Building a National Capability
to Monitor and Assess Medical
Countermeasure Use During a
Public Health Emergency

GOING BEYOND THE LAST MILE

PROCEEDINGS OF A WORKSHOP

Morgan Boname, Theresa Wizemann, and Justin Snair, Rapporteurs

Board on Health Sciences Policy

Health and Medicine Division

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

THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001

This project was supported by a contract between the National Academy of Sciences and the U.S. Department of Health and Human Services’ U.S. Food and Drug Administration (Contract No. 1R13FD005495-01). 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: 978-0-309-46642-4
International Standard Book Number-10: 0-309-46642-3
Digital Object Identifier: https://doi.org/10.17226/24912

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Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2017. Building a national capability to monitor and assess medical countermeasure use during a public health emergency: Going beyond the last mile: Proceedings of a workshop. Washington, DC: The National Academies Press. doi: https://doi.org.10.17226/24912.

PLANNING COMMITTEE ON BUILDING A NATIONAL CAPABILITY TO MONITOR AND ASSESS MEDICAL COUNTERMEASURE USE IN RESPONSE TO PUBLIC HEALTH EMERGENCIES¹

BORIS LUSHNIAK (Chair), Dean and Professor, University of Maryland School of Public Health

PHYLLIS ARTHUR, Managing Director, Infectious Diseases and Diagnostics Policy, Biotechnology Innovation Organization

SUZANNE BAKKEN, Alumni Professor of Nursing; Professor of Biomedical Informatics, Columbia University

JEREMY BROWN, Director, Office of Emergency Care Research, National Institute of General Medical Sciences, National Institutes of Health

J. PERREN COBB, Professor, Clinical Surgery; Director, University of Southern California Critical Care Institute; Director, Keck Surgical Intensive Care Unit, University of Southern California

JEFFREY COUGHLIN, Senior Director, Federal and State Affairs, Healthcare Information Management and Systems Society

AMANDA PEPPERCORN, Senior Medical Director, Infectious Diseases Research and Development, GlaxoSmithKline

RICHARD PLATT, Professor and Chair, Department of Population Medicine, Harvard Medical School

KIMBERLEY RICKETTS, Public Health Advisor, U.S. Centers for Disease Control and Prevention, U.S. Department of Health and Human Services

PAUL SELIGMAN, Executive Director, Amgen

UMAIR SHAH, Executive Director, Harris County Public Health and Environmental Services

YON YU, Associate Director, Regulatory Affairs, National Center for Emerging and Zoonotic Infectious Diseases, U.S. Centers for Disease Control and Prevention, U.S. Department of Health and Human Services

Health and Medicine Division Staff

JUSTIN SNAIR, Senior Program Officer (until September 2017)

SCOTT WOLLEK, Senior Program Officer

___________________

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.

MORGAN BONAME, Associate Program Officer

ALEXA EDMIER, Research Assistant

ANDREW POPE, Director, Board on Health Sciences Policy

Consultants

LAURA RUNNELS, LAR Consulting

THERESA WIZEMANN, Consulting Writer

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:

SUZANNE BAKKEN, Columbia University

JAY C. BUTLER, Alaska Department of Health and Social Services

J. PERREN COBB, University of Southern California

EVA LEE, Georgia Institute of Technology

BORIS LUSHNIAK, University of Maryland School of Public Health

VINIT P. NAIR, Humana

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 LINDA C. DEGUTIS, Henry M. Jackson Foundation. She was responsible for making certain that an independent examination of this proceedings was carried out in accordance with stan-

dards 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.

Contents

ACRONYMS AND ABBREVIATIONS

1 INTRODUCTION AND OVERVIEW

Workshop Background and Objectives

Organization of This Proceedings of a Workshop

Viewpoint from the U.S. Food and Drug Administration on MCM Monitoring and Assessment During a PHE

Overview of Cross-Cutting Topics Highlighted During Presentations and Discussions

2 SETTING THE STAGE: DEFINING TERMINOLOGIES AND SHARING STAKEHOLDER PERSPECTIVES

Defining Terminologies Used Throughout the Workshop

Stakeholder Perspectives

3 DATA NEEDS, DATA SOURCES, AND COLLECTION METHODOLOGIES FOR STAKEHOLDER DECISION MAKING

Defining and Answering Questions to Inform Data Needs for MCM Distribution and Monitoring

Considerations and Approaches to Data Collection

Integrating Data from Different Sources

Existing Data Sources and Datasets

Disseminating Data and Information

Unique Challenges and Opportunities for Data Collection

4 CONSIDERATIONS FOR CONDUCTING RAPID CLINICAL RESEARCH ON MCMs DURING A PHE

Potential Elements of Clinical Research on MCM Use

Data and Information Sharing

Governance

5 INSPIRING COLLECTIVE ACTION: PERSPECTIVES FROM FEDERAL STAKEHOLDERS AND REFLECTIONS FROM INDIVIDUAL WORKSHOP PARTICIPANTS

Federal Stakeholder Perspectives on Moving Forward

Prioritizing Issues for Action: Reflections from Individual Workshop Participants

Closing Remarks

APPENDIXES

A References

B Workshop Agenda

C Biographical Sketches of Workshop Speakers and Moderators

Boxes, Figures, and Table

BOXES

1-1 Workshop Statement of Task

1-2 H1N1 Influenza as a Case Study in MCM Development, Deployment, Monitoring, and Assessment

2-1 Types of Electronic Health Data

2-2 Collaboration Between a State Health Department and the Health Care Community During the 2012 Nationwide Fungal Infection Outbreak

2-3 Understanding Methodologies and Biases in Data Collection: Lessons Learned from a Depression Study

3-1 Examining the Impact of Safety and Efficacy Profiles on MCM Distribution: Lessons from Anthrax and H1N1

3-2 A Routine, All-Hazards Approach to Data Collection During a PHE in New York State

3-3 Modifying a CDC Database to Meet Evolving Data Needs in the 2009 H1N1 Influenza Pandemic

3-4 Using Existing Data Collection Systems for Tracking MCM Distribution at the U.S. Department of Defense

4-1 Rapid Development of Clinical Research Protocols During the 2009 H1N1 Pandemic: Federal Perspective

4-2 Highlighting the Importance of Predefined Protocols in the 2009 H1N1 Pandemic: Industry Perspective

4-3 MCM Data and HIPAA: Human Subjects Research or Public Health Practice?

4-4 Exercising for Administrative Preparedness in PHEs

FIGURES

1-1 Traditional medical product life cycle

1-2 Timeline of FDA response to the 2009 H1N1 influenza pandemic

TABLE

1-1 Differences Between FDA Product Assessment in a PHE Versus Traditional Research and Development

Acronyms and Abbreviations