Dr. Goldberg is Director of Skin Laser & Surgery Specialists of New York and New Jersey, Director of Mohs Surgery and laser research, Mt. Sinai School of Medicine, and Adjunct Professor of Law, Fordham Law School.
In this month's Legal Eagle article, David J. Goldberg, MD, JD, examines physicians being sued for using injectables for off-label procedures, such as for oily skin.
Dr Face, a prominent cosmetic dermatologist, has been using neuromodulators recently to treat oily skin and enlarged pores. He knows that several botulinum toxins have been FDA approved for a variety of indications. He also understands that there are many other published potential adverse indications, including oily skin and enlarged pores. Recently he injected a woman’s cheeks and she experienced complications. She has sued Face for medical malpractice and alleged that he used the product for a non-FDA approved purpose. Should Face be concerned about this allegation?
The FDA is the federal agency that is responsible for regulating the safety, efficacy, and marketing and distribution of medical drugs including hyaluronic acid fillers and devices. It imposes rigorous standards for the approval and clearance of medical drugs and devices. However, it does permit physicians to exercise their authority in determining the most proper form of treatment for their patients. As stated in its information for patients, “From the FDA perspective, once the FDA approves a drug, health care providers generally may prescribe the drug for an unapproved use when they judge that it is medically appropriate for their patient.”1
Such treatments often involve the use of drugs or devices for uses other than those for which they received FDA approval. So-called “off-label” use of drugs in this manner does not require additional approval by the FDA. The use of drugs and devices for conditions that are not indicated in approvals is widespread among physicians and generally is not considered experimental or investigational.
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).2
A common misconception is that off-label use of drugs or devices is 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 administration states specifically that doctors are expected to use their “best knowledge and judgment” when practicing their specialties.
Off-label use by physicians gives patients access to a great number of drugs and devices that they would not have as treatment options if the FDA were to expand on its current regulatory authority.
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. They are involved in the practice of medicine on a daily basis and have person-to-person contact with the recipients of medications and devices that are being administered for off-label uses.
Physicians regularly consult colleagues, attend continuing education courses, and research scholarly journals, so they are best able to determine the safety and effectiveness of off-label drug and device use. The FDA is incapable of reviewing proposals for every potential use of a drug or device.
Face is not likely to lose the medical malpractice suit brought against him specifically because he used neuromodulators fillers for an off-label purpose.
1. Food and Drug Administration. Understanding Unapproved Use of Approved Drugs “Off Label”. May 2, 2018. Accessed April 15, 2021. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-unapproved-use-approved-drugs-label
2. Food and Drug Administration. The Food and Drug Administration Modernization Act of 1997. November 21, 1997. Accessed April 2, 2021. https://www.govinfo.gov/content/pkg/PLAW-105publ115/pdf/PLAW-105publ115.pdf