Association Between US Drug Price and Measures of Efficacy for Oncology Drugs Approved by the US Food and Drug Administration From 2015 to 2020

This cross-sectional study estimates all US Food and Drug Administration anticancer approvals in recent years and evaluates if an association exists between their cost and efficacy.

The US has worse cancer-related outcomes than other high-income countries while bearing the highest cost of cancer care in the world. 1,2 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), 3 an assumption that may be debated, given the uncertainty regarding health benefits associated with PFS. 4 Another study of all cancer drug approvals from 2009 to 2013 found no association between measures of efficacy and pricing of cancer drugs. 5 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.
Methods | In this retrospective, cross-sectional analysis of all cancer drugs approved by the FDA from January 1, 2015, to December 31, 2020, we assembled metrics of their activity or efficacy and their cost per annum or course of treatment as previously described. 5,6 All prices were rounded to the nearest $1000. For categorical variables, the Kruskal-Wallis test was used to determine significance differences between median annual cost of subgroups, with P < .05 considered statistically significant. Linear models for the continuous variables of annual cost and percentage of improvement were stratified by end points. Analysis was conducted in R, version 4.1.2 (R Foundation). The search was performed in December 2021. Because we used publicly available data, and this was not human participants research, we did not submit this study to an institutional review board.
Results | We analyzed 224 FDA oncological drug approvals across 119 individual drugs. Across all tumor types, the median annual cost for a course of an oncology drug was $196 000 (IQR, $170 000-$277 000; Table).
Discussion | In value-based pricing, drugs that are associated with greater improvements to the same end point or more improved objective end points (ie, PFS rather than ORR, OS rather than PFS) would be expected to cost more. The results of this analysis suggest the opposite: oncology drugs approved based on OS improvement had the lowest median annual cost of the 3 end points. This finding was similar to prior work that found a significantly higher median price of drugs with ORR-based approvals. 5 Furthermore, we did not detect a meaningful association between cancer drug prices and the magnitude of benefit for any of the end points. Among the drugs approved on the basis of response rate, there was only a weak correlation between cost and the magnitude of the response rate gain; the same was true in the categories of drugs approved on the basis of PFS and OS gains. This suggests that cancer drugs are priced based predominantly on what the market will bear. Correcting this trend is vital for the solvency of health care and pharmaceutical development. Limitations of the study include restriction of analysis to a 6-year period, and restriction of analyzed factors affecting drug price to end points only. Outliers (n = 7) were excluded from the figure for clarity, and each dot represents 1 approved drug. ORR indicates overall response rate; OS, overall survival; PFS, progression-free survival.