As a family physician, I often prescribe medications to treat conditions for which the medications do not have FDA approval. Such off-label prescribing is common, comprising approximately 1 in 5 prescriptions for adults and an even higher percentage for children. But patients can be confused by such off-label use, and with limited time during patient visits, providing informed consent becomes a challenge. To me, having them understand is essential.

Because obtaining FDA approval for a specific indication is a lengthy and costly process, many pharmaceutical companies will obtain approval for only one indication. Since companies are required by law to market the medication only under its approved indication, the FDA-approved indication is often the only information readily available to patients.

There may, however, be a large evidence base for other areas of efficacy for that medication.

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Once a medication has become generic, it is unlikely that funds will be made available for further studies to support additional indications. Though there is no law that says patients must be told that the medications they have been prescribed are for an off-label use, and the FDA does not regulate the practice of medicine, I feel it is important to provide at least a brief explanation to my patients. Otherwise, they might become confused or frightened if I prescribe a medication to treat a condition that is not explicitly approved by the FDA.

A good example is when I prescribe fluoxetine (Prozac) for generalized anxiety disorder or social anxiety disorder. Fluoxetine is quite effective for many anxiety disorders, including anxiety disorders in children, and is generally well tolerated. However, if a patient looks up the indications for this long-used medication, he or she will find FDA indications only for major depressive disorder, obsessive compulsive disorder, panic disorder, bulimia nervosa and premenstrual dysphoric disorder.

Patients may only know of fluoxetine as an antidepressant and feel apprehensive. Some of my patients have objected, “But Doctor, I’m not depressed!” Or asked, “Do you think I’m depressed?” I have found that some clarification often helps to avoid confusion and delay in initiation of treatment.

Another example of off-label prescribing is the widespread use of gabapentin (Neurontin) for neuropathic pain. In fact, off-label use of gabapentin is now more common than use for its FDA-approved indications — adjunctive treatment of partial seizures and postherpetic neuralgia. Common evidence-based, non-FDA approved uses of gabapentin include migraine prophylaxis, diabetic nerve pain, mild mood stabilization, restless leg syndrome and anxiety reduction.  Gabapentin can be very effective at reducing symptoms for these conditions. 

As always, I find that taking an extra moment to educate my patients about what the term “off-label” means reduces their confusion and worry regarding my treatment recommendations. It also leads to less delay in initiation of potentially valuable treatments following the visit.

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