National Academies Press: OpenBook
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

ENVISIONING A TRANSFORMED
CLINICAL TRIALS ENTERPRISE
IN THE UNITED STATES

ESTABLISHING AN AGENDA FOR 2020

img

WORKSHOP SUMMARY

Neil Weisfeld, Rebecca A. English, and Anne B. Claiborne, Rapporteurs


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. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

THE NATIONAL ACADEMIES PRESS     500 Fifth Street, NW     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 study was supported by contracts between the National Academy of Sciences and Department of Health and Human Services (Contract Nos. N01-OD-4-2139 TO #158 and HHSF223001003T), American Society for Microbiology, Amgen Inc., Association of American Medical Colleges, Bristol-Myers Squibb, Burroughs ­Wellcome Fund, Celtic Therapeutics, LLLP, Critical Path Institute, Doris Duke Charitable Foundation, Eli Lilly & Co., FasterCures, Foundation for the NIH, Friends of Cancer Research, GlaxoSmithKline, Janssen Research & Development, LLC, Merck & Co., Inc., Novartis Pharmaceuticals Corporation, and Pfizer Inc. The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for the project.

International Standard Book Number-13:978-0-309-25315-4
International Standard Book Number-10:0-309-25315-2

Additional copies of this report are available 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 2012 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). 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: ­Workshop Summary. Washington, DC: The National Academies Press.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

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

                                      —Goethe





img

INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES

Advising the Nation. Improving Health.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

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. Charles M. Vest 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. Charles M. Vest are chair and vice chair, respectively, of the National Research Council.

www.national-academies.org

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

PLANNING COMMITTEE FOR THE WORKSHOP SERIES ON IMPROVING THE CLINICAL TRIAL PROCESS IN THE UNITED STATES1

JEFFREY M. DRAZEN (Chair), New England Journal of Medicine, Boston, MA

BARBARA ALVING,2 National Center for Research Resources, Bethesda, MD

ANN BONHAM, Association of American Medical Colleges, Washington, DC

LINDA BRADY, National Institute of Mental Health, Bethesda, MD

ROBERT CALIFF, Duke University Medical Center, Durham, NC

SCOTT CAMPBELL,3 Foundation for the National Institutes of Health, Bethesda, MD

GAIL H. CASSELL, Harvard Medical School (visiting), Carmel, IN

JAMES H. DOROSHOW, National Cancer Institute, Bethesda, MD

PAUL R. EISENBERG, Amgen Inc., Thousand Oaks, CA

GARY L. FILERMAN, Atlas Health Foundation, McLean, VA

GARRET A. FITZGERALD, University of Pennsylvania School of Medicine, Philadelphia

PETRA KAUFMANN, National Institute of Neurological Disorders and Stroke, Bethesda, MD

RONALD L. KRALL, University of Pennsylvania Center for Bioethics, Steamboat Springs, CO

FREDA LEWIS-HALL, Pfizer Inc., New York, NY

ELLEN SIGAL, Friends of Cancer Research, Washington, DC

JANET TOBIAS, Ikana Media and Mount Sinai School of Medicine, New York, NY

JANET WOODCOCK, Food and Drug Administration, White Oak, MD

IOM Staff

ANNE B. CLAIBORNE, Forum Director

REBECCA A. ENGLISH, Associate Program Officer

ELIZABETH F. C. TYSON, Research Associate

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

ROBIN GUYSE, Senior Program Assistant

______________________

1Institute 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.

2Barbara Alving was with the National Center for Research Resources until September 30, 2011.

3Scott Campbell was with Foundation for the National Institutes of Health as of the dates of the workshop.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION1

JEFFREY M. DRAZEN (Co-Chair), New England Journal of Medicine, Boston, MA

STEVEN K. GALSON (Co-Chair), Amgen Inc., Thousand Oaks, CA

MARGARET ANDERSON, FasterCures, Washington, DC

HUGH AUCHINCLOSS, National Institute of Allergy and Infectious Diseases, Bethesda, MD

