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The New England Journal of Statistics in Data Science


Seamless Clinical Trials with Doubly Adaptive Biased Coin Designs
Volume 1, Issue 3 (2023), pp. 314–322
Pub. online: 1 March 2023      Type: Biomedical Research      Open accessOpen Access
Accepted
29 January 2023
Published
1 March 2023

Abstract

In addition to scientific questions, clinical trialists often explore or require other design features, such as increasing the power while controlling the type I error rate, minimizing unnecessary exposure to inferior treatments, and comparing multiple treatments in one clinical trial. We propose implementing adaptive seamless design (ASD) with response adaptive randomization (RAR) to satisfy various clinical trials’ design objectives. However, the combination of ASD and RAR poses a challenge in controlling the type I error rate. In this paper, we investigated how to utilize the advantages of the two adaptive methods and control the type I error rate. We offered the theoretical foundation for this procedure. Numerical studies demonstrated that our methods could achieve efficient and ethical objectives while controlling the type I error rate.

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Keywords
Adaptive design Ethics Efficiency Response adaptive randomizations Type I error

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