Summary

This volume reports on discussions among multiple stakeholders about ways they might help transform health care in the United States. The U.S. healthcare system consists of a complex network of decentralized and loosely associated organizations, services, relationships, and participants. Each of the healthcare system’s component sectors—patients, healthcare professionals, healthcare delivery organizations, healthcare product developers, clinical investigators and evaluators, regulators, insurers, employers and employees, and individuals involved in information technology—conducts activities that support a common goal: to improve patient health and well-being. Implicit in this goal is the commitment of each stakeholder group to contribute to the evidence base for health care, that is, to assist with the development and application of information about the efficacy, safety, effectiveness, value, and appropriateness of the health care delivered.

Because the nation falls far short of the possible in this respect, the Institute of Medicine (IOM) Roundtable on Evidence-Based Medicine was established in 2006 as a unique and neutral venue where the key stakeholders could work cooperatively to help transform the way in which evidence on clinical effectiveness is generated and used to improve health and health care and to drive improvements in the effectiveness and efficiency of medical care in the United States (Fisher, 2005; IOM, 2007; McGlynn et al., 2003; Wennberg et al., 2002).

The planning committee’s role was limited to planning the workshop, and the workshop summary has been prepared by the workshop rapporteur and Roundtable staff as a factual summary of workshop discussions



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Summary This volume reports on discussions among multiple stakeholders about ways they might help transform health care in the United States. The U.S. healthcare system consists of a complex network of decentralized and loosely associated organizations, services, relationships, and participants. Each of the healthcare system’s component sectors—patients, healthcare profes- sionals, healthcare delivery organizations, healthcare product developers, clinical investigators and evaluators, regulators, insurers, employers and employees, and individuals involved in information technology—conducts activities that support a common goal: to improve patient health and well- being. Implicit in this goal is the commitment of each stakeholder group to contribute to the evidence base for health care, that is, to assist with the development and application of information about the efficacy, safety, effectiveness, value, and appropriateness of the health care delivered. Because the nation falls far short of the possible in this respect, the Institute of Medicine (IOM) Roundtable on Evidence-Based Medicine was established in 2006 as a unique and neutral venue where the key stake- holders could work cooperatively to help transform the way in which evi- dence on clinical effectiveness is generated and used to improve health and health care and to drive improvements in the effectiveness and efficiency of medical care in the United States (Fisher, 2005; IOM, 2007; McGlynn et al., 2003; Wennberg et al., 2002). The planning committee’s role was limited to planning the workshop, and the workshop summary has been prepared by the workshop rapporteur and Roundtable staff as a factual summary of workshop discussions 

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2 LEADERSHIP COMMITMENTS TO IMPROVE VALUE IN HEALTH CARE Central to the Roundtable’s work are the notions that, collectively, the healthcare sectors possess the knowledge, expertise, and leadership necessary to transform the healthcare system and that what is most acutely needed is a shared commitment to improving the development and use of information about the efficacy, safety, effectiveness, value, and appropriate- ness of the health care delivered. Roundtable members have developed a vision for a learning healthcare system needed to achieve their goal: by the year 2020, 90 percent of clinical decisions will be supported by accurate, timely, and up-to-date clinical information and will reflect the best avail- able evidence. Fostering the collaborative work necessary to achieve this goal is the aim of the IOM Roundtable’s sectoral strategies process, which took place through the activities of nine sector-specific groups over several months in 2007 and culminated on July 23 and 24, 2007, in the third workshop in the Learning Healthcare System series, titled Leadership Commitments to Improve Value in Health Care: Finding Common Ground. The ideas pre- sented and discussed at the workshop are summarized here. The three goals of the workshop were (1) to consider stakeholder capacity for stronger progress toward a learning healthcare system; (2) to explore transforma- tional opportunities; and (3) to identify possibilities for collective initiatives that might be considered by Roundtable sectors. In the months before the workshop, Roundtable members were asked to reach out to colleagues in their sectors to describe perspectives on the key challenges and opportunities for healthcare improvement, as well as how each sector might contribute to advancing progress toward the Roundtable’s goal. Background papers summarizing these discussions were prepared and presented at the July workshop to provide context for cross- sector discussions. The elements of this process are presented in Appendix A and summarized as follows January: the initial formation of nine Roundtable sectoral discus- • sion groups February and March: reaching out to other sectoral participants in • preparing background material April: completion and circulation of strategy background paper to • sector participants May: circulation of sector review draft to Roundtable members in • each sector group June: consolidation of draft sectoral strategy background papers • and dissemination to all Roundtable members July: presentation of authored background papers for public dis- • cussion at an IOM workshop on sectoral strategies

