What Does Off-Label Use Mean? RealSelf Doctors Have the Answers You Need to Know

Elisabeth Kramer on 22 Jan 2015 at 5:00pm

FDA
If you’ve spent enough time in a doctor’s office, you’ve probably heard the term “off-label.” But what does it mean exactly? We quizzed our team of board-certified doctors to figure out what it is and why it matters.

So, what does “off-label” mean?
Off-label applies to a product used in a way it isn’t advertised for. Using clear nail polish to stop a run in your tights? Off-label. Toothpaste as an acne cure? Off-label. In the realm of cosmetic treatments, think of specific FDA-approved products or devices (Botox, Ultherapy, etc.) being used in ways the FDA hasn’t approved.

Take Botox. Back in 2002, the FDA signed off on the product treating frown lines and only frown lines. Soon, however, doctors discovered Botox could help with problems not listed on the label. Fight migraines? Sure. Reduce crow’s feet? It does that, too. Patients received the benefits of these “off-label” usages for years, but it wasn’t until 2010 and 2013, respectively, that the FDA would give its own thumbs-up.

Why the delay?
So what’s behind the lag in the FDA catching up to real world usage? Time and money.

“Each testing area requires more money and more study time,” explains RealSelf doctor F. Victor Rueckl. “When a company has done their own testing and takes it to the FDA for approval, they want to get it out as quickly as possible, so they only do one area.”

Restylane's an example. In 2003, the injectable gel got the all-clear from the FDA to smooth wrinkles around the nose and mouth. Of course, as those who’ve used the filler probably know, Restylane is also commonly used to plump the lips. The FDA didn’t sign off on that particular use, however, until 2011 — eight years after its initial approval and only when Restylane’s off-label use held up through the FDA’s clinical tests.

RELATED: Off-Label Usage and Who You Should Trust When Considering It

That’s great and all, but is using a product or device off-label actually safe?
Yes, if you ask RealSelf doctors. All of those who answered our question about what off-label means made sure to explain that it’s safe.

“Off-label does NOT mean that the drug or product is not perfectly safe and perfectly suited to treat the particular disease or symptom,” said Dr. Jason M. Petrungaro.

“Off-label use simply means that the FDA approval process has not been done for that specific use,” added Top Doctor Joshua L. Fox. “This does not necessarily mean the treatment is not perfectly safe and appropriate.”

Our doctors aren’t alone in their opinions. One of the few studies conducted on off-label usage found that one in five prescriptions written in the U.S. qualifies as off-label. Yes, we said one in five. That means chances are good that somewhere in the course of your own medical history, you’ve used a drug in a way the FDA would consider off-label.

OK, off-label usage is safe, but what about legal?
Don’t worry. Off-label usage of a drug or device is legal in the U.S. and many other countries around the world. What’s against the law are ads that make it sound like a prescription drug treats a condition if the FDA hasn’t verified that’s in fact the case. So if Botox had advertised that it could treat migraines before it got the green light from the FDA? That would have been illegal. Using Botox to treat migraines in the privacy of your own doctor’s office? A-OK in the eyes of the law.

RELATED: Does the Law Have Any Chance Against False Beauty Advertising?

Does my doctor have to tell me about off-label use?
No, doctors are not obligated to tell you if the treatment they prescribe is FDA-approved. This makes it all the more important that you ask for yourself so you have all the information you need to make your decision.

What if something goes wrong? Will my insurance cover off-label use?
While it’s legal, there remains one big risk when using off-label products: You might end up paying more. For example, if you receive a treatment with a drug in a way not listed on that drug’s FDA-approved label, your insurance company may insist you pay for the whole thing yourself. That applies to any negative side effects too, adds RealSelf’s Dr. Petrungaro.

As the American Cancer Society explains, insurance may be unwilling to fork over the money for expensive drugs used in what they deem “experimental” or “investigational” ways. While you may be willing to pay extra when the medicine in question could kill your tumor, you may want to do some more research before signing up for an off-label filler.

What it all comes down to…
Build a relationship with your doctor. “Trust in your physician is key!” advises Dr. Rueckl while RealSelf Top Doctor Jason Emer suggests discussing how any treatment meets your specific beauty goals.

Don’t be scared to ask questions, either. Off-label practice has a long history of providing safe, effective results so don’t be frightened when you hear the term batted around. Still, as we always promote here at RealSelf, be informed about the choices you make. It’s the best way to make sure you get what you while staying safe and healthy!