IOM ROUNDTABLE ON EVIDENCE-BASED MEDICINE
THE LEARNING HEALTHCARE SYSTEM
THE NATIONAL ACADEMIES PRESS
THE NATIONAL ACADEMIES PRESS
500 Fifth Street, N.W. Washington, DC 20001
NOTICE: The project that is the subject of this report was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine.
This project was supported by the contracts between the National Academy of Sciences and Agency for Healthcare Research and Quality, America’s Health Insurance Plans, AstraZeneca, Blue Shield of California Foundation, Burroughs Wellcome Fund, California Health Care Foundation, Centers for Medicare and Medicaid Services, Department of Veterans Affairs, Food and Drug Administration, Johnson & Johnson, sanofi-aventis, and Stryker. Any opinions, findings, conclusions, or recommendations expressed in this publication are those of the author(s) and do not necessarily reflect the view of the organizations or agencies that provided support for this project.
Library of Congress Cataloging-in-Publication Data
The learning healthcare system : workshop summary / [edited by] LeighAnne Olsen, Dara Aisner, and J. Michael McGinnis ; Roundtable on Evidence-Based Medicine.
p. ; cm.
Includes bibliographical references.
ISBN-13: 978-0-309-10300-8 (pbk. : alk. paper)
ISBN-10: 0-309-10300-2 (pbk. : alk. paper) 1. Evidence-based medicine. 2. Medical care—Research—Methodology. 3. Health education. I. Olsen, LeighAnne. II. Aisner, Dara. III. McGinnis, J. Michael. IV. Institute of Medicine (U.S.). Roundtable on Evidence-Based Medicine.
[DNLM: 1. Delivery of Health Care—organization & administration. 2. Biomedical Research. 3. Evidence-Based Medicine. 4. Learning. 5. Quality of Health Care. W 84.1 L438 2007]
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Copyright 2007 by the National Academy of Sciences. All rights reserved.
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The serpent has been a symbol of long life, healing, and knowledge among almost all cultures and religions since the beginning of recorded history. The serpent adopted as a logotype by the Institute of Medicine is a relief carving from ancient Greece, now held by the Staatliche Museen in Berlin.
Suggested citation: Institute of Medicine (IOM). 2007. The Learning Healthcare System: Workshop Summary. Washington, DC: The National Academies Press.
THE NATIONAL ACADEMIES
Advisers to the Nation on Science, Engineering, and Medicine
The National Academy of Sciences is a private, nonprofit, self-perpetuating society of distinguished scholars engaged in scientific and engineering research, dedicated to the furtherance of science and technology and to their use for the general welfare. Upon the authority of the charter granted to it by the Congress in 1863, the Academy has a mandate that requires it to advise the federal government on scientific and technical matters. Dr. Ralph J. Cicerone is president of the National Academy of Sciences.
The National Academy of Engineering was established in 1964, under the charter of the National Academy of Sciences, as a parallel organization of outstanding engineers. It is autonomous in its administration and in the selection of its members, sharing with the National Academy of Sciences the responsibility for advising the federal government. The National Academy of Engineering also sponsors engineering programs aimed at meeting national needs, encourages education and research, and recognizes the superior achievements of engineers. Dr. Wm. A. Wulf is president of the National Academy of Engineering.
The Institute of Medicine was established in 1970 by the National Academy of Sciences to secure the services of eminent members of appropriate professions in the examination of policy matters pertaining to the health of the public. The Institute acts under the responsibility given to the National Academy of Sciences by its congressional charter to be an adviser to the federal government and, upon its own initiative, to identify issues of medical care, research, and education. Dr. Harvey V. Fineberg is president of the Institute of Medicine.
