Posted on: Tuesday, 7 June 2011, 15:20 CDT

A study of more than 1,000 patients with colon cancer that had spread to distant sites found that one in eight was treated with at least one drug regimen that was not recommended. Those patients were exposed to significant risk without proven benefits, at an estimated cost—just for the drugs—of more than $2 million.

The study, presented June 7, 2011, by University of Chicago researchers at the American Society for Clinical Oncology’s annual meeting in Chicago, focused on three chemotherapy regimens that were not supported by evidence from prior clinical studies or clinical practice guidelines. One treatment was rated "insufficient data to support," one had been "shown to be ineffective," and one was supported by "no data, nor is there a compelling rationale."

"Patients with advanced cancers that do not respond to standard therapies should either be looking for clinical trials, where there is a chance for a benefit, or should have been thinking about shifting toward palliative care," said study author Jonas De Souza, M.D., a hematology/oncology fellow at the University of Chicago. "Patients should not face the risks, discomforts and costs of aggressive and often quite toxic chemotherapy with treatment regimens that did not provide a benefit in previous studies."

Under an agreement with UnitedHealthcare, a health benefits business, the researchers used de-identified medical and pharmaceutical claims data in the collaborative project. They examined claims from 7,642 colon cancer cases treated between January 2007 and June 2010, including 1,041 who developed metastatic disease. Of those 1,041 patients, 140 (13%) received treatments that were not supported by the evidence from clinical studies. Many of them received multiple cycles of non-beneficial chemotherapy.

The researchers focused on three chemotherapy regimens with specific recommendations against their use in the National Comprehensive Cancer Network (NCCN) guidelines. The regimen with insufficient data involved bevacizumab (trade name Avastin) used after the patient had progressed on a combination of that drug and chemotherapy. The treatment shown to be "ineffective" was capecitabine (trade name Xeloda) after progression on the same class of drugs. The regimen with no compelling rationale was panitumumab or cetuximab (trade name Erbitux) after progression on similar drugs.

The 140 patients received 869 cycles of chemotherapy. Some received two or more unproven treatments.

    * Ninety-one of those patients went through 632 intravenous cycles of bevacizumab, at an estimated cost of $1.3 million. Potential side effects include hypertension, heightened risk of bleeding and bowel perforation.    * Fifty-nine patients received 218 non-evidence-based cycles of capecitabine, at a cost of more than $600,000. This drug, taken orally, can cause diarrhea, nausea, vomiting, fatigue, rash and swelling of the hands or feet.    * Six patients underwent 19 cycles of panitumumab, at a cost of almost $70,000. This drug can trigger itching, dermatitis and rash.

"We did not study why these physicians and patients turned to unproven therapies," said co-author Caleb Alexander, M.D., associate professor of medicine at the University of Chicago. "I suspect that both patients and care providers, when facing life-threatening disease with limited options, are more willing to step outside guidelines."

"There could also be financial implications," he added. "A physician who has run out of options may be hesitant to send a patient to a center that has access to greater resources but may be far away."

The researchers emphasized, however, that as the costs of cancer care continue to rise, the impetus to base treatment decisions on solid evidence can only increase. "It’s important to get the right medicines to the right patients," said Alexander.

"DeSouza’s research highlights the importance of evidence-based treatment for cancer patients," said Lee Newcomer, M.D., UnitedHealthcare’s senior vice president, oncology. "Expert oncology opinions tell us that the extra therapies that these patients received potentially exposed these patients to unnecessary side effects. We should be relieving symptoms and not causing new ones, even as we try to address the underlying disease."

• University of Chicago Medical Center

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Tags: health benefits, clinical practice guidelines, Health Medical Pharma, associate professor, chicago researchers, Colon Cancer

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