Recent scientific and technological advances have accelerated our understanding of the causes of disease development and progression, and resulted in innovative treatments and therapies. Ongoing work to elucidate the effects of individual genetic variation on patient outcomes suggests the rapid pace of discovery in the biomedical sciences will only accelerate. However, these advances belie an important and increasing shortfall between the expansion in therapy and treatment options and knowledge about how these interventions might be applied appropriately to individual patients. The impressive gains made in Americans' health over the past decades provide only a preview of what might be possible when data on treatment effects and patient outcomes are systematically captured and used to evaluate their effectiveness. Needed for progress are advances as dramatic as those experienced in biomedicine in our approach to assessing clinical effectiveness. In the emerging era of tailored treatments and rapidly evolving practice, ensuring the translation of scientific discovery into improved health outcomes requires a new approach to clinical evaluation. A paradigm that supports a continual learning process about what works best for individual patients will not only take advantage of the rigor of trials, but also incorporate other methods that might bring insights relevant to clinical care and endeavor to match the right method to the question at hand. The Institute of Medicine Roundtable on Value & Science-Driven Health Care's vision for a learning healthcare system, in which evidence is applied and generated as a natural course of care, is premised on the development of a research capacity that is structured to provide timely and accurate evidence relevant to the clinical decisions faced by patients and providers. As part of the Roundtable's Learning Healthcare System series of workshops, clinical researchers, academics, and policy makers gathered for the workshop "Redesigning the Clinical Effectiveness Research Paradigm: Innovation and Practice-Based Approaches." Participants explored cutting-edge research designs and methods and discussed strategies for development of a research paradigm to better accommodate the diverse array of emerging data resources, study designs, tools, and techniques. Presentations and discussions are summarized in this volume.

It is essential for patients and clinicians to have the resources needed to make informed, collaborative care decisions. Despite this need, only a small fraction of health-related expenditures in the United States have been devoted to comparative effectiveness research (CER). To improve the effectiveness and value of the care delivered, the nation needs to build its capacity for ongoing study and monitoring of the relative effectiveness of clinical interventions and care processes through expanded trials and studies, systematic reviews, innovative research strategies, and clinical registries, as well as improving its ability to apply what is learned from such study through the translation and provision of information and decision support. As part of its Learning Health System series of workshops, the Institute of Medicine's (IOM's) Roundtable on Value & Science-Driven Health Care hosted a workshop to discuss capacity priorities to build the evidence base necessary for care that is more effective and delivers higher value for patients. Learning What Works summarizes the proceedings of the seventh workshop in the Learning Health System series. This workshop focused on the infrastructure needs-including methods, coordination capacities, data resources and linkages, and workforce-for developing an expanded and efficient national capacity for CER. Learning What Works also assesses the current and needed capacity to expand and improve this work, and identifies priority next steps. Learning What Works is a valuable resource for health care professionals, as well as health care policy makers. Table of Contents Front Matter Summary 1 The Need and Potential Returns for Comparative Effectiveness Research 2 The Work Required 3 The Information Networks Required 4 The Talent Required 5 Implementation Priorities 6 Moving Forward Appendix A: Learning What Works Best: The Nation's Need for Evidence on Comparative Effectiveness in Health Care Appendix B: Comparative Effectiveness Studies Inventory Project Appendix C: Comparative Effectiveness Research Priorities: IOM Recommendations (2009) Appendix D: Comparative Effectiveness Research Priorities: FCCCER Recommendations (2009) Appendix E: Affordable Care Act (ACA) (2010) Provisions for the Patient-Centered Outcomes Research Institute (PCORI) Appendix F: Workshop Agenda Appendix G: Biographical Sketches of Workshop Participants Appendix H: Workshop Attendee List Other Publications in The Learning Health System Series

