Legal: Off-label drug use: FDA allows physicians to determine best course of treatment for patients

Dr. Head, a prominent dermatologist, has five years of experience using botulinum toxin injections for the treatment of migraine headaches. He knows that botulinum toxin injections are approved for the treatment of hyperhidrosis and hyperkinetic glabellar lines, but he also is very comfortable treating a variety of wrinkles and headaches with these injections.

Key Points

Two years ago, he treated a headache sufferer who, three days after her injections, after progressive worsening of her headaches, was determined to have suffered from a ruptured cerebral aneurism. She has since recovered and has sued Dr. Head for medical malpractice. She alleges that he used the product for a purpose not approved by the Food and Drug Administration (FDA). Should Dr. Head be concerned about this particular allegation?

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 the FDA has approved them.

Use of drugs in this manner does not require additional approval by the FDA, and "off-label" use is not only recognized by, but, as has been suggested by some, is nearly encouraged by the FDA. 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 adults only, and because terminally ill patients have often exhausted the repertoire of available treatments.

Balancing act

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 actually 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).

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 wellbeing 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 physician use, are given access to a great number of drugs and devices that they would not otherwise have access to if the FDA were to expand its current regulatory authority. Recent studies estimate that anywhere from 25 percent to 60 percent of prescribed drugs are for off-label uses.

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. Physicians, courts have noted, 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, it is argued, regularly consult colleagues, attend continuing education courses and research scholarly journals. They are, therefore, 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

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 suit brought against him. However, he is not likely to lose the case because he used botulinum toxin for an off-label purpose.

Dr. Goldberg is the director of SkinLaser & Surgery Specialists of New York and New Jersey; director of Mohs surgery and laser research, Mount Sinai School of Medicine; and adjunct professor of law, Fordham Law School.

Related Videos
© 2023 MJH Life Sciences

All rights reserved.