Learning What Works Infrastructure Required for Comparative Effectiveness Research: Workshop Summary (2011) / Chapter Skim
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1 The Need and Potential Returns for Comparative Effectiveness Research
1 The Need and Potential Returns for Comparative Effectiveness Research
Pages 57-86
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From page 57... ...
. While each highlights a different problem or concern with the current healthcare system, collectively these findings reveal systemic inadequacies in current approaches to developing evidence to help guide the health decisions of policy makers, physicians, and patients.
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. Recently, policy makers have demonstrated substantial interest in comparative effectiveness research (CER)
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The Roundtable has defined science-driven health care broadly to mean "to the greatest extent possible, the decisions that shape the health and health care of Americans -- by patients, providers, payers, and policy makers alike -- will be grounded on a reliable evidence base, will account appropriately for individual variation in patient needs, and will support the generation of new insights on clinical effectiveness" (IOM Roundtable on Value & Science-Driven Health Care, 2009)
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. This background brief also provided context for the July 30–31 workshop, Learning What Works: Infrastructure Required for Comparative Effectiveness Research, and was included in the meeting's briefing materials.
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If only 1 percent of the nation's healthcare spending were devoted to understanding the effectiveness of the care purchased, the total for effectiveness research would come to approximately $20 billion annually -- about 10 times the amount in 2009. In contrast, even accounting for the support from all private and public sources, the aggregate national commitment to assessing the effectiveness of clinical interventions is far below the standard that any company would expect to invest in work to evaluate and improve its products.
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The integrity and reputation of healthcare delivery organizations is dependent on their ability to ensure the quality and appropriateness of the care delivered within their walls. Any decision support system is only as good as the information built into the model and should include the comparative advantages or disadvantages of different diagnostic and therapeutic options.
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, AHRQ, and the Centers for Disease Control and Prevention -- for all health services research amounted to about $1.5 billion, and only a modest portion of this was devoted to clinical effectiveness research, far below the industry level. Additional work, also modest, is undertaken by certain of the larger healthcare delivery organizations.
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. Models for a Stronger Approach to Comparative Effectiveness Research To narrow the rapidly growing gap between the available evidence on clinical effectiveness and the evidence necessary for sound clinical decision
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Other possibilities include approaches that are privately funded, although this raises issues of independence and objectivity, as well as approaches with a blend of public and private funding, which could have various governing and execution structures. In the latter category are those
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, which are funded primarily by the federal government but which are allowed to have up to 30 percent of their funding from private sources. The FFRDCs are private entities managed by nongovernmental organizations and are based on the examples of free-standing independent quasi-governmental entities such as the Federal Reserve Board, which serves as the nation's central banking system, and the IOM and the Transportation Research Board (TRB)
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health expenditures or in the context of the efficiencies that could be gained. Suggestions for funding mechanisms range from direct annual federal appropriation or a small set-aside from the Medicare Trust Fund to the structuring of proportionately matching contributions, including set-asides from Medicare fund expenditures, from private health insurance premiums, or from manufacturers' R&D expenditures (Health Industry Forum, 2006; Hopayian, 2001; Kupersmith et al., 2005; Wilensky, 2006)
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As outlined in the Act, PCORI will set a national agenda for research priorities, fund entities that conduct priority research, improve clinical effectiveness research methods, and ensure transparency and broad dissemination of its findings. It will be overseen by a Governing Board, comprised of 19 members appointed by head of the Government Accountability Office, as well as 2 ex officio representatives from the Agency for Healthcare Research and Quality and the National Institutes of Health.
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. research on the clinical effectiveness of interventions brings many potential opportunities for improvement across a wide spectrum of healthcare needs.
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Data and Information Networks Distributed data networks for securely and efficiently sharing relevant clinical and claims data are necessary for moving to a system that uses information captured at the point of care to influence practice patterns. These networks will be crucial to the CER infrastructure because they allow data owners -- commercial health plans, the federal government, and others -- to share only summary data in response to specific queries; individual-level data remains protected within the data owners' systems.
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Research Methods Capitalizing on the opportunities presented by emerging clinical data and information networks requires innovative approaches to clinical trials in order to allow them to be conducted under conditions of actual practice, enabling estimates of real-world effectiveness. It will also be necessary to have improved statistical and epidemiologic methods to address the limitations of nonrandomized studies employing heterogeneous but much richer and larger-scale data sources.
