A utility‐based design for randomized comparative trials with ordinal outcomes and prognostic subgroups

Thomas A. Murray, Ying Yuan, Peter F. Thall, Joan H. Elizondo and Wayne L. Hofstetter

Biometrics, 2018, vol. 74, issue 3, 1095-1103

Abstract: A design is proposed for randomized comparative trials with ordinal outcomes and prognostic subgroups. The design accounts for patient heterogeneity by allowing possibly different comparative conclusions within subgroups. The comparative testing criterion is based on utilities for the levels of the ordinal outcome and a Bayesian probability model. Designs based on two alternative models that include treatment‐subgroup interactions are considered, the proportional odds model and a non‐proportional odds model with a hierarchical prior that shrinks toward the proportional odds model. A third design that assumes homogeneity and ignores possible treatment‐subgroup interactions also is considered. The three approaches are applied to construct group sequential designs for a trial of nutritional prehabilitation versus standard of care for esophageal cancer patients undergoing chemoradiation and surgery, including both untreated patients and salvage patients whose disease has recurred following previous therapy. A simulation study is presented that compares the three designs, including evaluation of within‐subgroup type I and II error probabilities under a variety of scenarios including different combinations of treatment‐subgroup interactions.

Date: 2018
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