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Institute of Medicine (US) Roundtable on Evidence-Based Medicine. Leadership Commitments to Improve Value in Healthcare: Finding Common Ground: Workshop Summary. Washington (DC): National Academies Press (US); 2009.

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Leadership Commitments to Improve Value in Healthcare: Finding Common Ground: Workshop Summary.

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3Transformational Opportunities

The second goal of the workshop was to identify and discuss potentially transformational opportunities for the sectors to help improve value from health care. The Roundtable on Evidence-Based Medicine’s vision for health care is a system that draws upon the best available evidence to provide the care most appropriate to each patient, emphasizes prevention and health promotion, delivers the most value, and adds to learning throughout the healthcare delivery system. Workshop participants felt that achieving this vision will require transformational change—from incentives aligned for the application and generation of the best evidence and a greater emphasis on wellness and disease prevention to the adoption of interoperable personal and electronic health record systems that support both individual patient care and improvement of the evidence base. Progress toward this long-term vision is possible only if reform efforts focus on broad, crosscutting initiatives that seek to catalyze transformative, systemwide change. For example, information gleaned as part of routine practice might be made more central to discovery, innovation, and research; the culture of health care might define new stakeholder roles and responsibilities to better support value in health care; and increased investment might produce comparative effectiveness information to better inform decision making by patients and health-care professionals about the risks and benefits of a particular treatment and the role of patient preference in determining courses of treatment.

As part of the discussion of sectoral strategies, workshop participants were asked to specifically consider what initiatives and opportunities represented the most promising approaches to bringing about transformative change within health care. During the 2-day workshop, individuals from each sector presented for discussion the highest-priority transformational opportunities identified by members of that sector. This chapter summarizes those opportunities and relevant workshop discussions not only to illustrate where leadership is needed but also to offer some possible priority items for immediate action.

Briefly, these items highlight the need for a set of principles and priorities and the need to address issues related to stakeholder engagement and capacity development. Each sector coordinator addressed the need to clarify certain core concepts, such as the value proposition in health care and principles for evidence stewardship. The establishment of a set of national priorities for evidence development and application was viewed by most as a means of coordinating stakeholder action. Similarly, participants from different sectors called for better streamlining and coordination of the research enterprise to produce more timely and relevant information. Finally, advancing healthcare informatics and shifting the culture of health care to support evidence-driven team care were often mentioned as essential for systemwide transformation. This chapter explores some of those opportunities in more detail.


Workshop participants viewed an effort to drive clarity and consensus around the principles and elements of the value proposition common among all stakeholders as a first priority for multistakeholder collaboration. Because participants cited increased value as a desired outcome of sector activities, a focus on value offers an opportunity to align stakeholder interests around a common goal, a step considered potentially transformative in building a greater sense of trust and a willingness of the sectors to collaborate. Initial discussions revealed substantial disagreement on what constitutes value in health care. It might best be expressed as improvements in physical and mental health and a sense of well-being. This means getting the right care at the right time to the right patient for the right price. However, what is “right” about care, time, and price depends on perspective and circumstances. What weight is given to clinical outcomes, increased productivity, improved safety, better service, innovation, or cost savings? The perceived importance of these outcomes varies according to one’s point of view as a patient, caregiver, family member, employer, healthcare manager, healthcare product developer, or regulator. Moreover, the determination of value is often complicated by the fact that a benefit received is the result of a cost shared or borne elsewhere. Although most participants believed that determination of the value proposition for a specific intervention must begin with an understanding of its relative safety and effectiveness across populations, others believed that innovations in healthcare delivery, advances in understanding genetic variations, and accounting for patient preferences are opportunities to create value at entirely new levels.

Although a product developer’s definition of value might differ considerably from an insurer’s, a patient’s, or a practitioner’s, a focus on defining value in health care is important to help frame priorities, set standards, and develop incentives that can produce the desired outcomes for the system as a whole. Cross-sector conversations about value were viewed as particularly important, given the number of reform efforts that focus on measuring and rewarding value. As noted above, defining core concepts in health care was viewed as essential to establishing a common ground. Defining value, in particular, was viewed as having a strong potential to improve stakeholder trust and advance the collaboration and cooperation needed for progress.


As noted in Chapter 2, increased transparency in the processes and decision-making rules used in health care will also promote trust and cooperation among stakeholders. The interpretation and use of evidence are particularly important, because the effectiveness and efficiency of health care depend on the quality and reliability of the underlying evidence base. Because the care, integrity, and timeliness with which medical evidence is produced, interpreted, and applied are fundamental to identifying, confirming, and improving on best practices for different circumstances, evidence stewardship is the shared responsibility of all stakeholders.

