Medians and Milestones in Describing the Path to Cancer Cures

Abstract

Recent successes in drug development have revolutionized the treatment landscape for patients with advanced cancers. When describing clinical trial results, oncologists often default to median outcomes, both in our own minds and when discussing with patients. It is time to change that metric. Characterization of outcomes by medians fails to capture important distinctions that occur when the survival curve plateaus and does not reflect what an individual patient, hopeful for a chance of long-term survival, most wants to know. We believe transition from the de facto reliance on medians to describe patient outcomes is needed and propose increased use of survival estimates at specific milestones (eg, 1, 2, and 5 years after therapy). Although medians can represent an important shift in the survival curve, they also bias us toward therapies that produce modest benefits in a plurality of patients and obscure long-term benefits occurring in a minority of patients. Improving the chance for durable responses, and ultimately cure, takes a different form—raising the tail of the survival curve.¹ A focus on the tail of the survival curve yields a distinct perspective on the benefit of anticancer therapies and prioritizes therapies with potential to profoundly alter the natural history of disease, even if uncommonly.