A Patient’s Guide to Off-Label Drug Use and Prescribing

Let’s talk about off-label drug use and prescribing. What if your healthcare provider was prescribing drugs for uses not explicitly approved by the Food and Drug Administration (FDA)? Would you be alarmed? Should you be? Why would a healthcare provider do that? 

Believe it or not, prescribing drugs for uses other than those listed on an FDA label isn’t that unusual — it’s a practice called “off-label drug use.” 

Sometimes, medications approved for one purpose by the FDA are found to be effective treatment options for non-FDA approved conditions.

Essentially, off-label drug use is when a healthcare provider determines the best course of action for you is using an FDA approved drug for a purpose other than the drug’s primary use. 

This isn’t your healthcare provider throwing random medications at you or being irresponsible with their Rx pad. If a healthcare provider is prescribing certain medications for off-label use, it’s likely there’s support that the drug is effective for that use.

Still, anytime you start a new medication, it’s good to know what you’re getting yourself into. And this may be especially true with the off-label use of prescription drugs.

TL;DR What You Need to Know

• “Off-label” drug use refers to being prescribed a drug for an ailment or condition not listed on its FDA label. 

• Before a drug is approved by the FDA, it undergoes a ton of research and intense scrutiny. The label that accompanies a drug includes, among other things, its FDA approved  use, risks and precautions. 

• Sometimes drugs are found to be effective for uses not originally researched and then approved by the FDA.

• While there is some controversy surrounding off-label drug use, healthcare providers prescribing drugs for off-label use is widely accepted and practiced among physicians. 

• As with any prescription drug regimen, do your homework before starting an off-label prescription. 

What Does “Off-Label” Mean?

All drugs sold by pharmaceutical companies must be approved by the FDA. When a pharmaceutical manufacturer develops a new medication, they can’t just holler “Yee gads, we’ve got it!” and then rush the drug into the nearest Walgreens. Getting FDA approval is a long and in-depth process. 

Usually, the research process starts with a drug maker testing the medication in the lab, then in animals and only lastly in humans. 

Once they’re convinced of its effectiveness and safety, they submit the results of all their testing (and a whole bunch of other paperwork) to the FDA’s Center for Drug Evaluation and Research (CDER). 

At the CDER, all of the evidence is reviewed by doctors, statisticians, chemists, pharmacologists and other scientists. They’re reviewing what the drugmaker sent over for a long list of things, but primarily hoping to answer: 

• Does the drug treat the condition it is reported to? 

• Are there other drugs already doing this?

• What are the risks? 

• What can be done to manage those risks?

• Do the benefits outweigh those risks? 

• Is the testing conducted by the drug maker sufficient? 

Once a drug is approved, the FDA develops a label that includes all of the important information a healthcare provider needs to know. 

The label includes information on indications and usage, drug interactions, warnings, precautions, side effects, overdose, potential for abuse, dosage forms and strengths, clinical studies, use in specific populations (such as pregnant women or children) and patient counseling information. 

In other words, the label isn’t just the sticker on the side of your pill bottle — it’s designed to help healthcare providers understand all of the ins and outs of a drug. 

If a healthcare provider decides to prescribe a prescription drug for something other than what’s listed as approved on the label, it’s considered an “off-label” use.

Examples Of Off-Label Drugs 

There are many drugs that are prescribed by healthcare providers for purposes other than what the FDA approved them for. Here are some examples: 

• Bupropion is a drug approved to treat depression but is sometimes used in the treatment of ADHD. 

• Diphenhydramine is approved for the treatment of allergies but is prescribed off-label for insomnia and the effects of chemotherapy.

• Gabapentin is approved for the control of seizures but is prescribed off-label for bipolar disorder, migraines and restless leg syndrome.

• Prazosin is approved to treat high blood pressure, but also used in the treatment of PTSD nightmares and scorpion poisoning.

• Propranolol is a drug approved for high blood pressure, but healthcare providers sometimes prescribe it to treat the physical symptoms of performance anxiety, also known as “stage fright.”

• Singulair® is a brand name drug approved for use in the treatment of asthma, however, it’s prescribed off-label for the treatment of COPD.

• Zyprexa® is a brand name drug approved for use in schizophrenia but is sometimes prescribed off-label for depression treatment.

What Does the FDA Say? 

The FDA does not regulate the practice of medicine — healthcare providers can make prescribing decisions using their clinical judgment about what they think is best for their patients. 

Per the FDA, “Once the FDA approves a drug, healthcare providers generally may prescribe the drug for an unapproved (off-label) use when they judge that it is medically appropriate for their patient.”

Essentially, it’s up to healthcare providers to decide whether or not to give patients medications for purposes other than what’s specifically on the label.

Are Off-Label Drugs Safe? 

All medications come with risks, even when you’re using them for the explicitly approved reasons on the FDA drug label. So, off-label drugs come with risks, too. 

There’s a bit of controversy surrounding off-label drug use. Some believe it’s risky. They say because the drug hasn’t gone through the rigorous FDA process for that particular off-label use, its safety for that particular use is unknown. 

Most healthcare providers, however, support off-label drug use under the supervision of a healthcare provider. Healthcare providers are able to carefully weigh the potential risks vs. benefits of any prescription drug regimen and help patients make the safest decisions possible. 

The fact that off-label drug use is accepted by healthcare providers doesn’t mean it’s completely without risks, though. Remember, all drugs come with risks, potential side effects and possible complications. No prescription drugs are completely risk-free.

What to Know Before Taking a Drug “Off-Label” 

As with any prescription, you should be fully informed before taking a drug for an off-label use. Talk to your healthcare provider about the possible risks and benefits of taking the drug for any use that’s not on the label. 

If you prefer to do your research online, use trusted sources. Scientific journals and MedlinePlus from the National Library of Medicine, are all good places to start. Modern medicine is a marvel.

Often, the solutions for any single given ailment are many. Sometimes, however, the only (or the best) solution for a health problem is one the solution wasn’t initially designed for. 

Off-label prescription drugs successfully treat scores of people, and are a viable option widely accepted by the medical community.