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A new inferential approach for response-adaptive clinical trials: the variance-stabilized bootstrap

Author

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  • Alessandro Baldi Antognini

    (University of Bologna)

  • Marco Novelli

    (University of Bologna)

  • Maroussa Zagoraiou

    (University of Bologna)

Abstract

This paper discusses disadvantages and limitations of the available inferential approaches in sequential clinical trials for treatment comparisons managed via response-adaptive randomization. Then, we propose an inferential methodology for response-adaptive designs which, by exploiting a variance stabilizing transformation into a bootstrap framework, is able to overcome the above-mentioned drawbacks, regardless of the chosen allocation procedure as well as the desired target. We derive the theoretical properties of the suggested proposal, showing its superiority with respect to likelihood, randomization and design-based inferential approaches. Several illustrative examples and simulation studies are provided in order to confirm the relevance of our results.

Suggested Citation

  • Alessandro Baldi Antognini & Marco Novelli & Maroussa Zagoraiou, 2022. "A new inferential approach for response-adaptive clinical trials: the variance-stabilized bootstrap," TEST: An Official Journal of the Spanish Society of Statistics and Operations Research, Springer;Sociedad de Estadística e Investigación Operativa, vol. 31(1), pages 235-254, March.
    • Handle: RePEc:spr:testjl:v:31:y:2022:i:1:d:10.1007_s11749-021-00777-9
    DOI: 10.1007/s11749-021-00777-9
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    References listed on IDEAS

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    1. Lanju Zhang & William F. Rosenberger, 2006. "Response-Adaptive Randomization for Clinical Trials with Continuous Outcomes," Biometrics, The International Biometric Society, vol. 62(2), pages 562-569, June.
    2. Anthony C. Atkinson & Atanu Biswas, 2005. "Bayesian Adaptive Biased-Coin Designs for Clinical Trials with Normal Responses," Biometrics, The International Biometric Society, vol. 61(1), pages 118-125, March.
    3. Alessandro Baldi Antognini & Alessandra Giovagnoli, 2010. "Compound optimal allocation for individual and collective ethics in binary clinical trials," Biometrika, Biometrika Trust, vol. 97(4), pages 935-946.
    4. William F. Rosenberger & Nigel Stallard & Anastasia Ivanova & Cherice N. Harper & Michelle L. Ricks, 2001. "Optimal Adaptive Designs for Binary Response Trials," Biometrics, The International Biometric Society, vol. 57(3), pages 909-913, September.
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