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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
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The Role of NIH
in Drug Development Innovation
and Its Impact on Patient Access

PROCEEDINGS OF A WORKSHOP

Francis K. Amankwah, Alexandra Andrada, Sharyl J. Nass, and
Theresa Wizemann, Rapporteurs

Board on Health Care Services

Board on Health Sciences Policy

Health and Medicine Division

images

THE NATIONAL ACADEMIES PRESS
Washington, DC
www.nap.edu

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
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THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001

This activity was supported by Arnold Ventures (formerly the Laura and John Arnold Foundation). Any opinions, findings, conclusions, or recommendations expressed in this publication do not necessarily reflect the views of any organization or agency that provided support for the project.

International Standard Book Number-13: 978-0-309-49848-7
International Standard Book Number-10: 0-309-49848-1
Digital Object Identifier: https://doi.org/10.17226/25591

Additional copies of this publication 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.

Copyright 2020 by the National Academy of Sciences. All rights reserved.

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Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2020. The role of NIH in drug development innovation and its impact on patient access: Proceedings of a workshop. Washington, DC: The National Academies Press. https://doi.org/10.17226/25591.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×

Image

The National Academy of Sciences was established in 1863 by an Act of Congress, signed by President Lincoln, as a private, nongovernmental institution to advise the nation on issues related to science and technology. Members are elected by their peers for outstanding contributions to research. Dr. Marcia McNutt is president.

The National Academy of Engineering was established in 1964 under the charter of the National Academy of Sciences to bring the practices of engineering to advising the nation. Members are elected by their peers for extraordinary contributions to engineering. Dr. John L. Anderson is president.

The National Academy of Medicine (formerly the Institute of Medicine) was established in 1970 under the charter of the National Academy of Sciences to advise the nation on medical and health issues. Members are elected by their peers for distinguished contributions to medicine and health. Dr. Victor J. Dzau is president.

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Learn more about the National Academies of Sciences, Engineering, and Medicine at www.nationalacademies.org.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×

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Consensus Study Reports published by the National Academies of Sciences, Engineering, and Medicine document the evidence-based consensus on the study’s statement of task by an authoring committee of experts. Reports typically include findings, conclusions, and recommendations based on information gathered by the committee and the committee’s deliberations. Each report has been subjected to a rigorous and independent peer-review process and it represents the position of the National Academies on the statement of task.

Proceedings published by the National Academies of Sciences, Engineering, and Medicine chronicle the presentations and discussions at a workshop, symposium, or other event convened by the National Academies. The statements and opinions contained in proceedings are those of the participants and are not endorsed by other participants, the planning committee, or the National Academies.

For information about other products and activities of the National Academies, please visit www.nationalacademies.org/about/whatwedo.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×

WORKSHOP PLANNING COMMITTEE1

JEFF BINGAMAN (Chair), Former U.S. Senator, New Mexico

AMITABH CHANDRA, Ethel Zimmerman Wiener Professor of Public Policy, Harvard Kennedy School; Henry and Allison McCance Professor of Business Administration, Harvard Business School

PATRICIA DANZON, Celia Moh Professor Emerita, Wharton School, University of Pennsylvania

STEVEN GALSON, Senior Vice President, Global Regulatory Affairs and Safety, Amgen

STEPHEN MERRILL, Senior Research Fellow and Former Executive Director, Center for Innovation Policy at Duke Law

JENNIFER MOORE, Executive Director, Institute for Medicaid Innovation; Research Professor, University of Michigan Medical School

JOHN NIEDERHUBER, Former Director, National Cancer Institute; Executive Vice President, Inova Health System; Chief Executive Officer, Inova Translational Medicine Institute

AMEET SARPATWARI, Instructor in Medicine, Harvard Medical School; Assistant Director, Program on Regulation, Therapeutics, and Law, Brigham and Women’s Hospital

NEERAJ SOOD, Vice Dean for Research, Sol Price School of Public Policy, and Director of Research, Leonard D. Schaeffer Center for Health Policy & Economics, University of Southern California

REED TUCKSON, Managing Director, Tuckson Health Connections, LLC; Former Executive Vice President and Chief of Medical Affairs, UnitedHealth Group

