Measures of survival benefit in cancer drug development and their limitations

摘要

The investigator comparing outcomes across arms of a drug development trial has available a variety of measures of survival benefit. In this article we systematically review the most common measures of comparative survival used in published studies. We distinguish between relative and absolute survival differences, and measures of instantaneous and cumulative risk. We consider settings in which the endpoint is overall survival as well as those in which disease-specific endpoints are of primary interest. We note that different measures capture different aspects of benefit and some may be more reliable than others or more representative of clinically relevant benefit. Rather than simply using procedures that have become standard, analyses should identify the most clinically relevant measures of impact and apply procedures that reliably estimate these.