Guiding principles on the application of available evidence are needed not only to help decision makers determine when they should apply a proposed diagnostic or treatment intervention, but also to guide other processes informed by some interpretation of the evidence, such as market approval, insurance coverage, provider use, and patient acceptance. Such clarity is needed because without mutual understanding among all parties involved of the bases on which the evidence will be interpreted and applied, the generation of new evidence may result in little or no benefit to patient care. Without a mutual understanding, payers may lack consistency in their coverage decisions, regulators may request more studies, manufacturers may demur on product development because of what they see as ambiguous requirements and decision-making criteria, caregivers may be uncertain about the appropriate intervention, and patients may feel confused about what is best for them. All of these dynamics impede innovation, progress, efficiency, and the dissemination of results that should be expected from the healthcare delivery system.

The workshop discussion emphasized the importance of the need for guiding principles; participants noted that the term “evidence based” is increasingly used as a driver or benchmark for proposed reforms in guideline development, coverage decisions, and incentives in the healthcare system. For example, the potential impact of stronger linkages between evidence and incentives was the basis for a number of different efforts, including aligning existing policies to pay for care on the basis of the evidence, structuring reimbursement decisions to encourage widespread sharing of best practices and notable outcomes, designing benefits that link coverage to the determination of effectiveness and the strength of the evidence or that reward positive consumer behaviors supported by evidence-based medicine, and assigning preferential status to hospitals or physicians who meet evidence-based quality and safety standards.

In these discussions, some participants voiced concern over the potential implications for patient care, depending on how the evidence is interpreted and used. For example, disease-specific evidence or determinations of efficacy in a broad population cannot be generalized to all settings and all patients but nevertheless are often used to develop practice guidelines. If such evidence is linked too tightly with rewards and incentives, the care provided might not be appropriate for individual patients. Some participants believed that the interpretation and use of evidence should be structured so that individual care needs are not superseded by population-level evidence.

When coupled with the discussion of the myriad ways in which evidence might be used to transform health care, the uncertainty around what constitutes appropriate evidence underscores the need to establish principles and transparent processes to guide its interpretation and use. This was viewed as a precondition not only for coordination and synchronization of reform efforts but also for the effectiveness of such efforts in bringing about transformative change. Achieving a learning healthcare system requires not only a robust capacity to generate and apply new insights but also clear principles to guide the use of evidence. Using the neutral forum provided by the Roundtable, stakeholders could be queried on the key elements to be used in establishing a common set of principles for evidence interpretation and use.


Many issues confound the delivery of appropriate care to patients, but perhaps the most fundamental issue is the absence of information that could be used to guide treatment decisions. In most instances, the available information is insufficient or not appropriately organized to guide the selection of choices from among competing treatment options. Often, when guiding evidence exists, it remains unused. The identification of national priorities for evidence development and improved application of that evidence constitute a simple but potentially powerful step toward better use of the healthcare system’s collective resources. For example, which medical care dilemmas have the most pressing needs for better evidence to guide the selection of choices from among available and emerging diagnostic and treatment options? What proven best practices are not adequately applied, and what approaches are needed to accelerate the adoption and diffusion of best practices into care?

To tackle these challenges effectively, many participants supported the development of national priorities that would identify both specific interventions in need of research and current best practices that are underused in practice. Ideally, each list would be limited in length to keep its focus crisp and would be sufficiently flexible so that new items could be added as existing treatment options are addressed. The specific areas in need of exploration and prioritization are discussed in more detail below.

Many participants spoke in favor of a list of priority assessments—to identify specific research areas in which the need for clinical effectiveness information, that could be used to inform decision making, is the most acute. Such a list would form the basis of a cohesive plan and action agenda for an improved healthcare system. The process of developing inclusion criteria presents the opportunity to gather expertise from relevant sectors for collective identification of the most important answerable scientific questions. The resulting inventory would provide a common reference point for identifying key evidentiary uncertainties and gaps, as well as serve as a basis for identifying how stakeholders might initiate the needed studies. The latter is particularly important, given the current emphasis on understanding the comparative clinical effectiveness of new interventions but the limited resources available to support head-to-head assessments. Greater consensus provided by what several participants called a “national problem list” might allow the nation to focus limited resources on those interventions that are of the highest priority.

