PhRMA, BIO Outline Ideal Off-Label Promotion Regulations

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By Nathaniel Weixel

July 28 — Two of the drug industry's heavyweight trade groups want the FDA to re-evaluate how it regulates off-label communication between drugmakers and health-care professionals.

The Pharmaceutical Research and Manufacturers of America (PhRMA) and the Biotechnology Innovation Organization (BIO) July 27 released a set of principles aimed at changing and clarifying what information about drugmakers' products is allowed to be shared with payers and providers. The principles pertain primarily to data and information outside of FDA-approved labeling.

Biopharmaceutical companies have been pushing in recent years to loosen what they see as overly burdensome and unclear Food and Drug Administration regulations on the types of information about their products that they can discuss with doctors and other health professionals.

The principles “are intended to form the basis for defining new and clear regulatory standards governing responsible, truthful and non-misleading communications to inform health care professionals about the safe and effective use of medicines,” the organizations wrote. The principles pertain primarily to data and information outside of FDA-approved labeling.

According to the industry groups, when companies share off-label information, it should be held to high standards. The document centers around three core concepts:

  •  a commitment to science-based communication;
  •  a commitment to provide appropriate context about data; and
  •  a commitment to accurate representation of data.

Science-based information about alternative uses of their products can help better inform decision-making, the industry groups said.

First Amendment Protection

Under longstanding policy at the FDA, companies can be subject to criminal prosecution and civil liability if they promote their products for uses the FDA hasn’t specifically approved.

However, drugmakers have alleged the FDA's off-label regulations unconstitutionally prohibit them from making completely truthful and non-misleading statements to health-care providers about the unapproved uses of their products.

“FDA's regulatory regime is out of step, and inconsistent with the First Amendment,” Deborah Shelton, deputy general counsel for health care at BIO, told Bloomberg BNA July 27. “It's creating barriers to where we’re moving in 21st century medicine.”

Companies typically share information through FDA-approved labels, but continue to gather additional data and analyses “that can benefit patient care and enhance the efficiency of our health care system—including information on alternative uses beyond the approved indication or dosing,” the groups said. “Because this information can help health care professionals make informed decisions about the best treatments for their patients, companies should be able to communicate about such medically accepted alternative uses in a truthful and non-misleading manner.”

Shelton said the principles are meant to offer “perspective” on what a “modern FDA framework could look like.”

She said companies especially want to be able to share information about pipeline products that haven't been approved yet, so that insurers can make coverage decisions in advance based on sound scientific information.

No New Guidance

The FDA suffered multiple setbacks to its off-label promotion policy recently, most notably in cases involving the drug companies Amarin Pharma Inc. and Pacira Pharmaceuticals Inc.

In March, Amarin reached a settlement with the FDA in its lawsuit challenging the constitutionality of FDA off-label promotion regulations (47 HCDR, 3/10/16).

Despite these setbacks for the FDA, the agency hasn't released updated policies in the form of guidance or rules that would clarify its thinking on off-label uses.

The agency said it would issue revised guidance by the end of 2014, but industry is still waiting.

“Everyone has been looking for additional guidance on what manufacturers can say about their products. FDA has talked about it for years, and it's certainly on their radar. They can’t, nor have they, ignored the different court cases, which they’ve lost,” health-care attorney Linda Bentley told Bloomberg BNA.

However, Bentley doubted whether the agency would want to incorporate much, if any, of the joint BIO/PhRMA principles.

“If you look at all the different scenarios [outlined in the document], it’s a wish list. [The groups'] members would like specific guidance on specific issues, but I wouldn’t expect FDA to adopt this document” because it's very industry friendly, Bentley said July 28.

“Nothing's been blessed by the FDA. It might help FDA focus on certain real world situations” but it's not clear whether anyone at the agency will give it much attention,” Bentley said. Bentley is an attorney with Mintz, Levin, Cohn, Ferris, Glovsky and Popeo PC in Boston.

Shelton declined to say whether BIO has been in communication with the FDA about the off-label principles.

“We're consistently reaching out to all stakeholders. We've had some good, productive discussions,” Shelton said.

To contact the reporter on this story: Nathaniel Weixel in Washington at

To contact the editor responsible for this story: Brian Broderick at

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