National Academies Press: OpenBook
Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

LARGE SIMPLE TRIALS AND
KNOWLEDGE GENERATION IN
A LEARNING HEALTH SYSTEM

Workshop Summary

Claudia Grossmann, Julia Sanders, and Rebecca A. English, Rapporteurs

Roundtable on Value and Science-Driven Health Care

Forum on Drug Discovery, Development, and Translation

Board on Health Sciences Policy

INSTITUTE OF MEDICINE
              OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS

Washington, D.C.

www.nap.edu

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001

NOTICE: The workshop that is the subject of this workshop summary 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 activity was supported by contracts between the National Academy of Sciences and the U.S. Department of Health and Human Services (Contract No. N01-OD-4-2139 TO #203 and HHSN26300023 [Under Base No. HHSN263201200074I] and Contract No. N01-OD-4-2139 TO #276; HHSF22301026T [Under Base No. HHSF223200810020I]), AbbVie Inc., Agency for Healthcare Research and Quality, American Diabetes Association, American Society for Microbiology, Amgen Inc., Association of American Medical Colleges, AstraZeneca, Blue Shield of California, Blue Shield of California Foundation, Bristol-Myers Squibb, Burroughs Wellcome Fund, Celtic Therapeutics, LLLP, Centers for Medicare & Medicaid Services, Critical Path Institute, Doris Duke Charitable Foundation, Eli Lilly and Company, Epic Systems, Inc., FasterCures, Friends of Cancer Research, GlaxoSmithKline, Gordon and Betty Moore Foundation, Health Resources and Services Administration, Hospital Corporation of America, Inc., Johnson & Johnson, Kaiser Permanente (East Bay Community Foundation), March of Dimes Foundation, Merck & Co., Inc., Novartis Pharmaceuticals Corporation, Partners HealthCare, Patient-Centered Outcomes Research Institute, Pfizer Inc., Premier, Sanofi, United Health Foundation, and WellPoint, Inc. The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for the activity.

International Standard Book Number-13: 978-0-309-28911-5

International Standard Book Number-10: 0-309-28911-4

Additional copies of this workshop summary are available for sale from the National Academies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313; http://www.nap.edu.

For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu.

Copyright 2013 by the National Academy of Sciences. All rights reserved.

Printed in the United States of America

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: IOM (Institute of Medicine). 2013. Large simple trials and knowledge generation in a learning health system: Workshop summary. Washington, DC: The National Academies Press.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

Knowing is not enough; we must apply.
Willing is not enough; we must do.
”      

                                                —Goethe

image

INSTITUTE OF MEDICINE
              OF THE NATIONAL ACADEMIES

Advising the Nation. Improving Health.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

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. C. D. Mote, Jr., 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. C. D. Mote, Jr., are chair and vice chair, respectively, of the National Research Council.

www.national-academies.org

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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PLANNING COMMITTEE FOR THE WORKSHOP
ON LARGE SIMPLE TRIALS AND KNOWLEDGE
GENERATION IN A LEARNING SYSTEM1

DAVID L. DEMETS (Co-Chair), Professor, Biostatistics and Medical Informatics, University of Wisconsin School of Public Health

RICHARD E. KUNTZ (Co-Chair), Senior Vice President and Chief Scientific, Clinical, and Regulatory Officer, Medtronic

WILLIAM H. CROWN, President, HEOR and Late Phase Research, OPTUMInsight Life Sciences

JEFFREY M. DRAZEN, Editor-in-Chief, New England Journal of Medicine

RALPH I. HORWITZ, Senior Vice President, Clinical Sciences Evaluation, GlaxoSmithKline

PETRA KAUFMANN, Director, Office of Clinical Research, National Institute of Neurological Disorders and Stroke

JUDITH M. KRAMER, Senior Scientific Advisor, Clinical Trials Transformation Initiative, Duke Translational Medicine Institute

MICHAEL S. LAUER, Director, Division of Cardiovascular Sciences, National Heart, Lung, and Blood Institute

JOANN E. MANSON, Professor of Medicine, Harvard Medical School

JOHN J. ORLOFF, Chief Medical Officer and Senior Vice President, Global Development, U.S. Medical and Drug Regulatory Affairs, Novartis Pharmaceuticals Corporation

ERIC D. PETERSON, Professor of Medicine, Division of Cardiology, Duke University Medical School

RICHARD PLATT, Chair, Population Medicine, Harvard Medical School

JOE V. SELBY, Executive Director, Patient-Centered Outcomes Research Institute

RACHEL E. SHERMAN, Director, Office of Medical Policy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

JOSE M. VEGA, Vice President, Global Safety, Amgen Inc.

