Journal of clinical oncology : official journal of the American Society of Clinical Oncology
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The objectives of this article are (1) to examine the similarities and differences between comparative effectiveness research (CER) and evidence-based medicine (EBM); (2) to describe the implications of CER for systematic review methodologies in oncology; and (3) to address the transition from systematic reviews to guideline development and the implications of CER in this process. ⋯ There is considerable overlap between the principles and methods of the two paradigms. The focus on best care options in the context of routine practice is a more central tenet of the CER paradigm. Thus, its value is not that it is the first paradigm to recognize the importance of a patient-focused approach in the research community, but rather, given the attention it has garnered, the CER paradigm may be precisely the reminder and push required to: one, influence how systematic questions are framed so that a more patient-relevant perspective is achieved; two, broaden the types of study designs that are valued and to include those, such as pragmatic trials and observational studies, that are better able to answer effectiveness questions; three, accelerate the development and application of statistical methods that enable indirect comparisons of cancer care options; and four, create clinical practice guidelines that are better positioned to improve quality of care and system performance. Over time, we will see if the CER paradigm lives up to its potential.
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Examining the patient's subjective experience in prospective clinical comparative effectiveness research (CER) of oncology treatments or process interventions is essential for informing decision making. Patient-reported outcome (PRO) measures are the standard tools for directly eliciting the patient experience. There are currently no widely accepted standards for developing or implementing PRO measures in CER. ⋯ Key recommendations are to include assessment of patient-reported symptoms as well as health-related quality of life in all prospective clinical CER studies in adult oncology; to identify symptoms relevant to a particular study population and context based on literature review and/or qualitative and quantitative methods; to assure that PRO measures used are valid, reliable, and sensitive in a comparable population (measures particularly recommended include EORTC QLQ-C30, FACT, MDASI, PRO-CTCAE, and PROMIS); to collect PRO data electronically whenever possible; to employ methods that minimize missing patient reports and include a plan for analyzing and reporting missing PRO data; to report the proportion of responders and cumulative distribution of responses in addition to mean changes in scores; and to publish results of PRO analyses simultaneously with other clinical outcomes. Twelve core symptoms are recommended for consideration in studies in advanced or metastatic cancers. Adherence to methodologic standards for the selection, implementation, and analysis/reporting of PRO measures will lead to an understanding of the patient experience that informs better decisions by patients, providers, regulators, and payers.
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Comparative effectiveness research (CER) seeks to assist consumers, clinicians, purchasers, and policy makers to make informed decisions to improve health care at both the individual and population levels. CER includes evidence generation and evidence synthesis. Randomized controlled trials are central to CER because of the lack of selection bias, with the recent development of adaptive and pragmatic trials increasing their relevance to real-world decision making. ⋯ These estimates can be tailored to patient characteristics and can include economic outcomes to assess cost effectiveness. The choice of method for CER is driven by the relative weight placed on concerns about selection bias and generalizability, as well as pragmatic concerns related to data availability and timing. Value of information methods can identify priority areas for investigation and inform research methods.
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Comparative effectiveness research (CER) has been promoted as a way to improve the translation gap between clinical research and everyday clinical practice as well as to deliver more cost-effective health care. CER will account for a significant portion of funding allocated by the US government for health care research. Oncology has a rich history of improving clinical outcomes and advancing research through randomized controlled trials (RCTs). In this article, we review the role of RCTs in achieving the goals of CER, with particular emphasis on the role of publicly funded clinical trials.
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The goal of comparative effectiveness research is to inform clinical decisions between alternate treatment strategies using data that reflect real patient populations and real-world clinical scenarios for the purpose of improving patient outcomes. Observational studies using population-based registry data are increasingly relied on to fill the information gaps created by lack of evidence from randomized controlled trials. Administrative data sets have many advantages, including large sample sizes, long-term follow-up, and inclusion of data on physician and systems characteristics as well as cost. ⋯ The analysis of well-designed studies of comparative effectiveness is complex. However, careful framing, appropriate study design, and application of sophisticated analytic techniques can improve the accuracy of nonrandomized studies. There are multiple areas where the unique characteristics of observational studies can inform medical decision making and health policy, and it is critical to appreciate the opportunities, strengths, and limitations of observational research.