The National Research Council was organized by the National Academy of Sciences in 1916 to associate the broad community of science and technology with the Academy’s purposes of furthering knowledge and advising the federal government. Functioning in accordance with general policies determined by the Academy, the Council has become the principal operating agency of both the National Academy of Sciences and the National Academy of Engineering in providing services to the government, the public, and the scientific and engineering communities. The Council is administered jointly by both Academies and the Institute of Medicine. Dr. Ralph J. Cicerone and Dr. Wm. A. Wulf are chair and vice chair, respectively, of the National Research Council.
ROUNDTABLE ON EVIDENCE-BASED MEDICINE
Denis A. Cortese (Chair), President and Chief Executive Officer,
Adam Bosworth, Vice President of Engineering,
David R. Brennan, Chief Executive Officer,
Carolyn M. Clancy, Director,
Agency for Healthcare Research and Quality
Helen Darling, President,
National Business Group on Health
James A. Guest, President,
George C. Halvorson, Chairman and Chief Executive Officer,
Carmen Hooker Odom, Secretary,
Health and Human Services, State of North Carolina
Michael M. E. Johns, Executive Vice President for Health Affairs,
Michael J. Kussman, Acting Under Secretary for Health,
Department of Veterans Affairs
Cato T. Laurencin, Professor, Chairman of Orthopedic Surgery,
University of Virginia
Stephen P. MacMillan, President and Chief Executive Officer,
Mark B. McClellan,
Senior Fellow, AEI-Brookings Joint Center for Regulatory Studies
Elizabeth G. Nabel, Director,
National Heart, Lung, and Blood Institute
Peter M. Neupert, Corporate Vice President,
Health Solutions Group, Microsoft Corporation
Nancy H. Nielsen, Speaker of the House of Delegates,
American Medical Association
Leslie V. Norwalk, Acting Administrator,
Centers for Medicare and Medicaid Services
Jonathan B. Perlin, Chief Medical Officer and Senior Vice President for Quality,
Richard Platt, Professor and Chair,
Harvard Medical School and Harvard Pilgrim Health Care
John C. Rother, Group Executive Officer,
American Association of Retired Persons
Tim Rothwell, Chairman,
John W. Rowe, Professor,
Columbia University, Mailman School of Public Health
Donald M. Steinwachs, Professor and Director of Health Service Research and Development Center,
Johns Hopkins University
Andrew L. Stern, President,
Service Employees International Union
I. Steven Udvarhelyi, Senior Vice President and Chief Medical Officer,
Independence Blue Cross
Frances M. Visco, President,
National Breast Cancer Coalition
William C. Weldon, Chairman and Chief Executive Officer,
Johnson & Johnson
Janet Woodcock, Deputy Commissioner and Chief Medical Officer,
Food and Drug Administration
Dara L. Aisner, Program Officer
Pamela Bradley, Science and Technology Policy Fellow
Katharine Bothner, Senior Program Assistant
J. Michael McGinnis, Senior Scholar
LeighAnne Olsen, Program Officer
Julie Wiltshire, Financial Associate
This report has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise, in accordance with procedures approved by the National Research Council’s Report Review Committee. The purpose of this independent review is to provide candid and critical comments that will assist the institution in making its published report as sound as possible and to ensure that the report meets institutional standards for objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the deliberative process. We wish to thank the following individuals for their review of this report:
Carmella Bocchino, America’s Health Insurance Plans
Kathy Buto, Johnson & Johnson
Don Goldmann, Institute for Healthcare Improvement
Paul Wallace, The Care Management Institute and KP-Health Solutions, Permanente Federation, Kasier Permanente
Although the reviewers listed above have provided many constructive comments and suggestions, they were not asked to endorse the final draft of the report before its release. The review of this report was overseen by Dr. Elaine L. Larson, School of Nursing, Columbia University. Appointed by the National Research Council and the Institute of Medicine, she was responsible for making certain that an independent examination of this report was carried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this report rests entirely with the authors and the institution.
