Sept. 27, 2011 -- A new research review shows that powerful drugs approved to treat schizophrenia, bipolar disorder, and depression may have modest benefits when they are prescribed for certain other mental and behavioral problems.
The study is published in the Journal of the American Medical Association.
Off-label refers to drugs prescribed for conditions for which they were not approved by the FDA to treat. It is legal to use a medication for these off-label reasons as long as the drug has been FDA-approved for at least one condition.
In patients with dementia, for example, doctors sometimes use atypical antipsychotics to treat symptoms of agitation and aggression. More controversially, the drugs have been prescribed to treat attention deficit hyperactivity disorder in kids.
The growing use of off-label drugs has come under scrutiny from federal regulators in recent years.
Drugmakers have long defended the practice of off-label prescribing. They say it offers doctors important flexibility in making treatment decisions for individual patients.
"Patient safety is a core priority for AstraZeneca and we believe that Seroquel and Seroquel XR are safe and effective medications when used as recommended in the prescribing information," says Stephanie Andrzejewski, director of brand corporate affairs.
"Further, we trust doctors to use their medical judgment to determine when it is appropriate to prescribe medications," she says.
"Bristol-Myers Squibb and Otsuka Pharmaceutical support the use of Abilify (aripiprazole) for its approved indications, when used in accordance with the prescribing information," says Sonia Choi, director of public affairs for those companies.
Eli Lilly, the maker of Zyprexa, says in a statement emailed to WebMD: "Lilly is committed to the highest ethical standards and to promoting
our medications only for approved uses."
The statement notes that "a substantial body of evidence supports Zyprexa's overall safety and efficacy profile for the indications for which it is currently approved. Because patients may respond differently to different medications, it's necessary for physicians to evaluate and discuss risks versus
benefits openly with patients and their families to understand what the best treatment options might be for each individual."
Measuring Benefits and Harms
Information from 162 studies was included in the review.
Researchers say there's some evidence that certain atypical antipsychotics may offer modest amounts of help for dementia, generalized anxiety disorder, posttraumatic stress disorder (PTSD), and obsessive-compulsive disorder (OCD).
"We didn't see dramatic benefits," says researcher Alicia Ruelaz Maher, MD, a psychiatrist and researcher at RAND, a nonprofit research organization. She is also a clinical associate professor at the University of California, Los Angeles.
For symptoms of agitation associated with dementia, for example, researchers found patients treated with certain atypical antipsychotics saw about a 35% improvement in their symptoms. Trials of these medications for generalized anxiety disorder and obsessive-compulsive disorder found similar degrees of improvement.
But the review also found significant risks associated with taking the drugs.
The researchers found that elderly patients with dementia who took atypical antipsychotics had an increased risk of death compared to those who were taking a placebo.
In an analysis of 15 studies, 3.5% of elderly patients with dementia on atypical antipsychotics died compared to 2.3% in the placebo group.
That represents one extra death for every 87 elderly patients with dementia who take the drugs, researchers say.
The bottom line, researchers say, is that these powerful medications should be used off-label only as a last resort.
"If there is something on-label that can be used, obviously you'd rather do that first, particularly with these medications because they do have significant side effects," Maher says.
Researchers say they hope the review will better inform doctors and patients who are considering using one of these drugs.