The Food and Drug Administration wants to allow drug companies to give doctors information about unapproved uses of prescription drugs, a controversial move that is already drawing objections from Capitol Hill.
Companies generally aren't allowed to advertise or market such "off-label" uses, though doctors can prescribe drugs for any condition. The FDA has proposed guidelines allowing manufacturers to give physicians reprints of medical journal articles about uses of drugs and medical devices that haven't won the agency's approval.
The regulator is stepping into a high-stakes business issue, because off-label uses of prescription drugs are a mainstay of the industry -- an estimated 21% of drug use overall, according to a 2006 analysis published in the Archives of Internal Medicine.
Drug makers are generally expected to welcome the proposal because it clarifies a blurry legal area. At least under certain conditions, it promises a haven for a controversial promotional practice. It also contains an argument from the agency that may help the companies in court cases about marketing practices: There are "important public policy reasons for allowing manufacturers to disseminate truthful and non-misleading medical journal articles" and reference materials about off-label uses.
Randall Lutter, an FDA deputy commissioner, said in a news release that the articles "can contribute to the practice of medicine and may even constitute a medically recognized standard of care."
The ability to get flattering data about off-label uses in front of doctors' eyes is important to the companies because of the business heft of such uses. For instance, an estimated 31% of psychiatric-drug prescriptions, including antidepressants, antianxiety and antipsychotic medications, are off-label, according to the 2006 analysis, and IMS Health says that group had 2006 U.S. sales of about $26 billion. The percentages were even higher for some other types of medication: An estimated 42% of asthma medicines were used off-label, according to the 2006 study -- a class that IMS estimates brought in $7.8 billion in U.S. sales that year.
Risperdal, a Johnson & Johnson medicine that is approved to treat schizophrenia, bipolar disorder and irritability associated with autism, was used off-label 66% of the time, according to the Archives study. In 2006, the drug and its long-acting form brought in $4.2 billion total.
The FDA's move already is raising objections from industry critics. Democratic Rep. Henry Waxman of California wrote to the agency urging it to hold off on issuing the guidelines, which he argued would create a "large loophole" in the laws against off-label promotion. "It's a conflict of interest for the company to be promoting sales when they haven't been able to establish that a drug is safe and effective through the rigorous FDA process," Mr. Waxman said.
Industry officials said that doctors and patients benefit when physicians get reliable scientific information. "Companies do want to provide information that's truthful and nonmisleading," said Jeffrey Francer, assistant general counsel for the Pharmaceutical Research and Manufacturers of America. Peer-reviewed medical journal articles "should be distinguished from materials that are promotional."
The FDA's new guidelines may also add fuel to a broader debate over medical journals' role in policing studies sponsored by drug companies. A growing spate of academic research focuses on evidence that published industry-sponsored trials are often more likely to produce positive results than studies paid for by others.
Catherine DeAngelis, editor in chief of JAMA, the Journal of the American Medical Association, said the guidelines would provide "a way to assure at least some oversight of the off-label use of these publications." Still, she said of the drug companies that provide the articles: "Of course they're doing it promotionally. Why else would they do it?"
The status of off-label communications is fuzzy partly because of the issue's complicated legal history. In the late 1990s, the FDA was sued on First Amendment grounds over efforts to limit drug makers' promotions, but the 2000 final decision in the case left the rules still vague. Meanwhile, a 1997 law that promised limited protections for drug makers that distributed medical-journal articles on off-label uses has lapsed, leaving the practice's status potentially more tenuous. Congress didn't renew the provision when it updated the law last year.
The Justice Department, jumping on a spate of whistleblower suits, has ramped up the stakes with a number of big legal settlements in cases where drug companies were accused of off-label marketing, among other violations. For instance, Pfizer Inc. settled a case about the epilepsy drug Neurontin in 2004 for $430 million.
Today, industry lawyers say that a number of drug companies continue to distribute journal articles about off-label uses -- legally, they argue. Under the new proposal, the companies would be able to distribute only peer-reviewed studies, rather than lesser publications such as letters to the editor, and debatable articles would have to be balanced by others espousing the opposite point of view. "There's tremendous variation" in company practices, says Jeffrey Wasserstein, a lawyer who represents industry clients. The guidelines would "probably restrict the more aggressive companies."