Using medications “off-label” isn’t new—it’s actually been a common and accepted practice for many years. For example, Botox was originally approved in 1989 to treat certain eye conditions, but doctors used it for other purposes for about a decade before it was officially approved for cosmetic use. Another example is semaglutide medications, which are approved for diabetes but are sometimes used for weight loss.
What Does “Off-Label” Mean?
The FDA defines off-label use as using an approved medication or product in a way that isn’t specifically listed on its official label. This could mean using it for a different condition, at a different dose, in a different group of patients, or in another way not originally approved.
Even though the FDA approves drugs for specific uses, once a medication is approved, licensed healthcare providers can prescribe it however they believe is best for their patient. Their decisions are based on medical judgment and what other trained professionals would reasonably do—not just what the drug manufacturer recommends. The FDA allows this but does not guide or recommend individual treatments.
Why Is Off-Label Use Sometimes a Concern?
One challenge is that off-label medications are often used in groups that weren’t included in clinical trials, such as children, older adults, pregnant women, or people with complex health conditions. These groups are often excluded from studies for safety reasons, which means there’s sometimes less research available on how drugs affect them.
Because of this, Off-label uses of FDA-approved medications are not something carriers like to cover right now, and if they do, there will likely be a higher premium for that coverage.
Despite this, off-label use is often appropriate and even considered standard practice in some areas. For example, many medications used in pediatrics, cancer treatment, and HIV care are commonly prescribed off-label.
What Healthcare Providers Need to Consider
If a clinician is thinking about prescribing a medication off-label, they should:
Make sure the drug is already FDA-approved for some use (not experimental)
Be especially careful with medications that carry serious warnings
Fully understand both the approved and off-label uses, including risks and side effects
Check whether the off-label use is widely accepted in their field
Review the patient’s medical history to avoid harmful interactions
Clearly explain everything to the patient, including risks, benefits, and alternatives
Get informed consent in writing and make sure to include the following key points:
An off-label use is when a drug or device is used differently from its FDA-approved label, so not all side effects and complications are known.
The use involves a different route, dose, or condition, and may change the risk profile of the drug.
The details of the treatment have been fully explained and the patient’s understanding was verified and contemporaneously noted in the patient record.
The patient was not given guarantees about the potential results of the medication or device.
The patient is not required to take the medication or use the device as a condition of continued treatment by the practitioner.
The patient received written instructions about the types of conditions, signs, or symptoms that could indicate a possible adverse reaction with directions on what to do and who to contact in the event a reaction occurs.
Consider costs and whether insurance will cover the treatment
Monitor the patient closely for any unexpected
Off-label use has proved beneficial to patients. When approached in a manner consistent with patient safety, it can provide avenues for treatment and optimum clinical results not otherwise available.
