What exactly are off-label weight-loss medications?

The term “off-label medication” simply means that a healthcare provider is prescribing a medication for a condition that isn’t on the drug label—hence, it’s off-label. And the practice is fairly common: Research shows that 21% to 32.2% of all prescriptions in the U.S. are written for off-label use, including off-label weight-loss medication.

Here’s what the experts say about why this happens, whether it’s safe, and what to know before you consider taking a drug off-label.

What off-label really means

When a medication is FDA-approved, that means it’s been found to be safe and effective for a specific condition. That intended use is what shows up on approved drug labeling and is, generally, what doctors use when deciding which medicine to prescribe for which condition.

But there isn’t a law saying that providers can only prescribe a medication for its FDA-approved conditions. In fact, the FDA says that once a drug is approved, a healthcare provider can prescribe it for other uses as long as it’s medically appropriate. Any time that happens, it’s known as off-label use.

“Off-label just means that the medication is being used in a manner that it wasn't specified in the FDA-packaging approval,” says Dr. Lydia Alexander, M.D., an obesity medicine specialist and past-president of the American Board of Obesity Medicine. There are various reasons why a doctor may prescribe a drug for an off-label use, including that:

• A drug may not have been studied and approved for specific groups of people, like young children or pregnant women
• It has been shown to have additional benefits
• An on-label drug isn’t covered by your insurance but an off-label one with the same efficacy for your condition is
• Your medical condition doesn’t have any approved drugs yet
• You tried all the available on-label medications without success
• Your healthcare provider wants you to take the drug at a different dose than what’s on the label

In certain cases, a drug manufacturer may perform additional studies to show a drug is safe and effective for an off-label use and apply for it to get approved for that purpose by the FDA. This can take time and money, though, so it’s only done if the company thinks it will be worth it.

But wait–read this before you start an off-label medication

Anytime your doctor prescribes you a new medication—on-label or off—ask these questions:

1. Is it safe? Any drug approved by the FDA has gone through rigorous safety tests, but every drug has the potential for side effects. Talk to your provider about what those side effects may be and which medication option is best for you.
   
   
2. Has it been studied for off-label use? Just because the FDA has not approved a drug for a specific health condition doesn’t mean that there isn't any research on its efficacy and safety for that condition. Ask your healthcare provider what they know about the drug’s efficacy and research the drug on fda.gov to fill in any holes and get more answers.
   
   
3. Why this medication versus one that’s FDA-approved? If your provider recommends an off-label medication, ask them for their reasons.
   
   

Is it cheaper to take off-label medication?

This all depends on whether your insurance covers the medication for off-label use. Sometimes the medication that’s FDA-approved for a health condition isn’t covered by a patient’s insurance, but a medication that’s not approved for that condition is covered.

It’s best to talk about the cost of any medication, including off-label ones, with both your healthcare provider and your insurance provider. They can help you understand the potential costs and explore options for reducing those out-of-pocket expenses.

Bottom line

Healthcare providers may sometimes prescribe a medication off-label, which means they’re prescribing it for a condition that the FDA has not approved. They do this for various reasons, including to save a patient money if the off-label medication is covered by insurance while alternatives are not. Regardless of whether a medication is taken on-label or off-label, however, choosing a medication is a highly personal decision that’s between a healthcare provider and their patient.