Panel examines pearls and pitfalls of this complex issue

“If you are going to use a product in an off-label manner, it is your responsibility to your patient to make sure your plan is satisfactorily supported by evidence of safety and potential efficacy,” said Daniel J. Berry, MD, during a 2009 Annual Meeting symposium on “Off-label device use: When clinical need outpaces regulatory approval.”

“Document, disclose, document, disclose,” added attorney Patrick Hurd, JD. The two were part of a panel discussion moderated by A. Seth Greenwald, DPhil Oxon, and Michael J. Yaszemski, MD, PhD.

Off-label use requires vigilance
Dr. Berry suggested that key questions must be examined when a physician is considering using a product off-label for a patient. These include the following:

• What are you allowed to do?
• What are you not allowed to do?
• How do you decide what to do for a specific patient?

“You are allowed to use products that are approved by the Food and Drug Administration (FDA) for one indication for a different indication. That is off-label use,” he explained.

But he also examined what physicians are prohibited from doing. Off-label use is different than using a non-FDA approved product. “Remember, it is not permissible to use a non-FDA approved product without further regulatory approval.

“Using any devices or off-label products is prohibited if your primary objective is to test a hypothesis or obtain general knowledge. Those situations require that you conduct a study under Human Subjects Protection, Institutional Review Board, or other oversight,” Dr. Berry said.

Off-label use can be justified, according to Dr. Berry, when convincing clinical data and research support off-label use and when those data are available ahead of regulatory approval. It can also be used when on-label treatment is a less satisfactory alternative for the patient.

It is also very important to get informed consent, which varies from state to state. “The less well-established the treatment that you are considering, the more critical it is to have a detailed discussion with your patient about the possibility of using a device or drug in an off-label manner,” said Dr. Berry.

Off-label use requires being informed, protected
Although the FDA does regulate the safety and efficacy of products, it does not regulate the practice of medicine. “Continue to practice medicine,” advised Mr. Hurd, “relying on your training, experience, and judgment.”

“Know your science. Make your decisions based on sound science. Talk about your results of off-label use. Share your experiences—scientific, political—in various forums. Put it in writing—even in a letter to the editor of a journal. You are protected by the First Amendment,” he said.

Mr. Hurd emphasized the critical nature of the January 2009 FDA Guidance for Industry, “Good reprint practices for distribution of medical journal articles and medical or scientific reference publications on unapproved new uses of approved drugs and approved or cleared medical devices.”

“Get this guidance document and read it,” he urged. “Make sure you understand every provision of it, especially when you interact with device representatives and suppliers or if you are asked about particular off-label uses or devices. Know what is said in the guidance.”

Mr. Hurd also offered the following cautions when using off-label devices. “If you are going to use a device off-label, document your use. Disclose to your patient that you are using a device off-label.

“Document that you disclosed your use of the off-label device to your patient. Document how the surgery and the follow-up visits went. Disclose the relationship you have with the device supplier as it related to the patient’s device.

“Create your own record so that if anyone questions what you did, you have a written record to protect yourself from ‘whistleblowers,’ someone from your office, or a medical device company,” he explained.

Other panelists included Tanisha Carino, PhD; Steven J. MacDonald, MD; Mark Melkerson, MS; Bernard F. Morrey, MD; K. Daniel Riew, MD; and Stuart L. Weinstein, MD.

Dr. Berry reported the following disclosures: DePuy, A Johnson & Johnson Company; Smith & Nephew; Stryker, and Zimmer.

Annie Hayashi is the senior science writer for AAOS Now. She can be reached at hayashi@aaos.org