What Does Off-Label Drug Use Mean?

Dr. Bill Quick Health Pro
  • The "off-label use" of medications is common. I'll define off-label use as a recommendation by a physician to their patient to use a prescription medication to treat a medical condition that hasn't been approved for that medication by the FDA or other Health Authority. As most folks know, regulators review studies on a medication that are submitted by the manufacturer, and if the regulators consider the quality of the data from the studies to be sufficient, they allow the manufacturer to add additional information to the label, including new indications for the drug that weren't previously authorized.


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    Common examples of off-label use in the diabetes area include:


    * use of metformin (a widely-used medication for type 2 diabetes) in people diagnosed with pre-diabetes. No medications are approved for use in pre-diabetes, although major studies such as the DPP have found that drugs can help.


    * use of glyburide (a sulfonylurea medication for type 2 diabetes) in pregnant women with diabetes. Studies have found that this pill works almost as well as insulin in women with gestational diabetes.


    * use of insulin and Byetta (exenatide) at the same time: Byetta is not approved for use with insulin.


    * use of Lantus (insulin glargine) on a twice-a-day basis: it's only approved for once-daily dosing.


    * use of Lantus by children under age 6: it's only approved for ages 6 and older.


    Please be aware, physicians are not required to follow the label's restrictions concerning how to use a medication. However, if something goes wrong, and the physician is sued, the attorney will surely show the jury the approved label - and will insinuate that the physician wasn't smart enough to read (and to follow) the label.


    How does the physician defend the decision to use a medication off-label? He/she can't just say "well, it sounded like it's a good idea" or "everybody's doing it." One thing for sure, he/she should be comfortable with any published literature that describes the pro's and con's of using the medication.


    And another way to handle off-label use of medications is through informed consent. The patient should be informed that the proposed use of the medication is off-label. The fact that it hasn't been studied to the level of detail that the FDA wants should be mentioned, and that sometimes the manufacturers won't do the studies that would be needed to support the use: for instance, as glyburide is off-patent and available generically, it's unlikely any manufacturer would set up a study of its use in the high-risk setting of pregnancy. But the physician should be honest, and review both the advantages and disadvantages of the proposed off-label use, and the unknowns.


    And the patient should have the patient sign an informed consent form. Informed consent forms, widely used in surgery and for clinical trials, means that the physician reviews the rationale and risks with the patient, and the patient indicates their understanding by signing the form.


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    Now, back to why I chose the title for this essay ("I probably should do that"). I was at a diabetes meeting this past weekend, and was chatting with another physician-attendee about using a diabetes drug in an off-label setting. I pointed out that he might want to get the patient to sign an informed consent; his response was "I probably should do that."


    When I was in office practice, I routinely had patients sign off on their care plans (as well as me signing off on the plans), and if a drug was to be used off-label, that was part of the care plan. So it doesn't need to be an official looking document labeled with a pretentious title like "Off-Label Use Informed Consent Plan Form." But it should be very clearly documented that the patient was aware of the off-label use, and that the patient understood the risks as well as the benefits, and agreed to the off-label use.


    That's the ethical thing to do.

Published On: February 09, 2009