Institute of Medicine Roundtable on Evidence-Based Medicine
Charter and Vision Statement
The Institute of Medicine’s Roundtable on Evidence-Based Medicine has been convened to help transform the way evidence on clinical effectiveness is generated and used to improve health and health care. Participants have set a goal that, by the year 2020, ninety percent of clinical decisions will be supported by accurate, timely, and up-to-date clinical information, and will reflect the best available evidence. Roundtable members will work with their colleagues to identify the issues not being adequately addressed, the nature of the barriers and possible solutions, and the priorities for action, and will marshal the resources of the sectors represented on the Roundtable to work for sustained public-private cooperation for change.
The Institute of Medicine’s Roundtable on Evidence-Based Medicine has been convened to help transform the way evidence on clinical effectiveness is generated and used to improve health and health care. We seek the development of a learning healthcare system that is designed to generate and apply the best evidence for the collaborative healthcare choices of each patient and provider; to drive the process of discovery as a natural outgrowth of patient care; and to ensure innovation, quality, safety, and value in health care.
Vision: Our vision is for a healthcare system that draws on the best evidence to provide the care most appropriate to 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.
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 available evidence. We feel that this presents a tangible focus for progress toward our vision, that Americans ought to expect at least this level of performance, that it should be feasible with existing resources and emerging tools, and that measures can be developed to track and stimulate progress.
Context: As unprecedented developments in the diagnosis, treatment, and long-term management of disease bring Americans closer than ever to the promise of personalized health care, we are faced with similarly unprecedented challenges to identify and deliver the care most appropriate for individual needs and conditions. Care that is important is often not delivered. Care that is delivered is often not important. In part, this is due to our failure to apply the evidence we have about the medical care that is most effective—a failure related to shortfalls in provider knowledge and accountability, inadequate care coordination and support, lack of insurance, poorly aligned payment incen
tives, and misplaced patient expectations. Increasingly, it is also a result of our limited capacity for timely generation of evidence on the relative effectiveness, efficiency, and safety of available and emerging interventions. Improving the value of the return on our healthcare investment is a vital imperative that will require much greater capacity to evaluate high priority clinical interventions, stronger links between clinical research and practice, and reorientation of the incentives to apply new insights. We must quicken our efforts to position evidence development and application as natural outgrowths of clinical care—to foster health care that learns.
Approach: The IOM Roundtable on Evidence-Based Medicine serves as a forum to facilitate the collaborative assessment and action around issues central to achieving the vision and goal stated. The challenges are myriad and include issues that must be addressed to improve evidence development, evidence application, and the capacity to advance progress on both dimensions. To address these challenges, as leaders in their fields, Roundtable members will work with their colleagues to identify the issues not being adequately addressed, the nature of the barriers and possible solutions, and the priorities for action, and will marshal the resources of the sectors represented on the Roundtable to work for sustained public–private cooperation for change.
Activities include collaborative exploration of new and expedited approaches to assessing the effectiveness of diagnostic and treatment interventions, better use of the patient care experience to generate evidence on effectiveness, identification of assessment priorities, and communication strategies to enhance provider and patient understanding and support for interventions proven to work best and deliver value in health care.
Core concepts and principles: For the purpose of the Roundtable activities, we define evidence-based medicine broadly to mean that, to the greatest extent possible, the decisions that shape the health and health care of Americans—by patients, providers, payers, and policy makers alike—will be grounded on a reliable evidence base, will account appropriately for individual variation in patient needs, and will support the generation of new insights on clinical effectiveness. Evidence is generally considered to be information from clinical experience that has met some established test of validity, and the appropriate standard is determined according to the requirements of the intervention and clinical circumstance. Processes that involve the development and use of evidence should be accessible and transparent to all stakeholders.
A common commitment to certain principles and priorities guides the activities of the Roundtable and its members, including the commitment to the right health care for each person; putting the best evidence into practice; establishing the effectiveness, efficiency, and safety of medical care delivered; building constant measurement into our healthcare investments; the establishment of healthcare data as a public good; shared responsibility distributed equitably across stakeholders, both public and private; collaborative stakeholder involvement in priority setting; transparency in the execution of activities and reporting of results; and subjugation of individual political or stakeholder perspectives in favor of the common good.
