Why off-label isn’t off base
Dear Dr. Mossman:
When I was a resident, attending physicians occasionally cited journal articles in their consultation notes to substantiate their treatment choices. Since then, I’ve done this at times when I’ve prescribed a drug off-label.
Recently, I mentioned this practice to a physician who is trained as a lawyer. He thought citing articles in a patient’s chart was a bad idea, because by doing so I was automatically making the referred-to article the “expert witness.” If a lawsuit occurred, I might be called upon to justify the article’s validity, statistical details, methodology, etc. My intent is to show that I have a detailed, well-thought-out justification for my treatment choice.
Am I placing myself at greater risk of incurring liability should a lawsuit occur?—Submitted by “Dr. W”
Dr. W wants to know how he can minimize malpractice risk when prescribing a medication off label and wonders if citing an article in a patient’s chart is a good or bad idea. In law school, attorneys-in-training learn to answer very general legal questions with, “It depends.” There’s little certainty about how to avoid successful malpractice litigation, because few if any strategies have been tested systematically. However, this article will explain—and hopefully help you avoid—the medicolegal pitfalls of off-label prescribing.
Off-label: ‘Accepted and necessary’
Off-label prescribing occurs when a physician prescribes a medication or uses a medical device outside the scope of FDA-approved labeling. Most commonly, off-label use involves prescribing a medication for something other than its FDA-approved indication—such as sildenafil for women with antidepressant-induced sexual dysfunction.1
Other examples are prescribing a drug:
- at an unapproved dose
- in an unapproved format, such as mixing capsule contents with applesauce
- outside the approved age group
- for longer than the approved interval
- at a different dose schedule, such as qhs instead of bid or tid.
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Typically, it takes years for a new drug to gain FDA approval and additional time for an already-approved drug to gain approval for a new indication. In the mean-time, clinicians treat their patients with available drugs prescribed off-label.
Off-label prescribing is legal. FDA approval means drugs may be sold and marketed in specific ways, but the FDA does not tell physicians how they can use approved drugs. As each edition of the Physicians’ Desk Reference explains, “Once a product has been approved for marketing, a physician may prescribe it for uses or in treatment regimens or patient populations that are not included in approved labeling.”2 Federal statutes state that FDA approval does not “limit or interfere with the authority of a health care practitioner to prescribe” approved drugs or devices “for any condition or disease.”3
Courts endorse off-label prescribing. As 1 appellate decision states, “Because the pace of medical discovery runs ahead of the FDA’s regulatory machinery, the off-label use of some drugs is frequently considered to be ‘state-of-the-art’ treatment.”4 The U.S. Supreme Court has concluded that off-label prescribing “is an accepted and necessary corollary of the FDA’s mission to regulate.”5
Limited testing for safety and effectiveness. Experiences such as “Fen-phen” for weight loss11 and estrogens for preventing vascular disease in postmenopausal women12 remind physicians that some untested treatments may do more harm than good.
Commercial influence. Pharmaceutical companies have used advisory boards, consultant meetings, and continuing medical education events to promote unproven off-label indications for drugs.13,14 Many studies ostensibly designed and proposed by researchers show evidence of “ghost authorship” by commercial concerns.15
Study bias. Even published, peer-reviewed, double-blind studies might not sufficiently support off-label prescribing practices, because sponsors of such studies can structure them or use statistical analyses to make results look favorable. Former editors of the British Medical Journal and the Lancet have acknowledged that their publications unwittingly served as “an extension of the marketing arm” or “laundering operations” for drug manufacturers.16,17 Even for FDA-approved indications, a selective, positive-result publication bias and non-reporting of negative results may make drugs seem more effective than the full range of studies would justify.18
Legal use of labeling. Though off-label prescribing is accepted medical practice, doctors “may be found negligent if their decision to use a drug off-label is sufficiently careless, imprudent, or unprofessional.”4 During a malpractice lawsuit, plaintiff’s counsel could try to use FDA-approved labeling or prescribing information to establish a presumptive standard of care. Such evidence usually is admissible if it is supported by expert testimony. It places the burden of proof on the defendant physician to show how an off-label use met the standard of care.19
