Physicians have some leeway when prescribing drugs off-label

Key Points

Since then, he has been treating some patients with occasional great results. Unfortunately, one of his patients, who showed success, also had a toxin-induced lip drop for one month. The patient was furious and sought to sue Dr. Nose.

The patient-turned-plaintiff sought guidance from several attorneys. All refused to take the case because the economic damages of such a lawsuit were felt to be minimal at best.

No FDA recourse

It should be noted that Dr. Nose's former patient could not file a claim against him with the FDA for using botulinum toxins for a non-FDA approved purpose. The FDA does not have the authority to regulate the actual practice of medicine.

Practicing physicians are allowed to exercise their professional judgment and prescribe approved drugs for unapproved, or off-label, purposes. Physicians have been judicially accorded broad and unconstrained prescribing authority for years; "off-label" simply means that a drug is being used or prescribed in a manner inconsistent with its FDA-approved indication. Though it remains a surprise to many, the prescription of drugs for off-label use is commonplace, even in the absence of adequate supporting data.

Off-label statistics

At first blush, the concept of off-label drug use seems to undermine the central purpose of the FDA's approval process. However, off-label prescription is a critical tool for physicians. In fact, in 1995, the American Medical Association estimated that in the United States, 40 to 60 percent of prescriptions were for off-label uses. Furthermore, physicians rely heavily on off-label use when treating children, rare diseases and even some malignancies.

About 50 percent of cancer treatment drugs, 80 to 90 percent of drugs used to treat rare diseases and 80 percent of drugs used in pediatrics are prescribed for unapproved uses. Moreover, off-label uses may account for more than 25 percent of the approximately 1.6 billion prescriptions written each year.

Risks involved

Although off-label prescription is exceedingly common, it is not without risk. The primary argument against the practice is the lack of scientific justification for most off-label uses. Proponents argue that off-label drug use advances public health, while opponents fear off-label use presents an unacceptable risk to it. Each side of the debate can point to evidence to support their positions.

When fenfluramine (fen) was prescribed off-label in combination with phentermine, the FDA estimated that 285,000 fen-phen users suffered damage to heart valves during the brief period in which the combination was widely prescribed. Conversely, proponents point to the off-label use of aspirin, which has been proven to reduce the risk of heart attack and is credited with saving lives. Still, some critics argue that regardless of the result off-label prescriptions achieve, the current model is, in essence, experimentation on the public.

The debate is more a clash of perspectives than anything else, in that neither side is objectively wrong. Overall, the near-universal acceptance of off-label prescriptions tends to endorse the practice, and expanded use seems more likely than more restrictive use. In some cases, off-label use of a drug may actually represent the standard of care for treating a particular condition.

Ultimately, off-label prescription supplies what the FDA cannot: a timely solution. Off-label prescription compensates for the administrative slothfulness of the FDA approval process and enables immediate, if imperfect, treatment. If physicians were confined to the FDA-approved indication or severely restricted from deviation, the public would undoubtedly suffer.

The FDA does recognize the importance of off-label use and understands the importance of physician autonomy. Congress has exempted the practice of medicine from FDA oversight so as not to limit a physician's ability to treat patients. It is the State Board of Medical Examiners that provides oversight of physician practice. However, it is unlikely that any State Medical Board will sanction a physician for off-label use that appears to have some merit.

Dr. Nose is not likely to have any action taken against his medical license.

David Goldberg, M.D., J.D., is director of Skin Laser & Surgery Specialists of New York and New Jersey; director of laser research, Mount Sinai School of Medicine; and adjunct professor of law, Fordham Law School.

For more information:

O'Reilly J, Dalal A. Off-label or out of bounds? Prescriber and marketer liability for unapproved uses of FDA-approved drugs. Ann Health Law. 2003;12(2):295-298.