Ranking
Vaccines

A Prioritization Software Tool

Phase II: Prototype of a Decision-Support System

Committee on Identifying and Prioritizing
New Preventive Vaccines for Development, Phase II

Board on Population Health and Public Health Practice
Board on Global Health

Guruprasad Madhavan, Kinpritma Sangha, Charles Phelps,
Dennis Fryback, Rino Rappuoli, Rose Marie Martinez, and
Lonnie King, Editors

INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS

Washington, D.C.

www.nap.edu



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Committee on Identifying and Prioritizing New Preventive Vaccines for Development, Phase II Board on Population Health and Public Health Practice Board on Global Health Guruprasad Madhavan, Kinpritma Sangha, Charles Phelps, Dennis Fryback, Rino Rappuoli, Rose Marie Martinez, and Lonnie King, Editors

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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. The members of the committee responsible for the report were chosen for their special competences and with regard for appropriate balance. This study was supported by Contract No. HHSP23337024T, TO #45 between the National Academy of Sciences and the National Vaccine Pro- gram Office of the Department of Health and Human Services. Any opin- ions, findings, conclusions, or recommendations expressed in this publica- tion are those of the editors and do not necessarily reflect the view of the organizations or agencies that provided support for this project. International Standard Book Number-13: 978-0-309-26638-3 International Standard Book Number-10: 0-309-26638-6 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; www.nap.edu. SMART Vaccines 1.0 can be downloaded from www.iom.edu/SMART Vaccines or www.nap.edu/SMARTVaccines. For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu. Copyright 2013 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). 2013. Ranking vaccines: A prioritization software tool: Phase II: Prototype of a decision-support system. Washington, DC: The National Academies Press.

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“Knowing is not enough; we must apply. Willing is not enough; we must do.” —Goethe Advising the Nation. Improving Health.

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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 govern- ment 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 out- standing 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. C. D. Mote, Jr., 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 pro- viding 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. C. D. Mote, Jr., are chair and vice chair, respectively, of the National Research Council. www.national-academies.org

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Committee on Identifying and Prioritizing New Preventive Vaccines for Development, Phase II LONNIE KING (Chair), Dean, College of Veterinary Medicine, and Executive Dean, Health Sciences College, Ohio State University JONATHAN CARLSON, Researcher, eScience Research Group, Microsoft Research PAUL CITRON, Retired Vice President, Medtronic, Inc.; Senior Fellow, William J. von Liebig Center for Entrepreneurism and Technology, and Adjunct Professor of Bioengineering, University of California, San Diego RITA COLWELL, Chair Emeritus, Canon U.S. Life Sciences, Inc.; Distinguished Professor, University of Maryland, College Park, and Johns Hopkins University Bloomberg School of Public Health; and Former Director, National Science Foundation KATHRYN EDWARDS, Sarah H. Sell Professor of Pediatrics, Vanderbilt University School of Medicine DENNIS FRYBACK, Professor Emeritus of Population Health Sciences and Industrial Engineering, University of Wisconsin–Madison GLENDA GRAY, Executive Director, Perinatal HIV Research Unit, and Associate Professor of Pediatrics, University of the Witwatersrand, South Africa MICHEL GUILLOT, Associate Professor of Sociology, University of Pennsylvania VICTORIA HALE, Founder and Chief Executive Officer, Medicines 360 JOSEPH JASINSKI, IBM Distinguished Engineer and Global Industry Executive, Smarter Healthcare and Life Sciences, IBM Research TRACY LIEU, Director, Division of Research, Kaiser Permanente Northern California CHARLES PHELPS, University Professor and Provost Emeritus, University of Rochester RINO RAPPUOLI, Global Head, Vaccines Research, Novartis Vaccines, Italy ARTHUR REINGOLD, Edward Penhoet Distinguished Professor of Global Health and Infectious Diseases, University of California, Berkeley EDWARD SHORTLIFFE, Professor, Arizona State University; Adjunct Professor of Biomedical Informatics, Columbia University; and Scholar in Residence, New York Academy of Medicine ROBERT STEINGLASS, Immunization Team Leader, Maternal and Child Health Integrated Program, John Snow, Inc. v

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OYEWALE TOMORI, President, Nigerian Academy of Sciences; Professor and Vice Chancellor Emeritus, Redeemer’s University, Nigeria DETLOF VON WINTERFELDT, Professor of Industrial and Systems Engineering, and Public Policy and Management, University of Southern California Staff GURUPRASAD MADHAVAN, Study Director KINPRITMA SANGHA, Research Associate and Data Manager ANGELA MARTIN, Senior Program Assistant HOPE HARE, Administrative Assistant DORIS ROMERO, Financial Associate ROSE MARIE MARTINEZ, Senior Director, Board on Population Health and Public Health Practice PATRICK KELLEY, Senior Director, Board on Global Health Consultants JON ANDRUS, Deputy Director, Pan American Health Organization MARK FEINBERG, Vice President, Chief Public Health and Science Officer, Merck Vaccines, Merck & Company, Inc. DAVID HEYMANN, Chairman, Health Protection Agency; Head and Senior Fellow, Centre on Global Health Security, Chatham House; Professor, London School of Hygiene and Tropical Medicine, United Kingdom SCOTT LEVIN, Associate Professor of Emergency Medicine, Johns Hopkins University TYLER MARTIN, President and Chief Medical Officer, Dynavax Technologies SIMON MERCER, Director, Health and Wellbeing, Microsoft Research Connections  PAUL RADSPINNER, President and Chief Executive Officer, FluGen, Inc. JOHN SPIKA, Director General, Centre for Immunization and Respiratory Infectious Diseases, Public Health Agency of Canada vi

