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FDA, drug companies clash over off-label uses

Drug makers are complaining that the Food and Drug Administration is leaving them speechless.

In a closely watched battle, two small companies have petitioned to give doctors information about unapproved, or off-label, uses for their medicines. Last week, Pacira Pharmaceuticals Inc. sued the FDA for the right to promote a post-surgery pain drug for a wider range of operations than federal regulators allow. The other company, Amarin Corp., wants to promote a fish oil pill.

Typically, drug makers must prove to the FDA that their medicines work to treat a specific disease before they can market them for that purpose. The agency thus bars them from promoting drugs for unapproved uses, though doctors routinely prescribe treatments for such off-label purposes.


At issue is whether the government's drug watchdog is properly exercising its authority to rein in illegal promotions or is unfairly squelching First Amendment rights in the name of protecting public health.

There is a lot riding on this clash of opinions.

Beyond matters of constitutional law and governmental oversight, the pharmaceutical industry is clearly angling to boost prescription sales. Of course, this would fatten bottom lines, a good thing if you happen to be a shareholder.

More broadly, the outcome of this skirmish could help determine how doctors deliver patient care. There may be quicker access to drugs, but safety risks may emerge, along with increased spending for more expensive medicines.

"It's a very high-stakes policy debate," said Coleen Klasmeier, an attorney at Sidley & Austin who specializes in FDA regulatory issues. "This can have huge implications for patients."

The dispute over off-label marketing has been festering for a while. But it accelerated in 2012 after a federal appeals court overturned the criminal conviction of a sales representative who promoted a narcolepsy drug to treat insomnia and fibromyalgia, among other conditions, even though the FDA had not OK'd the drug for those uses.


The court ruled that his actions constituted protected speech, because the information conveyed to doctors was considered truthful and not misleading. This is now seen as the litmus test for determining if a drug maker has acted appropriately.

And so, those two small drug companies are trying to force the issue, and Amarin, which sued the FDA, has already won a significant victory. In August, a federal judge ruled that Amarin could promote the broad benefits of a fish oil pill designed to lower triglycerides, provided the company doesn't give doctors information that is false or misleading. Settlement talks are under way between Amarin and the FDA, and a progress report is supposed to be filed in court in late October.

Given that the FDA appears on the defensive, some health experts fear we are about to slide down a slippery slope toward less oversight.

"This case is a stalking horse for an entire movement that wants to weaken FDA capacity to regulate the promotion of drugs and open the door for companies to talk about other uses for their products," said Dr. Jerry Avorn, a professor of medicine at Harvard Medical School who tracks the pharmaceutical industry. "It's a very worrisome situation."

What happens from here, however, is uncertain.

For the moment, it seems unlikely that other drug companies will suddenly arm their sales reps with medical journal reprints demonstrating the benefits of off-label drug uses.


A key reason is that Amarin was in an unusual circumstance. The drug maker already had results of a solid clinical trial that successfully demonstrated an unapproved use. And Amarin was very willing to provide specific disclaimers about risks.

"I don't think the floodgates will open and companies will disseminate anything they want about off-label use," said David Gibbons of Hyman Phelps & McNamara, a law firm that specializes in FDA regulation. "They'll need to be sure they present a full picture of information, because FDA will be very keen to find any lapses in truthful and non-misleading information."

In effect, the Amarin case may have set a high bar for others to follow.

Then again, the judge suggested that drug makers might avoid legal trouble by sitting down with FDA officials to vet materials to be distributed to doctors. The FDA also acknowledged as much in a letter filed in court.

This might encourage companies to push the agency toward greater leniency — and even to push some boundaries of pharmaceutical promotion.

"I do think companies will become emboldened," said Richard Samp of the Washington Legal Foundation, which supports drug makers in their quest to press free-speech rights. "If they are absolutely convinced everything is truthful and FDA doesn't disagree, they will distribute materials FDA does not totally approve of."

Although the Amarin and Pacira cases are not resolved, the upshot is that FDA oversight is likely to diminish.

"There is the potential for a big sea change in how drugs are promoted," said Patti Zettler, a former associate chief counsel at the FDA and now an associate professor at the Georgia State University College of Law.


If companies can boost their drugs without the same degree of regulatory scrutiny, and if doctors in turn push more medicines on their patients, consumers might be left wondering what to believe and whom to trust.

With fewer restrictions on pharmaceutical marketing, the question is: Will patients be left speechless, too?

Ed Silverman can be reached at ed.silverman@statnews.com. Follow him on Twitter @Pharmalot. Follow STAT on Twitter @statnews.