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 SUMMARY The workshop presentations of these sector-oriented perspectives were designed by presenters to highlight their views of the key advances, transformational opportunities, and cross-sector collaborations needed to achieve the stated goal. The nine sectoral discussion groups were not Academy-appointed com- mittees, and there was no attempt to ensure group consensus. The back- ground papers reflect the views of the individuals who participated in the discussions prior to and during the workshop, as interpreted by the group coordinators and staff. The purpose of this summary is to present lessons from experience; out- line the range of key issues, stakeholder concerns, barriers, and challenges; and offer some potential responses as described by workshop participants. This chapter briefly summarizes workshop presentations, discussions, and relevant background materials and their relation to the workshop goals and to the overall Learning Healthcare System series of meetings. It has been prepared in consultation with the authors of sectoral background papers and reviewed independently by a committee appointed by the National Research Council to ensure that it is accurate and faithful to the meeting’s purpose and content. It does not, however, represent an Academy consensus document, nor does it contain recommendations. Later chapters describe the presentations in more depth. The greater part of workshop discussions focused on finding areas of common ground in which participants might join together on activities most important to the improvements necessary to fulfill the Roundtable’s goal. Part One of this publication, Finding Value in Common Ground, presents a synthesis of the workshop discussions in the context of the Roundtable’s focus and the workshop goals: the perspective guiding the sectoral strate- gies process (Chapter 1), important conceptual foundation stones needed for progress (Chapter 2), transformational opportunities recognized by participants as priority areas for focus and immediate work (Chapter 3), and areas for enhanced cross-sector collaboration (Chapter 4). Drafts of the authored background papers were revised to incorporate the workshop discussions, and final versions are included in their entirety in Part Two of this publication, Leadership Commitments to Improve Health Care (Chapters 5 to 13). Appendix B provides the workshop agenda, Appendix C presents biographical sketches of the speakers, and Appen- dix D lists the workshop participants. COMMON CONCERNS AND THEMES The spirit of the workshop discussion was one of open exchange, and over the course of the 2-day meeting, participants underscored many press- ing concerns common to all sectors, the following in particular:

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 LEADERSHIP COMMITMENTS TO IMPROVE VALUE IN HEALTH CARE Rising costs and limited resources. Whether they are borne by those • receiving or providing care or accrued during research on or the development of treatments and therapies, participants cited costs as limiting factors for access to and innovation in health care. System inefficiencies. The quality of health care in the United States • is uneven and delivered by a system characterized by inefficiency and waste. The existing evidence is poorly applied, and the delivery of care for similar conditions varies widely throughout the coun- try. Standards for care, healthcare system components, and even research are often inconsistent. Increasing complexity. Whether it is because of the increased • importance of genetic variation, the rapidly evolving landscape of medical technologies, or the growing prevalence of chronic disease, medicine is becoming increasingly complex. Expanding evidence gap. Across the practice of health care, infor- • mation is lacking for many key personal health or policy deci- sions. The “inference gap” between the evidence available and that needed to treat real-world populations will only widen as new interventions are introduced into the marketplace and health care moves further in the direction of personalized treatments. Limited system capacity and flexibility. The number of questions • that need to be addressed to ensure appropriate care continues to expand exponentially, rendering impractical the current approach to the development of evidence. Although randomized controlled trials are important in certain circumstances, they cannot provide all the information necessary. The availability of technologies lags the demand. Whether through habit or other circumstances, evi- dence is neither getting translated to the extent that it needs to be nor distributed as widely as it should be. Entrenched cultures. Health care has various customs and prac- • tices often not conducive to reform. Caregiving and caregivers are often “siloed,” with inadequate communications among the various functional areas of the healthcare system. Information is not shared as widely as it should be within specific healthcare systems, let alone between systems, contributing to inefficiency and distrust in the system. In general, providers, patients, and other sectors do not yet believe that the development of evidence is an activity relevant to their experience in the routine delivery of care. Several general themes were recurrent over the course of the meeting as issues important across stakeholder categories (Box S-1).

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 SUMMARY BOX S-1 Common General Themes • Build trust and collaboration • Foster agreement on “value” in health care • Improve public understanding of evidence • Characterize the impact of shortfalls in the evidence • Identify the priorities for evidence development • Improve the level, quality, and efficiency of the research • Clarify and promote transparency • Establish principles for the interpretation and use of evidence • Improve engagement in the full life cycle of interventions • Focus on frontline providers • Foster a trusted intermediary for evidence • Build the capacity to meet the demand • Create incentives for change • Accelerate advances in health information technology Build trust and collaboration. How can the distrust that has • emerged in health care—for example, distrust between and among patients and providers, providers and insurers, insurers and manu- facturers, and manufacturers and regulators—be reduced? Health care depends for its effectiveness on the close cooperation of all parties involved. Building trust and facilitating transformative change will require broader-based collaboration and cooperative stakeholder engagement. Foster agreement on “value” in health care. What constitutes value • in health care: reduced death or disease, better function, less pain, a better sense of well-being, fewer hospital days, or lower costs? Although all participants agreed on the centrality and importance of the value achieved from health care, different groups think of value in different ways. A multistakeholder effort might drive clarity and consensus on the principles and elements of value common to all stakeholders. Improve public understanding of evidence. What can be done • to improve public understanding, acceptance, and demand for evidence-based care? Too often, people perceive that certain common terms such as “evidence based,” “research,” “medical necessity,” and “risk” suggest a restrictive or experimental element to their care. It will take a systematic and coordinated communi-