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The remainder of this paper will be devoted to examining four key gaps in evidence that could be closed by a learning health system and that should inform CER infrastructure development. Establishing Baselines for Evaluations -- Disease Models and Natural Histories To move beyond evaluating the average impact of a treatment in a population and toward targeted, personalized medicine, researchers need to understand how particular types of patients are being treated.
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At a recent meeting of the American Health Information Community, a number of research groups discussed how a better understanding of particular disease models with particular kinds of patients is beginning to emerge. This initial success in bridging a particular type of gap in evidence can be accelerated with appropriate infrastructure development.
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These shortcomings in the evidence base create certain challenges that can be addressed with infrastructure development. A primary challenge is the relatively small effect of alternative treatments on patient outcomes observed in comparative effectiveness studies.
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As presence on a medical product label is viewed as the gold standard for determining whether evidence is of the highest quality available, the absence of particular evidence from a label can make it difficult for such evidence to be widely accepted by physicians. A considerable amount of work is currently under way to conduct comparative effectiveness trials and to use data that has been collected for other purposes to develop comparative effectiveness information.
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Such assessments should be part of the science of healthcare delivery, and knowledge gained through such studies could influence practice. These questions can be studied in real-world medical practice, where similar patient populations are exposed to different health policies and therefore may face different treatment options and strategies.
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THE POTENTIAL RETURNS FROM EVIDENCE-DRIVEN HEALTH CARE Gail R Wilensky, Ph.D., Senior Fellow, Project HOPE Interest in the potential of comparative clinical effectiveness information as a strategy to help Americans learn to "spend smarter" has been growing among those at both ends of the political spectrum, and it can best be understood as part of the concern about healthcare quality and value, and the drive toward the increased use of evidenced-based medicine.
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Finally, some determination will be needed as to whether the likely differences are ones that would be relevant at a clinical or policy level. With that introduction, let me turn now to the kinds of data that will be relevant for comparative effectiveness analyses, remembering that the focus for these analyses is generally a medical condition and the various alternative strategies that can be used for treating that medical condition.
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Efforts by Bryan Luce in developing his Pragmatic Approaches to Comparative Effectiveness initiative, along with the work of Don Berry that makes use of Bayesian statistical approaches to establish shorter end points in certain types of clinical trials, represent other important efforts in this vein. Even with these strategies to reduce the costs of new prospective trials, it is the anticipated need for a substantial amount of new data that makes the cost involved with comparative effectiveness significant.
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An important early step for more effective CER will be the creation of either a new center or a series of centers -- my preference would be for a single center -- that is part of the government or is a public–private enterprise and that is responsible for funding comparative clinical effectiveness studies. Unlike some of my colleagues, I believe it would be unwise -- both at a technical level and, even more importantly, at a political level -- to include cost-effectiveness analysis as part of the activities of a center for comparative clinical effectiveness.
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If it appeared that a direct appropriation to CMS for costeffectiveness analysis was unreliable, a small portion of the funding stream could be diverted to CMS in order to fund the cost-effectiveness studies that are important for Medicare. It will also be important to ensure that the information on cost effectiveness that is generated is valid in terms of objectivity and credibility -- just as information needs to be for comparative clinical effectiveness -- or it will not be trusted.
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Such a monopoly would be inappropriate in a country as large and diverse as ours, and it would also be a political nightmare for politicians. As part of the need to realign financial incentives so that physicians and other clinicians, as well as institutions, are rewarded for producing good clinical outcomes, a first step could be to have information available from comparative clinical effectiveness as part of a change in reimbursement policies.
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It will be particularly important to begin to give CMS the legislative authority to introduce what is known about clinical and cost effectiveness into its reimbursement decisions. As indicated, this would be preferable to having comparative clinical effectiveness become part of the coverage decision, which is too heavy a burden to use going forward.
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:w589-w592. FCC (Federal Coordinating Council for Comparative Effectiveness Research)
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2007. Comparative clinical effectiveness and cost effectiveness research: Back ground, history and overview.
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2006. Developing a center for comparative effectiveness information.
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