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Are Doctors Careful Enough With Off-Label Prescriptions?

Too often, doctors aren't using sound science when they prescribe a drug for unapproved uses, a new study finds.
(Photo: NVinacco/Flickr)

(Photo: NVinacco/Flickr)

When your doctor writes you a prescription to take to the pharmacy, you know it is for a drug that's been studied and approved by the Food and Drug Administration. But did you know that the prescribed drug might not be approved to treat your particular illness?

Officials at the FDA only approve a prescription drug after looking at studies on the drug's effectiveness (and level of safety) in treating specific illnesses. But once the drug is approved, doctors can legally prescribe it for any illness, not just the ones the FDA studied. Prescribing an approved drug for a use besides FDA-studied ones is called off-label prescribing, and there are good reasons to do it. Maybe standard treatment isn't working, or there's new data suggesting an off-label's effectiveness in treating illnesses aside from those given the FDA stamp of approval.

Doctors prescribing off-label drugs use strong science less than 20 percent of the time.

Still, some experts question whether doctors do too much off-label prescribing, and question the risk factor of such practices. A 2003 investigation by McClatchy's found examples of people suffering from heart attacks, strokes, and permanent nerve damage after taking drugs their doctors prescribed off-label. But it's always been difficult to get exact numbers on off-label prescriptions, or to show, in a quantitative way, whether they're good or bad for patients. Now, a new study offers exactly those numbers—and the results don't look good.

The study found that off-label prescriptions are as safe as on-label ones—but only when doctors base their off-label prescriptions on sound scientific evidence. Otherwise, patients are more likely to suffer ill side effects than patients who are taking drugs for approved uses, according to the study (which was conducted in Canada, so it looked at whether drugs were approved by Health Canada). Unfortunately, doctors prescribing off-label drugs use strong science less than 20 percent of the time. Overall, about 12 percent of new prescriptions are off-label, the study found.

The results come from an analysis of data of more than 46,000 adult patients in Quebec's electronic health records system. In an essay accompanying the study in the journal JAMA Internal Medicine, two University of Pittsburgh researchers who weren't part of the study call this finding "the most extensive and informative study to evaluate the safety of off-label drug use in an adult population to date."

The study scientists, a team of epidemiologists from McGill University, suggest a couple of policy fixes to reduce the risk of off-label prescriptions. "Physicians and physician organizations should recognize the enormity of the problem and be active participants in the promotion of cautious prescribing of drugs for off-label uses lacking strong scientific evidence," they write. In addition, they suggest electronic health records systems be designed to gather data about drugs' safety after they're Health Canada—or FDA—approved. The infrastructure is already set up for us to find the data we've long sought. We should use it.


Quick Studies is an award-winning series that sheds light on new research and discoveries that change the way we look at the world.