I Used Botulinum Toxin for Migraines—Now I’ve Been Sued

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“Dr Head,” a prominent dermatologist, has 5 years of experience using botulinum toxin injections for the treatment of migraine headaches. He knows botulinum toxin injections are approved for the treatment of hyperhidrosis and hyperkinetic glabellar lines, but he is also very comfortable treating a variety of wrinkles and headaches with botulinum injections. Two years ago, Dr Head treated a headache sufferer who, 3 days after her injections and after progressive worsening of her headaches, was determined to have a ruptured cerebral aneurysm. She has since recovered and has sued Dr Head for medical malpractice. She alleges that he used the product for a non-FDA approved purpose. Should Dr Head be concerned about this allegation?

The Food and Drug Administration (FDA) is the federal agency that is responsible for regulating the safety, efficacy, and marketing and distribution of medical devices and drugs (botulinum toxins). Although the FDA imposes very rigorous standards for the approval (more appropriately termed clearance) of medical drugs and devices, it does permit physicians to exercise their authority in determining the most proper form of treatment for their patients. Such treatments often involve the use of drugs or devices for uses other than those for which they have been approved by the FDA.

Use of drugs in this manner does not require additional approval by the FDA. The FDA not only recognizes off-label use but—as has been suggested by some—also nearly encourages it. The use of drugs and devices for conditions that are not otherwise indicated is widespread among physicians and is generally not considered experimental or investigational. Off-label use of drugs is even a necessity in some medical specialties, especially in the areas of pediatrics and terminal illnesses. Off-label use is particularly necessary in these areas because the majority of medications that undergo review and approval by the FDA are tested on only adults, and because patients who are terminally ill have often exhausted the repertoire of available treatments.

Courts and the FDA face numerous challenges in balancing the interest in regulating the distribution and marketing of drugs and devices with the interest in allowing physicians to determine the best course of treatment for their patients. The FDA is prohibited from interfering with physician authority concerning off-label use of drugs or devices, as enumerated in §214 of the Food and Drug Administration Modernization Act of 1997 (FDAMA).¹

A common misconception is that off-label use of drugs or devices is somehow experimental or investigative. It is important to note that the focus of medical therapy, or the practice of medicine, is the well-being of the patient, whereas the focus of investigative research is to test a particular hypothesis. The use of drugs or devices for indications not specifically approved by the FDA does not generally constitute investigative research. Because the FDA expects that physicians will exercise discretion in their prescribing patterns, the FDA states very specifically that doctors are expected to use their “best knowledge and judgment” when practicing their specialties.

Patients, through off-label use, are given access to a great number of drugs and devices that they would not otherwise have access to if the FDA was to expand on its current regulatory authority. Recent studies estimate that anywhere from 20% to 50% of prescribed drugs are for off-label uses.²

It should also be noted that despite the common view among many physicians, doctors do not have an obligation to disclose the FDA regulatory status for a drug or device to their patients. Courts have noted that physicians are in the best position to determine treatments for their patients, as they are involved in the practice of medicine daily and have person-to-person contact with the recipients of medications and devices being administered for off-label uses. It is argued that physicians regularly consult colleagues, attend continuing education courses, and research scholarly journals, therefore they are best able to determine the safety and effectiveness of off-label drug and device use. The FDA itself is clearly incapable of reviewing proposals for every potential use of a drug or device.

A bigger issue may arise as to whether a dermatologist without the knowledge base of a neurologist should be giving botulinum toxin injections for headaches. Both Dr Head’s expert witness and the suing patient’s expert witness will argue both sides of this view. A jury will need to decide this issue. Dr Head may or may not lose the medical malpractice brought against him, but he is not likely to lose the case because he used botulinum toxins for an off-label purpose.

References

1. Food and Drug Administration Modernization Act of 1997 (FDAMA). FDA. Updated March 29, 2018. Accessed July 10, 2024. https://www.fda.gov/regulatory-information/selected-amendments-fdc-act/food-and-drug-administration-modernization-act-fdama-1997

2. Van Norman GA. Off-label use vs off-label marketing of drugs: part 1: off-label use-patient harms and prescriber responsibilities. JACC Basic Transl Sci. 2023;8(2):224-233. doi:10.1016/j.jacbts.2022.12.011

David J. Goldberg, MD, JD, is medical director of Skin Laser and Surgery Specialists of New York and New Jersey; director of cosmetic dermatology and clinical research at Schweiger Dermatology Group in New York, New York; and clinical professor of dermatology and past director of Mohs Surgery and Laser Research at the Icahn School of Medicine at Mount Sinai in New York, New York.