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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.
In learning-phase clinical trials in drug development, adaptive designs can be efficient and highly informative when used appropriately. In this article, we extend the multiple comparison procedures with modeling techniques (MCP-Mod) procedure with g
Response adaptive randomization is appealing in confirmatory adaptive clinical trials from statistical, ethical, and pragmatic perspectives, in the sense that subjects are more likely to be randomized to better performing treatment groups based on ac
Phase I dose-finding trials are increasingly challenging as the relationship between efficacy and toxicity of new compounds (or combination of them) becomes more complex. Despite this, most commonly used methods in practice focus on identifying a Max
Use of historical data and real-world evidence holds great potential to improve the efficiency of clinical trials. One major challenge is how to effectively borrow information from historical data while maintaining a reasonable type I error. We propo
Most clinical trials involve the comparison of a new treatment to a control arm (e.g., the standard of care) and the estimation of a treatment effect. External data, including historical clinical trial data and real-world observational data, are comm