Prevalence of Off-Label Use and Spending in 2010 Among Patent-Protected Chemotherapies in a Population-Based Cohort of Medical Oncologists

Abstract

A B S T R A C T PurposeThe prevalence of off-label anticancer drug use is not well characterized. The extent of off-label use is a policy concern because the clinical benefits of such use to patients may not outweigh costs or adverse health outcomes. MethodsPrescribing data from IntrinsiQ Intellidose data systems, a pharmacy software provider maintaining a population-based cohort database of medical oncologists, was analyzed. Use of the most commonly prescribed anticancer drugs ("chemotherapies") that were patent protected and administered intravenously to patients in 2010 was examined. Use was classified as "on-label" if the cancer site, stage, and therapy line met the US Food and Drug Administration (FDA)-approved indication. All other use was "off-label." Off-label use was divided by whether it conformed to National Comprehensive Care Network (NCCN) Compendium recommendations, a basis of insurer coverage policies. IMS Health National Sales Perspectives was used to estimate national spending by use category. ResultsTen chemotherapies met inclusion criteria. On-label use amounted to 70%, and off-label use amounted to 30%. Fourteen percent of use conformed to an NCCN-supported off-label indication, and 10% of off-label use was associated with an FDA-approved cancer site, but an NCCNunsupported cancer stage and/or line of therapy. Total national spending on these chemotherapies amounted to $12 billion (B; $7.3B on-label, $2B off-label and NCCN supported; $2.5B off-label and NCCN unsupported). ConclusionCommonly used, novel chemotherapies are more often used on-label than off-label in contemporary practice. Off-label use is composed of a roughly equal mix of chemotherapy applied in clinical settings supported by the NCCN and those that are not.

Payment for Oncolytics in the United States: A History of Buy and Bill and Proposals for Reform

“…There are no out-ofpocket caps for beneficiaries, and coverage of oncolytics is essentially guaranteed for US Food and Drug Administrationapproved indications and many off-label uses as well. 5 The ASP plus 6% reimbursement has recently come under attack. In the 2012 budget sequester, Congress mandated that CMS cut Part B drug reimbursement by 2%.…”

Off-label use of targeted therapies in oncology

“…The frequence of off-label use was 30% of that half was clinically supported by the National Comprehensive Care Network (NCCN). Among agents, the rate of off-label utilization also varied considerably between trastuzumab (1%) and rituximab (67%) [12] . Another American study based on insurance administrative database found a rate of off-label use of rituximab of 25.3% during the period 2001-2007 [13] .…”

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The Ethics of Clinical Trials for Cancer Therapy

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