Progression-free survival: too much risk, not enough reward?

摘要

Given the increasing number of potential therapies for glioblastoma and the slow rate of progress to date, clinical trials must become more efficient while providing clinically relevant answers. In this context, careful selection of clinical trial endpoints is extremely important. Adoption of new therapies requires trials that demonstrate real, clinically meaningful improvements in outcome attributable to the experimental therapy. While survival is undeniably clinically meaningful, there is interest in using other endpoints such as response rate or progression-free survival as more efficient 'surrogates' that limit non-treatment related variation and confounding by post-progression therapy.