Dose Finding Experiments: Responses of Mixed Type

  • Valerii V. Fedorov
  • Yuehui Wu
  • Rongmei Zhang
Conference paper
Part of the Contributions to Statistics book series (CONTRIB.STAT.)


Multiple-endpoint models are widely used in drug development and other fields. It is common that the endpoints have different characteristics such as all continuous, all binary, or a mixture of them. This study investigates mixed responses, one continuous and one binary, correlated and observed simultaneously. It is an extension of our previous studies based on a bivariate probit model for two binary endpoints. We quantify the study goal with a utility function, construct locally two-stage D-optimal designs under the constraints, and use them as benchmarks for the two-stage designs with interim adjustment and fully adaptive designs. The simulation results suggest that the two-stage design is almost as efficient as the locally two-stage optimal design, as well as being logistically simpler than the fully adaptive design. We do not analyze asymptotic properties but confine ourselves to Monte-Carlo simulations to evaluate their properties for reasonable (practical) sample sizes.


Optimal Design Penalty Function Design Point Information Matrix Single Observation 
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Copyright information

© Springer-Verlag Berlin Heidelberg 2010

Authors and Affiliations

  1. 1.GlaxoSmithKlineCollegevilleUSA
  2. 2.CCEB, University of PennsylvaniaPhiladlephiaUSA

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