Off-Label Use: What It Means and What FDA Says | Dose Track
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Off-label use

Also called off label, unapproved use of an approved drug · Reviewed

Definition: Off-label use is using an FDA-approved drug in a way its label does not cover: for a different condition, in a different form or at a different dose. FDA says providers generally may prescribe this way when they judge it medically appropriate, but FDA has not found the drug safe and effective for that use.

What counts as off-label

FDA gives three kinds of example:

  • A different condition: a cancer drug approved for one type of cancer used for another.
  • A different form: a drug approved as a capsule given as an oral solution.
  • A different dose: a label that says one tablet a day, when the provider says two.

The approved uses are listed in section 1, Indications and usage, of the prescribing information.

Why it happens

FDA names two common reasons: there may be no approved drug for the condition, or a person may have tried the approved treatments without benefit. FDA suggests asking the provider about the evidence for the use, the possible benefits and risks, and whether insurance will cover it.

Why it matters for your log

Label numbers, such as side-effect rates and dosing steps, come from trials of the approved use. When a drug is used another way, those numbers are a starting point, not a promise. Off-label use is also different from products sold for research use only, which are not approved drugs at all.

Read more on Dose Track: Side effects from FDA labels · Side-by-side comparisons

General education drawn from labels and published studies, not personal medical advice. Your prescriber, pharmacist and the label that came with your product are the final word.
Prescribing informationPrescribing information is the FDA-approved label for a prescription drug, written mainly for health care… Research use only (RUO)Research use only (RUO) is a label phrase. In US rules it comes from labeling for in vitro diagnostic… Compounding pharmacyA compounding pharmacy makes a medicine from raw ingredients for a specific patient, rather than… Medication guideA Medication Guide is FDA-approved patient labeling that must be handed to the patient with certain…

Frequently asked questions

Is off-label prescribing legal?+
FDA says that once a drug is approved, health care providers generally may prescribe it for an unapproved use when they judge it medically appropriate.
Has FDA checked off-label uses for safety?+
No. FDA says it has not determined that the drug is safe and effective for the unapproved use.

Sources

  1. FDA: Understanding unapproved use of approved drugs "off label"
  2. 21 CFR 201.57: content and format of prescription drug labeling, Cornell LII

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