Journal of Statistics, Danish Society for Theoretical Statistics;Finnish Statistical Society;Norwegian Statistical Association;Swedish Statistical Association, vol. 41(1), pages 104-140, March.
    • Handle: RePEc:bla:scjsta:v:41:y:2014:i:1:p:104-140
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    File URL: http://hdl.handle.net/10.1111/sjos.12013
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    References listed on IDEAS

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    1. Chambaz Antoine & van der Laan Mark J., 2011. "Targeting the Optimal Design in Randomized Clinical Trials with Binary Outcomes and No Covariate: Theoretical Study," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-32, January.
    2. Jun Shao & Xinxin Yu & Bob Zhong, 2010. "A theory for testing hypotheses under covariate-adaptive randomization," Biometrika, Biometrika Trust, vol. 97(2), pages 347-360.
    3. van der Laan Mark J. & Rubin Daniel, 2006. "Targeted Maximum Likelihood Learning," The International Journal of Biostatistics, De Gruyter, vol. 2(1), pages 1-40, December.
    4. Chambaz Antoine & van der Laan Mark J., 2011. "Targeting the Optimal Design in Randomized Clinical Trials with Binary Outcomes and No Covariate: Simulation Study," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-30, January.
    5. van der Laan Mark J., 2006. "Statistical Inference for Variable Importance," The International Journal of Biostatistics, De Gruyter, vol. 2(1), pages 1-33, February.
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    Cited by:

    1. Hai Zhu & Hongjian Zhu, 2023. "Covariate‐adjusted response‐adaptive designs based on semiparametric approaches," Biometrics, The International Biometric Society, vol. 79(4), pages 2895-2906, December.
    2. Fengqing Zhang & Jiangtao Gou, 2021. "Refined critical boundary with enhanced statistical power for non-directional two-sided tests in group sequential designs with multiple endpoints," Statistical Papers, Springer, vol. 62(3), pages 1265-1290, June.

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