LESLIE Z. BENET, University of California-San Francisco

ANN BONHAM, Association of American Medical Colleges, Washington, DC

LINDA BRADY, National Institute of Mental Health, Bethesda, MD

ROBERT CALIFF, Duke University Medical Center, Durham, NC

C. THOMAS CASKEY,1Baylor College of Medicine, Houston, TX

GAIL H. CASSELL, Harvard Medical School (visiting), Carmel, IN

PETER B. CORR, Celtic Therapeutics, LLLP, New York, NY

ANDREW M. DAHLEM, Eli Lilly & Co., Indianapolis, IN

TAMARA DARSOW, American Diabetes Association, Alexandria, VA

JAMES H. DOROSHOW, National Cancer Institute, Bethesda, MD

GARY L. FILERMAN, Atlas Health Foundation, McLean, VA

GARRET A. FITZGERALD, University of Pennsylvania School of Medicine, Philadelphia

MARK J. GOLDBERGER, Abbott, Rockville, MD

HARRY B. GREENBERG, Stanford University School of Medicine, CA

STEPHEN GROFT, National Center for Advancing Translational Sciences, Bethesda, MD

LYNN HUDSON, Critical Path Institute, Tuscon, AZ

THOMAS INSEL, National Center for Advancing Translational Sciences, Bethesda, MD

MICHAEL KATZ, March of Dimes Foundation, White Plains, NY

PETRA KAUFMANN, National Institute of Neurological Disorders and Stroke, Bethesda, MD

JACK D. KEENE, Duke University Medical Center, Durham, NC

RONALD L. KRALL, University of Pennsylvania Center for Bioethics, Steamboat Springs, CO

FREDA LEWIS-HALL, Pfizer Inc., New York, NY

MARK B. MCCLELLAN, The Brookings Institution, Washington, DC

CAROL MIMURA, University of California, Berkeley

______________________

1Institute 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. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

ELIZABETH (BETSY) MYERS, Doris Duke Charitable Foundation, New York, NY

JOHN ORLOFF, Novartis Pharmaceuticals Corporation, East Hanover, NJ

AMY PATTERSON, National Institutes of Health, Bethesda, MD

MICHAEL ROSENBLATT, Merck & Co., Inc., Whitehouse Station, NJ

JANET SHOEMAKER, American Society for Microbiology, Washington, DC

ELLEN SIGAL, Friends of Cancer Research, Washington, DC

ELLIOTT SIGAL, Bristol-Myers Squibb, Princeton, NJ

ELLEN R. STRAHLMAN, GlaxoSmithKline, Research Triangle Park, NC

NANCY SUNG, Burroughs Wellcome Fund, Research Triangle Park, NC

JANET TOBIAS, Ikana Media and Mount Sinai School of Medicine, New York, NY

JOANNE WALDSTREICHER, Janssen Research & Development, LLC, Raritan, NJ

JANET WOODCOCK, Food and Drug Administration, White Oak, MD

IOM Staff

ANNE B. CLAIBORNE, Forum Director

RITA S. GUENTHER, Program Officer

REBECCA A. ENGLISH, Associate Program Officer

ELIZABETH F. C. TYSON, Research Associate

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

ROBIN GUYSE, Senior Program Assistant

Page viii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

This page intentionally left blank.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

Reviewers

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 process. We wish to thank the following individuals for their review of this report:

Deborah D. Ascheim, Department of Health Evidence and Policy and the Cardiovascular Institute, Mount Sinai School of Medicine

Ralph I. Horwitz, GlaxoSmithKline

James R. O’Dell, Rheumatoid Arthritis Investigational Network, University of Nebraska Medical Center

Deborah A. Zarin, National Library of Medicine, ClinicalTrials.gov

Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the report before its release. The review of this report was overseen by Daniel R. Masys, University of Washington. Appointed by the Institute

