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Activity Number: 129 - Adaptive Design - 1
Type: Contributed
Date/Time: Monday, July 30, 2018 : 8:30 AM to 10:20 AM
Sponsor: Biopharmaceutical Section
Abstract #330706 Presentation
Title: Efficient Two-Stage Designs and Proper Inference for Animal Studies
Author(s): Chunyan Cai* and Jin Piao and Jing Ning and Xuelin Huang
Companies: UT Health Science Center at Houston and University of Southern California and The University of Texas M.D. Anderson Cancer Center and University of Texas MD Anderson Cancer Center
Keywords: two-stage designs; cost-effectiveness; family-wise error rate; Animal studies

Cost-effective yet efficient designs are critical to the success of animal studies. We propose a two-stage design for cost-effectiveness animal studies with continuous outcomes. Given the data from the two-stage design, we derive the exact distribution of the test statistic under null hypothesis to appropriately adjust for the design's adaptiveness. We further generalize the design and inferential procedure to the K-sample case with multiple comparison adjustment. We conduct simulation studies to evaluate the small sample behavior of the proposed design and test procedure. The results indicate that the proposed test procedure controls the type I error rate for the one-sample design and the family-wise error rate for K-sample design very well; whereas the naive approach that ignores the design's adaptiveness due to the interim look severely inflates the type I error rate or family-wise error rate. Compared with the standard one-stage design, the proposed design generally requires a smaller sample size.

Authors who are presenting talks have a * after their name.

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