Initial National Priorities for Comparative Effectiveness Research (2009) / Chapter Skim
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2 What Is Comparative Effectiveness Research?
2 What Is Comparative Effectiveness Research?
Pages 29-60
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From page 29... ...
The purpose of CER is to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels." CER's distinguishing characteristics include informing a specific clinical or policy decision, comparing at least two approaches or interventions, describing results at the subgroup level, measuring benefits in real-world populations, and applying appropriate methods and data sources. Several federal agencies and many other organizations are involved in important CER activities, which are summarized in this chapter.
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. Even the most thoughtfully conceived and sophisticated practice guidelines have inadequacies in their evidence base, whereas many guidelines that are evidence-based and well-supported are often not translated into clinical practice.
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By contrast, effectiveness research intends to measure the benefits and harms of an intervention in ordinary settings and broader populations, and therefore can often be more relevant to policy evaluation and the health care decisions of providers and patients. Care needs to be taken, however, in effectiveness research such as observational, database, registry, and other studies to recognize that without randomization, unidentified bias and confounders may weaken the level of evidence, and that efficacy studies may be strengthened by broadening eligibility of populations or settings for trials and other attempts to increase generalizability.
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, particularly in subgroups that preapproval studies often omit. Beyond specific medical interventions and technologies, there is a need for evidence evaluating the clinical and resource effects of innovations in health care delivery models, including new benefit designs, costsharing techniques, integrated organizational models, public health and population-level strategies, and interventions to improve the quality of care.
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From page 33... ...
is that it provides evidence that is better focused on the decisions of daily medical practice than existing evidence and therefore helps patients, caregivers, providers, payers, and policy makers make informed decisions about health care. defining comparative effectiveness research CER can be very broad in scope depending on what is "compared," how one defines "effectiveness," and what constitutes "research." Virtually any applied biomedical inquiry is fundamentally about improving health, avoiding unnecessary costs, or both.
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From page 34... ...
Existing Definitions Although research comparing the effectiveness of health care strategies and interventions has been conducted for more than a century, the term "comparative effectiveness research" has taken on new meaning in recent years. Table 2-1 displays several definitions, all of which were developed by leading public and private-sector authorities in the United States.
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From page 35... ...
Physicians effectiveness, safety, and cost of two or more medical services, drugs, devices, therapies, or procedures used to treat the same condition. Although the use of the term comparative effectiveness broadly refers to the evaluation of both the relative clinical and cost differences among different medical interventions, it is notable that most comparative effectiveness research engaged in and used by stakeholders in this country focuses solely on evaluating relative clinical differences to the exclusion of cost factors.
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. the core question of comparative effectiveness research (is)
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From page 37... ...
CER has the objective of directly informing a specific clinical deci sion from the patient perspective or a health policy decision from the population perspective. The range of potential objectives for CER studies gives the field a broad scope.
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Harms or risks of unintended consequences are also outcomes of interest, because they influ ence the net benefits of an intervention. Including and giving weight to patient-reported outcomes is particularly important for CER studies in which patient ratings of effectiveness or adverse events may differ from clinical measures.
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. For decisions that have been the topic of substantial previous research, synthesis of existing studies (systematic reviews and meta-analysis, technology assessments and decision analysis)
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In some cases, bias or confounders may limit the reliability of data obtained through retrospective means. Alternatively, prospective, randomized clinical trials may take so long or be so expensive as to render the study unfeasible or unethical.
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. IOM National Priorities Committee Definition Proceeding from the definitions in Table 2-1 and the preceding considerations, the committee developed the following working definition of CER to guide its deliberations: Comparative effectiveness research (CER)
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The selected methodology is based on the sources of information available to answer the question and dictates the rapidity and cost involved in study performance. Treatment of coronary artery disease has been the subject of numerous CER studies.
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Pooled Analysis of Patient-Level Data from Many Clinical Trials Even more recently, data from 10 RCTs were pooled to create one large dataset that included the patients from the 10 trials. The authors sought to identify patient-level predictors of outcomes (Hlatky et al., 2009)
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differences and odds ratios between PCI and CABG and the 95% CI for each outcome of interest at annual intervals; observational studies were summarized qualitatively. NOTE: AMI = acute myocardial infarction; CABG = coronary artery bypass graft; CI = confidence interval; CMS = Centers for Medicare & Medicaid Services; MT = medical therapy; PCI = percutaneous coronary intervention.
