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Activity Number: 456 - Design and Analysis of Cancer Immunotherapy Trials with Complex Survival Patterns
Type: Topic Contributed
Date/Time: Wednesday, July 31, 2019 : 8:30 AM to 10:20 AM
Sponsor: Biopharmaceutical Section
Abstract #304252
Title: Designing Cancer Immunotherapy Trials with Complex Survival Patterns
Author(s): Zhenzhen Xu * and BIN ZHU and YONGSOEK PARK
Companies: FDA and NIH/NCI and University of Pittsburg, Department of Biostatistics
Keywords: Cancer Immunotherapy ; Clinical Trials design and analysis; Nonproportional Hazards Assumption; Complex Survival Patterns; Sample size and power calculation; Immuno-oncology study

The presence of various complex survival patterns in immune-oncology (IO) studies violates the Proportional Hazards (PH) assumption required by conventional design and analysis strategies, making most conventionally designed IO studies underpowered or even falsely negative. An efficient study design and analysis strategy should not only salvage the power loss incurred as a result of various complex patterns but also provide a remedy to reduce the occurrence of such patterns. In this paper, we first examine the potential primary root cause behind the complex patterns then present a novel strategy to incorporate the root cause into study design and data analysis, aiming to salvage power loss when the PH assumption is violated. Finite sample efficiency of the proposed methods is evaluated in simulations. Compared with the standard practice based on the regular log-rank test, the proposed design and analysis procedures achieve an adequate study power when various complex patterns are present. In addition, the proposed strategies provide an insight into how the IO study efficiency can be improved.

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

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