One of the important functions of the Institute of Medicine is use of its convening capacity to draw together key stakeholders in a neutral venue, one that allows them to discuss issues and foster collaborative activities around issues in which they have a strong common interest. No issue better demonstrates the importance of such convening than that of evidence-based medicine. We all want to ensure that, as a society, we are doing everything we can to marshal the best evidence in support of the best care for Americans. Yet, we too often fall far short of that ideal. As the Roundtable members have noted in their vision statement, too much care that is important is often not delivered, and too much care that is delivered is often not important.
Part of the problem is due to our inability to provide the evidence we have, and part is due to the inadequacy of the evidence base to keep pace with new tools and approaches for diagnosis and treatment. Both are of central importance to meeting our potential. The latter challenge, in particular, is soon to become much more acute, as new pharmaceuticals, medical devices, biologics, and procedures are introduced into the marketplace— and as advances in genetics give us a better sense of individual differences in response to various interventions. We clearly need a very different approach to the way we develop evidence. Fortunately, the tools are developing to refashion our approaches. The emerging era of individualized medicine and widespread utilization of health information technology presents a dramatically different terrain for clinical research, practice, and healthcare delivery. We can see rich opportunities for improving health through the creation of new knowledge about what works best for whom under what circumstance,
and to apply it more expeditiously. Still, ongoing systemic issues pose significant barriers to our ability to generate and translate such knowledge to improved patient care. Improvements in health care are increasingly to be determined by our capacity to manage information and our ability to develop accurate, timely, and reliable information and expedite the application of evidence in clinical decision making. The IOM Roundtable on Evidence-Based Medicine was created in 2006 to bring together leaders from multiple sectors—patients, health providers, payers, employees, health product manufacturers, information technology companies, policy makers, and researchers—to identify and discuss the issues and approaches to help transform how evidence on clinical effectiveness is generated and used to improve health and health care. As part of the charge, the Roundtable has developed a vision for a healthcare system that has the capacity to draw on the best evidence to provide the care most appropriate to each patient as well as the ability to add to knowledge throughout the delivery of care—a healthcare system that learns.
This publication, The Learning Healthcare System, presents a summary of a workshop held in July 2006 to identify and discuss the broad range of issues that must be engaged if we are to meet the ever-growing demand for evidence that will help bring better health and economic value for our sizable investment in health care. In that workshop, experts from a variety of fields came together to discuss the current approach to evidence development, the standards that are used in drawing conclusions, new research methodologies, some promising initiatives that are under way, and what is needed to enhance the cooperative roles of patients and providers in this work. This volume is rich with insights and sets a solid stage for follow-on activities and discussions on the issues identified.
I would like to offer my personal thanks to the Roundtable members for the important service they are performing on behalf of better health for Americans, to the Roundtable staff for their excellent contributions in coordinating the activities, and importantly, to the sponsors who support this vital activity: the Agency for Healthcare Research and Quality, America’s Health Insurance Plans, AstraZeneca, Blue Shield of California Foundation, Burroughs Wellcome Fund, California Health Care Foundation, Centers for Medicare and Medicaid Services, Department of Veterans Affairs, Food and Drug Administration, Johnson & Johnson, sanofi-aventis, and Stryker. It is this sort of commitment and leadership that give us confidence in our healthcare future.
Harvey V. Fineberg, M.D., Ph.D.
