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Key takeaways
Off-label prescribing is when a doctor prescribes medication for a use that is not approved by the FDA, a practice that is legal and common in the United States.
The promotion or marketing of off-label drug uses by pharmaceutical companies is illegal, although doctors can prescribe drugs off-label for various reasons, such as the lengthy and expensive process of getting additional FDA approvals.
Off-label use of medications can offer benefits such as more treatment options for rare conditions, potential cost savings, and sometimes more favorable side effects.
There are risks associated with off-label drug use, including possible unexpected side effects, as these uses have not undergone the rigorous FDA approval process, but most off-label uses are for drugs with known side effect profiles.
Years ago, when I was struggling with a mood disorder, my doctor prescribed a drug that is commonly used to treat seizures. I was confused. Why was my physician giving me a medication indicated for such a different condition? Was it a normal standard of care to try drugs for new uses? Is such use safe?
Maybe it’s happened to you: Your medical practice sends a prescription to your pharmacy, then you learn that the medication is typically used to treat a completely different condition than your symptoms. This is called off-label prescribing (also known as prescribing for “unapproved use”). But what does that really mean?
What are off-label medications?
When the Food and Drug Administration (FDA) evaluates a new drug, the pharmaceutical company seeking approval must submit scientific evidence—such as the results of clinical trials—that the drug is effective for its intended use. When a medication receives FDA approval (after the lengthy approval process), it is for treatment of that particular medical condition, and that indication only. Any other use of drugs is off-label.
Is off-label drug promotion legal?
When a pharmaceutical company begins to sell the drug, it can exclusively mention the condition the product is approved to treat. Off-label marketing, or advertising drugs for uses that are unapproved, could leave a pharmaceutical company open to litigation. It’s illegal.
Are doctors allowed to prescribe off-label drugs?
When a doctor prescribes a medication for off-label drug use, that means he or she has prescribed the drug outside of its approved indications. It’s legal in the United States, and common, for doctors to prescribe off-label. In fact, many patients don’t even realize when a prescription falls outside its approved use.
Sometimes, FDA-indications are specific to dosages or patient characteristics (such as sex), rather than just the medical issue itself. A good example of this is age group—most clinical trials test medications on adults rather than minors, so prescribing these medications to younger patients technically falls under the umbrella of off-label, or unapproved, use.
Why do doctors prescribe medication off-label?
It’s important to know that just because a drug use is off-label, it doesn’t mean it will be an ineffective treatment. The reason why prescription drugs are widely used off-label is a practical one, says Amber Cann, Pharm.D, owner of Venus Vitality. “The process of getting a drug through the clinical trial process and approved by the FDA can take two decades, and is incredibly expensive,” she explains. “It is usually not within the drug manufacturer’s interest to seek further indications once the drug is on the market.”
“Occasionally, a drug maker will invest in an additional indication, so that they can specifically market the drug for that purpose,” Dr. Cann says. For example, the antidepressant Paxil (paroxetine), was available for some time before the manufacturer, GlaxoSmithKline, sought additional label indications on the package insert for generalized anxiety disorder and obsessive-compulsive disorder.
These updates are rare—and when they do happen, they take time. “It can take years before labeling catches up with recent clinical data suggesting a drug may be efficacious for a specific problem,” says Amarish Dave, MD, a neurologist and associate medical director at Mercyhealth in Illinois. “In those intervening years, using drugs off-label can provide patients with improved care.”
Questions to ask your doctor about off-label prescription use
If you are curious about off-label use—or nervous about it—it is well within your rights as a patient to ask questions. You might want to know:
- Is the medication off-label for this condition?
- What is the medication approved to treat?
- Why is your doctor prescribing it?
- How will the medication treat the condition or symptoms?
- What are the side effects?
- What are the benefits of off-label treatment?
- Will your health insurance cover this medication for this condition?
Most physicians and pharmacists are happy to discuss new indications and drug safety if a patient has concerns.
Are off-label drugs covered by insurance and Medicare?
You might need to do some research to find out if your insurance, Medicare, or Medicaid will cover an off-label prescription. Coverage depends on the drug—and what condition your physician prescribes it to treat. Reimbursement for medications prescribed off-label can sometimes be difficult, as insurance companies may be wary of paying out for fraudulent reasons. If this happens to you, speak with your healthcare provider or pharmacist about alternate medications. You can also use the SingleCare price lookup tool to see if our savings beat the cash price.
Benefits of off-label use
Prescribing off-label drugs gives medical practitioners more treatment options if one medication doesn’t work for you. It’s especially useful for rare conditions that have limited treatment options. For example, chemotherapy is often only approved for one type of cancer treatment. Physicians can use it off-label to target many different types.
Sometimes off-label use can save you money. “Often a generic medication that is used off-label that can serve the same purpose as an ‘approved’ medication can be substantially cheaper,” Dr. Dave says. “Other benefits include more favorable side effects.”
Risks of off-label use
As with any medication, there may be risks. Because off-label drug use hasn’t been subject to the rigorous process of clinical trials and testing, as was done for approved uses, there could be unexpected side effects. But Dr. Dave says, “Most off-label use is for drugs that already have a known side effect profile.”
Popular off-label drugs
There are many prescription scenarios that fall into the off-label category. A physician might prescribe tricyclic antidepressants to treat ADHD, or prescribe an anticonvulsant, like Topiramate, for depression.
In some cases, off-label use is so common it becomes the primary use. It’s not unheard of for off-label uses to eventually become FDA-approved. “Minoxidil is an old medication once used for high blood pressure,” Dr. Cann says. “It was found to have a [then] unfortunate side effect of stimulating hair growth. That drug is now very rarely used as a hypertension medication, but it is commonly used as a hair growth product.” We now know it as … Rogaine.
Other medications that prescribers commonly use off-label include:
| Drug Name | Approved Use | Prescribed Off-Label For |
| Prazosin HCL | Treatment of hypertension | Nightmares/PTSD |
| clomiphene-citrate | Treatment of infertility in women | Treatment of infertility in men |
| Namenda | Treatment of Alzheimer’s Disease | ADHD and OCD treatment for children |
| Catapres | Treatment of High Blood Pressure | ADHD, Hot Flashes, Tourette’s Syndrome |
| Seroquel | Antipsychotic medications for treatment of schizophrenia, Bipolar Disorder and Major Depressive Disorder | Insomnia, Anxiety Disorder, OCD, Psychosis from Parkinson’s Disease |
| topiramate | Anticonvulsant for treatment of seizures, migraine prevention | Mood disorders, PTSD, weight loss |
| sildenafil citrate | Erectile dysfunction (in men | Female sexual arousal disorder |
| gabapentin | Treatment of seizures | Bipolar Disorder, Hot Flashes, Neuropathy |
| propranolol | Treatment for hypertension | Performance anxiety |
| Tegretol | Anticonvulsant for treatment of seizures | Bipolar Disorder, ADHD |