________________

1 Institute of Medicine planning committees are solely responsible for organizing the workshop, identifying topics, and choosing speakers. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

IOM Staff

CLAUDIA GROSSMANN, Senior Program Officer

ANNE B. CLAIBORNE, Senior Program Officer

REBECCA A. ENGLISH, Associate Program Officer

ELIZABETH F. C. TYSON, Research Associate

JULIA SANDERS, Senior Program Assistant

ELIZABETH JOHNSTON, Program Assistant

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

ROUNDTABLE ON VALUE & SCIENCE-DRIVEN HEALTH CARE1

MARK B. MCCLELLAN (Chair), Senior Fellow and Director, Health Care Innovation and Value Initiative, The Brookings Institution

RAYMOND BAXTER, Senior Vice President, Community, Benefit, Research and Health Policy, Kaiser Permanente

DAVID BLUMENTHAL, President, The Commonwealth Fund

BRUCE G. BODAKEN, Former Chairman and Chief Executive Officer, Blue Shield of California

PAUL CHEW, Chief Science Officer and Chief Medical Officer, Sanofi U.S.

FRANCIS COLLINS, Director, National Institutes of Health (Ex Officio) (designee: Kathy Hudson)

HELEN DARLING, President, National Business Group on Health

SUSAN DEVORE, Chief Executive Officer, Premier, Inc.

JUDITH FAULKNER, Founder and Chief Executive Officer, Epic Health Systems

THOMAS R. FRIEDEN, Director, Centers for Disease Control and Prevention (Ex Officio) (designee: James Galloway)

PATRICIA A. GABOW, Former Chief Executive Officer, Denver Health

ATUL GAWANDE, General and Endocrine Surgeon, Brigham and Women’s Hospital

GARY L. GOTTLIEB, President and Chief Executive Officer, Partners HealthCare

JAMES A. GUEST, President and Chief Executive Officer, Consumers Union

GEORGE C. HALVORSON, Chairman and Chief Executive Officer, Kaiser Permanente

MARGARET A. HAMBURG, Commissioner, U.S. Food and Drug Administration (Ex Officio) (designee: Peter Lurie)

JAMES HEYWOOD, Cofounder and Chairman, PatientsLikeMe

RALPH I. HORWITZ, Senior Vice President, Clinical Evaluation Sciences, GlaxoSmithKline

PAUL HUDSON, Executive Vice President, AstraZeneca

BRENT C. JAMES, Chief Quality Officer, Intermountain Healthcare

CRAIG JONES, Director, Vermont Blueprint for Health

GARY KAPLAN, Chairman and Chief Executive Officer, Virginia Mason Health System

________________

1 Institute of Medicine forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.

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Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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DARRELL G. KIRCH, President and Chief Executive Officer, Association of American Medical Colleges

RICHARD KRONICK, Director, Agency for Healthcare Research and Quality (Ex Officio)

RICHARD C. LARSON, Mitsui Professor, Massachusetts Institute of Technology

JAMES L. MADARA, Chief Executive Officer, American Medical Association

FARZAD MOSTASHARI, National Coordinator, Office of the National Coordinator for Health IT (Ex Officio)

MARY D. NAYLOR, Director, NewCourtland Center, University of Pennsylvania

WILLIAM D. NOVELLI, Former Chief Executive Officer, AARP; Professor, Georgetown University

SAM R. NUSSBAUM, Chief Medical Officer, WellPoint, Inc.

JONATHAN B. PERLIN, President, Clinical and Physician Services and Chief Medical Officer, Hospital Corporation of America, Inc.

ROBERT A. PETZEL, Under Secretary for Health, U.S. Department of Veterans Affairs (Ex Officio)

RICHARD PLATT, Chair, Population Medicine, Harvard Medical School

MICHAEL ROSENBLATT, Executive Vice President and Chief Medical Officer, Merck & Co., Inc.

