The Food and Drug Administration is considering dramatic changes to its drug marketing rules that threaten to make the problem of already out-of-control drug spending even worse.
The agency is responding to the drug industry's demands for greater freedom to promote the off-label use of prescription drugs and to the courts' drift toward giving corporations unlimited freedom-of-speech rights. “Off-label” refers to uses that have never received approval or scrutiny from the FDA's regulatory scientists. Doctors are allowed to prescribe off-label.
Nothing less than patients' health, safety and pocketbooks are at stake. An estimated 20% of drugs are prescribed off-label. Studies have shown nearly three-quarters of drugs prescribed off-label have little or no scientific backing for the use.
Among cancer drugs—the latest versions of which cost $150,000 a year or more—an estimated 50% to 75% are prescribed off-label, often to patients with metastatic disease who are desperate for anything that might give them hope.
This week, the agency is holding a two-day hearing to solicit public comment on how far the pharmaceutical industry should be allowed to go in promoting off-label drug use. It's largely prohibited now, although industry sales representatives can respond to physician requests for information about an off-label use by handing out reprints of studies that have appeared in peer-reviewed medical journals.
There's a long and sordid history of drug companies ignoring those restrictions. Over the past 15 years, major drug companies paid more than $15 billion in civil and criminal fines for promoting off-label uses. For example, GlaxoSmithKline in 2012 paid $3 billion for promoting the antidepressant Paxil to physicians treating adolescents, even though it had never been approved for pediatric use, and there was clear evidence the drug caused some troubled teens to consider suicide.
The FDA is undertaking its “comprehensive review” now because of a string of recent court decisions saying the First Amendment gives industry sales representatives free rein to promote unproven uses to physicians. The agency may be betting that by weakening its current rules, it will forestall review by a U.S. Supreme Court that is moving rapidly to giving corporations the rights of individuals.
But that's the wrong calculus for an agency that's supposed to ensure every drug is safe and efficacious. The FDA is well aware that many drug companies pursue a deliberate strategy of winning agency approval for drugs by conducting small but scientifically rigorous studies for a limited use. They then fund other studies—often observational without placebo control arms—for much broader uses, which many second-tier medical journals are more than happy to print.
That paradigm lies at the heart of two recent court cases. In 2012, a federal appeals court in United States v. Caronia overturned the conviction of a former Orphan Medical sales representative who had verbally touted Xyrem (sodium oxybate), which had only been approved for a very rare form of narcolepsy, for insomnia, fibromyalgia and Parkinson's disease.
Earlier this year, Amarin Pharma sued the FDA for the right to use “supportive but not conclusive research” that prescription omega-3 fish oil reduced the risk of coronary heart disease even though the FDA had already rejected that claim. After a federal district court sided with the company, the FDA turned around and let Amarin proactively make those claims and distribute the very studies it had found inadequate.
Organizations representing the payers and providers who are struggling with high drug costs need to make their voices heard at this week's hearing. Physicians have many ways of learning about potentially beneficial off-label uses of drugs: from clinical practice guidelines written by experts who've systematically reviewed the literature; by attending scientific meetings; and, yes, from the journals they decide to read.
What no one needs—except the drug industry—is another lane on the superhighway to unnecessary utilization and higher drug spending.