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From page 45... ...
effective" by the American College of Cardiology and American Heart Association, but are not currently tracked by CMS. Pooled analysis of individual patient data Observational, patient-level analysis of data from 10 clinical trials that randomly abstracted from hospital administrative assigned patients with multivessel and clinical records; temporal trends in coronary disease to either CABG or PCI individual and composite processes of care and that reported at least 3 years of for both CMS and non-CMS measures at follow-up.
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From page 46... ...
Agency for Healthcare Research and Quality AHRQ's Effective Health Care Program has been the federal government's leading effort to conduct systematic comparative effectiveness reviews and database studies that compare health care interventions. Created by Congress in 2003 under the Medicare Prescription Drug, Improvement, and Modernization Act, the Effective Health Care Program had a budget of $30 million in 2008.
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From page 47... ...
The Eisenberg Center creates and disseminates a variety of products for each audience, ranging from summary guides to decision aids and other materials. To date, the AHRQ Effective Health Care Program has released approximately 30 products, including 14 comparative effectiveness reviews from the EPCs; 18 summary guides for clinicians, patients, and policy makers; and 10 reports from the program's DEcIDE research network. Many more projects are under way, and all of the program's products are publicly available at AHRQ's website: http://www.effectivehealthcare.ahrq.gov.
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From page 48... ...
. The Cooperative Group Program housed within the National Cancer Institute was established in 1955 to promote and support clinical trials of new cancer treatments, explore methods of prevention and detection, and assess quality of life during and following treatment (NCI, 2006)
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. In addition to exercising regulatory authority to ensure the safety and effectiveness of medical products, the FDA is developing standards and methods for CER studies, as well as their interpretation to support its regulatory mission.
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. Department of Veterans Affairs The Veterans Healthcare System at the VA is suited to both produce and apply evidence on treatment effectiveness because it cares for a very large patient population, has a sophisticated electronic health records system that supports clinical research, and provides an array of medical services.
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. The VA is well-suited for CER activities because it has broad clinical and research capacity and feeds patient data recorded in the VA electronic health record system into a data warehouse that contains health records on the entire VA population.
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Cochrane Collaboration Established in 1993, the Cochrane Collaboration is an independent, multinational nonprofit organization that creates and distributes systematic reviews of health care interventions. These reviews are prepared by 52 Cochrane Review Groups.
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From page 53... ...
The Federal Coordinating Council's June 30, 2009, report to the Secretary of HHS will also describe the CER activities of federal agencies. Even limiting itself to its description of federal CER activities and the study of systematic reviews in Knowing What Works in Health Care, the committee found considerable duplicated effort, which is one reason to propose a mechanism to coordinate CER activities throughout the nation.
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From page 54... ...
NIH, FDA, AHRQ's DEcIDE Research Network, CERTs, CMS, VA, and CMTP are among the organizations focused on the development of new evidence from welld esigned comparative clinical trials and observational studies. Priorities for new CER must address the research infrastructure required to generate new data to answer questions of interest to patients and policy makers and must recommend investment in new capacity where needed.
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From page 55... ...
Although RCTs, prospective cohort studies, and patient registries are among its most important tools, CER also uses other forms of information, such as systematic reviews, electronic health records, patient registries, and other observational datasets. Prospective studies based on new trials or primary analyses of patientlevel data are a very important aspect of CER.
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2007. Systematic review: The comparative effectiveness of percutaneous coronary interventions and coronary artery bypass graft surgery.
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Paper presented at Implementing Comparative Effectiveness Research: Priorities, Methods, and Impact, Engleberg Center for Health Care Reform at Brookings: Wash ington, DC. Garber, A
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2008. Cochrane handbook for systematic reviews of interventions: Version 5.0.1 Place Published: The Cochrane Collaboration, 2008.
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2005. Challenges in systematic reviews that evaluate drug efficacy or effectiveness.
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2009. Identi fying, selecting, and refining topics for comparative effectiveness systematic reviews: AHRQ and the effective health care program.
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