Off-Label Use Explained
What off-label use of an approved drug means in the United States, why prescribers do it, what is and is not known about safety and benefit, and questions to ask your prescriber.
Content last updated . This page has not been medically reviewed.
What "off-label" covers
The FDA gives three examples of an unapproved use of an approved drug. [1]| Type | FDA's example |
|---|---|
| A different disease or condition | A chemotherapy approved to treat one type of cancer, used for a different type of cancer [1] |
| A different way of giving it | A drug approved as a capsule, given instead as an oral solution [1] |
| A different dose | A drug approved at one tablet every day, with a prescriber telling the patient to take two [1] |
Off-label applies to a drug that is already approved for something. A product that has no approval for any use is a different case, called an investigational or unapproved product. [2] See Established vs Investigational Use.
Why prescribers sometimes do it
The FDA names two common reasons: there may be no approved drug for the person's condition, or the person may have tried all approved treatments without benefit. [1] Researchers also study approved products for new uses; a physician can file a research IND to study an approved product for a new indication or in a new patient population. [3]What you can and cannot assume
For the approved use, the FDA has carefully evaluated benefits and risks, the decision is supported by strong scientific data, and approved labeling tells health professionals how to use the drug safely and effectively. [1] For an off-label use none of that is automatic. The FDA's own advice is to ask what scientific studies support the use. [1]"Safe" in the FDA's sense never meant "no side effects." It means the FDA found the benefits of the drug for a particular use outweigh its risks. [1] For an unapproved use, the FDA has not made that benefit-risk judgment. [1]
Off-label is not the same as
- Compounded or look-alike products. A compounded version of a drug is not the same as the approved product. See Compounded vs Approved Products.
- Expanded access. That is a regulated route for an investigational product in serious illness. See Expanded Access ("Compassionate Use").
- A product sold for a purpose it has no approval or evidence for. See Unapproved Commercial Interventions.
- A claim that "a doctor offers it, so it is approved." See Claim Check: "A Doctor Offers It, Therefore It Is Approved".
Questions to ask your prescriber
The FDA suggests these, among others. [1]- What is the drug approved for?
- Are there other drugs or therapies approved for my condition?
- What scientific studies support using this drug for my condition?
- Is it likely to work better than an approved treatment?
- What are the potential benefits and risks?
- Will my health insurance cover it?
- Are there clinical trials of this use that I could enroll in?
Also read Approved Product vs Approved Indication and Talking With a Health Professional. Never change a prescribed medicine on your own.
Frequently asked questions
Is off-label use illegal?
From the FDA's perspective, once a drug is approved, health professionals generally may prescribe it for an unapproved use when they judge it medically appropriate. [1] This is not legal advice, and other countries differ.
Does off-label mean unsafe?
Not automatically. It means the FDA has not determined the drug is safe and effective for that use. [1] The evidence may be strong, thin or absent, so ask what studies exist.
Can a drug be off-label if it is not approved at all?
No. Off-label describes an approved drug used beyond its label. [1] A product with no approval is investigational or unapproved. [2]
What if a clinic says a treatment is "approved off-label"?
Ask for the exact product, the approved indication and any studies for the use being proposed. [1]
Where do clinical trials of off-label uses appear?
Registered studies are searchable at ClinicalTrials.gov, as the FDA notes for trial seekers. [4] A registration does not mean the use works. [5]
References
- U.S. Food and Drug Administration (FDA), Understanding Unapproved Use of Approved Drugs "Off Label" (page current as of 02/05/2018). Read 2026-10-22.
- U.S. FDA, Expanded Access: Information for Patients (page current as of 10/22/2026). Read 2026-10-22.
- U.S. FDA, Investigational New Drug (IND) Application. Read 2026-10-22.
- U.S. FDA, Clinical Trial Participation (page current as of 12/05/2024). Read 2026-10-22.
- U.S. FDA, Important Patient and Consumer Information About Regenerative Medicine Therapies (posted June 3, 2021; page current as of 04/08/2024). Read 2026-10-22.