Multiple‐objective response‐adaptive repeated measurement designs in clinical trials for binary responses |
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Authors: | Yuanyuan Liang Yin Li Jing Wang Keumhee C. Carriere |
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Affiliation: | 1. Department of Epidemiology and Biostatistics, University of Texas Health Science Center at San Antonio, , San Antonio, TX, U.S.A.;2. Department of Mathematical and Statistical Sciences, University of Alberta, , Edmonton, Alberta, Canada |
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Abstract: | A multiple‐objective allocation strategy was recently proposed for constructing response‐adaptive repeated measurement designs for continuous responses. We extend the allocation strategy to constructing response‐adaptive repeated measurement designs for binary responses. The approach with binary responses is quite different from the continuous case, as the information matrix is a function of responses, and it involves nonlinear modeling. To deal with these problems, we first build the design on the basis of success probabilities. Then we illustrate how various models can accommodate carryover effects on the basis of logits of response profiles as well as any correlation structure. Through computer simulations, we find that the allocation strategy developed for continuous responses also works well for binary responses. As expected, design efficiency in terms of mean squared error drops sharply, as more emphasis is placed on increasing treatment benefit than estimation precision. However, we find that it can successfully allocate more patients to better treatment sequences without sacrificing much estimation precision. Copyright © 2013 John Wiley & Sons, Ltd. |
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Keywords: | response‐adaptive repeated measurement designs multiple‐objective allocation strategy binary outcome |
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