Definition
- Use of an FDA-approved drug for a:
- Disease/condition not specifically approved
- Population group not included in approval (eg, pediatrics, geriatrics)
- Dosage/formulation different from official labeling
Rationale for Off-Label Use
- No approved pharmacologic treatment exists for a condition.
- Failure of standard/approved therapies.
- Evidence from clinical studies or expert practice supports alternative use.
Example
- Tricyclic antidepressants (TCAs) → used off-label for neuropathic/chronic pain, though primarily approved for depression.
Summary Table
| Aspect | On-Label Use | Off-Label Use |
|---|---|---|
| FDA approval | Yes | No |
| Indication | Approved disease | Alternative disease/condition |
| Population group | Studied group | Unapproved group (eg, children) |
| Dosage/formulation | As labeled | Modified regimen |
Learning Objective
At the end of this section, students should be able to: Define off-label drug use, explain its rationale, and recognize clinical scenarios (eg, TCAs for neuropathic pain) where it is applied.