JOHN W. ROWE, Former Chairman and Chief Executive Officer, Aetna; Professor, Columbia University

JOE V. SELBY, Executive Director, Patient-Centered Outcomes Research Institute

MARK D. SMITH, President and Chief Executive Officer, California HealthCare Foundation

GLENN D. STEELE, President and Chief Executive Officer, Geisinger Health System

MARILYN TAVENNER, Administrator, Centers for Medicare & Medicaid Services (Ex Officio) (designee: Patrick Conway)

REED V. TUCKSON, Former Executive Vice President, UnitedHealth Group; Managing Director, Tuckson Health

MARY WAKEFIELD, Administrator, Health Resources and Services Administration (Ex Officio)

DEBRA B. WHITMAN, Executive Vice President, Policy and International, AARP

JONATHAN WOODSON, Assistant Secretary for Health Affairs, U.S. Department of Defense (Ex Officio)

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

IOM Staff

KATHERINE BURNS, Program Assistant

CLAUDIA GROSSMANN, Senior Program Officer

DIEDTRA HENDERSON, Program Officer

ELIZABETH JOHNSTON, Program Assistant

ELIZABETH ROBINSON, Research Associate

VALERIE ROHRBACH, Senior Program Assistant

JULIA SANDERS, Senior Program Assistant (through August 2013)

ROBERT SAUNDERS, Senior Program Officer

BARRET ZIMMERMANN, Program Assistant (through October 2013)

J. MICHAEL MCGINNIS, Senior Scholar, Executive Director, Roundtable on Value & Science-Driven Health Care

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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FORUM ON DRUG DISCOVERY,
DEVELOPMENT, AND TRANSLATION1

JEFFREY M. DRAZEN (Co-Chair), Editor-in-Chief, New England Journal of Medicine

STEVEN K. GALSON (Co-Chair), Global Regulatory Vice President, Amgen Inc.

RUSS BIAGIO ALTMAN, The Kenneth Fong Professor of Bioengineering, Genetics, Medicine, and (by courtesy) Computer Science, Stanford University

MARGARET ANDERSON, Executive Director, FasterCures

HUGH AUCHINCLOSS, Deputy Director, National Institute of Allergy and Infectious Diseases

CHRISTOPHER P. AUSTIN, Director, National Center for Advancing Translational Sciences

ANN C. BONHAM, Chief Scientific Officer, Association of American Medical Colleges

LINDA BRADY, Director, Division of Neuroscience and Basic Behavioral Science, National Institute of Mental Health

GAIL H. CASSELL, Visiting Professor, Department of Social Medicine and Global Medicine, Harvard Medical School

PETER B. CORR, Cofounder, Managing General Partner, Auven Therapeutics, LLP

ANDREW M. DAHLEM, Vice President and Chief Operating Officer, Lilly Research Laboratories, Eli Lilly and Company

JAMES H. DOROSHOW, Deputy Director for Clinical and Translational Research, Division of Cancer Treatment and Diagnosis, National Cancer Institute

GARY L. FILERMAN, President, Atlas Health Foundation

MARK J. GOLDBERGER, Divisional Vice President, Regulatory Policy and Intelligence, Pharmaceutical Products Group, Abbott Pharmaceuticals

HARRY B. GREENBERG, Senior Associate Dean for Research and Joseph D. Grant Professor of Medicine, Microbiology, and Immunology, Stanford University School of Medicine

PETER K. HONIG, Global Vice President, Regulatory Affairs, AstraZeneca

KATHY L. HUDSON, Deputy Director for Science, Outreach, and Policy, National Institutes of Health

________________

1 Institute of Medicine forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
×

LYNN D. HUDSON, Chief Science Officer, Critical Path Institute

S. CLAIBORNE JOHNSTON, Professor of Neurology and Epidemiology, Associate Vice Chancellor of Research, and Director, Clinical and Translational Science Institute, University of California, San Francisco

MICHAEL KAT Z, Senior Advisor, Transdisciplinary Research, March of Dimes Foundation

PETRA KAUFMANN, Director, Office of Clinical Research, National Institute of Neurological Disorders and Stroke

JACK D. KEENE, James B. Duke Professor of Molecular Genetics and Microbiology, Duke University Medical Center

RUSTY KELLEY, Program Officer, Burroughs Wellcome Fund

RONALD L. KRALL, Associate Fellow, University of Pennsylvania Center for Bioethics

FREDA C. LEWIS-HALL, Executive Vice President and Chief Medical Officer, Pfizer Inc.

