Abstract
Response-adaptive randomization in clinical trials uses accumulated patient response data to adjust the allocation probability for the next patient, so that a particular objective, for example, more patients assigned to the better performing treatment arm, can be achieved. This ethically appealing randomization procedure has gained significant attention in academia, regulatory agencies, and industry in light of widespread of adaptive clinical trial designs with the FDA’s Critical Path Initiative (FDA: Innovation or stagnation: challenge and opportunity on the critical path to new medical products, 2004). However, this procedure has also generated unmatched controversy since its first application in the ECMO trial (Bartlett et al., Pediatrics 76:479–487, 1985). In this chapter, we will describe response-adaptive randomization procedures from both frequentist and Bayesian perspectives and provide a comprehensive assessment on situations where such procedures should be applied.
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Zhang, L., Rosenberger, W.F. (2014). Response-Adaptive Randomization for Clinical Trials. In: He, W., Pinheiro, J., Kuznetsova, O. (eds) Practical Considerations for Adaptive Trial Design and Implementation. Statistics for Biology and Health. Springer, New York, NY. https://doi.org/10.1007/978-1-4939-1100-4_10
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