Off-label drug use, which is widely practiced in oncology care, accounted for $5 billion in cancer drug costs in 2010. Now a team of researchers is recommending an overhaul of the process that determines which cancer drugs used off-label are reimbursed by federally-funded health insurance in the United States.
In an article published online in JAMA, physician-researchers from the University of North Carolina Lineberger Comprehensive Cancer Center raised concerns that there are inconsistencies between the five designated compendia that the Centers for Medicare & Medicaid Services (CMS) use to determine which which drugs it will reimburse for off-label uses in cancer care. They also cited the “weak quality” of evidence used in some cases to allow some off-label uses in oncology, which they argue could be leading to poor quality of patient care and high costs.
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“We could be causing substantial suffering for cancer patients because of the sometimes cavalier use of off-label drugs. A new, more rigorous approach is warranted in order to protect our patients,” said coauthor Ethan Basch, MD, MSc, in a press statement.
Federal law specifies that CMS should pay for a drug that is not used in accordance with the US Food and Drug Administration (FDA) label when one of the five reference guides states that the drug’s off-label use is medically appropriate. However, private payers also use the compendia recommendations to support coverage decisions, often using different criteria than CMS or combing compendia recommendations with conclusions based on their own internal review process.
The researchers outlined four steps for improving the review process: (1) develop methodological standards for evaluating the evidence and generating indications for off-label coverage; (2) combine the findings into one single, rigorously developed resource rather than five inconsistent reference guides; (3) assess whether those reviewing and determining off-label drug use policies have potential financial conflicts of interest; and (4) make compendia listings free to the public.
Dr Basch and colleagues also recommended that the FDA, or a public–private partnership, should oversee the decision-making process.
