Off label drugs: Why you have to question your doctor

When your doctor prescribes a medication, you assume that the Food and Drug Administration has deemed it safe and effective for what ails you. That's not always the case. More than 20 percent of the drugs prescribed are for "off-label" use — that is, for conditions other than the ones for which they received FDA approval, according to a 2006 study in the Archives of Internal Medicine.

The range of drugs used off-label is far wider than thought, the federally funded study shows. They include medication for allergies, convulsions, heart conditions, indigestion, ulcers, and asthma. In 73 percent of the off-label cases, doctors had little or no scientific evidence to back up their choices.

Drug marketing plays a role, says Randall S. Stafford, M.D., Ph.D., of the Stanford Prevention Research Center in Stanford, Calif., and co-author of the study. "While direct promotion of off-label uses is illegal," he says, "there are several gray areas that provide the industry with an opportunity to increase the off-label use of their products." Over the past few years, some drugmakers have paid millions in fines for promoting off-label uses, said an official at the U.S. Department of Justice.

Physicians are not always aware that a particular use of a drug is not FDA-approved, especially if the off-label use has become commonplace, says Edward Langston, M.D., a spokesman for the American Medical Association. But he says doctors "need the flexibility to prescribe drugs off-label where it seems appropriate and there's peer-reviewed literature to support its use."

Certain off-label uses are appropriate and can even save you a lot of money. For example, some eye specialists use Genentech's cancer drug bevacizumab (Avastin), to inhibit the overgrowth of blood vessels in wet macular degeneration, an eye disorder, says R. Linsy Farris, M.D., M.P.H., a clinical ophthalmologist at the College of Physicians and Surgeons of Columbia University in New York. Published studies on its use, coupled with results in practice, have been so encouraging that Medicare frequently covers the cost despite the lack of formal FDA approval, he says.

In 2006, a similar Genentech drug, ranibizumab (Lucentis), was approved for macular degeneration. One injection costs $1,950, compared with $50 for the off-label drug, according to the April 3, 2007, Annals of Internal Medicine.

CR’s Take:
Ask your doctor whether your medication is FDA-approved for your condition. Or check at the FDA's Web site or our drug reviews. If it wasn't, ask why it was prescribed and if there's scientific support.

Comments

[Catherine M Greene MD]

Most drugs are used for both approved uses and so called off label uses. The indications (approved uses) are determined by what indications the drug manufactor feels the drug will easily be approved for and is frequently not the only clinical condition for which drug will be effective. Drug companies rarely go after further indications unless it will be advantages for them as far as competion in the market place. Further indications are extremely expensive as further complete clinical trials are necessary. Doctors became aware of the other uses for drugs through medical literature and their own trials of the medication when other medications have failed to help. An example from psychiatric drugs (my specialty) are some of the antiseizure drugs. Psychiatrists use them in Bipolar illness, in refractory depression, for PTSD, and migraines to name a few of Non-FDA approved uses. These drugs are well known through the psychiatric literature as being extremely helpful in these conditions. To sum it up - The approved indications for many drugs is a matter of economics not true clinical usefulness.

[Catherine M Greene MD]

The FDA approved indications for a drug are frequently not the only clinical uses of a drug. When a company is attempting to get a drug approved they go for the indication that the drug is most likely to be approved for. There may be and at least some drugs have other uses that the company does that try to get to get the indication for as further clinical trials are required and this is expensive. In general only if they feel that getting a further indication makes economic sense do they pursue this. Many drugs in my specialty (psychiatry) have multiple uses and we also use drugs from catagories other than psychotrophic. An example of this is several of the antiseizure drugs - they are used for Bipolar illness, refractory depression, migraines, and PTSD to name a few. These uses come out of clinical trials that are in our literature but are not necessarily known to a layperson or even doctors in other specialties. The off-label usesage of drugs is an extremely important adjunct to trating very serious and even life threating illness. To sum it up - Indications are in large part economics not true clinical usefulness.

[Bill Watson]

It is good to see a doctor comment on your reports. I think I would like go to a doctor that reads widely, not just the technical medical stuff but also publications like yours.