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.
A Risky Practice?
There is debate about off-label drug use. Doctors emphasize that off-label
prescribing has its place in medical practice, but they also admit that using a
drug off-label can raise the risk of lawsuits should a patient have unwanted or
bad side effects.
"Off-label prescribing can expose patients to risky and ineffective
treatments," medical ethics professor Rebecca Dresser and Joel Frader, MD,
write in the fall 2009 issue of The Journal of Law, Medicine &
Fen-Phen is one of the best examples of off-label use with a poor outcome.
The FDA approved medications fenfluramine hydrochloride and phentermine
hydrocholoride as individual, short-term treatments for obesity. But doctors
eventually began prescribing the two drugs together after an article describing
the cocktail's dramatic weight loss effects appeared in a medical journal and
numerous mainstream publications.
That off-label drug combination had devastating results: Many patients ended
up with severe, and potentially deadly, heart valve damage, an outcome that
triggered a multi-billion dollar lawsuit. In 1997, the FDA ordered Fen-Phen off
Although some off-label therapies can be beneficial and even lifesaving for
some patients, in most cases, there is little or no scientific evidence to
prove they work. In a recent commentary in the Archives of Internal
Medicine, three scientists from the National Institutes of Health
(NIH) Clinical Center's department of bioethics say that "off-label use
has been identified as one aspect of problematic drug prescribing," in part
because of inadequate data regarding drug safety and effectiveness for the
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