Are physicians protected when prescribing drugs for off-label uses?

Key Points

Although all agree that the infection had nothing to do with the actual toxin per se, the patient finds out that no botulinum toxins are approved by the Food and Drug Administration (FDA) for this purpose.

The patient is angered over the fact that she has paid hundreds of dollars for a non-FDA-approved treatment. She files a complaint with the local state board of medical examiners. Dr. Nose contacts his medical malpractice carrier and is informed that his policy will not protect him against state medical board proceedings. Will Dr. Nose's actions be sanctioned by his state medical board?

It should be noted that the FDA does not require approval for all clinical purposes for which the drug may be used, only those intended uses for which the manufacturer sought and received FDA approval. Once a drug has been approved for a specific indication, physicians may, at their discretion, prescribe the drug for any use they deem medically safe and appropriate.

When a drug is prescribed for an indication, population, dose or method of administration that is not specifically included on the label, this is considered an off-label use. Physicians are permitted to prescribe off-label drug uses because "(n)either the FDA nor the federal government regulate the practice of medicine."¹

"New uses for drugs are often discovered, reported in medical journals and at medical meetings, and subsequently may be widely used by the medical profession ... When physicians go beyond the directions given in the package insert it does not mean they are acting illegally or unethically, and Congress does not intend to empower the FDA to interfere with medical practice by limiting the ability of physicians to prescribe according to their best judgment."²

This practice of off-label use is not only common, but it "is an accepted and necessary corollary of the FDA's mission to regulate in this area without directly interfering with the practice of medicine."³ A 2003 report on off-label use found that for the three leading drugs in each of the 15 leading drug classes, approximately 21 percent of prescriptions were for off-label use. Another recent study found that one in five prescriptions written in the United States - as many as 173 million each year - are for unapproved uses, and as many as one in seven drug prescriptions are written for reasons that have "little or no scientific support."⁴

In fact, the practice of off-label use is so widely accepted that even Medicare, Medicaid and private insurers are required to provide reimbursement for certain off-label uses. No law in any state specifically requires a doctor to inform a patient that a drug is being prescribed for off-label use.