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Appendix D The Learning Health System Series: Workshop Common Themes 1. VISION The Learning Healthcare System Adaptation to the pace of change: continuous learning and a much more dynamic approach to evidence development and application, tak- ing full advantage of developing information technology to match the rate at which new interventions are developed and new insights emerge about individual variation in response to those interventions. Stronger synchrony of efforts: better consis- tency and coordination of efforts to gener- ate, assess, and advise on the results of new knowledge in a way that does not produce conflict or confusion. Culture of shared responsibility: to enable the evolution of the learning environment as a common cause of patients, providers, and researchers and better engage all in improved communication about the importance of the nature of evidence and its evolution. 283

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284 PATIENTS CHARTING THE COURSE New clinical research paradigm: drawing clinical research closer to the experience of clinical practice, including the development of new study methodologies adapted to the practice environment and a better under- standing of when RCTs are most practical and desirable. Clinical decision support systems: to accommodate the reality that although professional judgment will always be vital to shaping care, the amount of information required for any given decision is moving beyond unassisted human capacity. Universal electronic health records: comprehensive deployment and effec- tive application of the full capabilities available in EHRs as an essential prerequisite for the evolution of the learning healthcare system. Tools for database linkage, mining, and use: advancing the potential for structured, large databases as new sources of evidence, including issues in fostering interoperable platforms and in developing new means of ongoing searching of those databases for patterns and clinical insights. Notion of clinical data as a public good: advancement of the notion of the use of clinical data as a central common resource for advancing knowl- edge and evidence for effective care—including directly addressing cur- rent challenges related to the treatment of data as a proprietary good and interpretations of the Health Insurance Portability and Accountability Act (HIPAA) and other patient privacy issues that currently present barriers to knowledge development. Incentives aligned for practice-based evidence: encouraging the develop- ment and use of evidence by drawing research and practice closer together, and developing the patient records and interoperable platforms necessary to foster more rapid learning and improve care. Public engagement: improved communication about the nature of evidence and its development, and the active roles of both patients and healthcare professionals in evidence development and dissemination. Trusted scientific broker: an agent or entity with the public and scientific confidence to provide guidance, shape priorities, and foster the shift in the clinical research paradigm.

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285 APPENDIX D Leadership: to marshal the vision, strategy, and actions necessary to create a learning healthcare system. 2. CARE COMPLEXITY Evidence-Based Medicine and the Changing Nature of Health Care Increasing complexity of health care: New pharmaceuticals, medical devices, technologies, and predictive data offer much promise for im- proving health care, but they also introduce high levels of complexity, requiring changes on the parts of both caregivers and their patients. Unjustified discrepancies in care patterns: The intensity of healthcare service delivered for similar conditions varies significantly across geographic regions, particularly in areas that require discretionary decision making. How- ever, the higher-spending regions often do not deliver better-quality care, hence offering sub- stantial opportunity for reduced spending with- out sacrificing health outcomes. Importance of better value from health care: The current healthcare system is not designed to deliver value, and the nation’s long-term fiscal challenges are serious and are being driven predominately by excessive medical spend- ing, often on interventions of no clinical benefit. Opportunities exist to eliminate wasteful spending with no reduction in health care, as well as to improve the overall health outcomes, but agreement is needed both on what constitutes best care and on what constitutes value in health care. Uncertainty exposed by the information environment: An irony of the information-rich environment is that information important to clinical decision making is often not available, or is provided in forms that are not relevant to the broad spectrum of patients—with differing levels of health, socioeconomic circumstances, and preferences—and the issues encountered in clinical practice. This is due to too little clinical effectiveness research, too poor dissemination of the evidence that is available, and too few incen- tives and decision supports for evidence-based care.