CAROL MIMURA, Assistant Vice Chancellor for Intellectual Property & Industry Research Alliances, University of California, Berkeley

BERNARD H. MUNOS, Founder, InnoThink Center for Research in Biomedical Innovation

ELIZABETH (BETSY) MYERS, Program Director for Medical Research, Doris Duke Charitable Foundation

JOHN J. ORLOFF, Chief Medical Officer and Senior Vice President, Global Development, U.S. Medical and Drug Regulatory Affairs, Novartis Pharmaceuticals Corporation

ROBERT E. RATNER, Chief Scientific Medical Officer, American Diabetes Association

MICHAEL ROSENBLATT, Executive Vice President and Chief Medical Officer, Merck & Co., Inc.

JAMES S. SHANNON, Chief Medical Officer, GlaxoSmithKline

JANET SHOEMAKER, Director, Office of Public Affairs, American Society for Microbiology

ELLEN V. SIGAL, Chair and Founder, Friends of Cancer Research

LANA R. SKIRBOLL, Vice President, Academic and Scientific Affairs, Sanofi

BRIAN L. STROM, Executive Vice Dean for Institutional Affairs and Professor, University of Pennsylvania Perelman School of Medicine

JANET TOBIAS, President, Sierra/Tango Productions, Ikana Media

JOANNE WALDSTREICHER, Chief Medical Officer, Johnson & Johnson

JANET WOODCOCK, Director, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

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Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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IOM Staff

ANNE B. CLAIBORNE, Forum Director

REBECCA A. ENGLISH, Associate Program Officer

ELIZABETH F. C. TYSON, Research Associate

ROBIN GUYSE, Senior Program Assistant

ANDREW M. POPE, Director, Board on Health Sciences Policy

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Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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Reviewers

This workshop summary 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 workshop summary as sound as possible and to ensure that the workshop summary 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 process. We wish to thank the following individuals for their review of this workshop summary:

STEFAN JAMES, Uppsala University Hospital

Elliott Levy, Bristol-Myers Squibb

Sean Tunis, Center for Medical Technology Policy

Paul J. Wallace, Optum Labs

Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the workshop summary before its release. The review of this workshop summary was overseen by Joel Kupersmith, Veterans Health Administration. Appointed by the Institute of Medicine, he was responsible for making certain that an independent examination of this workshop summary was carried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this workshop summary rests entirely with the rapporteurs and the institution.

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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Preface

The current paradigms for effectiveness research and drug development face increasingly acute challenges in cost, timing, and applicability, given their reliance on classic randomized controlled trials. Opportunities to engage these challenges by the use of large simple trial (LST) designs and the integration of research at the point of care prompted the Institute of Medicine’s Roundtable on Value & Science-Driven Health Care (the Roundtable) and the Forum on Drug Discovery, Development, and Translation (the Forum) to convene a workshop, Large Simple Trials and Knowledge Generation in a Learning Health System, which is summarized in this publication.

Experts from a wide range of disciplines—including health information technology, research funding, clinical research methods, statistics, patients, product development, medical product regulation, and clinical outcomes research—met to marshal a better understanding of the issues, options, and approaches to accelerating the use of LSTs. This publication summarizes discussions on the potential of LSTs to improve the speed and practicality of knowledge generation for medical decision making and medical product development, including efficacy and effectiveness assessments, in a continuously learning health system.

The work of the Roundtable is focused on moving toward a continuously learning health system, one in which every care encounter is an opportunity for learning and evidence is applied to ensure and improve best care practices. Since its inception in 2006, the Roundtable has set out to

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Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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help realize this vision through the support, involvement, and activities of senior leadership from key stakeholders in the health care system.

Since its creation in 2005, the Forum has provided a platform for dialogue and collaboration among thought leaders and stakeholders in government, academia, industry, foundations, and patient advocacy. Since 2009, the Forum has convened an ongoing initiative dedicated to addressing the challenges facing the U.S. clinical trials enterprise by bringing together the broad range of clinical research stakeholders to surface potentially transformative strategies to improve the efficiency and effectiveness of clinical trials. The Forum’s public meetings focus substantial public attention on critical areas of drug discovery, development, and translation and in doing so serve to educate the policy community about issues where science and policy intersect.

