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
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.