Off-label Promotion

FDA to Hold Post-election Hearing on Off-label Communication

Sept. 1, 2016 – In the wake of the agency’s recent settlement with Amarin and other rulings that support the “truthful and non-misleading” communication of unapproved uses, industry has been anxiously awaiting the FDA’s next move, especially after the agency stated in May 2015 that it would hold a hearing on this matter. The wait is over: The FDA announced in today’s Federal Register that it will hold a hearing Nov. 9-10 to gather input on the issues surrounding manufacturer communication of off-label uses for medical products that will inform FDA’s policy development on this topic.

“While it’s been a long time coming, we’re delighted the FDA is finally inching forward on these important issues,” said Coalition for Healthcare Communication Executive Director John Kamp. “We are hopeful the FDA will take the initiatives needed to avoid more First Amendment defeats in court.”

The Medical Information Working Group (MIWG), which for several years has urged the FDA to take prompt action to revise and clarify its policies regarding manufacturer communication of truthful, non-misleading information about marketed drugs and devices, has issued the following statement:

“More than two years ago, the agency granted two Citizen Petitions filed by the MIWG and agreed swiftly to address these issues. We hope and expect that the agency will not use the public meeting as an excuse for further delay but instead will move expeditiously to initiate Constitutionally-mandated and clinically-appropriate policy changes,” MIWG stated. “The issues are clear and the agency can act even without this hearing.  We believe that recent court cases have confirmed that there is value to the availability of truthful and non-misleading scientific information. We regard this as a fundamental public health issue as the availability of more reliable scientific information to both physicians and patients will lead to better health outcomes.”

FDA’s Position

The FDA maintains that when using a medical product for an approved use, patients and their healthcare providers “can be assured that the decision to use the product is supported by robust premarket review of scientific data and other appropriate scientific evidence by an independent scientific agency,” and that the benefits and risks of the use are described in FDA-approved labeling.

“These important assurances are absent for unapproved uses,” according to the agency, which appears to be making its case for its current position in the background section of the hearing notice, where it also states that the FDA is within the boundaries set by Congress to evaluate the safety and effectiveness of each intended use. “A drug with severe adverse effects may be considered safe and effective for treating metastatic lung cancer, but may be unlikely to have positive benefit risk balance for treating high blood pressure.”

The agency recognizes that healthcare professionals may prescribe or use medical products outside of their intended use, a practice which often is guided by scientific information about unapproved uses in scientific journals, clinical practice guidelines and compendia, or from companies upon request.

“FDA is interested in comment on the extent to which additional communications from firms about unapproved uses can provide access to information that is relevant, scientifically sound, responsibly presented, and provides as full an understanding as possible about the limitations of the available evidence,” the notice states. “FDA is interested in comment and information addressing whether and in what ways firms’ communications of unapproved use information are distinct and perhaps provide unique benefits compared to other sources.”

The agency also contends that “not all communications of information about unapproved uses help support public health” because they may focus on a product’s claimed benefits while minimizing a product’s known or potential adverse effects, and “may inappropriately influence prescribing or use decisions in a manner that is not in a patient’s best interest.” To that end, FDA is seeking comment on the “pros and cons” of “firms’ communications of unapproved use information and the kinds of limitations or requirements that would be appropriate to protect patients from harm.”

The FDA lists eight specific areas for which it seeks comment in the Federal Register notice (2016-21062). Several of these address what standards the agency should apply to minimize the potential for off-label communications to be misleading, one of the litmus tests that the courts have established.

To attend the hearing, registration is required by Oct. 19; electronic or written comments will be accepted after the hearing until Jan. 9, 2017. A link to a live Webcast of the hearing will be available at on the day of the hearing, and a video recording of the hearing will be available following the hearing at that same URL.