Resources used to inform coverage of off-label uses of cancer drugs are inconsistent and the evidence used to support these uses is of weak quality, according to a study published in the Journal of the American Medical Association.1

Off-label use of cancer drugs is common. The decision to use a cancer drug outside of its FDA-approved indication includes limited treatment options in a particular clinical setting or because a clinician wants to try an unconventional approach.

Five reference guides (compendia) are used by the Centers for Medicare and Medicaid Services (CMS) to determine reimbursement for off-label use of a drug. Private insurers may also use these compendia to inform their coverage decisions.

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A team of researchers at the University of North Carolina (UNC) Lineberger Comprehensive Cancer Center sought to determine the quality and consistency of the compendia used to support off-label use and CMS reimbursement. In previous research, the quality of evidence used to develop these guidelines was found to be less rigorous than that used to support FDA approvals.

In this analysis, the researchers found the level, quantity, consistency, and timeliness of evidence cited was limited. Their findings suggest 4 strategies that could improve the quality of the compendia resources used to guide decisions related to off-label use.

  • Develop a methodological standard for evaluating the evidence presented to support off-label drug recommendations
  • Combine compendia into a single rigorously developed resource
  • Assess the panelists reviewing and developing the recommendations for financial conflicts of interest
  • Compendia listing should be free to the public, not by subscription only

Lastly, the researchers recommend a public-private partnership or FDA oversight on the decision-making process. The UNC team advocates an approach that would be different from the drug-approval process but an improvement over the current system.


1. Green AK, Wood WA, Basch EM. Time to reassess the cancer compendia for off-label drug coverage in oncology. JAMA. 2016 Aug 25. doi: 10.1001/jama.2016.12770. [Epub ahead of print]