Does My Insurance Cover Off-Label Prescriptions

Healthcare professionals work hard to find the best treatments for your health conditions. Sometimes, that means prescribing medications for uses they weren't approved for. This is referred to as "off-label" prescribing.

While taking medication for an off-label use might be the best option for treating your health condition, your insurance plan may not cover it since it's not an FDA-approved use. And if your plan does cover the medication, there are specific criteria that must be met.

What are off-label prescriptions?

An off-label prescription is when a medication is prescribed in a way that isn't FDA-approved. But why would a healthcare professional recommend a medication for off-label use?

When a new medication is created, it's tested for specific uses. Then, it's FDA-approved for these specific uses if it's considered safe and effective. However, the medication may also be effective for other health conditions for which it's not FDA-approved. If it's prescribed for these uses, it's considered "off-label."

You might be wondering why medications with off-label uses don't just become FDA-approved for these uses. To get the FDA to approve these off-label uses, medication manufacturers need to do additional clinical trials. This takes time and money. As a result, some older medications that work well for unapproved uses remain off-label. This is often the case, for example, for many cancer medications. Insurance companies then have to decide how to handle coverage for these off-label uses.

Examples of off-label prescription use include:

• Using a medication for a different health condition than the ones it's approved for: For example, a chemotherapy medication approved for one type of cancer might be used to treat a different type of cancer.

• Giving a medication in a different form: For instance, a medication may come as a tablet and oral solution. They may be approved for different health conditions but used interchangeably.

• Prescribing a different dosage than the approved amount: For example, if a medication dosage is approved as 1 tablet daily, a healthcare professional might tell you to take 2 tablets daily because they think a higher dose is best for you.

• Using a medication approved for adults in children: For instance, a blood pressure medication approved for adults might be prescribed to treat the same condition in a child.

Off-label prescribing is legal and more common than many people realize. Healthcare professionals may choose this option when they believe it's the best treatment. This is especially true if approved treatments haven't worked.

Does insurance cover off-label use?

It might. But some insurance plans have lists of specific off-label uses they won't cover. Whether insurance covers off-label medications depends on several factors, including:

• Insurance provider

• Policy exclusions

• Intended use

For example, Ozempic (semaglutide) and Mounjaro (tirzepatide) are popular glucagon-like peptide-1 (GLP-1) medications used to treat Type 2 diabetes, but they're often prescribed off-label for weight loss. While your insurance may cover these medications for Type 2 diabetes, it's unlikely it will cover them for weight management, since they're not FDA-approved for that purpose. However, other GLP-1 medications - such as Wegovy (semaglutide) and Zepbound (tirzepatide) - are FDA-approved for chronic weight management, so there's a higher chance your insurance may cover them.

How do insurance plans evaluate off-label prescriptions?

A health plan might review off-label use requests by considering a few factors, such as:

• If the medication is FDA-approved

• Whether it's being used for a serious or rare condition

• If standard treatments haven't worked

• Whether the medication is listed in an approved compendium (a database compiled by experts who review and rate different medications and their uses, including some off-label uses)

How to get insurance to cover off-label prescriptions

Generally, to get insurance coverage for off-label prescriptions, you'll need a prior authorization. This means your prescriber will submit a request to your insurance company. The request explains why the off-label medication is medically necessary for you. Part of this review may include making sure you've tried FDA-approved options first.

It's a good idea to check with your health insurer about its policies on off-label coverage. Ask about any requirements or restrictions it may have. Also, find out if it has a list of approved off-label uses. This can help you understand your coverage options before starting an off-label medication.

Will Medicare cover off-label prescriptions?

Medicare may cover off-label prescriptions, but it depends on several factors. First, the medication must be FDA-approved for at least one use. Additionally, the medication must be "medically accepted." This means the use should be listed or supported in an approved compendium. It must also be backed by medical research or accepted as standard medical practice.

In some cases, healthcare professionals can ask for approval for off-label use by showing evidence from clinical trials. This evidence can come from at least two large studies (called Phase III trials) that show that the medication is safe and effective for the new use. If these aren't available, two smaller studies (Phase II trials) might be considered.

Medicare also considers whether respected medical groups recommend the off-label use.

For cancer treatments, Medicare has specific guidelines for covering off-label uses. These medications may be covered if they're listed in certain approved sources. Medicare also looks at evidence from peer-reviewed medical journals when deciding whether to cover an off-label cancer treatment. However, if a use is identified as unsafe or ineffective by Medicare, the FDA, or an approved database, the prescription won't be covered.

Will Medicaid cover off-label prescriptions?

Medicaid generally covers prescription medications that the FDA has approved. But for off-label uses, Medicaid has specific rules. Medicaid programs are only required to cover off-label prescriptions if there's strong support for that use in certain recognized sources.

However, Medicaid is a state-specific program. This means states have some flexibility in managing their Medicaid medication coverage. They can require prior authorization for any covered outpatient medication. States can also choose not to cover a medication for off-label use if it's not considered medically accepted or if the state has excluded it from its formulary (list of covered medications).

When can healthcare professionals prescribe medication for off-label use?

Healthcare professionals can prescribe medications for off-label use when they think it's the best treatment option. They use their medical judgment and scientific evidence to make this decision. Off-label prescriptions may also be prescribed when new research suggests a different use for an existing medication.

Here are some instances when off-label prescribing is used:

• Children: Many medications aren't tested in children. Because of this, healthcare professionals may prescribe adult medications off-label for children.

• Cancer treatment: Healthcare professionals might use a medication approved for one type of cancer to treat a different type.

• Mental health conditions: Some medications originally approved for one mental health condition may be prescribed off-label for other conditions.

• Rare health conditions: There may not be any FDA-approved treatments for some rare health conditions. These disorders aren't common but can be serious or life-threatening. In these cases, healthcare professionals might use medications off-label to help people with these conditions.

Examples of common medications that are prescribed off-label

Many medications approved for conditions such as pain, seizures, diabetes, or depression are often prescribed off-label to treat different health issues. Here are some common examples:

What are the advantages and disadvantages of off-label use?

Off-label use can be helpful in many ways. It gives healthcare professionals more choices when approved treatments don't work. This is often useful for rare health conditions that don't have many approved treatments. Sometimes, using medications off-label can be more cost-effective, particularly when older, less costly medications work just as well as newer, pricier ones. It may also mean quicker access to effective treatments without waiting for the lengthy FDA approval processes.

But, off-label use also comes with risks. Medications used off-label haven't been fully tested for the new use, so we're less sure about their safety and effectiveness. Figuring out the right dosage of an off-label medication can also be tricky. Plus, there might be unexpected side effects.

As mentioned, insurance coverage can be an issue. Also, you might not always understand that a medication is being prescribed off-label and what that means. That's why it's important to have open discussions with your prescriber and ask questions such as:

• Is this medication being prescribed for an FDA-approved use or off-label?

• Why do you think this off-label use is the best option for my condition?

• What are the potential benefits and risks of using this medication off-label?

• Are there any FDA-approved alternatives we should consider first?

• Will my insurance cover this off-label prescription, and if not, what are my options?

The answers to these questions can help you better understand your treatment and make an informed decision.