Like many other industries, health care is increasingly turning to digital information and the use of electronic resources. The Institute of Medicine's Roundtable on Value & Science-Driven Health Care hosted three workshops to explore current efforts and opportunities to accelerate progress in improving health and health care with information technology systems. Table of Contents Front Matter Synopsis and Highlights 1 Introduction 2 Visioning Perspectives on the Digital Health Utility 3 Technical Issues for the Digital Health Infrastructure 4 Engaging Patient and Population Needs 5 Weaving a Strong Trust Fabric 6 Stewardship and Governance in the Learning Health System 7 Perspectives on Innovation 8 Fostering the Global Dimension of the Health Data Trust 9 Growing the Digital Health Infrastructure 10 Accelerating Progress Appendix A: The Learning Health System and the Digital Health Utility Appendix B: Case Studies for the Digital Health Infrastructure Appendix C: Example Stakeholder Responsibilities and Opportunities Appendix D: Summary Overview of Meaningful Use Objectives Appendix E: PCAST Report Recommendations Appendix F: Workshop Agendas Appendix G: Workshop Participants Other Publications in The Learning Health System Series

As past, current, or future patients, the public should be the health care system's unwavering focus and serve as change agents in its care. Taking this into account, the quality of health care should be judged not only by whether clinical decisions are informed by the best available scientific evidence, but also by whether care is tailored to a patient's individual needs and perspectives. However, too often it is provider preference and convenience, rather than those of the patient, that drive what care is delivered. As part of its Learning Health System series of workshops, the Roundtable on Value & Science-Driven Health Care hosted a workshop to assess the prospects for improving health and lowering costs by advancing patient involvement in the elements of a learning health system. Table of Contents Front Matter Synopsis and Overview 1 The Learning Health System 2 Clinical Research, Patient Care, and Learning That Is Real-Time and Continuous 3 Clinical Data as a Public Good for Discovery 4 Engaging Patients to Improve Science and Value in a Learning Health System 5 Health Information Technology as the Engine for Learning 6 Patients, Clinical Decisions, and Health Information Management in the Information Age 7 Applying Evidence for Patient-Centered Care: Standards and Expectations 8 Team-Based Care and the Learning Culture 9 Incentives Aligned with Value and Learning 10 Common Themes and Opportunities for Action Appendixes Appendix A: Workshop Agenda Appendix B: Biographical Sketches of Workshop Participants Appendix C: Workshop Attendee List Appendix D: The Learning Health System Series: Workshop Common Themes OTHER PUBLICATIONS IN THE LEARNING HEALTH SYSTEM SERIES

Improving our nation's healthcare system is a challenge which, because of its scale and complexity, requires a creative approach and input from many different fields of expertise. Lessons from engineering have the potential to improve both the efficiency and quality of healthcare delivery. The fundamental notion of a high-performing healthcare system-one that increasingly is more effective, more efficient, safer, and higher quality-is rooted in continuous improvement principles that medicine shares with engineering. As part of its Learning Health System series of workshops, the Institute of Medicine's Roundtable on Value and Science-Driven Health Care and the National Academy of Engineering, hosted a workshop on lessons from systems and operations engineering that could be applied to health care. Building on previous work done in this area the workshop convened leading engineering practitioners, health professionals, and scholars to explore how the field might learn from and apply systems engineering principles in the design of a learning healthcare system. Engineering a Learning Healthcare System: A Look at the Future: Workshop Summary focuses on current major healthcare system challenges and what the field of engineering has to offer in the redesign of the system toward a learning healthcare system. Table of Contents Front Matter Summary 1 Engineering a Learning Healthcare System 2 Engaging Complex Systems Through Engineering Concepts 3 Healthcare System Complexities, Impediments, and Failures 4 Case Studies in Transformation Through Systems Engineering 5 Fostering Systems Change to Drive Continuous Learning in Health Care 6 Next Steps: Aligning Policies with Leadership Opportunities Appendixes Appendix A: Workshop Agenda Appendix B: Biographical Sketches of Workshop Participants Appendix C: Workshop Attendee List Other Publications in The Learning Health System Series

