Randomizing a clinical trial in neuro-degenerative disease

Anthony C. Atkinson, Belmiro P.M. Duarte, David Pedrosa and Marlena van Munster

LSE Research Online Documents on Economics from London School of Economics and Political Science, LSE Library

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.

Keywords: bias; biased-coin design; empirical multivariate distribution; loss; minimization; randomization (search for similar items in EconPapers)
JEL-codes: C1 (search for similar items in EconPapers)
Date: 2023-06-01
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Published in Contemporary Clinical Trials Communications, 1, June, 2023, 33. ISSN: 2451-8654

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