Off-Label Prescriptions: What to Know When Medications Are Used Beyond the Label

When a healthcare professional prescribes a medication, you might assume it’s being used exactly as the FDA approved it. But in some cases, it’s prescribed “off-label.” This means the medication is being used in a way that hasn’t been officially approved, such as for a different health condition, dose, or age group.

Off-label prescriptions are legal and often necessary, especially in areas of medicine where approved treatment options are limited. They’re also more common than you may think. In fact, 1 out of every 5 prescriptions is written for an off-label use.

While off-label use can be beneficial, it also comes with some uncertainties. Here’s what off-label use means, when and why it’s prescribed, and potential risks to know about.

What does ‘off-label’ mean?

“Off-label” use refers to when a medication is prescribed in a way that differs from its FDA-approved label. This could include:

• Treating a health condition that the medication isn’t approved for

• Using a different dose, schedule, or route of administration

• Prescribing a medication for a different age group or population (such as children instead of adults)

Off-label uses may eventually get approved by the FDA. But before that happens, the pharmaceutical company that makes a medication needs to invest time and money into doing the required research. This may not always happen, given the costs associated with approving every possible use of a medication.

Common reasons for off-label prescriptions

An off-label use may not be the first choice for treating a health condition. But it can be an important option — especially when other treatments haven’t worked or don’t exist. Here are a few common situations where off-label prescribing is frequently used.

Children

Off-label prescriptions are very common for children. That’s because most medications are initially studied and approved only for adults, leaving fewer options officially approved for children. Even so, many medications are still used safely and effectively in children when carefully prescribed.

Examples of off-label medication uses in children:

• Antibiotics

• Antipsychotic medications

• Antiseizure medications

• Blood thinners

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Cancer

Off-label prescribing is also common in cancer treatment and supportive care. Many cancer medications work against multiple types of cancer, even if they’re only approved for one type.

Cancer care also often involves combination therapy, where medications are used together to improve results. These combinations may not be approved when used together. But clinical experience and research often support their use.

Rare or hard-to-treat health conditions

Off-label prescribing is sometimes necessary for health conditions with limited or no approved treatments. In these cases, healthcare professionals may rely on smaller studies, case reports, or expert guidance when selecting a medication. Examples include:

• Fibromyalgia, where medications such as antidepressants and antiseizure medications may be used off-label

• Migraines, using blood pressure medications or antiseizure medications not specifically approved for that use

• Polycystic ovary syndrome (PCOS), often treated with metformin, which is only approved for Type 2 diabetes

• Post-traumatic stress disorder (PTSD), where medications such as prazosin (Minipress) and certain antidepressants are used off-label

What are the risks of off-label prescription use, and is it safe?

Off-label use is common and often helpful, but it doesn’t come without risks. When the FDA approves a medication, the agency reviews its safety, effectiveness, and instructions for use. For off-label uses, that same level of review hasn’t happened. This means that:

• The benefits and risks may not be fully known.

• There may be less evidence supporting the use.

• Side effects and interactions may differ from those with the approved uses.

In some cases, widespread off-label prescribing can also contribute to drug shortages. This is especially the case if a medication approved for a specific health condition is suddenly used for something else in large numbers, such as diabetes medications being used for weight loss. This can make it harder for people using the medication for its FDA-approved purpose to access it.

That said, many off-label uses are supported by good evidence and are widely accepted in clinical practice. The strength of this evidence can vary, though. It’s up to your healthcare professional to review the available data and determine whether the benefits outweigh the risks for your situation.

What are some popular medications with off-label uses?

Off-label prescriptions aren’t limited to rare conditions, cancer, or treatments for children. Many well-known medications are frequently used for other purposes.

What does the FDA say about off-label prescriptions?

The FDA approves medications for specific uses, but it doesn’t regulate how they’re prescribed in practice. This allows healthcare professionals to use their clinical judgment and prescribe medications off-label when they believe it’s the best choice for a specific person. But in general, off-label uses should be:

• Supported by scientific evidence

• Aligned with clinical guidelines or best practices

• Documented clearly in a person’s medical record

However, there are limits placed on how pharmaceutical companies promote medications. Drug manufacturers aren’t allowed to market or advertise off-label uses. Doing so can lead to serious legal and financial consequences. These restrictions help ensure that off-label prescribing decisions are made by healthcare professionals, not driven by marketing.

Does insurance pay for off-label use?

Insurance coverage for off-label prescriptions varies and can be complex. In general, you may have a harder time getting your plan to pay for a medication that hasn’t been FDA-approved to treat your health condition.

But off-label use may be necessary for some people when standard treatments aren’t effective or available. To manage off-label coverage, insurers typically use several tools, including:

• Prior authorization: Before covering an off-label prescription, insurers may require your prescriber to submit documentation explaining why the medication is being prescribed, including evidence that you’ve tried FDA-approved options first. This process helps the insurer determine if the off-label use is appropriate and medically necessary.

• Medically accepted indications: Insurance plans, including Medicare, may cover off-label uses if they’re supported by specific drug compendia. These are expert-reviewed reference guides that summarize evidence for both FDA-approved and off-label uses, ranking the strength of evidence for each use.

Coverage policies can vary between plans. So, it’s a good idea to check with your insurer about its specific requirements and whether your off-label prescription might be covered.

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Written by:

Alyssa Billingsley, PharmD

Alyssa Billingsley, PharmD, is the director of pharmacy content for GoodRx. She has over a decade of experience as a pharmacist and has worked in clinical, academic, and administrative roles.

Reviewed by:

Joshua Murdock, PharmD, BCBBS

Joshua Murdock, PharmD, BCBBS, is a licensed pharmacist in Arizona, Colorado, and Rhode Island. He has worked in the pharmacy industry for more than 10 years and currently serves as a pharmacy editor for GoodRx.

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