After years of anticipation, the US Food and Drug Administration will hold a public, two-day meeting in November to review the extent to which so-called off-label information about medicines may be disseminated to physicians.
Off-label information is regulatory parlance for materials that describe unapproved uses of a drug. Doctors are, in fact, allowed to prescribe a medicine for an unapproved use, but drug makers have long chafed at restrictions on their ability to distribute such information — reprints of medical studies, for example — and have lobbied Congress and the FDA to loosen regulations.
Despite such efforts, the FDA has taken a firm stance toward the issue. A key concern is that public health could be jeopardized if a company were to distribute information about an unapproved use that had not been proven to be safe and effective, a standard for regulatory approval.
The issue became highly contentious, however, after a 2012 ruling by a federal appeals court that overturned the criminal conviction of a Jazz Pharmaceutical sales rep, who was prosecuted for encouraging doctors to prescribe a drug for unapproved uses. The court ruled his speech was protected, since the information was truthful and not misleading.
Since then, drug makers have argued that conveying certain types of information is protected by the First Amendment. The debate accelerated last year when Amarin filed a lawsuit arguing it had the right to off-label marketing, so long as the information provided to doctors is truthful and not misleading. A federal judge agreed with the company and the FDA recently reached a settlement with Amarin.
Throughout these events, the FDA indicated it would issue regulations on off-label marketing and hold a public meeting to review the myriad issues.
However, drug makers and their supporters have grown impatient and fear that various court rulings might become a de facto standard. Last winter, an independent review panel was floated as a way to address the issue. Last May, two lawmakers accused the US Department of Health and Human Services of delaying new rules and issued a draft bill that would allow companies to market products for unapproved uses.
The upcoming meeting, which will be held on Nov. 9 and 10 at FDA offices in Silver Spring, Md., is supposed to give the public a long-awaited chance to convey their opinions and debate the issue. In a notice published Wednesday in the Federal Register, the agency poses several potential questions for which it hopes to receive responses about the pros and cons of off-label marketing.
“FDA is interested in comment on the extent to which additional communications from firms about unapproved uses can provide access to information that is relevant, scientifically sound, responsibly presented, and provides as full an understanding as possible about the limitations of the available evidence, as well as comment on the extent to which health care professionals currently face impediments to accessing such information, whether from firms or from other sources,” the notice says.
Among the questions posed: What are the benefits for clinical decision-making, research, coverage, reimbursement, or other purposes if firms communicate to health care professionals, payers, researchers, and patients more information, including preliminary or inconclusive information, about unapproved uses of approved/cleared medical products? What are the drawbacks and risks? Are there safeguards or requirements that would effectively mitigate any drawbacks or risks?
Another question: How could communication of information about unapproved uses of approved medical products affect patient incentives to enroll in clinical trials?
And another: “To what extent do changes occurring in the health care system that give payers and formulary committees more influence on prescribing decisions (including denying, limiting, or endorsing coverage of unapproved uses of approved products) provide incentives for companies to generate the high-quality data needed to demonstrate safety and effectiveness for new uses?”
And a few more: “What criteria should the agency consider in determining whether a study or analysis that is the basis of a company’s communication is scientifically appropriate to support the presentations or conclusions in the communication?”
“What do health care professionals generally understand about the quality and utility of different kinds or levels of scientific evidence related to unapproved uses? Can the same information be misleading to some audiences of health professionals and not others?”
It’s not clear how long it will take the FDA to issue new regulations.