Oral Targeted Anticancer Medications: Use and Spending by Evidence and Magnitude of Clinical Benefit, 2016-2021

An increasing number of oral targeted anticancer medications (OTAMs) are approved with uncertain evidence of clinical benefits, marketed at increasing prices, and subject to insurance coverage mandates. We determined how use of and spending on OTAMs varied by evidence and magnitude of clinical benefit.

Using claims data between 2016 and 2021 from a large, national insurer, we assessed volumes of use and spending on OTAMs approved by the (US) Food and Drug Administration (FDA) between 2010 and 2021. We categorized drug use by evidence of overall survival (OS) and progression-free survival benefits for each FDA-approved indication using FDA-approved drug labels. We assessed how use in terms of days supplied, spending, and patients exposed was related to the level of evidence available for specific drug indications.

Use of OTAMs increased from 50.7 patient-days supplied per 1,000 enrolled patients in 2016 to 120.4 patient-days supplied in 2020 (last full year of data). Approximately half of the dispensings (46.8% in 2016 and 55.6% in 2021) were for patients with indications for which the drugs had label-documented evidence of OS benefit at the time of dispensing. Spending on OTAMs increased from $394 million US dollars (USD) in 2016 to nearly $1 billion USD in 2020. Spending for indications with evidence of OS benefit fluctuated between 41.7% and 47.1% of total spending across all OTAM dispensings. Spending on off-label use was consistently 11%-12% of all OTAM spending annually.

Between 2016 and 2021, the largest shares of OTAM dispensings and spending were for indications with documented evidence of OS benefits. Nevertheless, significant spending and use were observed for drugs without confirmed OS benefit.