Taking advantage of existing resources will require not only a prioritization of areas in need of evidence development but also efforts to address the limited adoption and diffusion of many practice interventions of demonstrated benefit. Recent analyses have demonstrated not only that care varies significantly for reasons unrelated to appropriateness, but that even when the available evidence strongly supports a regimen of care—or best practice—such care is received, on average, only half of the time. To move toward a system that generates insights on what works best for whom and under what circumstances, work must focus on ensuring the ability of the healthcare system to select and use established best practices for care. The identification of a set of best clinical practices that are underapplied in practice would be an important first step in highlighting systems that effectively apply existing knowledge as well as the improvements needed by the healthcare system.

A set of national priorities not only would illuminate the most pressing common concerns, but also would provide a framework for illustrating the importance of a healthcare system oriented toward the application and generation of clinical evidence. Collaboration between sectors to help develop national priorities would serve to engage stakeholders in a common agenda and inform strategic decisions about who is responsible for generating evidence, synthesizing it, paying for its collection and interpretation, and ultimately ensuring that the evidence is translated into practice. In a world of limited resources for research and development, participants emphasized that priority setting would help stakeholders best apply the existing resources.


Evidence continually evolves over the life cycle of any one product or intervention. Beginning during the research and development phase for a product or intervention, investigators create evidence with which regulators may evaluate the safety and efficacy of the product or intervention for regulatory approval. Once approved for use, studies on effectiveness investigate the associated outcomes in clinical practice. In clinical environments, the effects of treatment with the product or intervention might vary because of differences within delivery systems or patient characteristics, such as genetic variation or multiple comorbidities, or for other reasons, such as level of adherence to the treatment or protocol. Some participants noted that studies conducted to assess various end points are often lengthy, resulting in a significant lag between when evidence is needed and when it is available, as well as significant additional expense. Moreover, with the rapid pace of technological changes in diagnostic and treatment patterns, incremental improvements present an additional challenge to the creation of evidence that is both timely and relevant.

Patients and providers are compelled to make healthcare decisions at specific junctures throughout the course of care, and because of the ever-evolving nature of evidence, most participants acknowledged that they often make decisions in the absence of sufficient information. As a result, many felt that any system designed to improve the way evidence is applied and generated for healthcare decision making needs to consider both how evidence should be produced for today’s decisions and how additional evidence should be developed and integrated throughout a product’s life cycle.

Engaging the Life Cycle of Interventions

Throughout the life cycle of an intervention, critical assessments and decisions occur at specific points in the care process (e.g., approval, coverage, and application). Facilitating innovation, access, and effective information gathering, while emphasizing patient safety, appropriate application, improved outcomes, and efficiency, will require a set of life cycle-oriented decision-making rules that have been more carefully considered than they are at present. In part, economic and policy incentives are needed to enhance the generation of new evidence. Approaches suggested included aligning purchasing incentives to value, using the reimbursement power of insurers and other financial incentives to generate new insights from medical care (e.g., coverage with evidence development), linking purchaser and payer decisions to performance incentives to support best practices and outcomes, and designing practical and immediately implementable solutions to support the generation of evidence.

Also emphasized were incentives for the better secondary use of the data that have been collected. Health data are collected routinely for financial and administrative purposes and are increasingly collected during the course of care through electronic health records and other mechanisms. However, the available data are not systematically used to assess the results of treatments employed in routine practice. In addition, regulatory change may be needed to obtain greater access to healthcare data. Although patient privacy should be adequately protected, a school of thought suggests that healthcare data represent a public good and therefore need to be made transparent and accessible to clinical researchers, albeit with stringent oversight. Proactive steps would have to be taken to ensure that access to patient healthcare data becomes more open. Suggestions for stakeholder action included a comprehensive review of privacy issues in the context of the practice of evidence-based medicine and the development of principles and standards for the proprietary treatment of data. Practical approaches are needed to improve the quality of data and the transparency of their stewardship. A February 2008 Learning Healthcare System workshop, Creating a Public Good: Clinical Data as a Basic Staple of Healthcare Improvement, explored the notion of clinical data as a public good and evaluated a variety of perspectives on the issue. Publication of the workshop summary is expected in 2009.

Another opportunity noted to make better secondary use of data might be the creation of a distributed healthcare data researcher network. Existing healthcare service delivery settings could make better use of the data already in their databases. Existing data obtained from real-world patient care could be used more widely as feedback for continuous improvement in clinical practice and for the generation of evidence. In addition, a more robust information technology infrastructure could likely lead to new ways of using the data that do exist and to wholly new ways of mining the data collected in the future. Keys to success in these endeavors might include stakeholder participation in developing data oversight principles and data governance procedures and the specification of a technical design that accommodates existing data systems, along with careful coordination of current federal and private data collection initiatives.

