Robust Treatment Comparison Based on Utilities of Semi-Competing Risks in Non-Small-Cell Lung Cancer

• Activity Number: 565 - JASA Applications and Case Studies
• Type: Invited
• Date/Time: Wednesday, August 1, 2018 : 2:00 PM to 3:50 PM
• Sponsor: JASA, Applications and Case Studies
• Author(s): Thomas Murray* and Ying Yuan and Peter F. Thall and Sarah McAvoy and Daniel R. Gomez
• Companies: University of Minnesota and University of Texas M.D. Anderson Cancer Center and The University of Texas MD Anderson Cancer Center and The University of Texas MD Anderson Cancer Center and The University of Texas MD Anderson Cancer Center

Abstract

This talk will describe a design for a randomized clinical trial comparing two second-line treatments for recurrent non-small-cell lung cancer, chemotherapy versus chemotherapy plus reirradiation. The research question is whether the potential efficacy benefit that adding reirradiation to chemotherapy may provide justifies its potential for increasing the risk of toxicity. The design uses two co-primary outcomes: time to disease progression or death, and time to severe toxicity. Because follow-up ends at disease progression or death, the two outcomes are semi-competing risks. A conditionally conjugate Bayesian model that is robust to misspecification is formulated using piecewise exponential distributions. A numerical utility function is elicited from the physicians that characterizes desirabilities of the possible co-primary outcome realizations. A comparative test based on posterior mean utilities is proposed. A simulation study is presented to evaluate test performance for a variety of treatment differences, and a sensitivity assessment to the elicited utility function is performed. General guidelines are given for designing a utility-based trial in a similar setting.