Successful development of clinical data as an engine for knowledge generation has the potential to transform health and health care in America. As part of its Learning Health System Series, the Roundtable on Value & Science-Driven Health Care hosted a workshop to discuss expanding the access to and use of clinical data as a foundation for care improvement. Table of Contents Front Matter Summary 1 Clinical Data as the Basic Staple of the Learning Health System 2 U.S. Healthcare Data Today: Current State of Play 3 Changing the Terms: Data System Transformation in Progress 4 Healthcare Data: Public Good or Private Property? 5 Healthcare Data as a Public Good: Privacy and Security 6 Creating a Next-Generation Data Utility: Building Blocks and the Action Agenda 7 Engaging the Public 8 Clinical Data as the Basic Staple of Health Learning: Ideas for Action Appendixes Appendix A: Workshop Agenda Appendix B: Biographical Sketches of Workshop Participants Appendix C: Workshop Attendee List Appendix D: The IOM Committee on Health Research and the Privacy of Health Information: The HIPAA Privacy Rule Other Publications in the Learning Healthcare System Series

The United States has the highest per capita spending on health care of any industrialized nation. Yet despite the unprecedented levels of spending, harmful medical errors abound, uncoordinated care continues to frustrate patients and providers, and U.S. healthcare costs continue to increase. The growing ranks of the uninsured, an aging population with a higher prevalence of chronic diseases, and many patients with multiple conditions together constitute more complicating factors in the trend to higher costs of care. A variety of strategies are beginning to be employed throughout the health system to address the central issue of value, with the goal of improving the net ratio of benefits obtained per dollar spent on health care. However, despite the obvious need, no single agreed-upon measure of value or comprehensive, coordinated systemwide approach to assess and improve the value of health care exists. Without this definition and approach, the path to achieving greater value will be characterized by encumbrance rather than progress. To address the issues central to defining, measuring, and improving value in health care, the Institute of Medicine convened a workshop to assemble prominent authorities on healthcare value and leaders of the patient, payer, provider, employer, manufacturer, government, health policy, economics, technology assessment, informatics, health services research, and health professions communities. The workshop, summarized in this volume, facilitated a discussion of stakeholder perspectives on measuring and improving value in health care, identifying the key barriers and outlining the opportunities for next steps.

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 wellbeing. 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.

Creating an environment in which children in the United States grow up healthy should be a high priority for the nation. Yet the prevailing pattern of food and beverage marketing to children in America represents, at best, a missed opportunity, and at worst, a direct threat to the health prospects of the next generation. Children's dietary and related health patterns are shaped by the interplay of many factors?their biologic affinities, their culture and values, their economic status, their physical and social environments, and their commercial media environments?all of which, apart from their genetic predispositions, have undergone significant transformations during the past three decades. Among these environments, none have more rapidly assumed central socializing roles among children and youth than the media. With the growth in the variety and the penetration of the media have come a parallel growth with their use for marketing, including the marketing of food and beverage products. What impact has food and beverage marketing had on the dietary patterns and health status of American children? The answer to this question has the potential to shape a generation and is the focus of Food Marketing to Children and Youth. This book will be of interest to parents, federal and state government agencies, educators and schools, health care professionals, industry companies, industry trade groups, media, and those involved in community and consumer advocacy. Table of Contents Front Matter Executive Summary 1 Setting the Stage 2 Health, Diet, and Eating Patterns of Children and Youth 3 Factors Shaping Food and Beverage Consumption of Children and Youth 4 Food and Beverage Marketing to Children and Youth 5 Influence of Marketing on the Diets and Diet- Related Health of Children and Youth 6 Public Policy Issues in Food and Beverage Marketing to Children and Youth 7 Findings, Recommendations, Next Steps A Acronyms B Glossary C Literature Review Appendix D Chapter 2 Appendix Appendix E Chapter 4 Appendix Appendix F Chapter 5 Appendix Appendix G Chapter 6 Appendix Appendix H Workshop Program Appendix I Biographical Sketches of Committee Members and Staff Index