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286 PATIENTS CHARTING THE COURSE Pressing need for evidence development: More and better evidence— including comparative and longitudinal data—is needed to determine the effectiveness and usefulness of new medical interventions, treatments, drugs, devices, and genetic information. There is an untapped potential to reduce healthcare costs and improve quality by developing evidence not only for specific medical interventions, but also for the way health care is delivered. Promise of health information technology: Electronic medical records (EMRs) and clinical data registries offer tremendous potential both to generate new evidence and to augment randomized clinical trials. Addressing privacy and proprietary issues that limit data access and sharing would help to support a system in which electronic medical records, clinical registries, and other types of electronic data could contribute to building a more robust evidence base. Need for more practice-based research: How might the system better sup- port the notion of a “living textbook of medicine” in which the experi- ence of healthcare diagnosis and treatment is routinely captured in order to better care for those in the future. To develop best evidence for the delivery of medicine that is geared toward the needs of individual patients, investment is needed into infrastructure for the gathering and analysis of healthcare data and information, and standards and protocols to ensure their accuracy and reliability. Shift to a culture of care that learns: This changing role will require health- care providers and patients to adopt a culture that supports the generation and application of evidence. Effective culture change must also be accom- panied by insurance and reimbursement system reform that encourages development and application of the systems necessary. New model of patient-provider partnership: With the increasing complexity of care, and the need and demand for more patient involvement, the tra- ditional “physician-as-sole-authority” model will need to adapt to support patients as integral partners in medical decisions. Leadership that stems from every quarter: Adapting to and taking advan- tage of, the changes in the healthcare environment will take broad leader- ship. A strategic focus on the development and application of evidence will require the involvement of both the public and private sectors working together, and with policy makers, providers, patients, insurers, and other stakeholders in the steps toward change.

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287 APPENDIX D 3. EFFECTIVENESS RESEARCH Redesigning the Clinical Effectiveness Research Paradigm: Innovation and Practice-Based Approaches Address current limitations in applicability of research results: Because clinical condi- tions and their interventions have complex and varying circumstances, there are different implications for the evidence needed, study designs, and the ways lessons are applied: the internal and external validity challenge. In addition, although our assessment of can- didate interventions is primarily through pre-market studies, the opportunity for dis- covery extends throughout the lifecycle of an intervention—development, approval, cover- age, and the full period of implementation. Counter inefficiencies in timeliness, costs, and volume: Much of current clinical effective- ness research has inherent limits and inefficiencies related to time, cost and volume. Small studies may have insufficient reliability or follow-up. Large experimental studies may be expensive and lengthy, but have limited applica- bility to practice circumstances. Studies sponsored by product manufacturers have to overcome perceived conflicts and may not be fully used. There is a strong need for more systematic approaches to better defining how, when, for whom, and in what setting an intervention is best used. Define a more strategic use to the clinical experimental model: Just as there are limits and challenges to observational data, there are limits to the use of experimental data. Challenges related to the scope of inferences possible, to discrepancies in the ability to detect near-term vs. long-term events, to the timeliness of our insights and our ability to keep pace with changes in technology and procedures, all must be managed. For the future of clinical effectiveness research, the important issues relate not to whether random- ized experimental studies are better than observational studies, or vice versa, but to what’s right for the circumstances (clinical and economic), and how the capacity can be systematically improved. Provide stimulus to new research designs, tools, and analytics: An exciting part of the advancement process has been the development of new tools and resources that may quicken the pace of our learning and add real value

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288 PATIENTS CHARTING THE COURSE by helping to better target, tailor, and refine approaches. Use of innovative research designs, statistical techniques, probability and other models may accelerate the timeliness and level of research insights. Some interesting ap- proaches using modeling for virtual intervention studies may hold prospects for revolutionary change in certain clinical outcomes research. Encourage innovation in clinical effectiveness research conduct: The kinds of “safe harbor” opportunities that exist in various fields for developing and testing innovative methodologies for addressing complex problems are rarely found in clinical research. Initiative is needed for the research com- munity to challenge and assess its approaches—a sort of meta-experimental strategy—including those related to analyzing large datasets, in order to learn about the purposes best served by different approaches. Innovation is also needed to counter the inefficiencies related to the volume of studies conducted. How might existing research be more systematically summa- rized; or, different research methods organized, phased, or coordinated to add incremental value to existing evidence? Promote the notion of effectiveness research as a routine part of practice: Taking full advantage of each clinical experience is the theoretical goal of a learning healthcare system. But for the theory to move closer to the prac- tice, tools and incentives are needed for caregiver engagement. A starting point is with the anchoring of the focus of clinical effectiveness research planning and priority setting on the point of service—the patient–provider interface—as the source of attention, guidance, and involvement on the key questions to engage. The work with patient registries by many specialty groups is an indication of the promise in this respect, but additional em- phasis is necessary in anticipation of the access and use of the technology that opens new possibilities. Improve access and use of clinical data as a knowledge resource: With the development of bigger and more numerous clinical data sets, the potential exists for larger scale data mining for new insights on the effectiveness of interventions. Taking advantage of the prospects will require improve- ments in data sharing arrangements and platform compatibilities, address- ing issues related to real and perceived barriers from interpretation of privacy and patient protection rules, enhanced access for secondary analysis to federally sponsored clinical data (e.g., Medicare part D, pharmaceutical, clinical trials), the necessary expertise, and stronger capacity to use clinical data for post-market surveillance. Foster the transformational research potential of information technology: Broad application and linkage of electronic health records holds the poten-

