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

ACRONYMS AND ABBREVIATIONS

PROCEEDINGS OF A WORKSHOP

OVERVIEW OF THE WORKSHOP

THE TRANSLATIONAL RESEARCH LANDSCAPE

Bridging the Valley of Death

Eroom’s Law

Translational Science: Increasing Efficiency, Decreasing Failure

NCATS Rare Disease Research Programs

Models for Drug Discovery

Moving Forward

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

Improving Data Resources

Reforming the Patent System

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

March-In Rights

Stakeholder Feedback on the Technology Transfer Process

Interpreting the Bayh-Dole Act

Standardizing the Licensing Process

THE ECONOMICS OF DRUG PRICING

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

REFERENCES

APPENDIX A: STATEMENT OF TASK

APPENDIX B: WORKSHOP AGENDA

Boxes and Figures

BOXES

1 Observations and Suggestions Made by Individual Workshop Participants

2 Definitions

3 Case Examples of Patenting Federally Funded Drugs

4 Some Findings from the NIST Report Return on Investment Initiative for Unleashing American Innovation

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

5 The antibiotic tripod

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Acronyms and Abbreviations