Doctors Going 'Off-Label' to Help Patients Patients may not realize it, but many of the prescriptions they get from their doctors are "off-label" -- that is, for uses not approved by the Food and Drug Administration. It's legal, and a new study shows it's an accepted part of medical practice: 21 percent of all prescriptions are for what are called off-label uses.
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Doctors Going 'Off-Label' to Help Patients

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Doctors Going 'Off-Label' to Help Patients

Doctors Going 'Off-Label' to Help Patients

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From NPR News, this is ALL THINGS CONSDIERED. I'm Melissa Block.


And I'm Robert Siegel.

A new study shows that it's very common for doctors to prescribe drugs off- label, that is for the treatment of diseases or conditions that have not been specifically approved by the Food and Drug Administration. The findings are being reported today in the archives of internal medicine.

NPR's Joanne Silberner has been looking into what they mean for doctors and patients.

JOANNE SILBERNER: Off-label use is common knowledge within the medical profession. What hasn't been known is how commonly it's used. Randall Stafford of Stanford University was one of three researchers who studied data collected from 3,500 physicians.

RANDALL STAFFORD: What we found is that in the United States, 21% of drug use was for indications where there's no FDA approval. That equates to approximately 150 million uses of these drugs in a single year.

SILBERNER: Stafford and his colleagues did have a surprise when they looked at how rarely those uses were backed up by consensus among physicians.

STAFFORD: Among the off-label uses, 73% of off-label uses were for indications where there's not strong scientific evidence.

SILBERNER: There may be some argument about that. Some doctors say the guidebook used for defining not strong evidence is too conservative. Others say it's too liberal. Medicine has always been part-art, part-science. And physicians have to make a lot of judgments with insufficient evidence.

Still, it's clear there's creative prescribing going on. Orthopedic surgeon Peter Lavine, speaking at his (unintelligible) in Bethesda, Maryland, says he uses drugs off-label when he has to.

PETER LAVINE: When you've tried the usual medications and you've gone through the list of what's approved and your patient still is not doing well, then, you know, you're sort of in a bind and you have to start looking for other options.

SILBERNER: Lavine treats a lot of dancers and performers. They often suffer pain from orthopedic injuries and where he hasn't been able to help them with old drugs, he's tried a new drug called Lyrica that came on the market a year and a half ago. It's approved only for seizures and special types of nerve pain caused by diabetes and infection. He's within his legal rights as a physician to prescribe Lyrica as he sees fit. He's photocopied pages from the standard drug reference found in almost every doctor's office and hospital and reads from it.

LAVINE: Once the product has been approved for market, a physician may chose to prescribe for uses in treatment regiments or patient populations that are not included in the approved labeling.

SILBERNER: Lavine says doctors need to be very careful. Take Vioxx, he points out. It was approved for use in people with chronic pain who were at risk of stomach problems from other medications. But doctors prescribed broadly for people with milder pain for whom the risk, it turned out, was not worth the benefit.

LAVINE: I think it's a mistake to immediately jump to an off-label use because you've had good success with it. I still think you should probably start with the original things that are known to work and have been proven to work and just sort of go down that algorithm and save the off-label use for those refractory cases where you're not making progress.

SILBERNER: Drug companies got into a lot of trouble a few years ago for sending salespeople into doctors' offices to push off-label uses and the FDA prohibits it now. But doctors still learn about off-label uses from other doctors working for drug makers. Lavine says he heard about Lyrica at a lecture sponsored by manufacturer Pfizer. But the local pain physician who gave the presentation was careful to say it hadn't been proven for generalized pain.

The FDA is well aware of off-label prescribing. Dr. John Jenkins heads the agency's office of new drugs. He says the agency sometimes contacts manufacturers when it hears of common off-label uses.

JOHN JENKINS: We're willing discuss with them ways to make it as least burdensome as possible for them to pull together the information and submit it for our review.

SILBERNER: But that research can take years to complete and Jenkins says it's hard to interest drug companies in testing new uses for medicines that have lost their patent. There's no payback for generic drugs. He says consumers can become their own investigators.

JENKINS: If they have concerns about being prescribed a drug for any use, whether it's approved by FDA or not approved by FDA, they should discuss that further with their physician and, you know, be educated consumers.

SILBERNER: As for Lyrica, the drug that orthopedic surgeon Peter Lavine prescribes off-label for some of his patients in pain, a company spokeswoman says Phizer is considering doing studies to broaden its approved uses.

Joanne Silberner, NPR News.

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