The US has worse cancer-related outcomes than other high-income countries while bearing the highest cost of cancer care in the world. A reason for increasing cost may be improved efficacy of expensive novel agents. One study found an association between measures of benefit and price, although the study considered gains in progression-free survival (PFS) to be the same as gains in overall survival (OS), an assumption that may be debated, given the uncertainty regarding health benefits associated with PFS. Another study of all cancer drug approvals from 2009 to 2013 found no association between measures of efficacy and pricing of cancer drugs.

The past 5 years have seen a marked increase in the number of cancer therapies on the market and a greater interest in value-based pricing. It is unclear whether the value proposition has shifted: have cancer drugs started to rely on improved benefit and decreased use of surrogate end points to justify skyrocketing costs? We described these trends by estimating all US Food and Drug Administration (FDA) anticancer approvals in recent years and evaluating if an association exists between their cost and efficacy.