I enjoyed reading “Off-Label: Technically Unproven, But Not Out of Bounds” (September 2010). But, why wasn’t anything mentioned about the medical and legal responsibility of doctors to inform the patient that the prescribed drug is not FDA-approved for the purpose at hand. Both legally and medically speaking, should the patient be informed about the off-label use, possible side effects, etc.?
—Mike Silverman, O.D.
Coral Springs, Fla

Jill Autry, O.D., R.Ph., Responds:
I do not discuss off-label medication use with patients unless there is a current FDA-approved medication for the condition and I am choosing another agent that is not FDA approved. For example, because there is not another agent approved for bacterial keratitis, I have no choice but to prescribe standard of care topical antibiotics off-label. This is true for other conditions, such as using AzaSite (azithromycin, Inspire) for posterior blepharitis, Mucomyst (acetylcysteine, Bristol-Myers Squibb) for filamentary keratitis or topical NSAIDs for CME.

In contrast, the use of Avastin (bevacizumab, Genentech) for wet AMD is not FDA approved, but Lucentis (ranibizumab, Novartis) is. This an instance where I would discuss off-label use with the patient and the pros/cons of Avastin vs. Lucentis.

Because off-label use is so common for many medications and conditions, the legal precedence would again follow the standard of care, which holds that the majority of practitioners do not generally discuss this altered use with patients.

In regard to side effects, I discuss these with the patient regardless of on-label or off-label because this status does not change the risk of adverse effects unless the drug is used in a different concentration or dosing schedule than what is generally prescribed on-label.