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 LEADERSHIP COMMITMENTS TO IMPROVE VALUE IN HEALTH CARE cation strategy to better convey the central concepts that medical evidence is dynamic, that evidence-based medicine is the provision of care that the evidence suggests is best for any given patient at any given time, and that health care is a joint patient-provider endeavor. Characterize the impact of shortfalls in the evidence. What might • be the tangible impact of broad improvements in the availability and application of appropriate evidence for healthcare decisions on patients, on providers, and on society? Documenting the con- sequences of provision of care on the basis of too little evidence or the potential benefits of providing care on the basis of the right evidence is a prerequisite to obtaining an improved under- standing of and demand for evidence-based care and stakeholder activation. Identify the priorities for evidence development. Which medical • care dilemmas represent the most challenging and pressing needs for better comparative information and guidance on choices among the available and the emerging diagnostic and treatment options? The first step toward a systematic and coordinated effort to con- duct the most important assessments is identification of priorities as a sort of consensus national problem list and research agenda for the most pressing issues for medical care decisions. Improve the level, quality, and efficiency of research. How can • the healthcare system take better advantage of emerging clinical record resources to gain insights into the evidence? Policies that facilitate the ability to use clinical data to monitor the effectiveness of interventions are needed. Novel approaches to the conduct of clinical trials are also needed. A more structured lexicon for “best practices” in undertaking observational studies may be necessary. Clarify and promote transparency. What principles define openness • in health care, clinical research, the interpretation of evidence, cov- erage decisions, regulatory policy, marketing practices, oversight, and the governance of use of clinical data? Consensus is needed to establish common principles of transparency and standards for how they should be applied in each sector. One starting point might be with principles for evidence interpretation. Establish principles for the interpretation and use of evidence. • What guiding principles related to application of the available evidence might be used to help decision makers determine when they should apply a proposed diagnostic or treatment intervention? Decisions about market approval, insurance coverage, provider use, and patient acceptance are all informed by some interpretation of the evidence. Clarity of the guiding principles is important.

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 SUMMARY Improve engagement in the full life cycle of interventions. How • should assessments and decisions on proposed healthcare services be tailored to ensure that each stage of the development and appli- cation process for a given intervention builds efficiently to the next? Many factors are at play for each intervention—for example, similarity to previously tested interventions, the safety and effec- tiveness of an intervention for some populations but not others, the availability of biomarkers predictive of efficacy, and costs that vary by scale and stage of application or by the need for later services. Facilitating innovation, access, and effective information gathering while emphasizing patient safety, appropriate applica- tion, improved outcomes, and efficiency will require a set of life cycle-oriented decision-making rules that are more carefully con- sidered than they are at present. Focus on frontline providers. What key levers might help ensure • that both primary care and specialty providers are taking full and appropriate advantage of the best available evidence in the care they provide? Accelerating the translation of clinical research into practice involves addressing matters of professional education, cre- dentialing, licensure, practice support, economic incentives, patient acceptance, and the culture of care. It will require the central and coordinated involvement of the organizations that represent those providers. Foster a trusted intermediary for evidence. How can patients, pro- • viders, healthcare organizations, employers, insurers, and others know when they have the best evidence on which to base the healthcare decisions they make? In this information age, health- related information is presented constantly through news reports, marketing, professional organizations, journals, and the Internet; but it is often confusing and even contradictory. A trusted infor- mation source—one that is independent but that engages all stakeholders—is needed to identify gaps; set priorities; establish standards; and guide the development, interpretation, and dissemi- nation of evidence on clinical effectiveness. Build the capacity to meet the demand. What mechanism is nec- • essary to close the current and emerging gaps in evidence on the relative effectiveness of various interventions, to ensure the qual- ity and integrity of the studies used to establish the evidence, and to provide a sustained capacity to meet the need? Currently, the combined resources of the various public and private organizations involved in studying comparative clinical effectiveness meet but a small and scattered fraction of the demand. The centrality of this problem to the quality and efficiency—the viability, according to

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 LEADERSHIP COMMITMENTS TO IMPROVE VALUE IN HEALTH CARE some—of the nation’s healthcare system may require the creation of a new independent entity devoted to the work. Create incentives for change. What practice-based economic and • policy incentives might help enhance the next generation of new evidence and transform the ability and commitment of providers to use the best available evidence and more fully engage patients in the clinical decision-making process? Approaches include the align- ment of purchasing incentives when value is determined; use of the reimbursement power of insurers and other financial incentives to generate new insights from medical care (e.g., coverage with evidence development); and the linkage of purchaser and payer decisions to performance incentives for best practices, outcomes, and the better secondary use of routinely collected data. Accelerate advances in health information technology. What can • stakeholders do to accelerate the nation’s progress toward the goal of the universal application of interoperable—or functionally accessible—personal and organizational electronic health records, as well as toward the goal of providing real-time electronic access to the best information available? Health information technology can facilitate the development of learning networks and accelerate the generation of evidence, enable data aggregation and utilization, deliver evidence to the point of care, and expand research capaci- ties. Coordinated stakeholder action—and financial incentives— should be able to speed the progress necessary on both the basic interoperability issues (e.g., standards and vocabulary) and, pos- sibly, the development of more radical data search innovations. Several opportunities for collaborative activities by Roundtable members and participating sectors were identified by participants in the discussions: • Development of a priority assessment inventory. Termed a “national problem list” by meeting participants, this is a multisector collab- orative effort to develop criteria and a list of the diagnostic and treatment interventions that might be viewed as particularly impor- tant for the development of comparative effectiveness studies. The list will serve as a means of illustrating and prompting discussion on the key evidence gaps and on the design, support, and execution of the studies needed. • Pursue agreement on the value proposition. Identify key concepts and elements to be considered in assessing and characterizing value from health care, setting the stage for discussions on approaches to assessing those elements and applying to add perspective and inform decision making. An IOM workshop, Value in Healthcare:

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9 SUMMARY Accounting for Cost, Quality, Safety, Outcomes, and Innovation, was convened in November 2008, with publication of the work- shop summary expected in 2009. • Identify common principles for evidence interpretation and use. Identify the core principles underpinning activities in interpretation and use of evidence, as background for discussion of the implica- tions and of the ways the principles might be applied in the devel- opment of a framework adaptive to different circumstances related either to the evidence base or the condition of interest. • Foster cooperative data sharing. Several issues are important in this regard: platform compatibilities, standards, economic incen- tives and disincentives, the regulatory and privacy environment. Health Insurance Portability and Accountability Act issues are being addressed by an IOM Committee expected to issue its report and recommendations in 2009, including those related to the use of clinical data for knowledge development. The Roundtable’s Febru- ary 2008 meeting, Clinical Data as the Basic Staple of Healthcare Learning: Creating and Protecting a Public Good, addressed a number of the other issues related to sound data stewardship. And collaborative work has been sponsored by the Roundtable on mining electronic health records for postmarket surveillance and clinical safety and effectiveness insights. • Pursue a public communication initiative on evidence-based medi- cine. Use the Roundtable membership’s collective communication expertise to explore improving terminology and advancing public awareness on the nature and importance of evidence in medical care, the key needs, and the centrality of patient and provider communication around the state of the evolving evidence for indi- vidual treatment choices. The Roundtable’s Evidence Communi- cation Collaborative has a working group actively working on a communication initiative proposal. • Support progress on a trusted intermediary for evidence promotion. The Roundtable’s Sustainable Capacity working group oversaw the development of a comprehensive Issue Brief, framing the issues and options under discussion related to enhancement of the national capacity to develop, evaluate, organize, validate, and disseminate information on the comparative effectiveness of health interven- tions. Technical assistance and related information is provided on an ongoing basis to the various policy discussions of the issue. • Identify the potential from best practices in the use of evidence. It is important to assess and underscore the best practices in evidence development and application, including consideration of ongoing methods of identifying and disseminating those best practices. A

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0 LEADERSHIP COMMITMENTS TO IMPROVE VALUE IN HEALTH CARE working group is underway to characterize the potential returns from implementing certain established best practices. • Enlist front-line healthcare providers more effectively. Charge the sectoral working group on providers with proposing approaches to convening a coalition of provider groups, perhaps under Round- table auspices, to consider sustained, coordinated work on health professions education, testing, credentialing, and practice setting tools and structure to improve focus, accessibility, use, and genera- tion by providers of the best evidence. A Roundtable collaborative of providers is being formed to engage this issue. PART ONE: FINDING VALUE IN COMMON GROUND The Learning Healthcare System series of workshops sponsored by the IOM Roundtable on Evidence-Based Medicine is designed to both identify and discuss the most important advances needed to transform health care, with sector leadership and collaboration in this work. This workshop was aimed at considering how various sectors could make a difference. The sectoral strategies perspective development process engaged participants in the development of authored background papers to be presented during the workshop, as a first step toward the national conversation needed on how to facilitate a better alignment and better collaboration among the vari- ous sectors of the healthcare system. Part One provides a synthesis of the workshop discussion among participants from different sectors. The mate- rial presents the individual views of the participants of the workshop and does not represent the consensus of the discussion groups, the workshop participants, the Roundtable, or the IOM. Guiding Perspective: The Learning Healthcare System The Roundtable’s goal for 2020 specifies what ought to be expected from a healthcare system that “draws upon the best evidence to provide care that is the most appropriate for each patient, emphasizes prevention and health promotion, delivers the most value, adds to learning throughout the delivery of care, and leads to improvements in the nation’s health.” Central to this vision is the development of a healthcare system that learns by generating and applying evidence as a natural component of the process of providing health care (IOM Roundtable on Evidence-Based Medicine, 2006).

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 SUMMARY The Learning Healthcare System The guiding perspective for the Roundtable’s vision and for the develop- ment of background papers from each sector is that of a learning healthcare system. Its key characteristics were discussed at the Roundtable’s inaugural workshop and summarized in the annual report of the Roundtable, Learn- ing Healthcare System Concepts v. 200 (Institute of Medicine, 2008): Continuous improvement in the value delivered. A learning health- • care system is one that maintains a constant focus on the health and economic value returned by care delivered and continuously improves in its performance. Learning in health care as a partnership enterprise. Broad cul- • ture change is needed to enable the evolution of the learning environment as a common partnership of patients, providers, and researchers alike. Developing the point of care as the knowledge engine. Given the • rate at which new interventions are developed, along with new insights about individual variation in response to interventions, the point of care must be the central focus for the continuous learning process. • Full application of information technology. The rate of learning— both the application and the development of evidence—will depend on the full and strategic application of information technology, including electronic health records central to long-term change. • Database linkage and use. The emergence of large, electronically based datasets offers important new sources for quality improvement and evidence development. Progress requires fostering interoperable platforms, linking analyses, establishing networks, and developing new approaches for ongoing searching of those databases for pat- terns and clinical insights. • Advancing clinical data as a public utility. Meeting the potential for using new datasets as central sources of evidence on the effec- tiveness and efficiency of medical care will require recognition of their qualities as a public good, including assessing issues related to ownership, availability, and use for real-time clinical insights. • Building innovative clinical effectiveness research into practice. Improving the speed and reliability of evidence development requires fostering development of a new clinical research paradigm—one that deploys careful criteria for trial conduct, draws clinical research more closely to the experience of clinical practice, advances new study methodologies adapted to the practice environment, and engages cultural incentives to foster more rapid learning.