Building Capacity

To take advantage of the new streams of healthcare data generated throughout a product’s life cycle and to close the gaps in evidence of the relative effectiveness of various interventions, stakeholders also noted the importance of ensuring that a sustainable capacity for clinical effectiveness research is developed. Currently, the combined resources of the various public and private organizations involved in assessing clinical effectiveness meet a small fraction of the demand. The centrality of this problem to the quality and efficiency—the viability, according to some—of the nation’s healthcare system suggests that more investment is needed. Specific capacity needs include innovation in the development of tools and methodologies that can be used to ensure the quality and integrity of clinical studies, development of the human capital needed to conduct complex studies and analyses, and expansion of the infrastructure for research.

Better tools and processes for collection and analysis of the data required to generate evidence are needed. One way to expand the evidence base would be to find ways to improve the capture of data relevant to comparative effectiveness research and to informing clinical experience, in part by drawing more comprehensively on the expertise of researchers, health-care delivery systems, and healthcare professionals as well as by making more effective use of information technologies. More funding, a priorities list, and better clinical tools would all contribute to a goal of seeing that the generation of evidence throughout the life cycle of an intervention is more of a focus and a priority.

A central challenge to building capacity is the need for innovative approaches for generating evidence. For example, an in-depth evaluation of what constitutes best practices in using observational studies might be undertaken and guidelines developed. Key questions in this regard are how better insights can be obtained from the evidence available in clinical records, and what research innovations can improve their utility. Accordingly, participants emphasized the need for policies that address the broad interoperability of information technology across various clinical and research data systems and also ensure a modernized and updated approach to health information policies and management. Also needed is the development of a better understanding of when the use of different methods of evidence development, including observational and data-mining methods, is appropriate. A related challenge is the need to blend new ways of incorporating new types of knowledge into older research approaches. Today’s evidence interpreters, for example, need to meld traditional ways of gathering and assessing evidence with such emerging approaches as genomics, pharmacogenetics, imaging, and proteomics. The factoring in of new technologies is also part of this mix.

Discussants referenced the need to enhance the nation’s ability to conduct formal prospective comparisons of treatments in common use when observational studies alone are insufficient. Examples of methods that might be well suited to such comparisons include pragmatic clinical trials and cluster randomized trials, in addition to conventional randomized clinical trials. Investments in technology will also broaden the ability to generate evidence. For example, the Food and Drug Administration (FDA) is working to develop standards and processes that can be used to optimize the retrieval and analysis of information from electronic healthcare databases. FDA has also put forth the concept of establishing a sentinel network for safety surveillance, which relies on a sophisticated ability to mine electronic databases, and created the Critical Path Initiative, which was designed to modernize the scientific process that transforms a potential human drug, biological product, or medical device from a discovery into a medical product.

A Trusted Source

As the capacity to produce evidence—particularly the ability to engage the life cycle of products and interventions—expands, the information available to guide decision making may become overwhelming. Particularly in the era of the Internet, many patients and other decision makers readily access healthcare information, but a means must be developed to help navigate emerging information and guide decision makers. Participants spoke to the need for a trusted source of information that patients, providers, healthcare organizations, employers, insurers, and others can rely on to ensure that they have the best evidence available. An entity of this sort could also act as an evidence intermediary that is independent but engages all stakeholders in identifying gaps; setting priorities; establishing standards; and guiding the development, interpretation, and dissemination of evidence on clinical effectiveness. The utility of such an organization in unifying and coordinating the disparate voices and interests that currently speak for the status of clinical evidence was also noted. Some participants cautioned that care must be taken not to stifle innovation.


Information technology is key to a learning healthcare system, in improving both the generation and the application of evidence to improve health care. The value of medical informatics—where the information sciences, technology, and health care intersect—is its ability to track and link the many processes and actors involved in the healthcare system. Because of this capability, most participants spoke to informatics as one of the most important drivers of progress. Throughout the workshop, participants mentioned ways in which medical informatics could enhance the development of evidence (through learning networks and the use of information gleaned from linked databases, registries, and electronic and personal health records); the application of evidence (through clinical decision support systems that encourage best practices at the point of care and assist with complex decision making); stakeholder engagement (by giving patients, providers, and the public access to the best available information); and systemwide tracking and improvement (via feedback mechanisms, performance measurement, and rewards and incentives).

