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Study Finds Increased Use of New Cancer Drugs Without Documented Clinical Benefit, With Major Cost Implications for Patients

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A new study, published in JAMA Internal Medicine, found that the estimated use of new cancer drugs without documented clinical benefit has substantially increased over the past decade, with major cost implications. 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).

The study sought to establish the proportion of US residents with employer-sponsored insurance between 2011 and 2018 who were dispensed new cancer drugs received drugs without documented overall survival (OS) benefit, and the associated costs. The cross-sectional study of 37,348 patients who received 1 or more of 44 new oral targeted cancer drugs, found 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.

The overall finding 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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