Building on this groundwork, the objectives of this workshop were to explore acceleration of the use of LSTs to improve the speed and practicality of knowledge generation for medical decision making and medical product development; consider the concepts of LST design, examples of successful LSTs, the relative advantages of LSTs, and the infrastructure needed to build LST capacity as a routine function of care; identify structural, cultural, and regulatory barriers hindering the development of an enhanced LST capacity; discuss needs and strategies in building public demand for and participation in LSTs; and consider near-term strategies for accelerating progress in the uptake of LSTs in the United States.

Multiple individuals donated valuable time toward the development of this publication. We acknowledge and offer strong appreciation to the contributors to this publication for their presence at the workshop and their efforts to further develop their presentations into the summaries contained in this publication. We are especially indebted to those who provided counsel by serving on the planning committee for this workshop.

A number of Roundtable and Forum staff played instrumental roles in coordinating the workshop and translating the workshop proceedings into this summary, including Claudia Grossmann, Anne Claiborne, Julia Sanders, Rebecca A. English, Elizabeth Johnston, Elizabeth Tyson, Valerie Rohrbach, Robin Guyse, Barret Zimmermann, Rob Saunders, Diedtra Henderson, and Elizabeth Robinson. Finally, we thank Daniel Bethea, Marton Cavani, Laura Harbold DeStefano, and Chelsea Frakes for helping to coordinate various aspects of review, production, and publication.

A fundamental component of a learning health system is the ability to improve the speed and practicality of knowledge generation. The increased use of LSTs that rely on larger, more representative populations,

Suggested Citation:"Front Matter." Institute of Medicine. 2013. Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18400.
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and optimize data collection could serve to speed the necessary transformation. We believe that Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary will be a valuable resource in informing this transformation.

David L. DeMets

Planning Committee Co-Chair

Professor

University of Wisconsin School of Public Health

Richard E. Kuntz

Planning Committee Co-Chair

Senior Vice President and

Chief Scientific, Clinical, and Regulatory Officer

Medtronic

J. Michael McGinnis

Senior Scholar

Executive Director

Roundtable on Value & Science-Driven Health Care

Institute of Medicine

Andrew M. Pope

Director

Board on Health Sciences Policy

Institute of Medicine

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Randomized clinical trials (RCTs) are often referred to as the "gold standard" of clinical research. However, in its current state, the U.S. clinical trials enterprise faces substantial challenges to the efficient and effective conduct of research. Streamlined approaches to RCTs, such as large simple trials (LSTs), may provide opportunities for progress on these challenges. Clinical trials support the development of new medical products and the evaluation of existing products by generating knowledge about safety and efficacy in pre- and post-marketing settings and serve to inform medical decision making and medical product development. Although well-designed and -implemented clinical trials can provide robust evidence, a gap exists between the evidence needs of a continuously learning health system, in which all medical decisions are based on the best available evidence, and the reality, in which the generation of timely and practical evidence faces significant barriers.

Large Simple Trials and Knowledge Generation in a Learning Health System is the summary of a workshop convened by the Institute of Medicine's Roundtable on Value & Science-Driven Health Care and the Forum on Drug Discovery, Development, and Translation. Experts from a wide range of disciplines--including health information technology, research funding, clinical research methods, statistics, patients, product development, medical product regulation, and clinical outcomes research--met to marshal a better understanding of the issues, options, and approaches to accelerating the use of LSTs. This publication summarizes discussions on the potential of LSTs to improve the speed and practicality of knowledge generation for medical decision making and medical product development, including efficacy and effectiveness assessments, in a continuously learning health system.

Large Simple Trials and Knowledge Generation in a Learning Health System explores acceleration of the use of LSTs to improve the speed and practicality of knowledge generation for medical decision making and medical product development; considers the concepts of LST design, examples of successful LSTs, the relative advantages of LSTs, and the infrastructure needed to build LST capacity as a routine function of care; identifies structural, cultural, and regulatory barriers hindering the development of an enhanced LST capacity; discusses needs and strategies in building public demand for and participation in LSTs; and considers near-term strategies for accelerating progress in the uptake of LSTs in the United States.

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