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2 LEADERSHIP COMMITMENTS TO IMPROVE VALUE IN HEALTH CARE As entities at the interface of patients, providers, and payers, healthcare delivery organizations have a unique opportunity to identify and imple- ment the needed change. The background paper from this sector empha- sized the interest in creating a national entity to develop and disseminate needed evidence, increasing the demand for evidence-based care through communication efforts, and increasing the support for evidence-based care through improved linkage of evidence with performance standards and incentives. Finally, large healthcare delivery organizations can lead the way in the adoption of electronic health records as well as encouraging their broader adoption by smaller physician groups through the provision of technical assistance and expertise and the provision of assistance with the establishment of learning networks of organizations that have implemented electronic health records to disseminate knowledge to all providers—both organized and nonorganized. Healthcare Product Developers More than 20,000 companies worldwide produce more than 80,000 brands and models of medical devices and diagnostics for the U.S. market. The biopharmaceutical portion of the market includes more than 2,000 companies worldwide that collectively introduce 25 to 30 new innova- tive products each year and that currently have some 2,000 products in development. In bringing pharmaceuticals to the market, developers often invest an average of 15 years and more than $800 million. By conducting high-quality clinical research to meet regulatory requirements, to adhere to coverage and coding policies, and for clinical decisions, product developers play a pivotal role in the development of evidence. Likewise, participants from the health product sector discussed their work to interpret evidence to meet formulary access and coverage requirements and to encourage the application of evidence through the dissemination and communication of information on specific clinical issues to providers, patients, and payers. Because the tasks of developing and translating evidence into practice are core capabilities of the industry, members of this sector underscored their potential to add far more value to healthcare delivery and the appropriate use of medications and devices than has yet been realized. Several key questions were presented as pivotal in developing a system that better generates and uses evidence in healthcare decision making: who will decide the priorities for more evidence, who is responsible for generat- ing evidence, who is responsible for synthesizing evidence, who pays for the generation and synthesis of evidence, and who is responsible for ensuring that evidence is translated into practice? Thus, three key transformational oppor- tunities were identified by participants for this sector’s work: (1) establishing principles for interpretation of the evidence, (2) developing new methods for

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2 SUMMARY the generation of evidence, and (3) accelerating the application of evidence by identifying behavioral approaches that speed the translation of clinical research into practice, thereby driving the learning healthcare system. The extensive experience of this sector across the spectrum of activities related to the development and application of evidence creates numerous opportunities for collaboration. As critical stakeholders, product developers believe that they can contribute extensively to discussions related to the interpretation and use of evidence. Their understanding of evidence devel- opment and the potential methodological or data limitations, for example, could be used to inform the development of standards of evidence for prod- uct approval, healthcare policy decision making, and patient care decisions. Other relevant conversations might focus on the development of principles governing how evidence is integrated into coverage decisions; the develop- ment of best-practice standards for interpretation of the evidence; and edu- cation about the uncertainties of decision making from studies conducted with nonrepresentative populations. The sector’s methodological expertise was also noted as potentially useful to discussions about how to develop evidence that better addresses individual patient needs. In the area of application of the evidence, the sector paper emphasized the needed development of a process for setting coverage and payment poli- cies that are open, transparent, and trustworthy; that consider a wide range of relevant evidence; and that can help foster a better understanding of real- world data requirements for the developers of clinical practice guidelines. Additionally, communications expertise developed through investments in sophisticated advertising offers possible utility in the development of refined methods of communicating evidence to consumers to assist with (1) consumer-based decision making and (2) the development and imple- mentation of a research agenda focused on systems changes and behavioral approaches important to improving the translation of evidence-based guide- lines into clinical practice and adherence to therapeutic regimens. To achieve all of these goals, discussions of healthcare product developer opportunities recognized the need to partner with many stakeholders, particularly the patient sector, healthcare delivery organizations, clinical investigators and evaluators, insurers, and regulators. Clinical Investigators and Evaluators The work of clinical investigators and evaluators includes quantitative and qualitative evaluations of specific healthcare interventions, projects that improve population health, cost-benefit analyses, and organizational studies. The principal activities of the sector include those involved in evidence development: design and implementation of clinical trials and registries, database development, study reviews, standards development,

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2 LEADERSHIP COMMITMENTS TO IMPROVE VALUE IN HEALTH CARE evaluation of the application of evidence in clinical practice, methodology development, and modeling and simulation studies. Sector discussants identified several important and overarching chal- lenges. Resources for research and development are limited both in terms of participation in research activities by healthcare providers and the public and in financial terms. Investigators must also contend with what is char- acterized as the inefficient use of existing data—including, for example, the inefficient secondary use of data. Systemic constraints inherent in the way healthcare organizations deliver care or use information are barriers to the creation of new evidence. Needs for new and improved research method- ologies permeate the sector’s work. Sector presenters suggested several major transformational initiatives if clinical investigators are to promote a learning healthcare system: improved and sustained investment in applied research and development; reengineer- ing of healthcare delivery to facilitate structured learning about best prac- tices; use of information developed during the routine delivery of health care to assess outcomes; clarifying the ways in which outcomes assessment can be performed in compliance with HIPAA regulations; better standard- ization of institutional review board practices; greater interaction between regulators, payers, and investigators in the generation of evidence; and the development of new policies and approaches concerning advanced cover- age for new therapies. Of central importance is expanding the use of a broad range of clinical research designs to compare approved treatments. Examples of these designs include not only conventional RCTs but also large pragmatic trials and cluster randomized trials. To allow this expan- sion, creating an environment in which both providers and patients see participation in such trials as an expected, desirable activity was considered especially important. To improve data quality, use, and access, presenters emphasized the development of improved database architectures, policies, and governance procedures. Finally, participants felt that increased activity is needed to expand the workforce of trained investigators and evaluators and to develop innovative methodologies that will improve research quality and accelerate the translation of evidence into practice. Regulators Federal agencies, including FDA and CMS, regulate different aspects of the healthcare system: the former regulates the introduction and use of medical products, whereas the latter regulates the quality of care through its reimbursement decisions for healthcare products and services under Medicare and Medicaid. With expenditures of approximately $650 billion in 2006 and serving more than 90 million beneficiaries, CMS naturally plays a key role in the overall direction of the healthcare system, particu-

