FDA Questions Need for Looser Off-Label Promotion Restrictions

Regulatory NewsRegulatory News | 14 November 2016 |  By 

Officials from the US Food and Drug Administration (FDA) last week questioned industry arguments for looser regulations on off-label promotion for drugs and devices at a two-day public hearing at FDA's White Oak campus.

Currently, FDA limits companies' ability to promote products to their approved indications, and restricts the types of data companies can share that steps outside of the products' approved labeling.


Earlier this year, FDA announced it was undergoing a review of these policies after recent legal setbacks (United States v. Caronia; United States v. Vascular Solutions, Inc.;Pacira Pharmaceuticals, Inc. v. FDA; and Amarin Pharma, Inc. v. FDA) andincreased pressure from industry.

Most recently, in March, FDA settled a lawsuit with drugmaker Amarin after the US District Court for the Southern District of New York found that off-label statements Amarin made to doctors about its drug Vascepa were "truthful and non-misleading."

More Flexibility?

In his opening remarks, FDA Commissioner Robert Califf acknowledged that there might be room for more flexibility regarding the information that companies can communicate off-label, though he cautioned that the issues that must be resolved in order to do so are "numerous and complex."

"The purpose of this review is to help ensure that our implementation of FDA's legal authorities best protects and promotes the public health and the wellbeing of patients," Califf said.

"I feel that much is at stake as we gather information over the next several days," Califf said, adding,"Relevant, truthful and non-misleading scientific or medical information regarding unapproved uses of approved medical products may help healthcare providers make better individual patient decisions in these instances."

Despite this acknowledgement, Califf said there is a fine line between helpful information, and information that could harm patients.

"Of course, not all communications about unapproved uses would help support public health or the specific health of individual patients. For example, communications that are not based on sound science, or otherwise mislead audiences about the benefits or risks of the product, or about the strength of the evidence that supports the use of the product can inappropriately influence prescribing or use decisions in a way that harms patients," Califf said.

Califf also expressed concern that allowing companies to promote their products for unapproved uses could disincentivize companies from doing the types of high quality studies required to support new indications.

"If firms are able to promote products for new uses without generating the kinds of data that are needed to make a robust assessment of the benefits and risks of new uses, and without applying for marketing authorization for new uses, the important public health interests that the FDA premarket review system advances would be compromised and patients could be harmed," Califf said.

Industry Arguments Meet FDA Skepticism

Representatives from three prominent industry groups, the Pharmaceutical Research and Manufacturers of America (PhRMA), the Biotechnology Innovation Organization (BIO) and the Advanced Medical Technology Association (AdvaMed), painted a different picture for off-label promotion.

Michael Labson, a partner at Covington and Burling, presented PhRMA's case, calling for safe harbors for communicating certain types of information outside a drug's approved labeling with healthcare professionals and payers.

This information, Labson said, should include predefined categories of communications and types of evidence such as so-called real world evidence and data from clinical trials that was not used in the product's marketing application.

Labson also said that companies should be able to share information with healthcare professionals about "medically accepted unapproved uses of FDA-approved medicines."

However, Rachel Sherman, FDA's deputy commissioner for medical product and tobacco, questioned this line of thinking. "What is a medically accepted unapproved use that should be communicated versus one that might not be?" she asked.

According to Labson, healthcare professionals currently lack ready access to some of this information.

"These groups require complete, current and reliable information about treatment options to inform coverage and prescribing decisions," he said, adding, "Biopharmaceutical manufacturers are uniquely situated to provide to these audiences robust and current information about the medicines they have researched and developed."

Sherman also questioned Labson about the availability of such data: "Can you give a better description of what the valid scientific information is that's not otherwise in the public domain? Do companies have valid scientific data they have not made available? Or is it just that you feel they should be the people transmitting [the data]?"

Later in the hearing, Califf questioned BIO Executive Vice President Cartier Esham over her similar testimony that companies are "uniquely positioned" to provide crucial information to healthcare decision makers, but limited by the current legal and regulatory frameworks.

Specifically, Califf asked whether allowing companies to share information about their products outside their approved labeling could lead to companies not publishing their data in peer-reviewed publications.

"Another pathway would be for the company to basically keep the information to itself and promote it without going through those steps of peer review, in a way that some people might regard as not being transparent," Califf said.


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