Off-Label Prescribing Explained
FDA says the unapproved use of an FDA-approved drug is often called off-label use, and it can mean a different condition, a different way of giving the drug, or a different dose. FDA has not determined that a drug is safe and effective for an unapproved use. [1]
What Off-Label Prescribing Means
FDA says health care providers generally may prescribe an approved drug for an unapproved use when they judge that it is medically appropriate for their patient. Because FDA describes off-label use as the unapproved use of an approved drug, the term applies only when an FDA-approved version of that drug exists. [1]
What FDA Drug Approval Covers
Before a drug can be approved, FDA says a company must submit clinical data and other information and show that the drug is safe and effective for its intended uses. [1] FDA says "safe" does not mean a drug has no side effects; it means FDA has determined that the benefits of using the drug for a particular use outweigh the potential risks. [1] According to FDA, a drug's approved labeling lists the specific diseases and conditions the drug is approved to treat, how to use it for them, its risks, and what providers should discuss with patients before they take it. [1] A drug's FDA approval applies to its intended use; FDA's review does not cover other uses. [1][2]
The Three Kinds of Off-Label Use
One kind of off-label use is treating a condition the drug is not approved for, such as using a chemotherapy approved for one type of cancer to treat a different type of cancer. Another kind of off-label use is giving a drug in a different way, such as giving a drug approved as a capsule as an oral solution instead. A third kind of off-label use is a different dose, such as a provider telling a patient to take two tablets a day when the drug is approved at one tablet a day. [1]
| Kind | Example |
|---|---|
| Condition | A different condition, such as a different type of cancer |
| Form or route | A different way of giving it, such as an oral solution instead of a capsule |
| Dose | A different dose, such as two tablets a day instead of the approved one |
Sources: [1]
Why a Provider Might Prescribe a Drug Off-Label
FDA says one reason a provider might prescribe a drug off-label is that there may be no approved drug for the patient's disease or condition. FDA says another reason is that a patient may have tried all approved treatments without seeing any benefit. FDA has not determined that a drug is safe and effective for an unapproved use. [1]
Questions FDA Suggests Asking a Provider
FDA suggests asking a provider what the drug is approved for, whether other approved drugs or therapies exist for the condition, and what scientific studies support the unapproved use. FDA also suggests asking whether the drug is likely to work better than an approved treatment, what the potential benefits and risks are, whether health insurance will cover it, and whether there is a clinical trial of the drug for the condition that the patient could join. [1]
- 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?
Approved Use Compared With Off-Label Use
This table compares what happens under each status.
| Question | Approved use | Off-label use |
|---|---|---|
| Agency evaluation | FDA has carefully evaluated benefits and risks for that use | FDA has not determined safety and effectiveness for that use |
| Evidence | Supported by strong scientific data, per FDA | Ask the prescriber what studies support the use |
| Labeling | Approved labeling explains how to use the drug for that use | Not reported |
| Advertising by the developer | Ads must be truthful, with effectiveness, side-effect and prescribing information [3] | By law, the developer may not advertise unapproved uses |
Prescribing Off-Label Is Different From Advertising an Unapproved Use
By law, a drug developer may not advertise uses of its product that FDA has not approved. A company that wants to study an approved drug for a new use, a new dosage strength or a new form does so under an Investigational New Drug application. FDA says significant changes to an approved drug's formulation, labeling or dosage strength generally must be approved by FDA before the company can make them. [3]
Off-Label Use Is Different From Compounded or Unapproved Drugs
A compounded drug is a different category from off-label use: FDA says compounded drugs are not FDA-approved, while off-label use means using an FDA-approved drug for an unapproved use. [4]
FDA states: "Compounded drugs are not FDA-approved. This means that FDA does not verify the safety, effectiveness or quality of compounded drugs before they are marketed." [4]
Off-label use of an approved drug is also different from using an unapproved drug: FDA says unapproved prescription drugs have not been reviewed by FDA for safety, effectiveness or quality. [5]
What We Could Not Find
We found no FDA figure, among the sources reviewed for this page, for how often drugs are prescribed off-label. We found no FDA list, among the sources reviewed for this page, of which off-label uses are supported by good evidence; FDA suggests asking the prescriber what scientific studies support the use.
Related pages
- FDA Drug Approval Process Explained
- Compounded Medications Explained
- What Does FDA-Approved Mean?
- How We Grade Medical Evidence
References
- U.S. Food and Drug Administration. Understanding Unapproved Use of Approved Drugs 'Off Label'. fda.gov. FDA page content current as of 02/05/2018. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-unapproved-use-approved-drugs-label. Accessed 2026-09-28.
- U.S. Food and Drug Administration. Is It Really 'FDA Approved'? fda.gov. FDA page content current as of 05/04/2026. https://www.fda.gov/consumers/consumer-updates/it-really-fda-approved. Accessed 2026-09-28.
- U.S. Food and Drug Administration. Step 5: FDA Post-Market Drug Safety Monitoring. fda.gov. FDA page content current as of 01/04/2018. https://www.fda.gov/patients/drug-development-process/step-5-fda-post-market-drug-safety-monitoring. Accessed 2026-09-28.
- U.S. Food and Drug Administration. Compounding and the FDA: Questions and Answers. fda.gov. FDA page content current as of 09/16/2025. https://www.fda.gov/drugs/human-drug-compounding/compounding-and-fda-questions-and-answers. Accessed 2026-09-28.
- U.S. Food and Drug Administration. Unapproved Drugs. fda.gov. FDA page content current as of 05/09/2024. https://www.fda.gov/drugs/enforcement-activities-fda/unapproved-drugs. Accessed 2026-09-28.