Off-label Drug Use
Off-label drug use happens when a doctor prescribes a medicine for a purpose, dosage, or method that isn’t included in its official FDA approval. When considering off-label drug use in cancer care, it’s important to understand the risks, benefits, and whether insurance will cover it.
What is off-label drug use?
Off-label drug use means using a medicine in a way that is not approved by the US Food and Drug Administration (FDA). It’s also called non-approved or unapproved use.
The FDA approves medicines only in specific doses and to treat specific health conditions. These are on-label uses. But doctors might find the medicine helpful in other ways that are not officially approved.
Use of a medicine is considered off-label if it is prescribed:
- For a different disease or medical condition than in the approved drug label
- In a different dose than in the approved drug label
- To be given in a different way (such as by a different route) than in the approved drug label
What is a drug label?
A drug label is an official document that the maker of a drug creates for each approved medicine. Despite the name, the “label” is not the sticker on the bottle. It’s a detailed report, also called the prescribing information or package insert (PI) that is reviewed and approved by the FDA. It’s written for health care professionals who prescribe or sell the medicine.
The label includes:
- General information: The drug name (brand and generic), ingredients, dosage form, strength, how it’s given, and the name of the manufacturer.
- Approved uses and instructions: The conditions it’s approved to treat (the indications for use), how to take or administer it, dosing instructions, and how it should be stored.
- Risks and warnings: Possible side effects, warnings, drug interactions, and information about who should or shouldn’t use it.
The drug label is different from the consumer medication information (CMI) that comes with prescriptions at the pharmacy. CMI has much of the same information but is written in a more understandable way for patients and caregivers.
Why is the drug label important?
In the United States, new medicines and treatments must be tested in clinical trials before they are approved by the FDA for use in the general public. These trials are done to show that the drug:
- Works to treat a certain medical condition
- Works the way it’s expected to
- Is safe when used as directed
Once the FDA reviews the trial results and determines that the medicine works and is safe, it approves both the drug and its label. The label can only include information supported by research and approved by the FDA.
The drug label is one of the most reliable and easy-to-access sources of information for health professionals. The more understandable CMI version is the best information available for caregivers and patients – you can ask your doctor for a copy. However, it does not include off-label uses.
Is off-label use legal?
The FDA doesn't regulate how approved medicines are used once they're on the market, so off-label use is legal in the United States. However, it’s not legal for pharmaceutical companies to advertise or promote their drugs for off-label uses. Off-label marketing is very different from off-label use.
Can doctors prescribe off label?
Yes. Doctors and certain other health care professionals who hold and maintain a license to write prescriptions can prescribe drugs for off-label uses. The FDA allows this and trusts that the prescriber is using their medical judgment when using medicines and treatments they believe will benefit a patient.
Because information on off-label uses and outcomes can be limited, people might face a higher risk of medication errors, side effects, and unwanted drug reactions.
It’s important that you and your doctor talk about the possible risks and benefits of off-label use.
Why are medicines used off label?
Off-label use allows doctors to also prescribe approved medicines for an unapproved use if they think a medicine can help their patient.
The decision to offer a medicine to a patient in an off-label way is made by doctors based on the clinical trials and other research that’s been done on the medicine. They look at risks and benefits for the patient and also take into account a patient’s specific situation and preferences.
Off-label use in people with cancer
Off-label drug use is common in cancer care. It may have benefit when:
- A medicine works well against several kinds of cancers, even though it’s only approved for 1 or a few
- The most effective treatment includes a combination of drugs not all approved for that specific cancer type
- Few approved medicines are available, especially for less common cancers or cancer-related complications
Examples of common off-label use in cancer care include:
- Using a chemotherapy medicine approved for one type of cancer to treat a different type of cancer that has few treatment options
- Trying a treatment approved for a different cancer or stage when all approved treatments haven’t worked
- Adjusting the dose of a medicine due to a person’s age, weight, body mass index, or other health problems they may have
- Prescribing a medicine in liquid form when someone has trouble swallowing capsules
- Using a tricyclic antidepressant, approved for depression, to help manage certain types of pain
Does insurance cover off-label drug use?
Insurance companies often consider off-label use to be “experimental” or “investigational.” This means they don’t always pay (reimburse) for medicines prescribed in off-label use.
However, the Cancer Treatment Act was passed in 1993 requiring insurance to cover medically appropriate cancer therapies. This law includes off-label uses if the treatment has been tested in careful research studies and written up in well-respected drug reference books or medical journals. In 2008, Medicare rules were changed to cover more off-label uses of cancer treatment drugs.
Still, health insurance coverage laws and regulations are complex. Pre-authorization may be needed for off-label use. If your doctor is thinking about off-label drug use, check your health plan’s coverage carefully.
Questions to ask about off-label use
When considering off-label drug use, it’s important to understand risks, benefits, and other treatment options that might be available.
Start by asking if all the medicines recommended for your cancer treatment are approved for the planned use. If any are not, you can find out more by asking:
- Is there research to support this off-label use to treat my type of cancer?
- Do you have experience with using this medicine off label?
- Is this off-label treatment likely to work better than an approved one?
- What are the risks and benefits of this off-label medicine or treatment?
- Will my health insurance cover off-label use of this medicine?
- If my treatment plan involves more than one type of medicine, and one of them is being used off label, will my health insurance cover it?
- Written by
- References
The American Cancer Society medical and editorial content team
Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as editors and translators with extensive experience in medical writing.
American Society of Clinical Oncology. Reimbursement for cancer treatment: Coverage of off-label drug indications. J Clin Oncol. 2006;24:3206-3208.
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National Cancer Institute. Off-Label Drug Use in Cancer Treatment. 2022. Accessed at https://www.cancer.gov/about-cancer/treatment/drugs/off-label on April 8, 2025.
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US Food and Drug Administration (FDA). Understanding Unapproved Use of Approved Drugs ‘Off Label’. 2018. Accessed at https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-unapproved-use-approved-drugs-label on April 8, 2025.
Van Norman GA. Off-label use vs off-label marketing of drugs: part 1: off-label use-patient harms and prescriber responsibilities. JACC Basic Transl Sci. 2023;8(2):224-233. doi: 10.1016/j.jacbts.2022.12.011.
Last Revised: April 25, 2025
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