Pharma groups to FDA: Stop that new off-label rule in its tracks

Cheered by victories in off-label marketing lawsuits, pharma companies were expecting some loosening of the regulatory shackles when the FDA rolled out a new rule on the subject.

But that rule, published in the Federal Register last month, sent drugmakers into a minor tizzy—and industry groups are demanding that the FDA set its new off-label regime aside.

Of course, the petition itself may end up moot, what with the regulatory rollback stance of the Trump Administration and the prospect of an FDA commissioner with a decidedly anti-rules outlook.

But in the meantime, business as usual. So, in a citizen petition filed earlier this month, and recently posted to the agency’s website, the industry groups PhRMA and BIO and the Medical Information Working Group called on the FDA to stay the new rule and instead use a standard proposed in September 2015.

Two issues here: One, the definitions of “intended use” for drugs and devices. The FDA changed that definition to include a “totality of the evidence” standard. Two, the fact that those particular words hadn’t appeared in public proposals before the rule was published means companies had no chance to object. This, after a two-day hearing on the subject in November.

Why not “totality of the evidence?” That seems like a reasonable phrase on the face of it, but the industry groups—MIWG is an organization formed by companies specifically to address off-label rules—envision an outpouring of lawsuits and a self-imposed gag on free speech.

Specifically, the petition said the definition will embolden whistleblowers to file False Claims Act suits because they’ll be able to use more “circumstantial evidence.” And it’ll hamper “free speech about legitimate scientific data” on off-label uses. Anyway, the standard itself came out of the clear blue, not out of case law or the Food, Drug and Cosmetic Act, the petition said.

The free speech issue is the more important one, the National Law Review argued. Drugmakers likely can defend themselves in whistleblower cases, the review recently concluded. 

And indeed, the Justice Department lately has been announcing more settlements in kickback investigations than off-label cases. That's perhaps by chance, because judges are growing more strict about the detailed evidence required. But perhaps it's because of a pile-up of off-label lawsuit victories/settlements for Allergan, Par Pharmaceuticals, Pacira Pharmaceuticals and others over the past several years.

The FDA itself settled with Pacira, for example, to allow it to tout its drug Exparel for patients undergoing types of surgery for which it was not expressly approved.

And let’s not forget the SCOTUS decision in U.S. v. Caronia in favor of a pharma rep who talked up unapproved uses for the drug he was detailing. Drugmakers may have felt less threatened in light of those cases, which in general favored off-label communications.

But the “chilling of communications” on scientific data could be a big problem for companies, the law review stated. What’s more, the petition stated that the new standard “raises serious constitutional concerns.”

“The chilling effect of such a standard is difficult to overstate,” the petition said. “For example, if a company engages in scientific exchange about off-label use, forecasts on- and off-label sales, and scales production to meet the combined demand, a prosecutor could decide that this evidence reflects an off-label intended use.” 

That said, the FDA already reopened comments on the free speech aspects of off-label marketing, with a new comment period starting January 19. And the consumer group Public Citizen maintained that the FDA didn’t overstep its bounds.

“We don’t think there’s anything unique there that’s inconsistent with the existing regulation or what was proposed in 2015,” Michael Carome, who heads up the organization’s health research group, told Bloomberg BNA.

Plus, the FDA’s latest rule may never truly come into effect in any case. There’s a new sheriff in town, after all—a.k.a. President Donald Trump—and he’s already signed an executive order to trim federal regulations, if only a nominal one.

Trump hasn’t yet appointed an FDA commissioner, but like as not, the appointee will be less rule-minded than previous chiefs. If Trump chooses a completely off-the-wall candidate like Peter Thiel associate Jim O’Neill, the off-label shackles could be unlocked completely.