Cancer Health Insurance Pharmaceuticals

Off-label use of cancer drugs

When each drug is approved by the FDA, the drug is not approved to treat all patients.  Each drug receives an “indication” which basically represents the types of patients the drug can treat.  Giving the treatment to patients with said indication is known as “on label” prescribing.

Drugs developed to treat one disease may sometimes be used “off label” to treat another disease.  In cancer treatment, for instance, in cases where there may be no effective treatments for a patient’s tumor, other cancer treatments may be used “off label” to treat this patient. It is often unclear whether “off label” drug use is inappropriate or not.

Smieliauskas et al. (2017) looks at off-label cancer drug use is a recent study by:

…we identified 41% of utilization of infused chemotherapies as being off‐label, including 29% of use deemed as appropriate off‐label use and 17% of utilization characterized as inappropriate off‐label administration under reference compendia over the 1999–2007 timeframe. We observed an overall declining trend from 1999 to 2007 for all three measures of off‐label use: overall, appropriate, and inappropriate use.

The authors define inappropriate off-label drug use as any use outside of those recommended by drug compendia, such as the National Comprehensive Cancer Network (NCCN).  The authors rely on a crude measure as some off-label uses may be helpful to patients if physicians have private information not available to health insurers or recorded in claims data that may make the off label treatment decision reasonable.

The authors also investigate whether state laws that mandate insurers to cover cancer treatments for off-label indications recommended by drug compendia.  They find:

…no discernible effect of the implementation of state laws to mandate coverage of off‐label use of cancer drugs on utilization in eight states that were late adopters of these mandates…Overall, these results suggest null to small effects on off-label utilization of the state laws passed during the time window of our analysis.

The authors are surprised by this small effect, but it could be the case that most private insurers already cover off-label use of cancer drug therapies included in these drug compendia.


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