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Real-World Utilization of and Spending on New Targeted Cancer Drugs in the United States
Findings from a real-world study suggest that cancer drugs without documented overall survival (OS) benefits are increasingly adopted in the health system, accounting for substantial spending.(JAMA Intern Med. 2021. doi:10.1001/jamainternmed.2021.5983).
“Launch prices of new cancer drugs in the US have substantially increased in recent years despite growing concerns about the quantity and quality of evidence supporting their approval by the [FDA],” wrote Mengyuan Fu, PhD, Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute, Boston, MA, and colleagues.
This cross-sectional study was conducted from July 17, 2019, to July 23, 2021, examining the use of and spending on new oral targeted cancer drugs among US residents with employer-sponsored insurance, stratified by the strength of evidence of benefit.
Authors analyzed dispensing claims for oral targeted cancer drugs that were first approved by the FDA between 2011 and 2018, aggregating the number of patients with drugs dispensed and the total payment for all claims by calendar year, and arraying outcomes according to evidence underlying FDA approvals, including pivotal study design (availability of randomized clinical trials) and OS benefit, as documented in drug labels.
A total of 37,348 patients were included in this study, having taken at least one of the new oral targeted drugs. There were 44 new oral targeted drugs that were dispensed between 2011 and 2018.
Of the participants, 57.1% were men, and the mean age was 64.1 years. In total, 97% of participants received drugs for which evidence from randomized clinical trials existed. The percentage of patients who received drugs without documented OS benefit during the study period rose from 12.7% in 2011 to 58.8% in 2018.
Total spending on sample drugs reached $3.5 billion by the end of 2018, and 96.8% of that was spent on drugs that were approved based on a pivotal randomized clinical trial. By the end of 2018, spending on drugs without documented OS benefit reached $1.8 billion, and spending on drugs with documented OS benefit reached $1.7 billion.
“The findings of this cross-sectional study suggest that drugs used for treatment of cancer without documented OS benefits are adopted in the health system and account for substantial spending,” concluded Dr Fu and colleagues.
