Statistical Considerations in the Design and Analysis of Cancer Trials with Immune-Oncology Therapies
This chapter provides an overview of the basic principles involved in the design and analysis of phase III immune-oncology clinical trials, which includes the choice of the endpoints, the statistical analysis of the main analysis for the primary endpoint, and different design considerations. In particular, this chapter discusses four alternative methods for analysis of the primary endpoint in a phase III trial when the proportional hazards assumption is not met (often the case in immune-oncology trials), including the weighted log-rank test, the weighted Kaplan-Meier test, the MaxCombo test, and the restricted mean survival time test. Next, the potential advantages and limitations are discussed and are applied on a real dataset. Lastly, the chapter also provides an example of an intricate trial in its design stage.
