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Randomizing a clinical trial in neuro-degenerative disease

Author

Listed:
  • Atkinson, Anthony C.
  • Duarte, Belmiro P.M.
  • Pedrosa, David
  • van Munster, Marlena

Abstract

The paper studies randomization rules for a sequential two-treatment, two-site clinical trial in Parkinson’s disease. An important feature is that we have values of responses and five potential prognostic factors from a sample of 144 patients similar to those to be enrolled in the trial. Analysis of this sample provides a model for trial analysis. The comparison of allocation rules is made by simulation yielding measures of loss due to imbalance and of potential bias. A major novelty of the paper is the use of this sample, via a two-stage algorithm, to provide an empirical distribution of covariates for the simulation; sampling of a correlated multivariate normal distribution is followed by transformation to variables following the empirical marginal distributions. Six allocation rules are evaluated. The paper concludes with some comments on general aspects of the evaluation of such rules and provides a recommendation for two allocation rules, one for each site, depending on the target number of patients to be enrolled.

Suggested Citation

  • Atkinson, Anthony C. & Duarte, Belmiro P.M. & Pedrosa, David & van Munster, Marlena, 2023. "Randomizing a clinical trial in neuro-degenerative disease," LSE Research Online Documents on Economics 118653, London School of Economics and Political Science, LSE Library.
    • Handle: RePEc:ehl:lserod:118653
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    File URL: http://eprints.lse.ac.uk/118653/
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    References listed on IDEAS

    as
    1. Anthony C. Atkinson, 2002. "The comparison of designs for sequential clinical trials with covariate information," Journal of the Royal Statistical Society Series A, Royal Statistical Society, vol. 165(2), pages 349-373, June.
    2. Wei Ma & Yichen Qin & Yang Li & Feifang Hu, 2020. "Statistical Inference for Covariate-Adaptive Randomization Procedures," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 115(531), pages 1488-1497, July.
    Full references (including those not matched with items on IDEAS)

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    More about this item

    Keywords

    bias; biased-coin design; empirical multivariate distribution; loss; minimization; randomization;
    All these keywords.

    JEL classification:

    • C1 - Mathematical and Quantitative Methods - - Econometric and Statistical Methods and Methodology: General

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