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Activity Number:
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674
- Adaptive Design - 3
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Type:
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Contributed
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Date/Time:
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Thursday, August 2, 2018 : 10:30 AM to 12:20 PM
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Sponsor:
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Biopharmaceutical Section
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Abstract #328492
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Presentation
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Title:
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Practical Considerations of Subgroups Quantification, Selection and Adaptive Enrichment in Confirmatory Trials
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Author(s):
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Jianchang Lin* and Rachael Liu and Veronica Bunn
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Companies:
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Takeda Pharmaceuticals and Takeda Pharmaceuticals and Florida State University
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Keywords:
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Subgroup Analysis;
Bayesian Decision;
Sample Size Re-estimation ;
Reversal effects;
Enrichment;
Adaptive Design
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Abstract:
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Subgroup analyses are very commonly encountered and conducted in confirmatory clinical trials manner to ensure the appropriate interpretation of the study results. However, there are natural methodological complications that come with multiple analyses could result in incorrect scientific or regulatory conclusions for subgroup analysis. Typical issues could arise include but not limit to: (a) How to make sure subgroup results are reliable and reproducible? (b) How to quantify the reversal subgroup effect which might due to chance finding or the lack of power in subgroup and treatment interaction tests? (c) How to design efficient trials to establish treatment effect in subgroups or/and overall population with proper type I error control? In this paper, we discuss and present different approaches corresponding to these general issues of subgroup analysis, and their impacts and implications on the interpreting of clinical trials as well as the innovations and opportunities in the era of precision medicine. We also conduct simulations to illustrate the operating characteristics of different methodological tools and designs, along with further practical recommendations and guidance.
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Authors who are presenting talks have a * after their name.
