Response-adaptive randomization in clinical trials: from myths to practical considerations
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Abstract (arXiv)
Response-Adaptive Randomization (RAR) is part of a wider class of data-dependent sampling algorithms, for which clinical trials are typically used as a motivating application. In that context, patient allocation to treatments is determined by randomization probabilities that change based on the accrued response data in order to achieve experimental goals. RAR has received abundant theoretical attention from the biostatistical literature since the 1930's 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 well-known practical examples and its widespread use in machine learning. Papers on the subject present different views on its usefulness, and these are not easy to reconcile. This work aims to address this gap by providing a unified, broad and fresh review of methodological and practical issues to consider when debating the use of RAR in clinical trials.
Key facts. Cited by 10 papers in the map (data through 14 Oct 2026). First posted on arXiv 1 May 2020.
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Citing papers per year: 2022: 2; 2023: 1; 2024: 1; 2025: 1; 2026: 5.
10 papers.
- 2023Modern Bayesian Experimental Designcited by 125
- 2022Multi-disciplinary fairness considerations in machine learning for clinical trialscited by 8
- 2024Screening for Diabetes Mellitus in the U.S. Population Using Neural Network Models and Complex Survey Designscited by 3
- 2025Towards Regulatory-Confirmed Adaptive Clinical Trials: Machine Learning Opportunities and Solutionscited by 3
- 2022Some performance considerations when using multi-armed bandit algorithms in the presence of missing datacited by 1
- 2026Designing Time Series Experiments in A/B Testing with Transformer Reinforcement Learningcited by 1
- 2026Adaptive Experimentation for Censored Survival Outcomescited by 0
- 2026On Response-Adaptive Targeting Strategies for Multi-Treatment Experimentscited by 0
- 2026Full Bayesian Reinforcement Learning via LF-IBIScited by 0
- 2026Longitudinal Adaptive Experimental Design for Learning Multiple Target Estimands with Semiparametric Efficient Inferencecited by 0
- 2026Longitudinal Adaptive Experimental Design for Learning Multiple Target Estimands with Semiparametric Efficient Inference31 Jul 2026
- 2026Full Bayesian Reinforcement Learning via LF-IBIS2 Jul 2026
- 2026On Response-Adaptive Targeting Strategies for Multi-Treatment Experiments16 Jun 2026
- 2026Adaptive Experimentation for Censored Survival Outcomes18 May 2026
- 2026Designing Time Series Experiments in A/B Testing with Transformer Reinforcement Learning2 Feb 2026
- 2025Towards Regulatory-Confirmed Adaptive Clinical Trials: Machine Learning Opportunities and Solutions12 Mar 2025
- 2024Screening for Diabetes Mellitus in the U.S. Population Using Neural Network Models and Complex Survey Designs28 Mar 2024
- 2023Modern Bayesian Experimental Design28 Feb 2023
- 2022Multi-disciplinary fairness considerations in machine learning for clinical trials18 May 2022
- 2022Some performance considerations when using multi-armed bandit algorithms in the presence of missing data8 May 2022