Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2013 Mar 20;31(9):1134-9.
doi: 10.1200/JCO.2012.42.7252. Epub 2013 Feb 19.

Prevalence of off-label use and spending in 2010 among patent-protected chemotherapies in a population-based cohort of medical oncologists

Rena M Conti  1 , Arielle C Bernstein, Victoria M Villaflor, Richard L Schilsky, Meredith B Rosenthal, Peter B Bach
Affiliations

Prevalence of off-label use and spending in 2010 among patent-protected chemotherapies in a population-based cohort of medical oncologists

Rena M Conti et al. J Clin Oncol. .

Abstract

Purpose: The 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.

Methods: Prescribing 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.

Results: Ten 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 NCCN-unsupported 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).

Conclusion: Commonly 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.

PubMed Disclaimer

Conflict of interest statement

Authors' disclosures of potential conflicts of interest and author contributions are found at the end of this article.

Figures

Fig 1.
Fig 1.
Estimated use of chemotherapies on-label, off-label (National Comprehensive Cancer Network Drugs & Biologics Compendium [NCCN] supported), and off-label (NCCN unsupported).
Fig 2.
Fig 2.
Estimated off-label use of chemotherapies: off-label National Comprehensive Cancer Network (NCCN) Drugs & Biologics Compendium supported, off-label NCCN unsupported, US Food and Drug Administration (FDA)–approved cancer site, and other.
See this image and copyright information in PMC

Comment in

References

    1. Edwards BK, Brown ML, Wingo PA, et al. Annual report to the nation on the status of cancer, 1975-2002, featuring population-based trends in cancer treatment. J Natl Cancer Inst. 2005;97:1407–1427. - PubMed
    1. MEDPAC: Report to Congress: Variation and innovation in Medicare. 2006. http://www.medpac.gov/publications/congressional_reports/June03_Ch9.pdf.
    1. Schrag D. The price tag on progress: Chemotherapy for colorectal cancer. N Engl J Med. 2004;351:317–319. - PubMed
    1. Yabroff KR, Lamont EB, Mariotto A, et al. Cost of care for elderly cancer patients in the UnitedStates. J Natl Cancer Inst. 2008;100:630–641. - PubMed
    1. Aitken ML, Berndt ER, Cutler DM. Prescription drug spending trends in the US: Looking beyond the turning point. Health Affairs. 2009;28:151–160. - PubMed

Publication types