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289 APPENDIX D tial to foster movement toward real-time clinical effectiveness research that can generate vastly enhanced insights into the performance of interventions, caregivers, institutions, and systems—and how they vary by patient needs and circumstances. Capturing that potential requires working to better understand and foster the progress possible, through full application of electronic health records, development and application of standards that facilitate interoperability, agreement on and adherence to research data col- lection standards by researchers, developing new search strategies for data mining, and investing patients and caregivers as key supporters in learning. Engage patients as full partners in the learning culture: Access to up-to-date information by both caregiver and patient changes the state of play in sev- eral ways. The patient sometimes has greater time and motivation to access relevant information than the caregiver, and a sharing partnership is to the advantage of both. The more patients understand and communicate with their caregivers about the evolving nature of evidence, the less disruptive will be the frequency and amplitude of public response to research results that find themselves prematurely, or without appropriate interpretative guidance, in the headlines and short-term consciousness of Americans. Build toward continuous learning in all aspects of care: This foundational principle of a learning healthcare system will depend on system and culture change in each element of the care process with the potential to promote in- terest, activity, and involvement in the knowledge and evidence development process, from health professions education to care delivery and payment. 4. DATA UTILITY Clinical Data as the Basic Staple of Health Learning: Creating and Protecting a Public Good Clarity on the basic principles of clinical data stewardship: The starting point for expanded access and use of clinical data for knowledge develop- ment is agreement on some of the fundamental notions to guide the activi- ties for all individuals and organizations with responsibility for managing clinical data. Workshop participants repeatedly mentioned the need for consensus on approaches to such issues as data structure, standards, report- ing requirements, quality assurance, timeliness, de-identification or security measures, access and use procedures—all of which will determine the pace and nature of evidence development. Incentives for real-time use of clinical data in evidence development: Current barriers to the real-time use of clinical data for new knowledge discussed

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290 PATIENTS CHARTING THE COURSE at the workshop ranged from regulatory and commercial issues to cost and quality issues. Participants suggested the need for a dedi- cated program of activities, incentives, and strategies to improve the methods and ap- proaches, their testing and demonstration, the cooperative decision making on priorities and programs, and the collective approach to regulatory barriers. Transparency to the patient when data are applied for research: Patient acceptance is key to use of clinical data for knowledge develop- ment, and patient engagement and control are key to acceptance. In this respect, clarity to individual patients on the structure, risks, and benefits of access to data for knowledge development was noted by partici- pants as particularly important. Patient confidence and system accountabil- ity may be enhanced through transparent notification and audit processes in which patients are informed of when and by whom their information has been accessed for knowledge development. Addressing the market failure for expanding electronic health records: Currently, market incentives are inadequate to bring about the expansion of use of electronic health records necessary to make the point of care a locus for the development, sharing, and application of knowledge about what works best for individual patients. Shortfalls noted by participants included demand by providers or patients that is not sufficient to counter the expense to small organizations, competing platforms and asynchronous reporting requirements that work against their utility for broad quality and outcome determinations, and that even the larger payers—apart from government—do not possess the critical mass necessary to drive broader scale applicability and complementarity. It will likely take a deeper, more directed and coordinated strategy involving Medicare leadership to foster such changes. Personal records and portals that center patients in the learning process: Patient demand could be instrumental in spreading the availability of elec- tronic health records for improving patient care and knowledge develop- ment. Such demand will depend upon much greater patient access to, comfort with, and regular use of programs that allow either the mainte- nance of personal electronic health records or access through a dedicated portal to their provider-maintained electronic medical record. As noted

