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Activity Number: 29 - Statistical Issues Specific to Therapeutic Areas, Power and Sample Size Calculations, and Trial Monitoring
Type: Contributed
Date/Time: Sunday, August 8, 2021 : 1:30 PM to 3:20 PM
Sponsor: Biopharmaceutical Section
Abstract #318362
Title: Interim Analysis Incorporating Intermediate Binary Responses
Author(s): Gang (Gerry) Li* and Jiuzhou Wang and Jingnan (Jenna) Zhang
Companies: Alexion Pharmaceuticals and University of Minnesota Twin Cities and Alexion Pharmaceuticals
Keywords: Interim Analysis; Intermediate Response; Transitional Probability

In many clinical trials, the endpoint of interest is assessed longitudinally with the primary response at the last assessment visit and one or multiple intermediate responses at earlier visit(s) prior to the primary response. At an interim analysis (IA), the analysis set consists of study completers with both intermediate responses and the primary response, and study incompleters with only intermediate response(s). Traditionally the IA is performed based on the primary response from only study completers. However there has been a realization that in some cases incorporating intermediate response(s) from study incompleters could increase the efficiency of the IA. In this talk we will consider IA methods for trials with binary endpoint to incorporate one or two intermediate responses, where the primary response for study incompleters at IA will be predicted through estimated transition probabilities from observed data. Simulations are conducted under various scenarios of treatment effects and sample sizes to compare the performance of different IA methods and results will be presented. The extension of the research to cover more than 2 intermediate responses will also be discussed.

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

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