Off-Label Drug Use Needs Strong Oversight

WEDNESDAY April 2, 2008 -- U.S. Food and Drug Administration draft guidelines for oversight of off-label drug use would allow pharmaceutical companies to market more drugs for unapproved uses, one expert contends.

The proposed guidelines are a step in the wrong direction, Dr. Randall Stafford, an associate professor of medicine at the Stanford Prevention Research Center, wrote in an editorial published in the April 3 issue of the New England Journal of Medicine.

While the FDA approves drugs for specific purposes, doctors can use drugs off-label to treat other medical conditions that aren't included in the FDA approval. This is common practice, but what's known about the use of a drug for one condition may not apply to other situations, said Stafford, a Stanford University School of Medicine researcher. The use of antipsychotics for dementia and the use of antidepressants in children are key examples, he noted.

Stafford doesn't believe the FDA should start telling physicians how to practice, however. He said that physician judgment in such cases is critical, and off-label use of drugs can be helpful in certain cases where patients don't respond to approved therapies.

"But in other cases, off-label prescribing has become first-line therapy even in the absence of strong evidence of benefits and safety. This is problematic," Stafford said.

Such cases point to the need for a better method of evaluating and regulating off-label drug use, Stafford added. Ideally, a drug company would have to conduct additional studies and apply to the FDA for supplemental approval for a new clinical use of drug.

Currently, drug makers can't overtly promote drugs for off-label use but can give doctors educational materials about off-label use of a drug, most often in the form of published journal articles. This practice is permitted only if the drug maker submits the articles to FDA for review and is seeking formal FDA approval for the new use. However, FDA enforcement of these rules is limited, Stafford said.

The new draft guidelines eliminate both these requirements and replace remaining polices with non-binding recommendations.

"The FDA may be conceding to drug manufacturers the responsibility for regulating their own off-label marketing practices. The agency may also believe that its limited resources can be put to better or more effective use in confronting other ongoing challenges. Nevertheless, I believe that the FDA must take an active role in fostering evidence-based practice, eliminating subversion of the approval process, and requiring a balanced and fair presentation of the scientific evidence," Stafford wrote in the editorial.

Public comments on the FDA's draft guidelines are being accepted through April 21.

Posted: April 2008


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