Has your healthcare provider ever talked to you about using an FDA-approved drug for an unapproved use (sometimes called an “off-label” use) to treat your disease or medical condition?

It is important to know that before a drug can be approved, a company must submit clinical data and other information to FDA for review. The company must show that the drug is safe and effective for its intended uses. “Safe” does not mean that the drug has no side effects. Instead, it means the FDA has determined the benefits of using the drug for a particular use outweigh the potential risks.

When you are prescribed a drug for its approved use, you can be sure:

• That FDA has conducted a careful evaluation of its benefits and risks for that use.

• The decision to use the drug is supported by strong scientific data.

• There is approved drug labeling for healthcare providers on how to use the drug safely and effectively for that use.

The approved drug labeling for healthcare providers gives key information about the drug that includes:

• The specific diseases and conditions that the drug is approved to treat.

• How to use the drug to treat those specific diseases and conditions.

• Information about the risks of the drug.

• Information that healthcare providers should discuss with patients before they take a drug.

Some drugs may also have labeling information for patients such as Medication Guides, Patient Package Inserts and Instructions for Use.

What are Medication Guides?

Medication Guides are paper handouts that come with many prescription medicines. The guides address issues that are specific to particular drugs and drug classes, and they contain FDA-approved information that can help patients avoid serious adverse events.

Why do some medicines have Medication Guides?

FDA requires that Medication Guides be issued with certain prescribed drugs and biological products when the Agency determines that:

• certain information is necessary to prevent serious adverse effects

• patient decision-making should be informed by information about a known serious side effect with a product, or

• patient adherence to directions for the use of a product are essential to its effectiveness.

Where can I find Medication Guides?

Medication Guides are found within drug labeling - the section is usually located at the end of the drug label although this is not always the case. Note that the links in the Medication Guides database go directly to the first page of the Medication Guide within the drug label. Please check the number of Medication Guide pages before printing.

Drugs@FDA and DailyMed databases also contain medication guides as part of drug labeling.

How often are Medication Guides updated?

Medication Guides are updated as they appear in new drug labeling. The newest labeling may contain a Medication Guide, but this is not necessarily reflective of the date of the label.

Drugs can be legally sold in the United States only after the Food and Drug Administration (also known as the FDA) has approved them. Drugs are approved after research shows they are safe and effective for a specific use.

Off-label drug use refers to the practice of prescribing a drug for a different purpose than what the FDA approved. This practice is called “off-label” because the drug is being used in a way not described on its package insert. This insert is known as its “label.”

The label describes details about the drug, such as

• what the drug is made of
• how it works in the body
• the research studies that led to its approval
• side effects it may cause

The FDA must make sure that a drug is safe and effective for a specific use. However, it does not control the decision doctors make about which drugs to use for their patients. This means that once the FDA approves a drug, doctors can prescribe it for any purpose they think makes sense for the patient.

Off-label uses may include using an approved drug

• for a different type of cancer than the one it is approved to treat
• at a different dose or frequency
• to treat a child when it is approved to treat adults

Off-label uses of a drug can become approved uses if the company that makes it obtains approval from the FDA. To gain the added approvals, the company must conduct research studies to show that the treatment is safe and effective for the new uses. However, a company may decide not to invest time and money in this research.

From the FDA perspective, once the FDA approves a drug, healthcare providers generally may prescribe the drug for an unapproved use when they judge that it is medically appropriate for their patient.

You may be asking yourself why your healthcare provider would want to prescribe a drug to treat a disease or medical condition that the drug is not approved for. One reason is that there might not be an approved drug to treat your disease or medical condition. Another is that you may have tried all approved treatments without seeing any benefits. In situations like these, you and your healthcare provider may talk about using an approved drug for an unapproved use to treat your disease or medical condition.

Unapproved use of an approved drug is often called “off-label” use. This term can mean that the drug is:

• Used for a disease or medical condition that it is not approved to treat, such as when a chemotherapy is approved to treat one type of cancer, but healthcare providers use it to treat a different type of cancer.

• Given in a different way, such as when a drug is approved as a capsule, but it is given instead in an oral solution.

• Given in a different dose, such as when a drug is approved at a dose of one tablet every day, but a patient is told by their healthcare provider to take two tablets every day.

If you and your healthcare provider decide to use an approved drug for an unapproved use to treat your disease or medical condition, remember that FDA has not determined that the drug is safe and effective for the unapproved use.

Research has shown that off-label use of drugs is very common in cancer treatment. Often, usual care for a specific type or stage of cancer includes the off-label use of one or more drugs.

Off-label drug use is common in cancer treatment because

• many cancer drugs are effective against more than one type of cancer

• cancer treatment often involves the use of combination chemotherapy

  Combination chemotherapy (which is treatment using more than one drug) is effective in treating many types of cancer. Examples of combination chemotherapy include:

  • R-CVP to treat non-Hodgkin lymphoma
  • CMF and TAC to treat breast cancer
  • BEACOPP to treat Hodgkin lymphoma
  • FOLFOX to treat colon cancer

  These combinations might include one or more drugs not approved for the type of cancer they are being used to treat.

  The FDA usually does not approve combinations of chemotherapy. There are so many of them that it would not be practical to approve each combination.

• Research studies find new uses for drugs that are already approved. The results of research studies are published in medical journals and shared in the medical community. Doctors then adopt the new use and it may become an accepted and widely-used treatment for a different cancer, even if the FDA has not approved the drug for that use.

There are times when off-label drug use may cause harm, such as when

• it has not been shown to be effective against a certain cancer
• there is no reason to believe the drug might be effective
• the possible risks of giving the drug outweigh the possible benefits

However, if your doctor prescribes a drug for an off-label use to treat your cancer, they are basing the decision on knowledge of and experience with the drug, as well as on research that shows it might be helpful for your stage and type of cancer.

Health insurance coverage of off-label drugs in cancer treatment

Medicare and many insurance companies pay for off-label drugs for cancer treatment, as long as the off-label uses are listed in an approved compendium. A compendium is a collection of drug summaries put together by experts who have reviewed data about the drug’s use in patients.

If your doctor prescribes an off-label drug for your treatment, check your plan to make sure the drug is covered. If coverage is denied, it may be helpful for the doctor to provide the insurance company with copies of documents that support the suggested off-label use.

Questions to ask your doctor about off-label drugs

Discussing these questions with your doctor can help you understand why your doctor might prescribe an off-label drug for you.

• Why do you think the off-label use of this drug will help the type of cancer that I have?
• Is the off-label drug likely to work better than an approved drug?
• What are the risks and benefits of treatment with this drug?
• Will my health insurance cover my treatment with this drug?
• If my treatment involves combination chemotherapy and one of the drugs is off-label, will my health insurance cover it?

If your healthcare provider is thinking about using an approved drug for an unapproved use, you may want to ask your healthcare provider questions like these:

• What is the drug approved for?

• Are there other drugs or therapies that are approved to treat my disease or medical condition?

• What scientific studies are available to support the use of this drug to treat my disease or medical condition?

• Is it likely that this drug will work better to treat my disease or medical condition than using an approved treatment?

• What are the potential benefits and risks of treating my disease or medical condition with this drug?

• Will my health insurance cover treatment of my disease or medical condition with this drug?

• Are there any clinical trials studying the use of this drug for my disease or medical condition that I could enroll in?