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291 APPENDIX D during the workshop, many consumer-oriented products currently under development give patients and consumers more active roles in managing personal clinical information, and may help to demonstrate value in the speed and ease of personal access to the information, better accommodate patient preference in care, and foster a partnership spirit conducive to the broader EHR application. Coordinated EHR user organization evidence development work: The devel- opment of a vehicle to enhance collaboration among larger EHR users of dif- ferent vendors was raised during the workshop as a means to accelerate the emergence of more standardized agreements and approaches to integrating and sharing data across multiple platforms, common query strategies, virtual data warehousing rules and strategies, relational standards, and engagement of ways to reduce misperceptions on regulatory compliance issues. The business case for expanded data sharing in a distributed network: Demonstrating the net benefits of data sharing could promote its use. Benefits suggested by participants included cost savings or avoidance from facilitated feedback to providers on quality and outcomes; quick, continu- ous improvement information; and improved management, coordination, and assessment of patient care. Assuring publicly funded data for the public benefit: Federal and state funds that support medical care, as well as support insights into medical care through clinical research grant funding, are the source of substantial clinical data; yet, many participants observed that these resources are not yet ef- fectively applied to the generation of new knowledge for the common good. Broader semantic strategies for data mining: Platform incompatibilities for clinical data substantially limit the spread of electronic health records and their use for knowledge development. Yet discussion identified strategies using alternative semantic approaches for mining clinical data for health insights, which may warrant dedicated cooperative efforts to develop and apply them. Public engagement in evidence development strategies: Generating a base of support for and shared emphasis on developing a healthcare ecosystem in which all stakeholders play a contributory role was noted by many partici- pants as important for progress. Ultimately, the public will determine the broad acceptance and applicability of clinical data for knowledge develop- ment, underscoring the importance of keeping the public closely involved and informed on all relevant activities to use clinical data to generate new knowledge.

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292 PATIENTS CHARTING THE COURSE 5. EVIDENCE Learning What Works: Infrastructure Required for Comparative Effectiveness Research Coordinating work and ensuring standards are key components of the evidence infra- structure: Infrastructure for evidence devel- opment includes the capacity for greater coordination in the setting of study priori- ties; the development of systematic decisions for the conduct of comparative effectiveness research, systematic reviews, and guideline development; and ensuring the consistent translation of developed information. Learning about effectiveness must continue beyond the transition from testing to practice: Pre-market testing for the safety and effective- ness of various interventions cannot assess the results for all populations or the circum- stances of use and differences in practice patterns, so gathering information as interventions are applied in practice settings should represent a key focus in designing the infrastructure to learn which care is best. Timely and dynamic evidence of clinical effectiveness requires bridging research and practice: Although historical insulation of clinical research from the regular delivery of healthcare services evolved to facilitate data capture and control for confounding factors, it may not adequately inform the real-world setting of clinical practice. With the prospect of enhanced data capture electronically at the point of care, on real-world patient popu- lations, and statistical approaches to improve analysis, as well as increasing demand to keep pace with technologic innovation, the divide of clinical research from care practice increasingly limits the utility of research results. Current infrastructure planning must build to future needs and oppor- tunities: Emerging questions include those related to the management of multiple co-occurring chronic diseases of increasing prevalence in an aging population, the improved insights into individual variation relevant to both treatments and diagnostics, and the impact of innovation in shortening the lifecycle of any particular intervention. Emerging tools include innovations in trial design, the development of new statistical approaches to data analy- sis, and the development of electronic medical and personal health records.

