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Original Investigation
October 18, 2021

Real-world Use of and Spending on New Oral Targeted Cancer Drugs in the US, 2011-2018

Author Affiliations
  • 1Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute, Boston, Massachusetts
  • 2Department of Pharmacy Administration and Clinical Pharmacy, School of Pharmaceutical Sciences, Peking University, Beijing, China
  • 3Department of Health Policy, London School of Economics and Political Science, London, UK
  • 4Division of Cancer Care and Epidemiology, Departments of Oncology and Public Health Sciences, Queen’s University Cancer Research Institute, Kingston, Canada
JAMA Intern Med. 2021;181(12):1596-1604. doi:10.1001/jamainternmed.2021.5983
Key Points

Question  What proportion of US residents with employer-sponsored insurance who were dispensed new cancer drugs received drugs without documented overall survival benefit, and what were the costs between 2011 and 2018?

Findings  In this cross-sectional study among 37 348 patients who received 1 or more of 44 new oral targeted cancer drugs, the proportion of patients receiving drugs without documented overall survival benefit increased from 13% in 2011 to 59% in 2018, accounting for 52% of the $3.5 billion estimated cumulative spending on the new oral targeted cancer drugs by the end of 2018.

Meaning  Results of this study suggest that the estimated use of new cancer drugs without documented clinical benefit has substantially increased over the past decade, with major cost implications.


Importance  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 US Food and Drug Administration (FDA).

Objective  To assess the use of and spending on new oral targeted cancer drugs among US residents with employer-sponsored insurance between 2011 and 2018, stratified by the strength of available evidence of benefit.

Design, Setting, and Participants  In this cross-sectional study, dispensing claims for oral targeted cancer drugs first approved by the FDA between January 1, 2011, and December 31, 2018, were analyzed. The number of patients with drugs dispensed and the total payment for all claims were aggregated by calendar year, and these outcomes were arrayed according to evidence underlying FDA approvals, including pivotal study design (availability of randomized clinical trials) and overall survival (OS) benefit, as documented in drug labels. This study was conducted from July 17, 2019, to July 23, 2021.

Main Outcomes and Measures  Annual and cumulative numbers of patients who had dispensing events, and annual and cumulative sums of payment for eligible drugs.

Results  Of 37 348 patients who had at least 1 of the 44 new oral targeted drugs dispensed between 2011 and 2018, 21 324 were men (57.1%); mean (SD) age was 64.1 (13.1) years. Most individuals (36 246 [97.0%]) received drugs for which evidence from randomized clinical trials existed; however, a growing share of patients received drugs without documented OS benefit during the study period: from 12.7% in 2011 to 58.8% in 2018. Cumulative spending on all sample drugs totaled $3.5 billion by the end of 2018, of which 96.8% was spent on drugs that were approved based on a pivotal randomized clinical trial. Cumulative spending on drugs without documented OS benefit ($1.8 billion [51.6%]) surpassed that on drugs with documented OS benefit ($1.7 billion [48.4%]) by the end of 2018.

Conclusions and Relevance  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.

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