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

of Medicine, he 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

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×

Acronyms

ACO

Accountable Care Organization

AHRQ

Agency for Healthcare Research and Quality

AHSS

academic health science system

AMI

acute myocardial infarction

 
CDC

U.S. Centers for Disease Control and Prevention

CED

Coverage with Evidence Development

CER

comparative effectiveness research

CERN

European Organization for Nuclear Research

CF

cystic fibrosis

CMS

Centers for Medicare & Medicaid Services

CRO

contract research organization

CTE

clinical trials enterprise

CTSA

Clinical and Translational Science Award

CTTI

Clinical Trials Transformation Initiative

 
DoD

U.S. Department of Defense

EDC

electronic data capture

EHR

electronic health record

EMA

European Medicines Agency

 
FDA

U.S. Food and Drug Administration

FNIH

Foundation for the National Institutes of Health

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
GCP

Good Clinical Practice

GenISIS

Genomic Informatics System for Integrative Science

GMP

Good Manufacturing Practice

 
HCA

Hospital Corporation of America

HHS

Department of Health and Human Services

HIPAA

Health Insurance Portability and Accountability Act

HITECH

Health Information Technology for Economic and Clinical Health

HMORN

HMO (health maintenance organization) Research Network

 
i2b2

Informatics for Integrating Biology and the Bedside

IHS

integrated health system

IND

Investigational New Drug

INTERMACS

Interagency Registry for Mechanically Assisted Circulatory Support

IOM

Institute of Medicine

IRB

Institutional Review Board

IT

information technology

 
MRSA

methicillin-resistant Staphylococcus Aureus

NCI

National Cancer Institute

NHLBI

National Heart, Lung, and Blood Institute

NIAID

National Institute of Allergy and Infectious Diseases

NIH

National Institutes of Health

NINDS

National Institute of Neurological Disorders and Stroke

 
OAT

Occluded Artery Trial

PACeR

Partnership to Advance Clinical electronic Research

PCORI

Patient-Centered Outcomes Research Institute

 
RCT

randomized controlled trial

REDUCE-MRSA

Randomized Evaluation of Decolonization versus Universal Clearance to Eliminate-MRSA

 
SEMATECH

Semiconductor Manufacturing Technology consortium

 
VA

U.S. Department of Veterans Affairs

VINCI

VA Informatics and Computing Infrastructure

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R1
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R2
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R3
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R4
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R5
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R6
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R7
Page viii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R8
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R9
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R10
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R11
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R12
Page xiii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R13
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R14
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R15
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13345.
×
Page R16
Next: 1 Introduction »
Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020: Workshop Summary Get This Book
×
Buy Paperback | $49.00 Buy Ebook | $39.99
MyNAP members save 10% online.
Login or Register to save!
Download Free PDF

There is growing recognition that the United States' clinical trials enterprise (CTE) faces great challenges. There is a gap between what is desired - where medical care is provided solely based on high quality evidence - and the reality - where there is limited capacity to generate timely and practical evidence for drug development and to support medical treatment decisions.


With the need for transforming the CTE in the U.S. becoming more pressing, the IOM Forum on Drug Discovery, Development, and Translation held a two-day workshop in November 2011, bringing together leaders in research and health care. The workshop focused on how to transform the CTE and discussed a vision to make the enterprise more efficient, effective, and fully integrated into the health care system. Key issue areas addressed at the workshop included: the development of a robust clinical trials workforce, the alignment of cultural and financial incentives for clinical trials, and the creation of a sustainable infrastructure to support a transformed CTE. This document summarizes the workshop.

  1. ×

    Welcome to OpenBook!

    You're looking at OpenBook, NAP.edu's online reading room since 1999. Based on feedback from you, our users, we've made some improvements that make it easier than ever to read thousands of publications on our website.

    Do you want to take a quick tour of the OpenBook's features?

    No Thanks Take a Tour »
  2. ×

    Show this book's table of contents, where you can jump to any chapter by name.

    « Back Next »
  3. ×

    ...or use these buttons to go back to the previous chapter or skip to the next one.

    « Back Next »
  4. ×

    Jump up to the previous page or down to the next one. Also, you can type in a page number and press Enter to go directly to that page in the book.

    « Back Next »
  5. ×

    Switch between the Original Pages, where you can read the report as it appeared in print, and Text Pages for the web version, where you can highlight and search the text.

    « Back Next »
  6. ×

    To search the entire text of this book, type in your search term here and press Enter.

    « Back Next »
  7. ×

    Share a link to this book page on your preferred social network or via email.

    « Back Next »
  8. ×

    View our suggested citation for this chapter.

    « Back Next »
  9. ×

    Ready to take your reading offline? Click here to buy this book in print or download it as a free PDF, if available.

    « Back Next »
Stay Connected!