Use of New Cancer Drugs Without Documented Clinical Benefit Substantially Increases

A growing share of patients have been prescribed oral targeted cancer drugs without documented overall survival (OS) benefit — from 12.7% in 2011 to 58.8% in 2018, according to a JAMA study, which analyzed dispensing claims for oral cancer drugs first approved by the FDA between Jan. 1, 2011, and Dec. 31, 2018. Cumulative spending on all 44 sample drugs reached $3.5 billion by the end of 2018, and 96.8% of that spending was on drugs approved based on a pivotal randomized clinical trial (RCT). Meanwhile, cumulative spending on drugs without documented OS benefit surpassed that on drugs with a documented benefit by the end of 2018. Among the top 20 drugs by spending amounts in 2018, one drug lacked a pivotal RCT and 13 drugs had no documented OS evidence at the time.


NOTES: RCT refers to “at least one randomized controlled trial supporting approval for at least one indication by end of 2018.” OS refers to “evidence of overall survival benefit by end of 2018.” The study adjusted the spending to 2018 using the Consumer Price Index for prescription drugs, obtained from the U.S. Bureau of Labor Statistics.

SOURCE: “Real-world Use of and Spending on New Oral Targeted Cancer Drugs in the US, 2011-2018,” JAMA Intern Med. 2021;181(12):1596-1604. doi:10.1001/jamainternmed.2021.5983.

This story was reprinted from AIS Health’s biweekly publication RADAR on Drug Benefits.

Jinghong Chen

Jinghong Chen Reporter

Jinghong produces infographics and data stories on health insurance and specialty pharmacy for AIS Health. She graduated from Missouri School of Journalism with a focus on data journalism and international reporting. Before joining AIS in 2018, she worked at WBEZ, Al Jazeera English and The New York Times Chinese.

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