Comparative effectiveness research (CER) has received considerable research attention in recent months, and efforts to promote CER are part of the newly enacted Patient Protection and Affordable Care Act. In this paper, we define CER, how it complements traditional efficacy research in asthma, and discuss how CER can help provide the basis for rational decision-making about the care of individual patients with asthma and how best to deliver this care in real-world settings. We present information about the challenges and opportunities to conduct CER, including enhanced patient registries for observational CER and effectiveness trials (also called pragmatic trials). We discuss the urgent need to define the appropriate methodologies for CER and to develop and prioritize a research agenda for CER studies in asthma with the help of a diverse group of stakeholders.
KeywordsComparative effectiveness research; comparative clinical effectiveness research; observational studies; effectiveness trials; efficacy trials; pragmatic trials; explanatory trials; asthma Randomized controlled trials are the gold standard by which the benefits and harms of treatments have been established, because this approach greatly minimizes the risk of confounding and avoids selection bias. Such studies have traditionally employed an efficacy paradigm in which various approaches are used to amplify the signal-to-noise ratio in order to address the question "Can this intervention work?" Such studies are critical to the development of innovative approaches to patient care. A hallmark of the efficacy trials is the application of narrowly defined inclusion and exclusion criteria to select study subjects. A recent study found, for example, that a median of 6% (range 0 to 43%) of patients treated for asthma met eligibility criteria for major trials cited in evidence-based treatment guidelines. 1 A high level of selectiveness is employed in efficacy trials to ensure that study subjects are enriched with patients most likely to benefit and least likely to be harmed by the study interventions. MC 6076, Room M644, Chicago, IL 60637, jkrishna@medicine.bsd.uchicago.edu,. Publisher's Disclaimer: This is a PDF file of an unedited manuscript that has been accepted for publication. As a service to our customers we are providing this early version of the manuscript. The manuscript will undergo copyediting, typesetting, and review of the resulting proof before it is published in its final citable form. Please note that during the production process errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal pertain. Another feature of efficacy trials is the conduct of such studies in a research environment that promotes uniform application of the intervention to study subjects, often by expert clinicians and in resource intensive settings. The National Heart, Lung, and Blood Institutesponsored AsthmaNet, for example, is a multi-center network led by Principal Investigators with established research prog...