America's health care system has become too complex and costly to continue business as usual. Best Care at Lower Cost explains that inefficiencies, an overwhelming amount of data, and other economic and quality barriers hinder progress in improving health and threaten the nation's economic stability and global competitiveness. According to this report, the knowledge and tools exist to put the health system on the right course to achieve continuous improvement and better quality care at a lower cost. The costs of the system's current inefficiency underscore the urgent need for a systemwide transformation. About 30 percent of health spending in 2009--roughly $750 billion--was wasted on unnecessary services, excessive administrative costs, fraud, and other problems. Moreover, inefficiencies cause needless suffering. By one estimate, roughly 75,000 deaths might have been averted in 2005 if every state had delivered care at the quality level of the best performing state. This report states that the way health care providers currently train, practice, and learn new information cannot keep pace with the flood of research discoveries and technological advances. About 75 million Americans have more than one chronic condition, requiring coordination among multiple specialists and therapies, which can increase the potential for miscommunication, misdiagnosis, potentially conflicting interventions, and dangerous drug interactions. Best Care at Lower Cost emphasizes that a better use of data is a critical element of a continuously improving health system, such as mobile technologies and electronic health records that offer significant potential to capture and share health data better. In order for this to occur, the National Coordinator for Health Information Technology, IT developers, and standard-setting organizations should ensure that these systems are robust and interoperable. Clinicians and care organizations should fully adopt these technologies, and patients should be encouraged to use tools, such as personal health information portals, to actively engage in their care. This book is a call to action that will guide health care providers; administrators; caregivers; policy makers; health professionals; federal, state, and local government agencies; private and public health organizations; and educational institutions.

As our nation enters a new era of medical science that offers the real prospect of personalized health care, we will be confronted by an increasingly complex array of health care options and decisions. The Learning Healthcare System considers how health care is structured to develop and to apply evidence-from health profession training and infrastructure development to advances in research methodology, patient engagement, payment schemes, and measurement-and highlights opportunities for the creation of a sustainable learning health care system that gets the right care to people when they need it and then captures the results for improvement. This book will be of primary interest to hospital and insurance industry administrators, health care providers, those who train and educate health workers, researchers, and policymakers. The Learning Healthcare System is the first in a series that will focus on issues important to improving the development and application of evidence in health care decision making. The Roundtable on Evidence-Based Medicine serves as a neutral venue for cooperative work among key stakeholders on several dimensions: to help transform the availability and use of the best evidence for the collaborative health care choices of each patient and provider; to drive the process of discovery as a natural outgrowth of patient care; and, ultimately, to ensure innovation, quality, safety, and value in health care. Table of Contents Front Matter Summary 1 Hints of a Different Way-Case Studies in Practice-Based Evidence 2 The Evolving Evidence Base-Methodologic and Policy Challenges 3 Narrowing the Research-Practice Divide-Systems Considerations 4 New Approaches-Learning Systems in Progress 5 Developing the Test Bed: Linking Integrated Service Delivery Systems 6 The Patient as a Catalyst for Change 7 Training the Learning Health Professional 8 Structuring the Incentives for Change Appendix A Workshop Agenda Appendix B Biographical Sketches of Participants Appendix C Workshop Attendee List Appendix D IOM Roundtable on Evidence-Based Medicine

Risk assessment is a highly important activity of numerous governmental health and regulatory bodies. It is on the accuracy of quantitative and qualitative measurement that the decisions of government policymakers depend. Those decisions, of course, are intended to manage risks. That management frequently involves regulations over a wide range of individual and environmental exposures. Bailar and his colleagues examine the methodological challenges faced by federal agencies involved in risk assessment and the sometimes controversial implications and consequences of methodological considerations. The authors query how, given a choice of methods, one is chosen; the role that method-related issues and problems may have in the acceptance of risk assessment findings; and what impact the controversies regarding methods have on the role of risk assessment in overall risk management.

Ten hazards, as assessed by a range of federal agencies with a variety of assessment methods, give topicality and specificity to the analysis. Among the risks addressed are ethylene dibromide, formaldehyde, passive smoking, and the use of mammography for breast cancer screening. The authors conclude with a setting of priorities for risk assessment because risks to human health clearly outstrip resources available for accurate assessment.