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29 SUMMARY larly through management of coverage decisions, payment structures, and accountability measures under Medicare. With Medicaid, although CMS sets global policy, each state has substantial flexibility to determine the final form of the program as carried out in its jurisdiction. Moreover, the states directly regulate the practice of medicine, the healthcare workforce, and the commercial (including nonprofit) health insurance that is purchased from health insurance companies or commercial managed care health plans. Participants noted that regulators share a critical mandate and interest in the safety and effectiveness of the pharmaceuticals, devices, and services used in medical care. Because regulators are responsible for assessing medi- cal products at various points in their life cycles, they collect and analyze substantial amounts of data to evaluate whether a product is safe and effective for its indicated use. As the nation moves toward personalizing treatment, those developing the background paper for the regulatory sector underscored the centrality of a better evidence base to their work. FDA’s contribution to this effort resides primarily in its ability to improve the quality and type of evidence generated during the early phases of a medical product’s life cycle, as well as to improve the development, communication, and use of risk information throughout a product’s life cycle. CMS’s key contribution—at the state and federal levels—lies in its ability to leverage the broad healthcare system through the implementation of initiatives and incentives that advance evidence-based medicine, as well as the potential value of coverage requirements to generate new evidence Unique challenges in regulation lie in the tension between innovation and access to new therapies and the need for greater evidence about per- formance. For example, the FDA Critical Path Initiative includes programs aimed at reengineering and streamlining clinical trials, facilitating better understanding of product performance, hastening the implementation of personalized medicine, increasing the quality and quantity of information that can be derived from clinical trials and other data analyses, and easing administrative and other burdens associated with the conduct of complex, multisite studies. In addition, work is under way to modernize manage- ment of the nation’s medical product safety information by developing a national electronic standard for a medical product adverse event report, called the individual case safety report, as well as laying the foundation for a sentinel system, a national postmarketing surveillance system. CMS uses coverage with evidence development policies to accelerate the development of evidence and has several other programs aimed at modernizing and supporting the use of information technology capabilities. Projects under development include Lifecycle Evidence Development, which embodies a substantial culture change by way of continuous data acquisition, evalua- tion, and response to findings, and the Chronic Condition Data Warehouse,

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0 LEADERSHIP COMMITMENTS TO IMPROVE VALUE IN HEALTH CARE a new research resource. CMS is also working to build pay-for-performance incentives into its payment systems. Certain initiatives emphasized by participants focused on the state level. With Medicaid coverage decisions made locally, for example, it is anticipated that states will continue to work with each other and with not- for-profit organizations, federal partners, and others—as they are doing now—to expand the use and availability of evidence in clinical and admin- istrative decision making. Increasingly, deliberations among state insurance policy makers are shaped by the inclusion of research evidence, as is work to articulate state standards of effective medical practices. State regulators are playing a catalytic role in moving toward a more consistent, relevant, and accessible approach to measuring and communicating information about a physician’s competence throughout his or her career. The regulatory sector presentation focused on two key possibly trans- formational initiatives. First, a national think tank or large national col- laborative effort is considered important to identify evidence needs, agree on priorities, and assign projects to fill gaps in the evidence knowledge base. Second, a national problem list or national research agenda is needed both to illustrate the pressing need for more evidence development and to identify areas and projects of priority for research on the basis of the most significant evidence gaps in health care today. Insurers In 2005, private health insurance plans and other private spending, including consumers’ out-of-pocket costs, accounted for almost 55 percent of total U.S. healthcare expenditures (approximately $1.09 trillion of $2 tril- lion). Public spending, including spending by Medicare, Medicaid, the State Children’s Health Insurance Program, the U.S. Department of Defense, and the U.S. Department of Veterans Affairs health benefits program, accounted for the remaining 45 percent of total healthcare expenditures. The insur- ance industry operates in a variable and volatile marketplace. After a period of relatively low cost increases in the mid-1990s, healthcare costs again began to rise, resulting in the growth of health insurance premiums that peaked at 13.9 percent in 2003. The number of uninsured Americans grew during the same period, rising from 14 percent in 2000 to 15.3 percent in 2005. Although the growth in premiums slowed during from 2003 to 2006, healthcare costs continue to outpace inflation and place significant pres- sure on the cost of insurance coverage, as evidenced by the approximately 46 million Americans who remain uninsured and the recent increase in the numbers of uninsured or inadequately insured. Presentation and discussion noted that, in addition to the general chal- lenges related to rising costs, waste, and the provision of ineffective care

