Off-label use of medications and other products has been an accepted practice for many years. For example, Botox was first approved in December 1989 by the Food and Drug Administration (FDA) to treat blepharospasm and strabismus and in December 2000 to treat cervical dystonia. On April 15, 2002, the FDA approved Botox to treat frown lines; however, this has been a widely accepted off-label use for cosmetic purposes for almost 10 years. The FDA defines “off-label use” as the use of a medication or product for an indication, dosage amount or regimen, patient population, or other considered use not mentioned in the approved labeling.1
The FDA is empowered by law to review drug products for safety and effectiveness. FDA approval has been required since 1938, with the additional requirement in 1962 that new drugs be reviewed for both efficacy and safety. It was not until 1976 that the FDA had the authority to regulate medical devices.2
Once approved, a drug product may be prescribed by a licensed physician for any use that, based upon the physician’s professional opinion, is deemed to be appropriate. The standard is what other similarly trained physicians would do—not what the manufacturer of the medication or product recommends. Although the FDA recognizes that this action by a physician is considered to be a part of the practice of medicine, it does not regulate, comment on, or recommend any course of treatment for an individual.
The FDA recognizes that off-label use of drugs by prescribers is often appropriate and may represent the standard of practice. In today’s medicine, many pediatric uses are not on a label. The reason for this off-label practice is the issue of drug or product testing experimentation in children. Many standard treatments in oncology and HIV therapy involve off-label use of medications. There are no rigorous data available on the extent or profile of off-label uses.3
Prior to 1997, manufacturers were prohibited from discussing off-label uses with providers and could not distribute written materials that mention an off-label use or uses. With the Food and Drug Modernization Act of 1997, a manufacturer “may disseminate to a health care practitioner...written information concerning the safety, effectiveness, or benefit of a use not described in the approved labeling of a drug or device if the manufacturer meets...” certain requirements.4 Further clarification by the FDA was published in 2009 and revisited again in 2014.5
Physicians may also obtain information on an off-label use from the pharmaceutical company. Additionally, the FDA does not restrict other parties from discussing off-label uses or distributing written materials concerning them. Manufacturers may also disseminate off-label use information to a pharmacy benefit manager, health insurance issuer, group health plan, or federal and state government agency. Drug companies may not distribute or market off-label use information to patients or the general public.
The patient safety implications for physicians in prescribing a medication or using a medical device in an off-label manner include the following:
Off-label use has proven to be very beneficial in the medical community and when approached in a manner consistent with patient safety, can provide avenues of treatment not otherwise available.
The guidelines suggested here are not rules, do not constitute legal advice, and do not ensure a successful outcome. The ultimate decision regarding the appropriateness of any treatment must be made by each healthcare provider in light of all circumstances prevailing in the individual situation and in accordance with the laws of the jurisdiction in which the care is rendered.