SIDEBAR: The Good, the Bad, and the Ugly 

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Reviewing a study on off-label use of chemotherapy, Monika K. Krzyzanowska, MD, MPH, of Princess Margaret Cancer Centre University of Toronto, reflected on what she called the good, the bad, and the ugly of off-label drug use.

“Certainly there are some good aspects about off-label therapy,” she said. Among the good is “early access to medications that have been shown to be efficacious already. It takes a long time to get drug approval, so once the clinical trial is presented or published and the results look promising, if the drug is already on the market, this gives you an opportunity to get access to it early,” she said. Off-label use can also be helpful in treating rare diseases “where it may be your only option.”

Off-label use also presents “an opportunity in innovation in the molecular matching between drugs and molecular abnormalities,” Dr. Krzyzanowska said. “I think if we are going to use these drugs in that setting, we really need to be tracking what’s happening so that we learn from all of this.”

“In terms of the bad, there has been some work that suggests than when drug therapy that is being tested in a clinical trial is available off-label for another indication, it does have a negative impact on trial accrual,” Dr. Krzyzanowska stated. High costs can also be negative factor. In a study published in the Journal of Clinical Oncology,1 total spending on off-label use of 10 commonly prescribed intravenous drugs totaled $4.5 billion in 2010.

“In terms of the ugly part of this equation, we have the issue of harm,” Dr. Krzyzanowska said, because many of the drugs prescribed off-label also have significant side effects. “The second issue is the issue of motivation. Are we prescribing drugs off-label specifically because we think they are going to benefit patients? Or is this a way of avoiding having that difficult conversation that further treatment then would be futile and you really should be looking at supportive care and palliative care?” ■


1. Conti, RM, Bernstein AC, Villiflor VM, et al: Prevalence of off-label use and spending in 2010 among patent-protected chemotherapies in a population-based cohort of medical oncologists. J Clin Oncol 31:1134-1139, 2013.

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