Off-Label Indications

NOTE:
This article is only for educational purpose. Consult with your physician about using a medicine with its off-label indication first before do anything!

Off-label indications are the usage of medicine out of its own listed indications as approved by local regulatory. Other name for off-label indication is "additional indication". This indication is usually based on empirical experience of some key opinion leaders in an institution or region. Off-label indications can sometimes emerge as on-label indication, has it passed phase IV trial and has it been approved by local or international regulatory.

Legally, the risk or casualty caused by the usage of a medicine for an off-label indication cannot be protected ethically (including that, this use of medicine sometimes not covered by health insurance!). So practically, usage of a medicine with its off-label indications should be conducted more carefully. Ask your physician about safety of the off-label indications before ask them to treat you by that medicine.

Estimated Numbers of Prescriptions for On-Label and Off-Label Uses of Medications in Various Functional Classes, 2001 [Source: N Engl J Med 2008; 358: 1427-9]

Here we list several named off-label indications:

• Beta blockers, antihypertensive: have off label indications for migraines and anxiety (usually with propanolol).
• Bevacizumab, an anticancer (monoclonal antibody): has off-label indication as therapy for age-related macular degeneration.
• Bupropion, an antidepressant: has off-label indications as therapy of nicotine/smoking cessation and antiobesity (under investigation)
• Carbamazepine, an anticonvulsant for general epilepsy: has off-label indication as mood stabilizer. Several physicians also use this medicine for bipolar disorder.
• Dexamethasone and betamethasone, a corticosteroid: has off-label indication to enhance pulmonary maturation of the fetus (used in premature labour).
• Gabapentin, an anticonvulsant for partial epilepsy: has off-label indication for neuropathic pain and several diseases of muscles and neurons (restless leg syndrome, phantom limb pain, migraine prophylaxis, bipolar disorders, etc).
• Ketokonazole, a systemic antifungal: has off-label indications as aid therapy in Cushing syndrome and acute distress respiratory syndrome.
• Metformin, an oral antidiabetic: has off-label indications as antiobesity therapy, polycystic ovarian syndrome (PCOS).
• Misoprostol, an antiulcerant: has off-label indication as an aid to stop postpartum hemorrhage.
• Ondansetron, an antiemetic: has off-label indication for emesis in acute gastroenteritis and hyperemesis gravidarum, due to its FDA pregnancy safety index (category of B).
• Sertraline, an SSRI (antidepressant): has off-label indication for premature ejaculation in male.
• Sildenafil, an erectile dysfunction medicine: has off-label indication to enhance sexual desire in normal male.
• Topiramate, an anticonvulsant: has off label indications for migraines, bipolar disorder, depression, and neuropathic pain.

Have any comments about this issue? Your comments are welcome as usual.