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Comparative Effectiveness Studies Explore Cancer, Trauma Care

 |  By cclark@healthleadersmedia.com  
   April 18, 2012

To highlight the importance of comparative effectiveness research, the Journal of the American Medical Association partnered with the $3.5 billion Patient Centered Outcomes Research Institute on Tuesday to explain three studies in this week's themed issue and discuss the need for more research.

The studies' conclusions help "physicians, other clinicians, policymakers,  and patients make informed decisions" about the benefits and harms of the choices available to "prevent, diagnose, treat, or monitor a clinical condition," said Phil Fontanarosa, MD, the JAMA's executive editor. 

Avastin and Lung Cancer
One study, in this edition of the journal, for example, upset prevailing wisdom about the benefit of adding a costly drug, bevacizumab, sold under the brand name Avastin, to the standard two-drug cocktail (carboplatin and paclitaxel) for treating advanced non-small cell lung cancer in Medicare beneficiaries.

The drug was approved by the U.S. Food and Drug Administration and covered by the Centers for Medicare & Medicaid Services after a 2006 trial found that adding it to the regimen provided a statistically significant, albeit slight, survival advantage.

However, explained Deborah Schrag, MD, principal investigator of the Center for Outcomes Research at the Dana Farber Cancer Institute, most of the participants in the original trial were patients younger than age 65. Most of the patients who are diagnosed with this form of cancer, and most of the 165,000 annual lung cancer deaths, however, are older than 65.

Schrag's report, which looked at Medicare and Surveillance, Epidemiology and End Result data for lung cancer, found that the survival benefit for Medicare beneficiaries treated with the three-drug cocktail was less than two months compared with patients treated with the two-drug regimen.

"We found that adoption of bevacizumab did not have a substantial benefit in the Medicare population with advanced non-small cell lung cancer," Schrag said at a Tuesday briefing. And while the drug may still have some benefit for people under 65, most of the people diagnosed with this disease are over age 65.

"In the future, for malignancies like NSCLC that disproportionately affect elderly patients or where the CMS covers a large proportion of treatment costs, negotiations with pharmaceutical sponsors of pivotal trials might mandate adequate representation of elderly patients and/or pre-planned subgroup analyses relevant to the Medicare population," the paper said.

Helicopters and Trauma Patients
The second highlighted paper, from Johns Hopkins University School of Medicine, attempted to settle the debate about whether helicopters used to rescue trauma patients actually save lives, compared with the use of ground emergency medical service transport.

The issue is controversial, acknowledges Adil H. Haider, MD, one of the authors, because maintaining ready helicopter service for trauma centers is expensive, may have limited application due to geography, and because of questions about how quickly which types of trauma patients need to get to a hospital; perhaps life extending remedies some can be effectively treated in the field.

The report examined outcomes from treatment of 223,475 patients older than age 15 with injury severity scores higher than 15 taken to a level I or level II trauma center, as reflected by data from the American College of Surgeons National Trauma Data Bank.

While the raw numbers showed that mortality was higher for those injured patients transported by helicopter, helicopters carried more severely hurt patients. When adjusted for those variables, helicopter transport to level I trauma centers was associated with 16% better chance of survival compared with ground transport. For transport to level II trauma centers, there was a 15% better odds of survival.

"For patients transported to level I trauma centers by helicopter, 65 patients would need to be transported to save one life; for patients transported to level II trauma centers, the number needed to treat is 69," the researchers wrote.

The authors said that future research should hone in on what factors associated with helicopter medical rescue provide the additional benefit, such as improved pre-hospital intervention, total pre-hospital time, or crew configuration.

Prostate Cancer and Proton Beam Therapy
The third, and perhaps most controversial, paper focused on a comparison of three types of treatments for non-metastatic prostate cancer in Medicare beneficiaries and found no additional benefit despite the proliferation of construction of expensive proton beam centers across the country.

The paper compared older conformal radiation therapy, the newer and more common intensity-modulated radiation therapy (IMRT), with proton beam.

Ronald Chen, MD, of the University of North Carolina at Chapel Hill said that based on outcomes in 12,976 men treated between 2000 and 2008, IMRT resulted in fewer gastrointestinal co-morbidities and hip fractures, but was more likely to result in erectile dysfunction compared with older conformal radiation therapy.

Comparing proton beam radiation just with IMRT revealed fewer gastrointestinal complications with IMRT, and no significant differences in rates of other morbidities or additional therapies between the two treatments.

Chen said the researchers looked at side effects such as bowel obstructions, rectal bleeding, urinary incontinence, all of which have been associated with various forms of radiation therapy for prostate cancer.

He added that proton beam centers, which cost an estimated $400 million each and require new structures, have moved forward in recent years based on their theoretical benefits that they may be able to reduce damage to surrounding organs because the beams may be more accurately targeted.

Two additional studies, one at Massachusetts General Hospital and the other at UNC, are looking at quality of life issues for various treatment options. "Hopefully these two studies will allow patients to answer these important questions," he said.

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