Pharmaceuticals Public Policy

Off label prescribing: Q&A

The Duke-Margolis Center for Health Policy has a great overview of some of the issues related to off-label prescribing.  Below is a summary of some key points from this article.

What is off-label prescribing?

Off-label prescribing and use can take many forms, such as use of an approved drug for an unapproved clinical indication, use at an unapproved dosage, use as first-line therapy in narrowly defined clinical populations where available treatment options are lacking (e.g., pediatrics or geriatrics), or use in areas where treatment options are limited or have been exhausted (e.g., oncology or rare diseases). Off-label prescribing often involves areas of high unmet medical need, and is increasingly utilized with the goal of individualizing patient treatment based on a broad array of sources of evidence.

Is off-label prescribing harmful to patients?

The answer is, it depends.  Because the level of evidence to support off-label uses is typically lower than on-label uses, the risk of using the treatments is often higher.  In many cases, however, off-label drug use has significant benefits for patients.

Can pharmaceutical firms communicate the benefits of off-label drug use?

In 1982, FDA published a one-page guidance to manufacturers explaining that materials provided to physicians in response to unsolicited [my italics] requests would not be regarded as labeling and therefore could lawfully include information about off-label uses…Another important “safe harbor” policy permitting manufacturers to provide off-label information arises under the “scientific exchange” regulation promulgated around the same time; it applies to communications such as manufacturer publication of original trial results and letters to the editor of scientific publications in defense of public challenges.

Additional guidance is available in the FDA “Good Reprint Practices” document.

What is FDAMA and how does it relate to off-label communication?

The agency’s current stance also has roots in the Food and Drug Administration Modernization Act (FDAMA) passed by Congress in 1997.9 Section 401 of FDAMA enabled the dissemination of peer-reviewed journal articles related to off-label indications with a confirmed commitment to filing a Supplemental New Drug Application (SNDA).

How does FDAMA affect communication of health economic results

Section 114 enabled the sharing of health care economic information with formulary committees, managed care organizations and other large consumers of health products.

What recent court cases have effected off-label communication?

  • United States v. Caronia (2012): Rejected a government effort to criminalize accurate speech about off-label uses.
  • Amarin Pharmaceuticals Inc. v. FDA (2015): Amarin Pharmaceuticals and physician plaintiffs filed a complaint alleging that the firm was being prevented, by FDA regulation, from communicating truthful and non-misleading information about the prescription drug Vascepa. The pharmaceutical company was granted preliminary relief by a district court, which gave the firm permission, based on Caronia, to communicate truthful and nonmisleading speech via specific, court-sanctioned promotional language
  • Pacira Pharmaceuticals Inc. v FDA (2015): Pacira sought to promote its post-surgery analgesic drug, Exparel, for uses that the FDA had alleged were unapproved, although some evidence suggested that the approved labeling could be interpreted to include these uses. The proceedings were settled in December 2015 after the FDA revised the approved labeling to make clear that the contested uses were, in fact, on-label.


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