Response-adaptive randomization in clinical trials: from myths to practical considerations


Abstract in English

Response-adaptive randomization (RAR) is part of a wider class of data-dependent sampling algorithms, for which clinical trials are used as a motivating application. In that context, patient allocation to treatments is determined by randomization probabilities that are altered based on the accrued response data in order to achieve experimental goals. RAR has received abundant theoretical attention from the biostatistical literature since the 1930s and has been the subject of numerous debates. In the last decade, it has received renewed consideration from the applied and methodological communities, driven by successful practical examples and its widespread use in machine learning. Papers on the subject can give different views on its usefulness, and reconciling these may be difficult. This work aims to address this gap by providing a unified, broad and up-to-date review of methodological and practical issues to consider when debating the use of RAR in clinical trials.

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