When a medication is approved by the U.S. Food and Drug Administration (FDA) for use in one way, it is generally understood that research and experience may eventually guide doctors to other uses of that medication. This is especially true with advanced cancer treatments, when it might be necessary to exhaust all reasonable options to fight the cancer. The term "off-label use" refers to the use of a drug in a way that has not been officially approved by the FDA. Doctors will usually choose to use a medication off-label to treat specific cancer situations only when there is some good evidence from research and clinical experience that the medication can be used effectively in that situation. Off-label use in this manner is considered acceptable and appropriate.
The research reviewed in this article found that about one-third of women being treated for metastatic breast cancer received a cancer medicine used in an off-label way. Most of the time the off-label use was considered appropriate to the situation. The results were presented at the 2009 American Society of Clinical Oncology (ASCO) Annual Meeting.
The researchers reviewed the treatment histories of over 2,000 women age 65 and older who were diagnosed with and treated for metastatic breast cancer.
The results:
If you are being treated for breast cancer and are taking time to learn about the medications in your treatment plan, you may discover that one or more of your treatments were never approved by the FDA for use in someone with your specific situation. Even though a treatment may be off-label, it is very likely that your doctor has recommended it to you based on sound evidence and experience from use of that treatment in patients with situations similar to your own. Still, it is a good idea to talk with your doctor about the role of any off-label treatments in your overall treatment plan, and why an off-label treatment might make sense for you and your unique situation.
ORLANDO, June 5 (MedPage Today) -- A third of patients with metastatic breast cancer receive off-label therapy at some point during treatment, according to a study reported here.
More than 70% of the drugs used off label lacked evidence to support their use in breast cancer, but these drugs were used in only a small percentage of patients, Sharon Giordano, M.D., of the University of Texas M.D. Anderson Cancer Center in Houston, said at the American Society of Clinical Oncology meeting.
Few patients received what was judged to be medically inappropriate therapy, she said.
"Although off-label drug use is widespread among patients with metastatic breast cancer, the vast majority of patients who received off-label drugs were treated with chemotherapy that was considered appropriate for use in treatment of breast cancer," Dr. Giordano said in an interview.
Estimates of the frequency of off-label therapy in medical practice range from 21% to 51%. Off-label use of chemotherapy drugs is thought to be widespread, but few studies have examined the issue, particularly with respect to the medial appropriateness of off-label therapy.
The last study to evaluate off-label chemotherapy practices was conducted about 20 years ago and covered cancer in general, not just breast cancer, Dr. Giordano said.
In an effort to develop a knowledge base specific to breast cancer, she and her colleagues analyzed merged data from the NCI Surveillance, Epidemiology, and End Results program and Medicare. They limited the analysis to women ages 65 and older who had a diagnosis of metastatic breast cancer from 1991 to 2002.
Investigators used the DRUGDEX drug compendium to evaluate the appropriateness of off-label therapy.
The study involved 2,082 women. Overall, 34.9% of the patients received at least one off-label therapy. Off-label treatment was least common in patients older than 80 (24.4%) and most common those ages 65 to 70 (37.7%, P=0.004).
Off-label drug therapy became less frequent over time. More than half of the therapy in 1991 (52.4%) was off label. Off-label therapy reached a nadir of 29.7% in 1997, rose to 37.6% over the next two years, and then declined again, accounting for 31.5% of treatment in 2002 (P=0.025).
The frequency of off-label therapy varied geographically, from 42.7% in Connecticut to 12.5% in rural Georgia (P=0.019).
The analysis uncovered off-label use of 36 different chemotherapeutic agents. Used most frequently were vinorelbine (Navelbine), 15.98%; gemcitabine (Gemzar), 8.37%; carboplatin, 7.03%; and mitoxantrone, 6.83%.
All four drugs had supporting evidence for breast cancer.
Dr. Giordano said 71% of the drugs lacked evidence for use in breast cancer, making the use medically inappropriate. However, only 6.7% of the patients received medically inappropriate therapy.
Because the study was limited to women 65 and older, the findings may understate the extent of off-label therapy, the researchers acknowledged.
Dr. Giordano has plans for additional studies of off-label therapy across a spectrum of tumor types and stages. She also hopes to examine off-label use in younger breast cancer patients.
Dr. Giordano and her coinvestigators reported no disclosures.
Primary source: Journal of Clinical Oncology Source reference: Dean-Colomb W et al. "Off-label drug use in women with breast cancer" J Clin Oncol 2009; 27(15 suppl): Abstract 1016.
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