An Off-Label Regualtory Temper Tantrum

  • by: Peter Pitts |
  • 01/23/2017
FDA memo rebuffs many suggestions for expanding off-label marketing
By ED SILVERMAN @Pharmalot

After weeks of anticipation, the FDA has issued a lengthy memo about the extent to which so-called off-label information about medicines may be disseminated to physicians, one of the most contentious issues to roil both the agency and the pharmaceutical industry. But rather than spell out possible solutions to this particularly thorny topic, the 63-page missive essentially summarized key points framing the long-running debate and carefully rebuffed many of the suggestions made by drug makers and others that support expanding pharmaceutical marketing. Meanwhile, the agency continues to seek comments while working to develop a final guidance.

Drug company “communications that are designed to cause the audience to reach safety or efficacy conclusions independent of, or not supported by, the available data are misleading, have the potential to harm patients, and lead to a waste of health care resources,” the agency declared.

The memo follows a public meeting held last November to explore off-label promotion, which refers to materials that describe unapproved uses of a drug. Doctors are allowed to prescribe a medicine for an unapproved use, but drug makers have battled restrictions on their ability to distribute such information — such as reprints of medical studies — and have lobbied Congress and the FDA to loosen regulations.

The FDA has, so far, avoided doing so.

The agency has regularly voiced concern that public health could be jeopardized if a drug maker distributes information about an unapproved use that has not been proven safe or effective. For its part, the pharmaceutical industry argues that its free speech rights are being restricted, but has won significant court rulings that say truthful and non-misleading speech is protected. These court battles have placed the agency on the defensive.

But in its memo, the FDA recounts why drug makers must provide evidence of safe and effective medicines, and why certain marketing restrictions exist. “The history of public health tragedies caused by medical products demonstrates that there have been some unscrupulous players in the marketplace who have made deceptive or unsubstantiated claims about medical products,” the memo states.

And the agency pointed to concerns that physicians are not always well positioned to discern the information they receive. “Studies have found that health care providers overestimate their knowledge of what uses are FDA-approved for drugs and assume that many unapproved uses are supported by sound scientific evidence when they are supported by uncertain or no evidence,” the agency wrote.

The FDA then reiterated its concerns about the aftermath of unapproved uses of drugs: “More recent studies have… found that the majority of unapproved uses for which drugs are prescribed lack adequate evidence of effectiveness, and that the risk of adverse events is higher for unapproved versus approved uses, and even higher when the unapproved use is not supported by reliable scientific data.”

To underscore the point, the FDA assembled two appendices replete with examples in which unapproved uses led to patient harm.

The agency also shot down several suggestions from the meeting, notably one that would permit drug companies to actively promote unapproved uses so long as disclosure is made. The FDA essentially rejected this out of hand by writing that patients may be harmed by a “return to an environment” where the public encounters claims based “conjecture or extrapolation from limited data, most of which is later found to be false or misleading.”

“They’re pulling out all the stops in pointing to the price to be paid from expanding off label use,” said Dr. Sid Wolfe of Public Citizen Health Research Group, who spoke at the meeting last November against industry efforts to widen off-label marketing.

At the same time, the FDA did acknowledge that there is virtue in sharing some off-label information.

“The reality remains at any point in time that, for some patients, approved or cleared therapies are not available or have failed,” the agency wrote, adding that reliable scientific information about unapproved uses may help further scientific research into new or existing medicines.

Nonetheless, industry supporters were miffed.

“This memo is a regulatory temper tantrum,” said Peter Pitts, a former FDA associate commissioner who heads the Center for Medicine in the Public Interest, a think tank that is funded, in part, by industry. “The memo is written to show that allowing the sharing of truthful, accurate, and non-misleading of information will cause the earth to stop spinning on its axis, research into new indications to cease, and misleading and misbranded communications to proliferate.”

Yet another observer, whose research was cited by the FDA throughout the report, praised the effort.

“It appears to be extremely well-researched and well-argued defense of the importance of the FDA in protecting patients and promoting accurate flow of information about approved medical products, among other outcomes, by maintaining its current oversight of off-label communications,” said Aaron Kesselheim, an associate professor of medicine at Harvard Medical School who also heads the regulation, therapeutics, and law program at Brigham & Women’s Hospital. “And it’s probably not a coincidence that it’s being published now, since the three names I’ve seen all be associated with a possible change in FDA leadership after Jan. 20 all have a very anti-regulatory ideology, and may look to change the FDA’s approach to regulating off-label marketing.”

Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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