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293 APPENDIX D Keeping pace with technological innovation compels more than a head-to- head and time-to-time focus: With the rapid pace of change in the nature of interventions and the difficulty, expense, and time required to develop studies—and the challenges of ensuring the generalizability of results in the face of limitations of traditional approach to randomized controlled trials (RCTs)—a first-order priority for the effectiveness research is the establishment of infrastructure for a more dynamic, real-time approach to learning. Leveraging new tools such as health IT should allow for a more networked and distributed approach to information sharing and evidence creation. Real-time learning depends on health information technology investment: It was noted that collecting data is the most time-intensive part of trials and studies, and IT is critical to streamlining this work. The increasing complex- ity of the factors involved in understanding the effectiveness of clinical op- tions under different circumstances requires a blend of database access and computing power that can only be provided from broadly applied health information technology. A policy framework for privacy and security will be necessary to build and maintain public trust that information will be protected as it is shared. Developing and applying tools that foster real-time data analysis is an important element: The scope and scale of evidence needs suggests that innovation is needed across the range of research methods, from making clinical trials faster and less expensive, to moving beyond randomized trials to better address practical circumstances. To take advantage of health in- formation technology, statistical tools, and analytic algorithms that can be embedded in databases to allow real-time insights will be important. Simi- larly, tools are needed that will allow findings to be drawn from databases built on different vendor platforms, using semantic technology to integrate currently disparate medical data, and for developing the next generation of statistical tools for the analysis of clinical data, including the building of models that allow insights to be generated by virtual studies. A trained workforce is a vital link in the chain of evidence stewardship: Given the pace of change in the number and variety of clinical interventions as well as in the tools and approaches to assessing them, there is a need to ensure that these developing opportunities are matched by the skills of the workforce. This includes training and education in the methodologies of research design, translating research, guideline development, and maintain- ing and mining clinical records. It also includes attention to re-orienting the education of front-line caregivers around their emerging responsibilities for access, interpretation, and discussion with patients of a dynamic evidence

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301 APPENDIX D problems, which may result in treatment errors and duplicative services. Participants described how team-based care offers the potential to rectify this disconnected care and limit human error. Effective teams are aided by an appropriate information technology infrastructure, which facilitates efficient and effective communication of health information, as well as by collaborative organizational systems, from medical homes to accountable care organizations. Efficient: Patients, their families, and clinicians should expect care to be appropriate to need, resources, and time required. Participants underscored the fact that currently, much of the care that is delivered is neither neces- sary nor efficient. Among the chief complaints of patients are increasing out-of-pocket costs and lost time in the care process. This finding is not surprising given that the current incentive structure, focused on volume over value, encourages overuse and waste. As multiple participants noted, the United States spent roughly 17 percent of its gross domestic product on health care last year, yet this investment did not yield the health outcomes commensurate with the costs. To gain greater value, participants stressed, the costs and outcomes of care must be more transparent to patients, and new payment models—ranging from bundling payments for an entire epi- sode of care, to pay-for-performance systems, to global payment—must be implemented. Accountable: All relevant aspects of the clinical experience, including patient perspectives, should be captured and routinely assessed against expectations. This information is vital not only to achieving effective patient management, but also to judging whether experiments with new delivery system models, payment incentives, or standards of care are having their intended effect on improving patient health and promoting efficiency. Measuring performance and disseminating innovations that work (and eliminating those that do not) constitute a systematic way of improving healthcare delivery. One presenta- tion highlighted how this systematic approach to improvement allowed the speaker’s organization to enhance care by conducting comprehensive reviews of interventions for different conditions, adopting the best practices identi- fied by that review, and measuring the performance of the revised standard of care. Transparent: Information on the outcomes of care—both effectiveness and efficiency—should be readily accessible and understandable to patients and their families. Several speakers mentioned the frustration felt by patients regarding the lack of understandable information on the costs, quality, and outcomes of care, especially in light of reports about medical errors and the increasing personal burden of costs and inefficiencies of

