Off-Label Use

Off-label use means using an FDA-approved medicine in a way the FDA did not approve: for a different disease, a different patient group (such as children), a different dose, or a different route. It is common and legal for doctors. The FDA says that once it approves a drug, health care providers generally may prescribe it for an unapproved use when they judge it is medically appropriate for their patient. Sometimes no approved treatment exists, or approved treatments have failed.

The legal line runs through the manufacturer. Drug companies generally cannot market a drug for uses the FDA has not approved. When they do, it can lead to:

• Government fraud cases. If off-label promotion causes false claims to Medicare or Medicaid, the government, often with a whistleblower, can bring a False Claims Act case. Some of the largest health care fraud settlements in U.S. history involved off-label marketing.
• Injury lawsuits. Patients harmed by a drug used off label may claim the company pushed the unapproved use while downplaying risks.
• Consumer class actions by people or health plans who paid for a drug based on misleading marketing.

For patients, off-label use is not by itself a sign of wrongdoing. It matters in a lawsuit when there is evidence the manufacturer promoted the use, or when the doctor was not told about known risks. If you were prescribed a drug off label and suffered a serious side effect, keep your prescription records and any materials you received. See our pages on whistleblower and qui tam lawsuits, the Ozempic lawsuit, and what is MDL.