A systemic analysis of clinical oncology trials shows discrepancies in the structure of cancer trials as well as differences among oncology studies, according to a study published online in JAMA Internal Medicine. The findings suggest room for improvement to help stakeholders and policy makers understand how research affects practice and determine optimal use of healthcare resources.

Cancer costs topped $125 billion in 2010, yet data regarding the value of cancer care are limited, due to multiple factors. These include a lack of comparative effectiveness research, inefficiencies in trial execution, increasing use of off-label therapies and increasing reliance on less robust trial design, according to Bradford R. Hirsch, MD, MBA, from the Duke University Cancer Institute and Duke Cancer Care Research Program in Durham, N.C.

Hirsch and colleagues mined the ClinicalTrials.gov database to analyze the oncology clinical research portfolio. They identified 8,942 oncology-focused studies registered between October 2007 and September 2010, which comprised 21.8 percent of trials.

The researchers reviewed number of arms, masking, randomization, phase, comparator arms, purpose of intervention, enrollment status, location of trial sites, sponsor and completion status.

“Oncology studies were significantly more likely to be single arm (62.3 percent vs. 23.8 percent; P < .001), open label (87.8 percent vs. 47.3 percent; P < .001), and nonrandomized (63.9 percent vs. 22.7 percent; P < .001),” wrote Hirsch et al.

Other observations include a modest link between the number of trials and incidence. Lung cancer, for instance, accounted for 27.6 percent of cancer deaths in 2010, and was the focus of 9.2 percent of trials.

Although treatment-oriented trials dominate, other outcomes play an important role in some cancers, particularly breast cancer. One in four breast cancer trials assessed supportive care, diagnosis or prevention.  

Hirsch and colleagues characterized oncology trials as “predominantly early-phase studies that evaluate surrogate end points and are small, single arm, and open label.” This design diverges from trials in other types of medicine.

The implications are problematic. “There is an inherent tension between the desire to use new, life-saving treatments and the imperative to develop the evidence base that patients, clinicians, regulatory agencies, and advocacy groups need to make sound decisions.”

The dominant oncology trial design model may not provide the requisite answer to clinical questions. Economics also comes into play as a total of 70 percent of $125 billion in oncology spending in 2010 was spent on agents approved in the last decade, many of which carry price tags that soar past those of earlier treatments.

The researchers concluded with a few suggestions to help guide future trials. These are:

• Ensure that the ClinicalTrials.gov database is as complete, up to date, and useful as possible;
• Employ a more nuanced approach to trial design and attempt to match design to indication; and
• Leverage the ClinicalTrials.gov database to determine critical models in existing trials and use them to inform and monitor research.

For more about the skyrocketing costs in cancer care, please read “MDs blast $100K price tag on oncology drugs.”

In addition, look for “The New Economics in Radiation Oncology,” in the May/June issue of Health Imaging magazine.