Conference Program

A Tumor Burden–Integrated Estimand for Cancer Clinical Studies (303708)

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Bo Gao, Regeneron Pharmaceuticals, Inc. 
Joseph G. Ibrahim, University of North Carolina 
Jing-ou Liu, Regeneron Pharmaceuticals, Inc. 
Debra McIntyre, Regeneron Pharmaceuticals, Inc. 
Furong Sun, Regeneron Pharmaceuticals, Inc. 
*Chenguang Wang, Regeneron Pharmaceuticals 

Keywords: Cancer clinical studies; Composite variable strategy; Estimand; R package; Tumor burden;

In cancer clinical studies, tumor burden, such as the size of a tumor or the number of cancer cells, is an important biomarker for evaluating a patient’s response to the treatment and the foundation for RECIST evaluation. In this talk, we will review several existing methods that allow integrating tumor burden outcomes in survival analysis. We will also describe an innovative utility-based approach that synthesizes tumor burden, disease progression and survival in the treatment effect evaluation. An R package that implements the proposed method will be described. Illustration examples will be provided based on multiple cancer clinical studies.