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302 PATIENTS CHARTING THE COURSE care. It was noted that, when offered a choice, patients do not routinely choose more costly or more intensive interventions. However, choice and information about alternatives are rarely available. It is clear that action to improve value—better outcomes at lower cost—requires transparent information on the costs and outcomes of care. Trustworthy: Patients should expect a strong and secure trust fabric on all dimensions—safety, quality, security, efficiency, accountability, and equity. In few areas of human endeavor is trust on each of these dimensions more important. Yet one presenter noted that, even in the face of information that 50,000 to 90,000 deaths per year are caused by medical errors, health care lacks the basic trust elements of transparency and accountability needed to drive improvements in quality and safety. In a learning system that draws lessons from each care experience, public trust must be bolstered in all aspects of the healthcare enterprise: equitable access to reliable clinician knowledge and skills, safeguards on clinical processes, privacy and security of medical records, and validity and safety of clinical trials. Learning: In a learning health system, the patient is an active contributor to, and supporter of, the learning process. Each patient experience offers the potential to deepen the knowledge base that drives care quality and outcomes—at the individual, practice, and societal levels. A focus of the workshop was the stake of the patient in fostering a digital health utility that provides needed information to patients and their clinicians, ensures synchronization among providers, and generates knowledge for progress— for example, for comparative effectiveness insights, public health activities, or postmarket monitoring of approved technologies and drugs. Reference was made, for example, to the need for a common core data set for EHR- based data that would allow reliable, platform-independent research across large patient populations. These are issues in which patients have a strong stake, and they must have confidence in the system’s functionality for the generation of timely and reliable new insights. 9. COST AND OUTCOMES The Healthcare Imperative: Lowering Costs and Improving Outcomes Challenges for health costs and outcomes Health cost excesses with personal, institutional, and national conse - quences. In the past decade, U.S. health costs have increased by 92 percent, representing approximately four times the inflation rate for the economy as a whole. Out-of-pocket costs for individuals have increased by 40 percent.

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303 APPENDIX D Overall, Medicaid now takes almost 20 per- cent of state budgets, crowding out other state priorities such as education. Health outcomes far short of expectations. Despite health spending double the average for other developed nations, U.S. health out- comes rank below two to three dozen other countries on indices such as life expectancy, care for chronic disease, and persistent dis- parities in the access and outcomes of care. Fragmented decision points, inconsistent principles, and political distortions. Barriers to appropriate care include poor care coor- dination, lack of consistent evidence-based guidelines, payment systems that encourage volume over value, and politi- cal influences that sometimes overturn scientific determinations. Domains of waste and inefficiency in healthcare spending Unnecessary services: services reflecting choices or levels beyond those sup- ported by evidence or benchmarks. Inefficiently delivered services: inefficient labor use, time-flow discontinui- ties, duplicate services, medical errors. Excess administrative costs: billing and insurance-related costs for payers and providers, inefficient reporting requirements. Prices that are too high: for medical services, pharmaceuticals, products and devices, relative to benchmarks. Missed prevention opportunities: for preventable obesity, diabetes, heart disease, stroke, pulmonary disease, cancers, infections. Medical fraud: systemic over-billing, billing for undelivered services, use of unlicensed providers. Drivers of the problems Scientific uncertainty. Clinical evidence development is not keeping pace with new diagnostics, treatments, and insights into individual variation.

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304 PATIENTS CHARTING THE COURSE Perverse economic and practice incentives. The fee-for-service reimburse- ment system rewards service volume rather than value. System fragmentation. Multiple, disconnected, and uncoordinated decision points in healthcare delivery and finance are fundamental challenges to efficient and effective care. Opacity as to cost, quality, and outcomes. Without meaningful and trust- worthy sources of information on costs and outcomes of care, neither patients nor their clinicians can make fully informed decisions. Changes in health status. An aging population, the growing prevalence of obesity, and increases in multiple co-occurring, complex chronic diseases are accelerating the need for health services. Lack of patient involvement. The culture of care is not yet conducive to active patient participation in care decisions, despite growing use of web- accessible information and evidence of the positive effect of shared decision making on health outcomes. Under-investment in population health. Because health status is impor- tantly influenced by behavioral, social, and environmental factors, progress depends on a stronger commitment to population-wide health programs. Corrective levers Streamlined and harmonized health insurance regulation. Reduce com- plexities and inconsistencies in coverage standards and requirements often unique to a jurisdiction. Administrative simplification and consistency. Streamline and harmonize inconsistent payment and reporting requirements that create unnecessary and excessive administrative costs. Focus incentives on results and value. Focus payments on outcomes and value, and increase targeting those at highest risk of poor outcomes. Quality and consistency in treatment. Establish treatment guidelines as the starting point for effective care, tailoring as indicated, and capturing the care experience for continuous improvement. Evidence that is timely, independent, and understandable. Foster effective care through a dedicated, unified program that provides reliable guidance;

