By Dr. Sk Sabir Rahaman, MBBS, MD (Pharmacology), DFM(Family Medicine), FCFM, CCEBDM, CCLSD
📝 What is Off-Label Use?
Off-label drug use means prescribing a medicine in a way not officially approved by regulatory authorities. This can include:
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Using it for a different disease/condition
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Prescribing to a different age group (e.g., children)
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Giving it at a different dose
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Administering by a different route
👉 Example: Propranolol (approved for hypertension) is often used off-label for performance anxiety.
⚖️ Important: Off-label ≠ Illegal. It is a medically accepted practice in many countries, but it must be backed by scientific evidence and sound clinical judgment.
🎯 Why is Off-Label Use Important?
Doctors turn to off-label use when:
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Standard treatments are ineffective, unavailable, or unsuitable.
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The disease is rare or has no approved drug options.
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Special groups (children, elderly, cancer patients) need alternatives.
Most common in:
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Oncology (cancer care)
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Paediatrics
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Psychiatry
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Rare/Orphan diseases
⚖️ Benefits vs. Risks
| Benefits | Risks |
|---|---|
| Offers treatment options in rare diseases | Potential for adverse effects |
| Expands therapeutic scope where evidence exists | Limited scientific data in some cases |
| Useful for urgent/unique populations | Possible medicolegal liability |
| May reduce costs | Risk of treatment failure or harm if misused |
🚨 Example of risk: The off-label “Fen-phen” (fenfluramine + phentermine) combo caused serious heart valve damage.
🌍 Regulatory Perspective
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USA (FDA): Legal if prescribed by a physician, but pharma companies cannot promote off-label use.
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EU (EMA): Encourages research, especially in pediatrics.
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India (DCGI): Technically illegal under Drugs & Cosmetics Act, though often practiced by doctors in special cases.
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Japan: Very liberal, allows approval with minimal evidence.
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France: Restricts promotion.
👉 In India, the Indian Medical Association (IMA) supports off-label use when justified, but laws are still evolving.
💡 Examples of Off-Label Use
A. Different Indication
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Bevacizumab – Cancer → Age-related macular degeneration
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Gabapentin – Epilepsy → Migraine, bipolar disorder
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Methotrexate – Cancer → Ectopic pregnancy
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Propranolol – Hypertension → Performance anxiety
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Sertraline – Depression → Premature ejaculation
B. Different Route
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Tobramycin inhalation in cystic fibrosis (originally IV)
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Oral N-acetylcysteine for renal protection (originally inhaled)
C. Different Age Group
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SSRIs in children <18 yrs
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Quetiapine in children <10 yrs
D. Different Dose
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High-dose Calcitriol in renal failure
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Melatonin beyond approved 2 mg tablets
🔄 Off-Label Use vs. Drug Repurposing
| Feature | Off-Label Use | Drug Repurposing |
|---|---|---|
| Basis | Clinical practice, experience | Formal research & trials |
| Regulation | Not officially approved | Gains official approval |
| Time/Cost | Quick, low cost | Long (3–8 yrs), expensive |
| Evidence | May be limited | Requires robust data |
| Example | Gabapentin for migraine | Sildenafil (Viagra): from angina → erectile dysfunction |
⚖️ Ethical & Legal Considerations
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Patient consent is crucial.
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Prescribers should ensure:
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Strong scientific rationale
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Peer-reviewed evidence
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No better approved alternative
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Pharma companies cannot promote off-label uses.
📍 India: Marketing off-label use is prohibited under the Drug and Magic Remedies Act.
✅ Conclusion
Off-label prescribing is a valuable clinical practice, especially in oncology, pediatrics, and rare diseases. While not without risks, it can provide life-saving options when standard therapies fall short.
👉 In India and globally, there is an urgent need for clearer legal frameworks, robust clinical evidence, and patient safeguards to ensure that off-label use remains ethical, effective, and safe.
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