President, Institute of Medicine
The Learning Healthcare System is the first formal product of the Institute of Medicine (IOM) Roundtable on Evidence-Based Medicine. It is a summary of a two-day workshop held in July 2006, convened to consider the broad range of issues important to reengineering clinical research and healthcare delivery so that evidence is available when it is needed, and applied in health care that is both more effective and more efficient than we have today. Embedded in these pages can be found discussions of the myriad issues that must be engaged if we are to transform the way evidence is generated and used to improve health and health care—issues such as the potential for new research methods to enhance the speed and reliability with which evidence is developed, the standards of evidence to be used in making clinical recommendations and decisions, overcoming the technical and regulatory barriers to broader use of clinical data for research insights, and effective communication to providers and the public about the dynamic nature of evidence and how it can be used. Ultimately, our hope and expectation are that the process of generating and applying the best evidence will be natural and seamless components of the process of care itself, as part of a learning healthcare system.
The aim of the IOM Roundtable on Evidence-Based Medicine is to help accelerate our progress toward this vision. Formed last year, and comprised of some of the nation’s most senior leadership from key sectors—consumers and patients, health providers, payers, employees, health product manufacturers, information technology companies, policy makers, and researchers—the work of the Roundtable is anchored in a focus on three dimensions of the challenge:
Fostering progress toward the long-term vision of a learning healthcare system, in which evidence is both applied and developed as a natural product of the care process.
Advancing the discussion and activities necessary to meet the near-term need for expanded capacity to generate the evidence to support medical care that is maximally effective and produces the greatest value.
Improving public understanding of the nature of evidence-based medicine, the dynamic nature of the evidence development process, and the importance of supporting progress toward medical care that reflects the best evidence.
The workshop summarized here was intentionally designed to cast the net broadly across the key topics, to identify issues and commonalties in the perspectives of the various participants. As indicated in the Summary, in the course of workshop discussions, a number of fundamental challenges to effective health care in this country were heard, as were a number of uncertainties, and a number of compelling needs for change.
Among the many challenges heard from participants were that missed opportunity, preventable illness, and injury are too often features in health care, and inefficiency and waste are too familiar characteristics. Insufficient attention to the evidence—both its application and its development—is at the core of these problems. Without a stronger focus on getting and using the right evidence, the pattern is likely to be accentuated as intervention options become more complex and greater insight is gained into patient heterogeneity. In the face of this change, the prevailing approach to generating clinical evidence is impractical today, and may be irrelevant tomorrow. Current approaches to interpreting the evidence and producing guidelines and recommendations often yield inconsistencies and confusion. Meeting these challenges may be facilitated by promising developments in information technology, but those developments must be matched by broader commitments to make culture and practice changes that will allow us to move clinical practice and research into closer alignment.
Among the uncertainties participants underscored were some key questions: Should we continue to call the randomized controlled clinical trial (RCT) the “gold standard”? Although clearly useful and necessary in some circumstances, does this designation over-promise? What do we need to do to better characterize the range of alternatives to RCTs, and the applications and implications for each? What constitutes evidence, and how does it vary by circumstance? How much of evidence development and evidence application will ultimately fall outside of even a fully interoperable and universally adopted electronic health record (EHR)? What are the boundaries of a technical approach? What is the best strategy to get to the
right standards and interoperability for a clinical record system that can be a fully functioning part of evidence development and application? How much can some of the problems of post-marketing surveillance be obviated by the emergence of linked clinical information systems that might allow information about safety and effectiveness to emerge naturally in the course of care?
Engaging the challenges and uncertainties, participants identified a number of pressing needs: adapting to the pace of change, through continuous learning and a much more dynamic approach to evidence development and application; a culture of shared responsibility among patients, providers, and researchers; a new clinical research paradigm that draws clinical research more closely to the experience of clinical practice; clinical decision support systems that accommodate the pace of information growth; full and effective application of electronic health records as an essential prerequisite for the evolution of the learning healthcare system; advancing the notion of clinical data as a public good and a central common resource for advancing knowledge and evidence for effective care; database linkage, mining, and use; stronger incentives to draw research and practice closer together, forging interoperable patient record platforms to foster more rapid learning; better consistency and coordination in efforts to generate, assess, and advise on the results of new knowledge; and the importance of strong and trusted leadership to provide the guidance, shape the priorities, and marshal the vision and actions necessary to create a learning healthcare system.
