Significant Off-Label Use of Chemo in Elderly Cancer Patients

Zosia Chustecka

June 13, 2013

CHICAGO — A new study has revealed significant off-label use of chemotherapy, this time in elderly patients with a variety of different tumors and advanced disease. The data, which come from a Medicare database over a 10-year period, show that 18% of total drug costs were for the off-label use of chemotherapy, either for an indication that was not approved or at a dose that was not approved.

In the study of 42,634 patients with metastatic cancer, the use of off-label chemotherapy varied widely by cancer type, and was most common in patients with myeloma and prostate cancers.

The findings were presented by Dawn Hershman, MD, MS, from the Columbia University Medical Center in New York City, here at the 2013 Annual Meeting of the American Society of Clinical Oncology(ASCO®).

Dr. Hershman explained that in 1993, the US Congress institutionalized a requirement for Medicare to pay for off-label use if there was some support for that use in standard medical compendia.

She and her colleagues examined Medicare data from 1998 to 2008 to evaluate all chemotherapy claims for patients older than 65 years with metastatic cancer. Among the variety of tumors represented were lung (n = 16,904), ovarian (n = 7916), breast (n = 6524), prostate (n = 4067), colon (n = 3504), and uterine (n = 1363).

Off-label chemotherapy was used most often in patients with prostate cancer (33 unapproved drugs), ovarian cancer (28 unapproved drugs), breast cancer (20 unapproved drugs), and myeloma (18 unapproved drugs).

Overall, about 45% of all patients were treated with an unapproved drug. The highest use was in patients with myeloma (more than 80% received an unapproved drug); the lowest use was in patients with colon cancer (just over 10% received an unapproved drug).

When the researchers analyzed the chemotherapy administered to these patients, they found that the cost of approved drugs was $541.8 million, of compendium drugs (used for indications for which there was some clinical evidence and that were approved in the subsequent year) was around $150 million, and of unapproved drugs was $149.5 million, which is about 18% of total drug costs.

Off-Label Chemotherapy Use Is Common

Another recent study has shown that off-label use of chemotherapy is common, noted study discussant Monika Krzyzanowska, MD, MPH, from the Princess Margaret Cancer Center, University of Toronto.

In 2010, about 30% of intravenous chemotherapy with patent-protected drugs was off-label use, as reported by Medscape Medical News (J Clin Oncol. Published online February 19, 2013).

She also cited a survey of medical oncologists, which found that 80% of respondents reported using off-label chemotherapy at least once; a smaller percentage said they did so on a regular basis (J Clin Oncol. 2008;36:5994-6000). In addition, academic oncologists were more likely to do so than nonacademics.

Some of this off-label use of chemotherapy might be good, Dr. Krzyzanowska noted. It can allow patients access to a beneficial therapy before it receives official approval, she said. It can also be innovative, perhaps targeting a genetic mutation that is known to respond to the drug in another cancer type, although she emphasized that this should be done in the context of a clinical trial.

However, there are also some bad aspects of off-label use, including the financial cost and the negative impact it can have on clinical trial accrual. There is some evidence to support this, she added, citing a study in which enrolment in clinical trials was found to be slower for a therapy that was already being used off-label (J Clin Onocol. 2010;28;5067-5073).

Off-label use can be harmful, too, not only because of toxic effects, but because if can be used inappropriately. "We have to question the motivation here: Are we prescribing this drug because we are putting off an end-of-life discussion or out of a fear of litigation?" Dr. Krzyzanowska noted.

Dr. Hershman has disclosed no relevant financial relationships. Dr. Krzyzanowska reports serving as a consultant or in an advisory role for Bayer/Onyx; receiving honoraria from AstraZeneca, Novartis, and Sanofi; and receiving research funding from AstraZeneca and Exelis.

2013 Annual Meeting of the American Society of Clinical Oncology: Abstract 6509. Presented June 4, 2013.

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