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.
Benefits of Off-Label Drug Use continued...
Beta-blockers are another example of beneficial off-label prescribing. Such
medications are FDA-approved for the treatment of high blood pressure, but are
widely recognized by cardiologists as a standard of care for patients with
heart failure. And in fact, some beta blockers are now formally approved to
treat heart failure. It's not uncommon for off-label uses to eventually get
approved by the FDA.
Other drugs commonly prescribed off-label include tricyclic antidepressants
for chronic pain, and antipsychotics for attention deficit hyperactivity
disorder (ADHD) "Name the drug, and one can come up with off-label uses,"
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