Fundamentally, broader access, system interoperability, and standards are essential for progress in evidence-based medicine. On the issue of access, virtually all commenters spoke to the need to expand the adoption of technology so that it is used as universally as possible across the whole health-care system. Such expansion would, ideally, include across-the-board access to repositories of medical knowledge, evidence-based guidelines, decision support systems in all healthcare settings, and the wider and more equitable distribution of medical technology. Having wide access to robust, fully functional electronic healthcare databases that can provide clinical decision support and link to research findings is seen as a critical goal. It was noted that stakeholder commitment is needed to provide the financial support to foster the wider application of evidence in clinical practice through the use of medical informatics.

The lack of compatibility in technology is a well-known impediment to progress. Frequent reference was made to the need to facilitate clinical information technology standards and a common information technology vocabulary, as well as to standardize data collection, metrics design, and the development and application of formulas. The development of these and other global standardizations will help create a common understanding of the data among all users.

Today, some organizations already have the ability to aggregate data across practice sites and times and to understand in real time what is working or not working for patients. They also have the potential to use information technology to deliver evidence at the point of care and to use data from their large patient healthcare information databases to add to the evidence base. These are all positive trends, but the sense at the workshop was that wider access to these capacities is needed urgently and that further development is necessary to improve the existing medical informatics technologies and develop new capabilities.

Beyond the challenge of interoperability, participants also pointed out that electronic medical record systems should be improved. They need to be made more sophisticated and universal in design and to provide more comprehensive information. They should be linked more effectively to essential data in ways that readily help researchers connect the pieces of evidence in their assessment of individual datum points. Electronic health records have to be redesigned to reduce the need for entry of redundant data, to provide recommended practices, and to accommodate a common language once such language standards have been developed. There is also the need to ensure wider, universal access to electronic health records by healthcare professionals; increased investment is needed to advance this goal.

Medical informatics can provide important infrastructure for evidence development and application. However, to have an impact on practice and health, several systems changes are also required. Specific needs include guidelines for the use of evidence-based medicine in healthcare settings, more investment by government and insurers in providing access to electronic health records and in utilizing medical informatics, enhanced guidelines to increase the use of evidence-based practice recommendations, strategies to motivate and support expertise in the use of these practices among healthcare professionals, and better practical training in such practices for students in the medical and healthcare professions, including practice competence for licensure and certification.


The need to coordinate the many different actors and expertise in a healthcare system reflects the complexity of delivering the appropriate care to individual patients that often spans disciplines, organizations, and various levels of intensity. Participants emphasized that to ensure the best health outcomes, not only must healthcare practice be evidence-driven but the culture of care itself must shift to reflect and embrace the complexity of patient care. Rather than the current hierarchical approach, the provision of patient-focused health care would be coordinated by a team of healthcare professionals. In this approach, leadership and expertise might vary based on specific needs. Although the use of interdisciplinary teams may be an effective method for the delivery of evidence-based care, institutionalization of this approach will require a significant shift in the culture of health care. The fundamental nature of evidence is that it is constantly evolving, which creates a need for continuous learning to be built into the system. There is thus a need for reevaluation of the various roles and responsibilities of healthcare professionals and for stronger clinical education—both ongoing training for practitioners and better training for students.

The background paper for the healthcare professions sector in particular emphasized the importance of facilitating greater collaboration and discussion among the various professions. Collaborative work might focus on identifying opportunities in education, credentialing, and practice to encourage team approaches to the provision of evidence-based care. Greater healthcare provider engagement in these issues will be critical to embedding evidence into the structures and processes used to deliver health care. As a practical first step toward achieving this goal, participants proposed the convening of a coalition of healthcare provider groups to discuss the approaches, incentives, and supports needed.

Patient involvement in and engagement with providers’ decision making have to be enhanced, and stakeholders should create and implement strategies that support this goal. The better availability of decision support tools for patients would also help encourage patient involvement. The goal of helping patients become more engaged in the management of their own care will require enhanced physician-patient communication and better exchange of information, including information about the best treatment options. In this context, patients need to be able to access their personal health information and evidence-based decision support programs more easily. To help ensure the centrality of the patient, sector participants suggested that providers develop skills that allow them to assess their patients’ preferences and communicate better with them. Better communication will require providers to have tools, strategies, and even specific messages that will help improve provider-patient communications, particularly in ways that would keep those conversations focused on the evidence and inform physician and patient decisions with the available evidence.

Copyright © 2009, National Academy of Sciences.
Bookshelf ID: NBK52845


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