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 SUMMARY that characterize the healthcare system, insurers face issues related to new treatments and higher-priced technologies; the increased bargaining power of providers; increased consumer demand; an aging population; and chronic conditions associated with obesity, smoking, and substance abuse. Accom- panying pressures on the insurance industry are wide regional variations in treatment, the significant underuse and misuse of recommended best practices, and an undue reliance on treatments of little or no value. In some cases, legislative mandates and regulatory processes have contributed to the challenge of basing decisions on evidence. The payer sector has been deeply engaged in the promotion of medical policy based on evidence. For example, committees of physicians, pharma- cists, and other healthcare professionals research the scientific evidence on drugs, depending in part on comparative effectiveness data, to determine which drugs to place on formularies. New technologies are assessed before insurers pay for them. Patients and providers are offered customized tools to modify their behavior, encourage the use of preventive care, monitor potential medication interactions, and improve health. Both public and pri- vate health insurers have begun to offer performance-based incentives mea- sured by selected evidence-based standards and performance measures. The insurer sector discussion suggests four major transformational activities: (1) a comparative effectiveness board, to help coordinate reform and set priorities; (2) a national research strategy focused on gaps in the evidence that, if filled, have the potential to significantly improve patient outcomes; (3) the transparency of actionable information that is used to make healthcare decisions; and (4) increased investment in the health- care infrastructure, including improved workforce training. In addition, insurers called for focused, coordinated research efforts to address the gaps in evidence that have been identified and factors that drive physician decision making. One model might come from health insurance plans that are collaborating to implement a national strategy to aggregate data from multiple plans and other sources to produce and report on an increasingly sophisticated set of quality and cost measures throughout the country. Sector participants also supported policies that would both reinforce the FDA’s capacity to assess the long-term safety and effectiveness of new drugs and strengthen the FDA’s review of certain devices and its capacity to track device safety. Also emphasized were investments in several key areas of infrastructure, including improved systems for aggregating administrative data and electronic health record information reliably. With respect to cross-sector collaboration, sector participants under- scored, in addition to a national comparative effectiveness board, the devel- opment of a more transparent and consistent approach to judging evidence with broad-based involvement of plans, product developers, evaluators, patients, employers, and government; collaboration among providers,

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2 LEADERSHIP COMMITMENTS TO IMPROVE VALUE IN HEALTH CARE payers, and developers to balance evidence with the effects of demographics, genomics, patient preferences, family history, and other factors; and align- ing benefit language with the language of innovations in evidence-based medicine to ensure that the public understands that the goals of evidence- based medicine and comparative effectiveness are not a reduction in access, but an improvement of health care. Employers and Employees Employers and employees shoulder a large share of healthcare expendi- tures in the United States. In return, they depend on the healthcare system to ensure the well-being and productivity of the workforce. Rising costs over the last decade have led to an erosion of employer-sponsored healthcare coverage, and healthcare benefits are an increasingly major factor in labor negotiations between employers and potential employees. Currently, only two-thirds of employees under 65 years of age have employer-sponsored coverage, and 40 percent of the employer market is self-insured. Employer expenditures rose 140 percent over the last decade, and healthcare spend- ing is projected to continue to rise at a rate of 7 percent annually over the next decade, twice the rate of inflation. In 2006, employee premiums and out-of-pocket spending averaged $3,136, up 12 percent from 2005. Costs, however, are only part of the challenge. In the face of wasteful spending and poor outcomes because of the overuse, underuse, and misuse of healthcare services, employers have increasingly supported efforts to improve health- care quality, safety, and efficiency, including the use of combined purchasing power to strengthen the system by improving quality and managing costs. The primary opportunities for employers and employees to encourage evidence-based medicine include provider contracting, benefit plan design, employee decision support, and public policy advocacy. As provider con- tracts are set, vendors can be selected and rewarded as they incorporate standards of medical evidence. Through benefit design, differential cover- age encourages effective care—as, for example, when coverage tiers are based on the strength of the evidence of effectiveness; network selection is based on performance; employee cost sharing encourages the use of high performers; and physicians, hospitals, and networks recognized for excel- lence receive higher payments. By developing and using decision support tools and resources, employers and employees can promote decision making informed by the evidence as well as risk-benefit profiles; and in a broader sense, employers and employees can shape public policy and advocate for patient safety, healthcare information technology, and comparative effec- tiveness research. Participants from the employer and employee sector identified several opportunities for immediate progress. First, the expansion of the evidence

