Legal Definitions - off-label use

Off-label use refers to the practice where a prescription medication or medical device is used in a way that has not been specifically approved by the Food and Drug Administration (FDA).

While the FDA rigorously evaluates drugs and devices for safety and effectiveness for particular conditions, dosages, and patient populations, healthcare providers sometimes prescribe or use these products for purposes or in manners not explicitly listed on the product's official labeling or approved indications. This practice is legal and often based on scientific evidence, clinical experience, or emerging research, but it means the specific use has not undergone the full FDA approval process.

• Example 1: Medication for a different condition.

  Scenario: A physician prescribes a medication that is FDA-approved to treat high blood pressure to a patient suffering from severe migraines, because studies have shown it can help prevent migraine attacks.

  Explanation: In this case, the medication's primary FDA approval is for hypertension (high blood pressure). Using it to prevent migraines is considered "off-label" because the FDA has not specifically reviewed and approved the drug for that particular neurological condition, even if medical literature supports its efficacy for migraines.

• Example 2: Medication for a different age group or dosage.

  Scenario: A pediatrician prescribes a common antidepressant, which is FDA-approved for adults with major depressive disorder, to a teenager experiencing severe anxiety, but at a lower dose than typically prescribed for adults.

  Explanation: The FDA's approval for this antidepressant might be limited to adult patients and specific dosage ranges for depression. Prescribing it to a teenager (a different age group) for anxiety (a different condition) and potentially at a modified dose constitutes off-label use, as these specific parameters have not been formally evaluated and sanctioned by the FDA.

• Example 3: Medical device for an unapproved procedure.

  Scenario: A surgeon uses a specialized surgical mesh, which is FDA-approved for repairing abdominal hernias, to reinforce tissue during a complex reconstructive breast surgery.

  Explanation: The surgical mesh has been cleared by the FDA for its safety and effectiveness in hernia repair. However, using it in reconstructive breast surgery falls under "off-label use" because the FDA has not specifically approved or cleared the device for that particular surgical application, meaning its performance and safety for breast reconstruction have not undergone the agency's formal review process.