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305 APPENDIX D keeps up with innovation and changing science; and improves practice reli- ability, consistency, and impact. Clinical records that are reliable, sharable, and secure. Use electronic health records to enhance the effectiveness and efficiency of care, facilitate patient handoffs, provide clinical decision guidance, and foster patient involvement. Data that are protected but accessible for continuous learning. Create a digital utility with clinical data as a resource for real-time monitoring of the results of treatment, ongoing generation of new evidence for effective care, and continuous care improvement. Transparency requirements on cost, quality, and outcomes. Build an ac- cessible information resource, with transparency as to cost, outcomes, and value serving as a critical element of system change. Culture and activities framed by patient perspective. Position patient perspec- tives and needs as primary—and convenience and interest as secondary—for the design and execution of healthcare organization and delivery. Medical liability reform. Diminish defensive medicine as a detrimental, significant driver of unnecessary services and procedures—e.g., through harbors for best evidence practices, caps on non-economic damages, spe- cialized tribunals. Prevention. Elevate the focus on prevention, ranging from clinical preven- tive services to community health and wellness. 10. VALUE Value in Health Care: Accounting for Cost, Quality, Safety, Outcomes, and Innovation Urgency: Increasing healthcare costs consistently outpace inflation, squeez- ing out employer coverage, adding to the uninsured, and doubling out- of-pocket payments—without commensurate health improvements. The long-term consequences for federal budget obligations, driven by the growth in Medicare costs, amount to an estimated $34 trillion in unfunded obligations. Perceptions: We have heard that for patients perceived value in health care is often described in terms of the quality of their relationship with their physicians. Clinicians discussed value as diagnostic and treatment ap-

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306 PATIENTS CHARTING THE COURSE proaches that offer increased confidence in the effectiveness of services offered. Employ- ers viewed value improvement in terms of keeping workers and their families healthier and more productive at lower costs. For health insurers, we heard that value improve- ment means emphasizing interventions that are crisply defined and supported by a high level of evidence. Health product innovators spoke of value improvement as a product that is better for the individual patient, more profitable, and contributes to product differ- entiation and innovation. Elements: Value from health care has dimen- sions beyond the nature, cost, and effective- ness of a particular intervention, including those related to elements such as preference, satisfaction, and appropriateness to circumstance. Value determination also means determining the right price, and we heard that, from the demand side, the right price is a function of perspective. From the supply side, the right price is a function of the cost of production, the cost of delivery, and the incentive to innovation. Gain: Because reliable information is the starting point for improving value, discussants underscored the importance of adequate transparency and con- tinuous improvement of insights on the safety, efficacy, effectiveness, and comparative effectiveness of interventions. Decisions: Currently, decision rules seem to many stakeholders to be vague, inconsistent, and poorly tailored to the evidence. Information: Because the quality of evidence varies, as do the methods used to evaluate it, transparency as to source and process, care as to interpreta- tion, and clarity in communication were noted as key. Incentives: Often noted in the workshop discussions was that the incentives prevalent in the American healthcare system are poorly aligned to effec- tiveness and efficiency, encouraging care that is procedure- and specialty- intensive and discouraging primary care and prevention. Limits: We have heard that obtaining the value needed will continue to be elusive until better means are available to draw broadly on information as to services’ efficiency and effectiveness, to set priorities and streamline

