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:

  1. Fostering progress toward the long-term vision of a learning health-care system, in which evidence is both applied and developed as a natural product of the care process.
  2. 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.
  3. 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