Comparative effectiveness research in oncology

Abstract

Although randomized controlled trials represent the gold standard for comparative effective research (CER), a number of additional methods are available when randomized controlled trials are lacking or inconclusive because of the limitations of such trials. In addition to more relevant, efficient, and generalizable trials, there is a need for additional approaches utilizing rigorous methodology while fully recognizing their inherent limitations. CER is an important construct for defining and summarizing evidence on effectiveness and safety and comparing the value of competing strategies so that patients, providers, and policymakers can be offered appropriate recommendations for optimal patient care. Nevertheless, methodological as well as political and social challenges for CER remain. CER requires constant and sophisticated methodological oversight of study design and analysis similar to that required for randomized trials to reduce the potential for bias. At the same time, if appropriately conducted, CER offers an opportunity to identify the most effective and safe approach to patient care. Despite rising and unsustainable increases in health care costs, an even greater challenge to the implementation of CER arises from the social and political environment questioning the very motives and goals of CER. Oncologists and oncology professional societies are uniquely positioned to provide informed clinical and methodological expertise to steer the appropriate application of CER toward critical discussions related to health care costs, cost-effectiveness, and the comparative value of the available options for appropriate care of patients with cancer.

Keywords: Clinical trials; Comparative effectiveness; Cost-effectiveness; Health policy; Outcomes.

Figure 1.

Plot of total annual health care expenditure per capita between 1960 and 2010 for selected major economies based on data reported to The Organisation for Economic Co-operation and Development (OECD) (http://www.oecd.org/). Expenditures are reported in U.S. dollars based on purchasing power parity estimates as of August 10, 2012. Annual estimates for the U.S. are highlighted in red, and the average for other OECD countries highlighted in green.

Figure 2.

Plot of estimated life expectancy at birth by total health care expenditure per capita for 2010 for selected major economies reporting to OECD (http://www.oecd.org/). Expenditures are reported in U.S. dollars based on purchasing power parity estimates as of August 10, 2012. Estimates for the U.S. are highlighted in red, and estimates for other OECD countries are highlighted in green.