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307 APPENDIX D approaches to filling the evidence gaps, to ensure consistency in the ways evidence is interpreted and applied, and to marshal incentives to improve the delivery of high-value services while discouraging those of limited value. Communication: Patients and providers do not communicate well with each other about diagnosis and treatment options or cost implications. Communication is often absent between multiple providers for a single patient, increasing the prospect of service gaps, duplications, confusion, and harm, according to discussants. Further, communication between scientific and professional organizations producing and evaluating evidence is often limited, resulting in inefficiencies, missed opportunities, and contradictions in the production of guidance. Providers: We heard that the clearest barriers to provider-level value im- provement appear to lie in the lack of economic incentives for a focus on outcomes and also in cultural and structural disincentives to tend to the critical interfaces of the care process—the quality of the links in the chain of care elements. Patients: It was noted that patients most often think of value in terms of their relationship with their provider but ultimately the practical results of that relationship, in terms of costs and outcomes, hinge on the suc- cess of programs that improve practical, ongoing, and seamless access to information on best practices and costs and of payment structures that reward accordingly. Workshop discussants offered insights into the use of various financial approaches to sensitize and orient patient decisions on healthcare prices according to the evidence of the value delivered. Manufacturers: Health product manufacturers and innovators naturally focus on their profitability but because product demand is also derived from the ability to demonstrate advantage with respect to outcomes and efficiency, manufacturers expressed an interest in regulatory and payment approaches that enhance performance on outcomes resulting from product use. Tools: Despite the broad agreement on their importance, we heard that the analytical tools and capacity to evaluate, in either absolute or comparative terms, the basic elements of value—outcomes and costs—are substantially underdeveloped and will need greater attention. Opportunities: Although attaining better value in health care depends on reducing the fragmentation that is its central barrier, we heard a number of examples of measures that might be taken at different levels, both to achieve better value now and to set the stage for future progress.

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308 PATIENTS CHARTING THE COURSE 11. LEADERSHIP Leadership Commitments to Improve Value in Health Care: Finding Common Ground Build trust and collaboration: Health care depends for its effectiveness on the close co- operation 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: Although all participants agreed on the cen- trality and importance of the value achieved from health care, different groups often think of value in different ways. A multistakeholder effort might drive clarity and consensus on those principles and elements of value com- mon to all stakeholders. Improve public understanding of evidence: 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 systematic and coordinated communication 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 point in time, and that health care is a joint patient–provider endeavor. Characterize the impact of shortfalls in the evidence: Documenting the consequences 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 understanding of and demand for evidence-based care and stakeholder activation. Identify the priorities for evidence development: The first step to a system- atic and coordinated effort to conduct the most important assessments is the identification of the priorities as a sort of consensus national problem list and research agenda of the most pressing issues for medical care decisions. Improve the level, quality, and efficiency of the research: Policies that facilitate the ability to use clinical data to monitor the effectiveness of in-

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309 APPENDIX D terventions are needed. Novel approaches to the conduct of clinical trials are needed. A more structured lexicon for “best practices” in undertaking observational studies may be necessary. Clarify and promote transparency: Consensus is needed to establish com- mon 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: Decisions on market approval, insurance coverage, provider use, and patient acceptance are all informed by some interpretation of the evidence. Clarity of the guid- ing principles is important. Improve engagement in the full lifecycle of interventions: Many factors are at play for each intervention—for example, similarity to previously tested interventions, the safety and effectiveness of an intervention for some popu- lations 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 application, improved out- comes, and efficiency will require a set of lifecycle-oriented decision-making rules that are more carefully considered than they are at present. Focus on frontline providers: Accelerating the translation of clinical re- search into practice involves addressing matters of professional education, credentialing, licensure, practice support, economic incentives, patient ac- ceptance, 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: In this information age, health- related information is constantly presented through news reports, market- ing, professional organizations, journals, and the Internet, but it is often confusing and even contradictory. A trusted information source—one that is independent but that engages all stakeholders—is needed to identify gaps; set priorities; establish standards; and guide the development, interpreta- tion, and dissemination of evidence on clinical effectiveness. Build the capacity to meet the demand: Currently, the combined resources of the various public and private organizations involved in studying com- parative clinical effectiveness meet but a small and scattered fraction of the demand. The centrality of the problem to the quality and efficiency—

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310 PATIENTS CHARTING THE COURSE the viability, according to some—of the nation’s healthcare system may require the creation of a new independent entity devoted to the work. Create incentives for change: Economic and policy incentives to engage the use of the best available evidence and more fully engage patients in the clini- cal decision-making process include the alignment of purchasing incentives accordingly 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: Health informa- tion technology can facilitate the development of learning networks and accelerate the generation of evidence, enable data aggregation and utiliza- tion, deliver evidence to the point of care, and expand research capacities. Coordinated stakeholder action—and financial incentives—should be able to speed the progress toward universal application of electronic health re- cords and access to information both on basic interoperability issues (e.g., standards and vocabulary) and, possibly, the development of more radical data search innovations.

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