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Off-Label Drug Use: What You Need to Know

Prescription drugs are often prescribed for uses other than what the FDA has approved. Find out why.

An Issue for Doctors

Some doctors may also be confused about a drug's approval status. A surprising number of doctors think a medicine is FDA-approved for something when it is not, Alexander and colleagues recently reported in Pharmacoepidemiology and Drug Safety.

"This is not a trivial issue," Alexander says. "Our findings suggest that some off-label prescribing might be driven by mistaken beliefs about FDA approval and the level of evidence supporting off-label drug use."

Alexander says better strategies and tools are urgently needed to inform doctors about common, off-label uses that pose the greatest harm. Misunderstandings about a drug's approved use or side effects can increase the patient's risk of medication errors and dangerous outcomes.

For example, Alexander's research showed that nearly one in five doctors questioned who prescribed the drug quetiapine (Seroquel) for dementia with agitation mistakenly believed it was approved for such use. But the medicine carries a "black box" warning -- the FDA's sternest warning -- stating that the use of antipsychotic drugs was associated with an increased death risk in elderly patients with dementia.

Psychiatric medicines are among the most common drugs to be prescribed off-label, and their use in children is of special concern. Many drugs prescribed to children are used off-label because medications are less commonly tested in this age group. In March 2009, researchers reported in Academic Pediatrics that 62% of outpatient pediatric visits resulted in an off-label prescription. Children under age 6 were most likely to be prescribed a drug off-label.

Who Is in Control?

Off-label drug use can sometimes evoke negative connotations and may diminish public expectations that drugs will go through the rigorous testing required by the FDA, experts say. Murphy says she'd be more comfortable knowing the medicine she takes was specifically tested for her particular condition.  

The big question, Alexander says, is how to best regulate off-label drug use without unduly stifling clinical innovation. Some have called for a greater emphasis on evidence-based prescribing, with stricter rules governing off-label drug use. But others worry that tightening the reins could hinder patients' early access to novel therapies.

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