FDA Plans for Off-label Communication Draft Guidance, Meeting

May 11, 2015 – With off-label provisions in the 21st Century Cures legislation in the works and the courts bearing down on the FDA regarding First Amendment protection of industry’s truthful, non-misleading speech about off-label uses of approved products, it appears that a bridge between the agency’s long-held positions and industry’s desire to speak to physicians about their products may have its first brick.

At a recent industry conference, FDA Chief Counsel Elizabeth Dickinson stated that the agency planned to hold a public meeting in the near future to hear industry’s concerns regarding off-label communications, according to Reuters.

Indeed, after “a number of stakeholders have raised issues about the Agency’s policies regarding manufacturer communications with physicians as well as with other entities,” a Center for Drug Evaluation and Research (CDER) representative told the Coalition for Healthcare Communication that “FDA has publicly stated that it intends to soon issue new comprehensive guidance regarding manufacturer communication regarding unapproved uses of approved products.”

Although the off-label meeting has not been officially announced in the Federal Register, the CDER official stated that FDA’s objective “is to revise its policies to best protect and promote the public health.  Because of the wide range of views held by different stakeholders and the importance of the underlying public health issues, FDA plans to engage stakeholders by releasing its draft guidance for comment and by holding a public meeting.”

Jeffrey K. Francer, vice president and senior counsel, Pharmaceutical Research and Manufacturers of America (PhRMA), said that healthcare can deliver “amazing advances,” but remarked that “an important step in embracing these medical possibilities is updating the regulatory framework that governs communications between biopharmaceutical companies and healthcare professionals.”

Francer added that “the FDA should update its regulatory framework to enable biopharmaceutical companies to share scientifically accurate, non-misleading information with healthcare professionals who need it to improve patient care,” noting that these companies “have tremendous amounts of data,” some of which is derived from clinical research, observational studies, exposure registries and medical record research, and some of which comes from currently disallowed sources, “including meta-analyses, sub-population data, and information about medically-accepted unapproved uses and combination uses.”

He stated that “our healthcare system is best served by an open dialogue among all parties to ensure physicians have the most accurate and complete information to meet their patients’ needs.”

How willing the FDA is to get to this place and if so, how quickly regulatory change can be made both remain to be seen. “The FDA has said it’s going to hold a meeting on these issues, but in terms of the substance, that has yet to be defined,” said APCO Worldwide’s Wayne Pines. “The agency announced in April 2014 that they were going to be looking at the way promotion was regulated – now a year later they’ve said we are going to have a meeting. Things are not exactly moving at a rapid pace,” Pines said.

However, the 21st Century Cures draft legislation, which includes placeholder language for off-label communication changes, is shining a spotlight on this issue. Sandra C. Raymond, president and CEO of the Lupus Foundation of America, wrote in a July 2014 letter to Congress regarding the 21st Century Cures initiative that there are only four FDA-approved medications to treat lupus and just one of those is a drug specifically approved to treat the disease. As such, Raymond wrote, “it is critically important for all stakeholders to have the ability to freely share peer-reviewed information about the safety and efficacy of medications used to treat lupus.”

Further, recent activity in the courts may apply additional pressure on the agency to endorse meaningful off-label revisions in any new draft guidance. In U.S. v Caronia, the Second Circuit Court of Appeals stated in 2012 that the First Amendment protected truthful and non-misleading speech about off-label uses.

“While courts and plaintiffs bring pressure on FDA to reform its rules on off-label communication, the FDA itself seems more focused on promises, delays and discussion,” said Coalition for Healthcare Communication Executive Director John Kamp. “Unfortunately for FDA, if the agency doesn’t take decisive action soon, the courts will be forced to wade into medical policy waters that are better left to FDA.”

The most recent legal challenge to FDA’s off-label policy came in a case last Thursday, Amarin v. FDA. According to the pleading in the case, Amarin is making what is called an “as applied First Amendment challenge to FDA regulations that prohibit Amarin … from making completely truthful and non-misleading statements about its product to sophisticated healthcare professionals.” The case relates to the drug Vascepa to treat patients with persistently high triglycerides who are not sufficiently responsive to traditional statin drugs.

The profile of this case is significantly raised by Amarin counsel Floyd Abrams, a well-known and respected First Amendment scholar and litigator. “It’s important the FDA does not have the power to stifle speech,” Abrams said in the Wall Street Journal. “And FDA is effectively stifling speech, unless we get a firm ruling from a court, which is very important to ensure the public is well served.”