The workshop then laid out a number challenges requiring the attention and action of stakeholders such as those represented on the Roundtable. We will be following up with deeper consideration of many of these issues through other workshops, commissioned papers, collaborative activities, and public communication efforts. The challenges are large but the Roundtable is populated by committed members who will also reach out to involve their colleagues more widely in the work, assisted by what has been heard and reported through this initial contribution.
We would like to acknowledge all the individuals and organizations that donated their valuable time toward the development of this workshop summary. In particular, we acknowledge the contributors to this volume for their presence at the workshop as well as their efforts to further develop their presentations into the manuscripts contained in this summary. In this respect, we should emphasize that the workshop summary is a collection of individually authored papers and is intended to convey only the views and opinions of individuals participating in the workshop—not the deliberations of the Roundtable on Evidence-Based Medicine, its sponsors, or the Institute of Medicine. We would also like to acknowledge those that provided counsel during the planning stages of this workshop, including Carol Diamond (Markle Foundation), Steve Downs (Robert Wood Johnson
Foundation), Lynn Etheredge (George Washington University), Joe Francis (Department of Veterans Affairs), Brent James (Intermountain Healthcare), Missy Krasner (Google), Nancy Nielsen (American Medical Association), Richard Platt (Harvard), Jeff Shuren (Food and Drug Administration), Susan Shurin (National Institutes of Health), Steven Udverheyli (Independence Blue Cross), and Paul Wallace (Kaiser Permanente). A number of IOM staff were instrumental in the preparation and conduct of the two-day workshop in July, including Shenan Carroll, Amy Grossman, Leon James, Paul Lee, and David Tollerud. Roundtable staff, in particular LeighAnne Olsen along with Dara Aisner and Katharine Bothner helped translate the workshop proceedings and discussion into this workshop summary. We would also like to thank Lara Andersen, Michele de la Menardiere, Bronwyn Schrecker, and Tyjen Tsai for helping to coordinate the various aspects of review, production, and publication.
Encouraging signs exist in our quest toward a learning healthcare system. Progress has been accelerating and we need to sustain this momentum. We look forward to building on this workshop’s insights, and the vision of The Learning Healthcare System is a welcome first step along the path.
Denis A. Cortese, M.D.
Chair, Roundtable on Evidence-Based Medicine
J. Michael McGinnis, M.D., M.P.P.
Senior Scholar, Institute of Medicine
Coverage with Evidence Development,
Use of Large System Databases,
Quasi-Experimental Designs for Policy Assessment,
Practical Clinical Trials,
Computerized Protocols to Assist Clinical Research,
Evolving Methods: Mathematical Models to Help Fill the Gaps in Evidence,
Heterogeneity of Treatment Effects: Subgroup Analysis,
Heterogeneity of Treatment Effects: Pharmacogenetics,
Broader Post-Marketing Surveillance for Insights on Risk and Effectiveness,
Adjusting Evidence Generation to the Scale of Effects,
Protecting Privacy While Linking Patient Records,
Electronic Health Records and Evidence-Based Practice,
Standards of Evidence,
Implications for Accelerating Innovation,
Implementation of Evidence-Based Practice in the VA,
Practice-Based Research Networks,
Envisioning a Rapid Learning Healthcare System,
NIH and Reengineering Clinical Research,
AHRQ and the Use of Integrated Service Delivery Systems,
The HMO Research Network as a Test Bed,
Council of Accountable Physician Practices,
The Internet, eHealth, and Patient Empowerment,
Evidence and Shared Decision Making,
Clinicians and the Electronic Health Record as a Learning Tool,
Opportunities for Private Insurers,
Opportunities for CMS,
Opportunities for Pharmaceutical Companies,
Opportunities for Standards Organizations,