GABE TZEGHAI, Chief Executive Officer, Summit Innovation Labs, LLC

___________________

1 The National Academies of Sciences, Engineering, and Medicine’s planning committees are solely responsible for organizing the workshop, identifying topics, and choosing speakers. The responsibility for the published Proceedings of a Workshop rests with the workshop rapporteurs and the institution.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×

Project Staff

Board on Health Care Services

FRANCIS K. AMANKWAH, Program Officer

ALEXANDRA ANDRADA, Associate Program Officer (from September 2019)

CAROL SANDOVAL, Senior Program Assistant

ANNALEE GONZALES, Administrative Assistant

MICAH WINOGRAD, Financial Officer

SHARYL J. NASS, Senior Board Director

Board on Health Sciences Policy

EESHAN KHANDEKAR, Associate Program Officer

CAROLYN SHORE, Director, Forum on Drug Discovery, Development, and Translation

ANDREW M. POPE, Senior Board Director

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×

Reviewers

This Proceedings of a Workshop was reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise. The purpose of this independent review is to provide candid and critical comments that will assist the National Academies of Sciences, Engineering, and Medicine in making each published proceedings as sound as possible and to ensure that it meets the institutional standards for quality, objectivity, evidence, and responsiveness to the charge. The review comments and draft manuscript remain confidential to protect the integrity of the process.

We thank the following individuals for their review of this proceedings:

Although the reviewers listed above provided many constructive comments and suggestions, they were not asked to endorse the content of the proceedings, nor did they see the final draft before its release. The review of this proceedings was overseen by BRUCE CALONGE, The Colorado Trust. He was responsible for making certain that an independent examination of this proceedings was carried out in accordance with standards of the

Page viii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×

National Academies and that all review comments were carefully considered. Responsibility for the final content rests entirely with the rapporteurs and the National Academies.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×

Acknowledgments

We are grateful to the chair and members of the planning committee for the Workshop on the Role of NIH in Drug Development Innovation and Its Impact on Patient Access, who collaborated to ensure a workshop with informative presentations and rich discussions. We also thank the speakers and moderators, who generously shared their expertise and time to make the workshop a success. We are also grateful for the support of Arnold Ventures (formerly the Laura and John Arnold Foundation), without which we could not have undertaken this project.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×

Acronyms and Abbreviations

ASP average sales price
BARDA Biomedical Advanced Research and Development Authority
CAP cross-agency priority
CAR-T chimeric antigen receptor T-cell
CARB-X Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator
cDNA complementary DNA
CMS Centers for Medicare & Medicaid Services
CRADA cooperative research and development agreement
CTSA Clinical and Translational Science Awards
DoD U.S. Department of Defense
EHR electronic health record
ERP external reference pricing
FDA U.S. Food and Drug Administration
gDNA genomic DNA
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
HCV hepatitis C virus
HHS U.S. Department of Health and Human Services
I-MAK Initiative for Medicines, Access, & Knowledge, Inc.
iPSC induced pluripotent stem cell
IRB institutional review board
L2M Lab-to-Market
MeSH Medline Subject Headings
NCATS National Center for Advancing Translational Sciences
NIAID National Institute of Allergy and Infectious Diseases
NIH National Institutes of Health
NIST National Institute of Standards and Technology (U.S. Department of Commerce)
PBM pharmacy benefit manager
PDUFA Prescription Drug User Fee Act
PI principal investigator
PrEP pre-exposure prophylaxis
R&D research and development
ROI return on investment
SBIR Small Business Innovation Research program
SNS Strategic National Stockpile
STTR Small Business Technology Transfer program
UAEM Universities Allied for Essential Medicines
USPTO U.S. Patent and Trademark Office
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Page xiii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2020. The Role of NIH in Drug Development Innovation and Its Impact on Patient Access: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25591.
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To explore the role of the National Institutes of Health (NIH) in innovative drug development and its impact on patient access, the Board on Health Care Services and the Board on Health Sciences Policy of the National Academies jointly hosted a public workshop on July 24–25, 2019, in Washington, DC. Workshop speakers and participants discussed the ways in which federal investments in biomedical research are translated into innovative therapies and considered approaches to ensure that the public has affordable access to the resulting new drugs. This publication summarizes the presentations and discussions from the workshop.

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