What does "off label" mean?

"Off label" is a term frequently used in the practice of cosmetic medicine. But what exactly does "off label" mean and is it safe for patients?

Background:

All drugs and devices are tightly controlled by the US Food and Drug Administration to ensure safety and effectiveness. Drug and device companies spend years and untold millions of dollars running trials to satisfy the demands of the FDA in order to receive approval. However, FDA approval is limited to the indication for which those time-consuming and expensive studies are performed.

Example:

In the case of the available neuromodulators (Botox, Dysport, Xeomin) that treat facial wrinkles, FDA approval was sought for the treatment of glabellar lines. The FDA did approve these three medications for softening of the "11s" or lines between the eyebrows. Thus, their FDA-approved use is for one specific area of the face.

How does "off label" use evolve?

After years of safe and effective use for treatment of lines between the eyebrows, plastic surgeons and dermatologists began expanding use of Botox, Dysport and Xeomin to other areas of the face. In the United States, this is perfectly legal. Since FDA-approval is costly and time consuming, physicians will often use medications for reasons other than the initial FDA indication. Thus, we began using Botox for softening of horizontal lines in the forehead, wrinkles around the eyes, "bunny lines" or wrinkles in the nose, treatment of muscles around the mouth to accentuate the smile as well as treatment of the masseter muscle to slim the lower face. We also began using Botox to treat migraine headaches which after years of successful use recently received FDA approval.

What are the advantages of "off label" medication use?

If "off label" use was not available, the pace of medical advances would be dramatically slowed. Thus, physicians have the option in the United States to use an FDA-approved medication outside of its initial indication to provide cutting-edge treatment to patients. This is only done after significant thought. Typically, a new technique will be presented at a national meeting of plastic surgeons that catches on. A physician will present data and patient photos that demonstrate safety and effectiveness for a medication beyond what the FDA had considered. This is slowly adopted by physicians in practice and the drug company might, or might not, perform a study and submit the new indication for FDA-approval. Regardless of whether that costly study is performed, physicians and patients will benefit from the new information and it can be immediately deployed to improve patient care.