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Some Solutions Inspired by Survey Sampling Theory to Build Effective Clinical Trials

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  • Yves Tillé

Abstract

The organisation of a design of experiments, for example, for the realisation of a clinical trial, is crucial. It is often desirable to balance designs so that the means of the covariates are approximately the same in the test and control groups. In survey sampling theory, balanced sampling and calibration are two techniques that improve the precision of estimates. In this paper, we show the links between the two areas. We begin by assessing the gain in precision between a balanced design and a simple random sampling for the least squares estimators and the estimator by differences. We compare rerandomisation techniques and the cube method in order to balance the design. We propose a new method, particularly efficient, which combines the cube method with multivariate matching. A set of simulations is carried out in order to evaluate the different methods. The interest of the calibration is shown even if the design is almost balanced. It is thus shown that tools used by survey statisticians can be useful for experimental designs and clinical trials.

Suggested Citation

  • Yves Tillé, 2022. "Some Solutions Inspired by Survey Sampling Theory to Build Effective Clinical Trials," International Statistical Review, International Statistical Institute, vol. 90(3), pages 481-498, December.
    • Handle: RePEc:bla:istatr:v:90:y:2022:i:3:p:481-498
    DOI: 10.1111/insr.12498
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    References listed on IDEAS

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    1. Maria Michela Dickson & Yves Tillé, 2016. "Ordered spatial sampling by means of the traveling salesman problem," Computational Statistics, Springer, vol. 31(4), pages 1359-1372, December.
    2. Zhenzhen Xu & John D. Kalbfleisch, 2010. "Propensity Score Matching in Randomized Clinical Trials," Biometrics, The International Biometric Society, vol. 66(3), pages 813-823, September.
    3. Kari Lock Morgan & Donald B. Rubin, 2015. "Rerandomization to Balance Tiers of Covariates," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 110(512), pages 1412-1421, December.
    4. Anton Grafström & Niklas L. P. Lundström & Lina Schelin, 2012. "Spatially Balanced Sampling through the Pivotal Method," Biometrics, The International Biometric Society, vol. 68(2), pages 514-520, June.
    5. Hasler, Caren & Tillé, Yves, 2014. "Fast balanced sampling for highly stratified population," Computational Statistics & Data Analysis, Elsevier, vol. 74(C), pages 81-94.
    6. Anton Grafström & Lina Schelin, 2014. "How to Select Representative Samples," Scandinavian Journal of Statistics, Danish Society for Theoretical Statistics;Finnish Statistical Society;Norwegian Statistical Association;Swedish Statistical Association, vol. 41(2), pages 277-290, June.
    7. Raphaël Jauslin & Yves Tillé, 2020. "Spatial Spread Sampling Using Weakly Associated Vectors," Journal of Agricultural, Biological and Environmental Statistics, Springer;The International Biometric Society;American Statistical Association, vol. 25(3), pages 431-451, September.
    8. José R. Zubizarreta, 2015. "Stable Weights that Balance Covariates for Estimation With Incomplete Outcome Data," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 110(511), pages 910-922, September.
    9. Desislava Nedyalkova & Yves Tillé, 2008. "Optimal sampling and estimation strategies under the linear model," Biometrika, Biometrika Trust, vol. 95(3), pages 521-537.
    10. F. J. Breidt & G. Chauvet, 2012. "Penalized balanced sampling," Biometrika, Biometrika Trust, vol. 99(4), pages 945-958.
    11. Guillaume Chauvet & Yves Tillé, 2006. "A fast algorithm for balanced sampling," Computational Statistics, Springer, vol. 21(1), pages 53-62, March.
    12. Jean-Claude Deville & Yves Tille, 2004. "Efficient balanced sampling: The cube method," Biometrika, Biometrika Trust, vol. 91(4), pages 893-912, December.
    13. Kosuke Imai & Gary King & Elizabeth A. Stuart, 2008. "Misunderstandings between experimentalists and observationalists about causal inference," Journal of the Royal Statistical Society Series A, Royal Statistical Society, vol. 171(2), pages 481-502, April.
    14. Stevens, Don L. & Olsen, Anthony R., 2004. "Spatially Balanced Sampling of Natural Resources," Journal of the American Statistical Association, American Statistical Association, vol. 99, pages 262-278, January.
    15. Matti Langel & Yves Tillé, 2011. "Corrado Gini, a pioneer in balanced sampling and inequality theory," Metron - International Journal of Statistics, Dipartimento di Statistica, Probabilità e Statistiche Applicate - University of Rome, vol. 0(1), pages 45-65.
    16. Ho, Daniel E. & Imai, Kosuke & King, Gary & Stuart, Elizabeth A., 2007. "Matching as Nonparametric Preprocessing for Reducing Model Dependence in Parametric Causal Inference," Political Analysis, Cambridge University Press, vol. 15(3), pages 199-236, July.
    17. Wayne A. Fuller, 2009. "Some design properties of a rejective sampling procedure," Biometrika, Biometrika Trust, vol. 96(4), pages 933-944.
    18. Quan Zhou & Philip A Ernst & Kari Lock Morgan & Donald B Rubin & Anru Zhang, 2018. "Sequential rerandomization," Biometrika, Biometrika Trust, vol. 105(3), pages 745-752.
    19. Wayne A. Fuller & Jason C. Legg & Yang Li, 2017. "Bootstrap Variance Estimation for Rejective Sampling," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 112(520), pages 1562-1570, October.
    20. G. Chauvet & J.-C. Deville & D. Haziza, 2011. "On balanced random imputation in surveys," Biometrika, Biometrika Trust, vol. 98(2), pages 459-471.
    21. José R. Zubizarreta, 2012. "Using Mixed Integer Programming for Matching in an Observational Study of Kidney Failure After Surgery," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 107(500), pages 1360-1371, December.
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