Here’s What It Means When a Physician Prescribes You an Off-Label Drug

With constant headlines linking Ozempic with weight loss, you may have forgotten that the medication is intended to manage diabetes in adults. That's because Ozempic and its cousin Mounjaro are being prescribed so frequently by physicians "off-label," which is when you're given a drug that's been FDA-approved to treat a condition different from your own. 

The practice is generally legal and quite common, but doesn’t come without its risks. According to one earlier study from 2001, about one in five prescriptions for many common medications are considered off-label, but "actual off-label use might be even higher" than what's being reported, per the American Cancer Society. There are legitimate reasons why off-label prescribing happens, and some state legislatures have recently passed bills to make sure that the practice can continue and doctors are protected from liabilities. Still, there are some safety and efficacy factors to consider.

How and why drugs are prescribed off-label 

Physicians have a lot of discretion when it comes to prescribing drugs for off-label uses, says  Adriane Fugh-Berman, MD, a pharmacology and physiology professor at Georgetown University Medical Center. “A physician or another prescriber can prescribe any drug for anything they want to,” she says. “ It’s complicated because some off-label use is proven and is good, but there's a lot of off-label promotion of drugs that either haven't been proven to help a particular condition or even has been disproven to help a particular condition... It’s illegal for a drug company to promote a drug for a condition other than what the FDA approved it for.” 

“There's off-label use, and then there's off-label promotion,” she adds. “Off-label use is not illegal — and it shouldn't be.” 

And that’s because in some cases, it’s necessary. If they were required to follow FDA-approved labels more strictly, patients would miss out on accessing medications that could improve their conditions. Consider:

• Pregnant women and children. If a medication isn't specifically tested in these groups, they often aren't approved for these patients either, but "we don't want to withhold drugs from a population in which it wasn't tested," says Fugh-Berman. For example, depression is common during pregnancy, but antidepressants aren't explicitly approved for pregnant women and may pose some risks to an unborn baby. But you and your doctor may determine that the benefits of treating depression outweigh the potential side effects. Off-label prescribing allows for that calculation. 

• Rare diseases. An estimated 95% of rare diseases do not have any FDA-approved therapies. Pharmaceutical companies may not research, develop, and test treatments that target certain diseases simply due to their rarity. Off-label prescribing allows those with such conditions to have more treatment options than they would otherwise. In fact, off-label meds account for up to 90% of drugs prescribed for rare diseases, according to one 2007 estimate.

• Research-backed uses. Research supports off-label use in some cases, but drugmakers may not seek approval. For example, many studies show that misoprostol is safe and effective for early pregnancy abortion, but it is not FDA-approved on its own for this purpose.

Nevertheless, taking drugs for a condition they have not been approved to treat can be a gamble. Off-label prescriptions may carry a greater risk of unwanted or harmful side effects, and according to a 2008 estimate, about three-quarters aren't backed by adequate scientific evidence. And real harm has occurred. In the case of the off-label drug combo "fen-phen," the medications were widely prescribed in the '90s for weight loss and ended up being linked to heart damage for some patients, which ultimately led to a multi-billion dollar settlement with the drug manufacturers. 

How can patients better advocate for themselves?

• Become an expert on the medications you take. While doctors may prescribe many drugs, you only have to focus on those you’re prescribed. Thoroughly research those medications and become familiar with their approved use (try the DailyMed database).

• Ask your doctor for context. If they prescribed an off-label drug for your condition or symptoms, some questions you might ask are: ‘Is this common practice? Are there studies that back up this use? What are the potential risks?’

• Talk to your pharmacist. Pharmacists often know much more about medications than doctors, making them a good resource for any questions about your prescriptions, including off-label drugs, says Fugh-Berman.

theSkimm

Off-label prescribing is common and enables patients to access much-needed medications, but it's not without its risks. It's worth familiarizing yourself with the treatment options for your condition, as well as your prescriptions, and asking your doctor about why they're prescribing something and potential pros and cons.