Off-Label Use of Monoclonal Antibodies for Cancer and the Impact on Patient Out-of-Pocket Costs

Cheryl Fullmer, The University of Texas School of Public Health


Purpose: Access to cutting edge treatments is important for patients who are battling cancer in the United States. Newer targeted therapies may be approved for specific diagnosis, but may be reasonable to use for cancers with similar molecular profiles but found in different organs for example. The trends of off-label use for newer classes of anticancer drugs, specifically monoclonal antibody therapy, is not well described. The extent of off-label use is an important policy consideration as costs of the newer drugs are significantly higher than standard therapy and create a financial impact on insurers and the members on those plans. ^ Methods: This study utilizes the MarketScan Research nationwide database which contains individual-level commercial and private health care claims from hospital inpatient stays, outpatient services, and physician claims for enrolled employees, their spouses and dependents. Drug use was classified as on-label or off-label based on diagnosis and meeting combination or progression requirements per the label. After identifying which diagnoses were most common for off-label use, the proportion of off-label use was calculated from the larger diagnoses cohort of patients in treatment at that time. Univariate regression was used to describe factors impacting access and the cost to insurance and patients is described. ^ Results: The off-label use of bevacizumab for oncology diagnoses in the MarketScan database of privately insured patients was 26.9% for the years 2004–2014. The off-label use of ipilimumab was less at 11.8% since its approval in 2011. The insurance policy decisions to cover off-label use is evident as there are reimbursed claims for certain off-label uses before the U.S. Food and Drug Administration (FDA) approves a labeled indication. However, when looking at the matched population with the same diagnosis, only a fraction have received the off-label drug. In 2009, the year following the FDA approval for HER2 negative breast cancer, bevacizumab was used in 7.8% of metastatic breast cancer cases, and when disapproved in 2011 decreased to 2.1% and then to less than 1% in 2012. Patients with certain insurance plans had a higher rate of off-label use treatments and the majority of patients who received the drug did not pay out-of-pocket. Capturing the estimated cost of care for the 4,816 patients who received off-label use bevacizumab for metastatic breast cancer is estimated at $117.5 million–$239.3 million using median and mean insurance payments for this regimen. ^ Conclusion: The progress of immunotherapies for targeted treatment of cancer is encouraging but continued surveillance is clearly needed in order to curtail costs in oncology while maintaining appropriate use and improving outcomes. The policy implications for promoting evidence-based use and monitoring are extremely important as access and cost are clear issues.1 It is difficult to conclude what has a heavier impact on access to off-label anticancer drugs – the drug price, insurance coverage, or physician prescribing patterns as evidence of new indications are supported in publications before FDA approval for specified indications.^

Subject Area

Economics|Health sciences|Public policy

Recommended Citation

Fullmer, Cheryl, "Off-Label Use of Monoclonal Antibodies for Cancer and the Impact on Patient Out-of-Pocket Costs" (2017). Texas Medical Center Dissertations (via ProQuest). AAI10275148.