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Using biomarkers to allocate patients in a response-adaptive clinical trial

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<mark>Journal publication date</mark>2/12/2023
<mark>Journal</mark>Communications in Statistics: Simulation and Computation
Issue number12
Number of pages20
Pages (from-to)5946-5965
Publication StatusPublished
Early online date25/11/21
<mark>Original language</mark>English


In this paper, we discuss a response adaptive randomization method, and why it should be used in clinical trials for rare diseases compared to a randomized controlled trial with equal fixed randomization. The developed method uses a patient’s biomarkers to alter the allocation probability to each treatment, in order to emphasize the benefit to the trial population. The method starts with an initial burn-in period of a small number of patients, who with equal probability, are allocated to each treatment. We then use a regression method to predict the best outcome of the next patient, using their biomarkers and the information from the previous patients. This estimated best treatment is assigned to the next patient with high probability. A completed clinical trial for the effect of catumaxomab on the survival of cancer patients is used as an example to demonstrate the use of the method and the differences to a controlled trial with equal allocation. Different regression procedures are investigated and compared to a randomized controlled trial, using efficacy and ethical measures.