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. The Oncologist 2013;18:752-759 Implications for Practice: Escalating health care costs, largely driven by expensive new technologies and therapies, have prompted calls for more rigorous assessment of the effectiveness, safety, and overall value. At the same time, the time and resources required for large randomized controlled trials, as well as their recognized limitations, necessitate consideration of other forms of comparative effectiveness research for evaluating meaningful clinical outcomes. Through the results derived from selective and appropriate use of a range of comparative effectiveness methodological tools, clinicians, patients, payers, and policy makers can make more rationale choices in selecting the most appropriate, efficient, and cost-effective cancer care. At the same time, practicing oncologists and other stakeholders must be aware of the challenges presented by such research approaches as well as the considerable progress that has been made in addressing these limitations in order to appropriately weigh the totality evidence on critical health care issues in every day oncology practice. INTRODUCTIONComparative effectiveness research (CER) attempts to compare the benefits and harms of alternative strategies for diagnosing, treating, or preventing disease in patients. Driven primarily by new therapies and technologies, health care expenditures c...

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