The Lynx Group

Comparative Effectiveness Research Should Reap Benefits for Oncology Care

October 2013, Vol 4, No 8

San Francisco, CA—Comparative effectiveness research (CER) is an important construct for identifying and summarizing the evidence on the effectiveness, safety, and overall value of alternative strategies in oncology care, said Gary H. Lyman, MD, MPH, Professor of Medicine, Duke University and the Duke Cancer Research Institute, Durham, NC, during the 2013 Breast Cancer Symposium.

“The big challenge is deciding what treatment works in whom, under what circumstances, and whether we can afford it,” Dr Lyman said.

How Is Cost Driving CER?
The major driver of CER is the unsustainable rise in healthcare costs. “The rapid rise in healthcare costs, along with the emergence of exciting but costly technologies, has focused attention on the need for rigorous evaluation of benefits, harms, and overall value in a real-world patient setting—which is the aim of CER,” Dr Lyman said.

He indicated, however, that the problem of rising costs is complex, and CER must try to examine the various contributors.

It has been argued by some, including the American Society of Clinical Oncology (ASCO), that inappropriate overuse of expensive cancer interventions is a major driver of escalating healthcare costs, with the fee-for-service payment system providing perverse incentives for the use of expensive new drugs, even before the drugs receive specific label indications, according to Dr Lyman.

However, a recent study (Conti RM, et al. J Clin Oncol. 2013;31:1134-1139) showed that most patent-protected cancer therapies are used according to label indications, and most off-label use was compliant with guideline recommendations or compendia, he noted.

“The primary drivers of healthcare costs appear to be less that of overuse or misuse of expensive new drugs and technologies and more related to rising prices and required payments,” Dr Lyman said.

“Meaningful control of healthcare spending in cancer may need to focus more attention in other areas, such as the actual price of drugs, escalating insurance premiums, and market concentration through aggressive efforts on the part of the healthcare system,” he suggested.

Multiple Approaches to CER Are Needed
Although CER largely focuses on treatment, in breast cancer it has also been applied to disease prevention and screening, staging, prognostic and predictive biomarker determination, and supportive care. CER is also gaining attention as a way to improve the regulatory approval process.

Certainly, CER has been performed for years via randomized controlled trials. These remain the gold standard, but what has changed is the awareness that all the important questions in oncology cannot be answered by randomized controlled trials, Dr Lyman said.

Additional methodological tools, when properly used and interpreted, can help identify the most effective, safe, and valuable approaches, including quality-of-life studies, clinical decision simulation studies, and cost-effectiveness and cost-utility analyses.

“These may provide reasonable, valid, and more generalizable estimates of comparative effectiveness, safety, and cost and may also generate hypotheses that form the basis of future confirmatory randomized controlled trials,” Dr Lyman said.

ASCO Initiatives
Among new initiatives to provide more rapid evaluation of promising therapies without compromising safety is the ASCO Initiative for Defining and Measuring Clinical Value, which will examine, according to Lyman:

  • The clinical benefit of given treat­ments
  • Short-term and long-term toxicity
  • Direct and indirect costs, and value (ie, patient outcomes divided by costs of delivery)
  • Value benchmarks, which will utilize quality-adjusted life-years, weighted values, and hard and soft thresholds.

“We thought, who better to talk about cost and value than the persons providing the care? We thought it important that ASCO be involved in these processes,” he emphasized.

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