“I probably should do that”

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.

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.