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
THE NATIONAL ACADEMIES PRESS
Washington, DC
www.nap.edu
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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
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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.
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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.
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
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
National Academies and that all review comments were carefully considered. Responsibility for the final content rests entirely with the rapporteurs and the National Academies.
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.
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Contents
THE TRANSLATIONAL RESEARCH LANDSCAPE
Translational Science: Increasing Efficiency, Decreasing Failure
NCATS Rare Disease Research Programs
FEDERAL BIOMEDICAL RESEARCH FUNDING AND NEW DRUG DEVELOPMENT
Perspectives on the Federal Role in Advancing Drug Development Innovation
Accounting for Public Funding in Drug Pricing
Direct and Indirect Effects of Public-Sector Funding on Drug Development
Gauging the Returns on Federally Funded Basic Research
Quantifying the Impact of Targeted, Disease-Specific NIH Extramural Funding
IMPACT OF PATENT AND TECHNOLOGY TRANSFER POLICIES ON NIH-FUNDED INNOVATION
Patent-Eligible Subject Matter in Biomedical Research and Development
Technology Transfer at U.S. Academic Institutions
Stakeholder Feedback on the Technology Transfer Process
Interpreting the Bayh-Dole Act
Standardizing the Licensing Process
Pricing and Reimbursement of Pharmaceuticals in the United States
Consumer Perspective on Prescription Drug Price Trends
Providing Access and Value for Low-Income Persons with Complex Medical Needs
Financing Antibacterial Drug Discovery and Development
STRATEGIES AND POLICIES TO FACILITATE TRANSLATION
Reducing Risks and Costs and Improving Returns in Drug Development
Meeting U.S. Department of Defense Medical Support Needs
C-Path: Collaborating to Accelerate Medical Product Development
Repurposing Compounds for New Therapeutic Uses
Infrastructure for Drug Development Innovation
Partnering for Translational Research Success
Additional Strategies to Facilitate Translation
POTENTIAL POLICIES TO ENSURE AFFORDABLE ACCESS
Pricing of Taxpayer-Funded Drugs
Improving System Performance and Supporting Emerging Innovative Companies
Boxes and Figures
BOXES
1 Observations and Suggestions Made by Individual Workshop Participants
3 Case Examples of Patenting Federally Funded Drugs
5 Reimbursement Prototypes Outside the United States
FIGURES
1 Drug discovery, development, and deployment map (4DM) for small molecules and biologics
2 Overall trend in R&D efficiency (*inflation-adjusted)
3 Costs increase as translation proceeds (using small molecule drugs as an example)
4 U.S. post-launch price growth for branded versus generic drugs, 2008–2016
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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 |
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 |