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 SUMMARY base through comparative effectiveness research and the improved capture and use of information generated as part of clinical experience could, in part, be assisted by policy advocacy and the provision of support for the adoption and implementation of health information technologies. Second, the evidence could be appropriately incorporated into coverage and pay- ment policies by targeting purchasers and payers, building on existing performance measurement and payment programs, and establishing cross- sector agreement on transparent methods and standards. Last, creating a demand for evidence-based medicine through a collective communications campaign is fundamental to the success of the other initiatives. Such a campaign should build on existing efforts and bring to bear the research and marketing expertise of stakeholders to promote the development of a learning environment and sustained change toward an evidence-based healthcare system. Healthcare Information Technology As key players in the healthcare arena, those in information technol- ogy have evolved from a focus on the delivery of stand-alone “smart” medical equipment (echocardiography systems, radiology systems, etc.) to the development and delivery of increasingly integrated clinical systems, full-function electronic health records, and related complex and evolving systems for healthcare professionals. Key players in healthcare information technology include clinical source system providers that create systems for use in specific functional areas (e.g., laboratories, radiology departments, and surgeries); electronic health record companies, which provide consoli- dated and integrated clinical systems that support inpatient and outpatient practices; administration and chain data management companies, which develop administrative systems in support of clinical care and research; World Wide Web–based patient and consumer information companies; personal health record companies; and niche companies, such as those that provide education tools, data warehousing, enterprise information manage- ment, and data analysis. Fundamentally, the work of this sector focuses on improving consumer access to reliable health and disease management information, enhancing patient-provider communication and interaction, developing operational effectiveness and efficiency, improving the ability to manage and analyze large quantities of data, and improving research on clinical effectiveness and quality of care. The background paper from the information technology sector iden- tified seven priority areas or challenges on which current work aims to improve the sector’s ability to support the transformative change implied in the Roundtable’s goal. Perhaps the single most transformational step toward achieving the goal of a learning healthcare system lies in the development and

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 LEADERSHIP COMMITMENTS TO IMPROVE VALUE IN HEALTH CARE implementation of information technology industry standards. In conjunction with these standards, healthcare information technology sector participants underscored the need for collaboration with other sectors to develop a com- mon vocabulary to facilitate the interoperability of clinical systems and the interpretation of clinical data across multiple sites. In the area of workflow, the dual goals of seamless movement of data between various patient care environments and just-in-time delivery of the right information are important to ensure that the best decisions can be made in partnership with patients. In part, this requires streamlining the complex tools that enable the collection, aggregation, synthesis, delivery, and interpretation of and access to data as part of provider clinical decision support systems. It also requires develop- ing new ways to view clinical data, and their relationship to other data, to help users interpret the significance of relationships and make appropriate and informed patient care decisions. Other sector themes included address- ing questions in the area of connectivity to ensure that healthcare networks connecting various stakeholders provide the seamless transfer of relevant and appropriate information, minimizing the sources and number of data inputs while increasing data integrity and reliability. The paper from the information technology sector translated these challenges into three transformational initiatives. The first involves building and promoting the foundational technologies needed to enable healthcare information technology-assisted evidence-based medicine. Achieving the goal of a learning healthcare system would be enhanced by the develop- ment and implementation of information technology industry standards and common vocabularies in health care. This would support healthcare information technology at every level and provide building blocks for bring- ing computational intelligence to aid human cognition in evidence-based medicine. The second initiative involves the establishment of a government- industry collaborative ecosystem for the ongoing evolution and develop- ment of clinical information technology standards. A virtuous cycle, or the continual feeding of outputs back into the cycle as inputs—as illustrated by eBay, Flickr, and YouTube—would result in more users of the standards and therefore more feedback. In the term “ecosystem,” it is assumed that both community and technology are included, as they are in blogs and wikis. Participants suggested that an evaluation of technical barriers to the adop- tion of existing publicly supported, open-standard vocabularies and tools by healthcare information technology providers would be useful. Initia- tives could be provided to remedy technical barriers, and success could be measured by determination of the rates of adoption and utilization of the technologies. Because information in the healthcare system is partitioned into silos without connectivity, a clinical data and analytic infrastructure must be created to enable evidence-based medicine, especially given the fact

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 SUMMARY that doctors spend 60 percent of their time seeking data. To achieve this, convening or supporting initiatives would be useful, perhaps by the IOM, to identify metrics around accessibility of core clinical data and core analytic tools (e.g., reporting specifications and data visualization). To speed these initiatives and realize the goals of the information tech- nology sector, the third initiative discussed would involve the provision of an incentive for dramatic innovation similar to the robot race of the Defense Advanced Research Projects Agency in which researchers competed with each other to create a driverless car. The collective investment of resources by competitors and the innovative research that resulted far exceeded what could have been achieved by direct investment of the $1 million prize. This contest might act as a model for healthcare information technology because a diverse population should be engaged in the radical technological innova- tion needed in evidence-based medicine. Such innovation could guide clini- cal decisions based on individual data, along with relevant clinical evidence and experiential information gathered from the mining of data on previous patients with similar conditions, all with just-in-time evidence delivery, alerts, and flexible data views. One option might be a challenge sponsored by the IOM as an Advanced Technical Strategies Innovations Initiative. REFERENCES Fisher, E. S. 2005. More care is not better care: Regional differences show that spending more does not improve—and may hurt—patients. More accountability can help. Expert voices (7). Washington, DC: National Institute for Health Care Management Foundation. Institute of Medicine. 2007. The learning healthcare system. Washington, DC: The National Academies Press. ———. 2008. Learning healthcare system concepts, v. 200. Washington, DC: The National Academies Press. IOM Roundtable on Evidence-Based Medicine. 2006. Charter and vision statement: Round- table on evidence-based medicine. Washington, DC. McGlynn, E., S. Asch, J. Adams, J. Keesey, J. Hicks, A. DeCristofaro, and E. Kerr. 2003. The quality of health care delivered to adults in the United States. New England Journal of Medicine 348(26):2635-2645. Wennberg, J., E. Fisher, and J. Skinner. 2002. Geography and the debate over Medicare reform. Health Affairs Suppl. Web Exclusives:W96-W114.

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