Published 8/1/2009
Mary Ann Porucznik

AAOS adopts ‘off-label’ statement

Supports use in “the best interest of patients,” advises counseling and reporting

The orthopaedic community faces many complex challenges in navigating and reconciling changing regulatory requirements and technological and scientific developments. One area of regulatory focus and ambiguity is the intersection of the practice of medicine and a physician’s legal use of products that may be unapproved for certain indications or “off-label” when in the best interest of the patient.

At its meeting on June 19, the AAOS Board of Directors adopted a position statement on the “Off-label Use of Medical Products” that affirms longstanding professional standards in the orthopaedic community. The statement also includes a summary of the U.S. Food and Drug Administration’s recognition of the practice of medicine and regulatory principles. In addition, the statement covers the appropriate dissemination of scientific and clinical information, and conflict of interest principles that are relevant to current legal, policy, and ethical interests in the off-label use of products.

The statement was developed as an educational tool by the Council on Research, Quality Assessment, and Technology, the office of government relations, and the office of general counsel, and is not a product of systematic review.

Defining “off-label” use
The statement defines “off-label” use for prescription drugs, biologics, and approved medical devices as “any use that is not specified in the labeling approved by the U.S. Food and Drug Administration (FDA). For cleared medical devices, ‘off-label’ means any use that is not included in the cleared ‘indications for use.’ Labeling is considered any written material which accompanies, supplements, or explains the product.”

Because “technological developments may rapidly outpace traditional educational opportunities for discussion and the regulatory review framework,” the statement recognizes that some uses of medical products may become standard of care before the use has been approved or cleared. The statement also notes that state laws regulate the practice of medicine and advises surgeons to adhere to all applicable state and federal laws and regulations.

In certain patient populations—such as oncology and pediatric patients—off-label therapies are used extensively and have been recognized as generally accepted within the physician community. The AAOS statement advises surgeons to “appropriately counsel patients about the benefits and risks of the proposed treatment, and alternative treatments that might be available. Surgeons are encouraged to report adverse events occurring with the off-label usage of medical products to manufacturers and/or appropriate federal authorities.”

Cautioning surgeons
The statement cautions surgeons that “certain interactions may create an actual or potential conflict of interest in advancing new scientific and clinical information to the orthopaedic surgeon community.” It encourages them to “avoid compromising situations that may call independent medical judgment into question” and references the AAOS Standards of Professionalism relating to Orthopaedist-Industry Conflicts of Interest.

Noting that “physicians and surgeons are not insulated from the law if they are engaging in sales and marketing activities on behalf of or in conjunction with manufacturers,” the statement advises that “orthopaedic surgeons with compensated arrangements with industry should undertake appropriate precautions and financial disclosures in continuing medical education, grand rounds, and other medical education and professional activities that may involve communications and interactions regarding the off-label use of products and avoid scenarios in which the surgeon is used in white coat marketing activities that are promotional in nature.”

“The best interest of the patient”
In conclusion, the statement affirms the AAOS belief that “surgeons may prescribe or administer any legally marketed product for an off-label use within the authorized practice of medicine in the exercise of appropriate medical judgment for the best interest of the patient.”

Surgeons who use a product “off-label,” however, “have the responsibility to be well informed about the product, to base its use on firm scientific rationale and on sound medical evidence, and to maintain awareness of the product’s use and effects.”

Finally, the statement makes the following three recommendations:

  • Surgeons should appropriately counsel patients about the benefits and risks of the proposed treatment, and alternative treatments that might be available.
  • In the case of an adverse event with an off-label use, surgeons can submit a report to the manufacturer and/or the FDA.
  • Orthopaedic surgeons should disclose all conflicts of interest to patients, institutions, and medical associations and adhere to all state and federal laws and regulations.

View the complete statement.

Mary Ann Porucznik is the managing editor of AAOS Now. She can be reached at porucznik@aaos.org