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Reviewers This report has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise, in accordance with proce- dures approved by the National Research Council’s Report Review Com- mittee. 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 deliberative process. We wish to thank the following individuals for their review of this report: Gerald Brown, U.S. Naval Postgraduate School Maryellen Giger, The University of Chicago Philip Hosbach, Sanofi Pasteur Emmett Keeler, RAND Corporation and University of California,  Los Angeles Osman Mansoor, United Nations Children’s Fund (UNICEF) Neela Patel, Abbott Laboratories Gregory Poland, Mayo Clinic Jaime Sepulveda, University of California, San Francisco,  School of Medicine Nirav Shah, New York State Department of Health David Shoultz, Bill & Melinda Gates Foundation Guy Steele, Oracle Labs Jeffrey Sturchio, Rabin-Martin Although the reviewers listed above have provided many construc- tive comments and suggestions, they were not asked to endorse the con- clusions or recommendations nor did they see the final draft of the report before its release. The review of this report was overseen by Alfred Berg, vii

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viii Reviewers University of Washington School of Medicine, and Stephen Fienberg, Carnegie Mellon University. Appointed by the Institute of Medicine and the National Research Council, they were responsible for making certain that an independent examination of this report was carried out in accor- dance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this report rests entirely with the authoring committee and the institution.

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Foreword Making choices among investments—whether in research, business, medi- cine, or daily life—typically involves uncertainties, preferences, and trade- offs. Decision science and modeling provide means to take account of these numerous elements, their interdependencies and interactions, and allow a decision maker to probe and assess each element without losing sight of the whole. This product of the Institute of Medicine, Ranking Vaccines: A Prioritization Software Tool, utilizes decision science and modeling to help inform choices among candidates for new vaccine development. This computer-based guide, called SMART Vaccines—Strategic Multi-Attribute Ranking Tool for Vaccines—builds on the blueprint presented in 2012 as Ranking Vaccines: A Prioritization Framework. As a software system, SMART Vaccines provides a customizable tool—with various built-in and user-defined attributes—for a vaccine enterprise that currently has no shared standards to support decision mak- ing. As a facilitator of informed discussion and decision making, SMART Vaccines has the potential to engage different users independently or coop- eratively when they wish to reduce barriers for new vaccine development and delivery. Unlike many previous recommended priorities, SMART Vac- cines does not impose a predetermined value system on decision makers. Instead, users are able to weigh and rank preferences that are relevant to the specific contexts in which they are making decisions. The usefulness of SMART Vaccines hinges on the availability of reli- able data for evaluation. Indeed, by carefully analyzing the variables that go into decisions about new vaccine priorities, the tool exposes those data ele- ments that are especially pertinent to inform choices. Additional work in establishing a data infrastructure—including new partnerships and mecha- nisms for generating and updating data—will be essential if this tool is to achieve its potential. Over time, users and other experts will be able to con- ix

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x Foreword tinue to refine this model, as its flexibility and capacity for improvement are key design features. SMART Vaccines has the potential to contribute to strategic plan- ning in a vaccine enterprise that confronts difficult choices and many con- straints. I commend the experts on the committee and the staff who led this pioneering effort at the Institute of Medicine and hope that it will prove useful to policy makers and leaders in the field. Harvey V. Fineberg, M.D., Ph.D. President, Institute of Medicine

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Preface We live in an era of rapid change and frequent disruptions caused by glo- balization, changing markets, demographics, economies, and innovations in new technology development. These considerations make efforts to prioritize the development and delivery of new vaccines and other health technologies extremely challenging and progressively more complex. Although the science and engineering underlying vaccine devel- opment is progressing in promising new directions, significant barri- ers remain. Among the key issues that must be addressed are the supply, delivery, safety, and cost of vaccines. Furthermore, despite the significant progress being made toward tackling the threat of infectious diseases, more work remains to be done on improving global public health. In light of these challenges, the development of new vaccines will be even more important in the future, and deciding which vaccines to prioritize will be especially critical. Currently, however, there are no standardized mecha- nisms in place to support decision making on vaccine prioritization and no systems that effectively involve stakeholders and users in this process. This study was organized as part of the 2010 National Vaccine Plan and has been conducted in two phases, with separate but closely related sub-studies. In response to the charge provided by the National Vaccine Program Office of the Department of Health and Human Services, an 18-member committee for Phase II was created that included some mem- bers from the Phase I committee, who ensured continuity from the first phase, and also new members who greatly expanded the group’s expertise. The Phase II committee extended the proof-of-concept presented in the Phase I report Ranking Vaccines: A Prioritization Framework, which was released in 2012. The model developed in Phase I, which was based on multi-attribute utility theory, served as the foundation for the creation of the blueprint for a software tool called SMART Vaccines Beta. The Phase II xi

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xii Preface committee refined this beta version and created its next iteration, SMART Vaccines 1.0. This report, Ranking Vaccines: A Prioritization Software Tool, dis- cusses the methods underlying the development, validation, and evalu- ation of SMART Vaccines 1.0. It also discusses how SMART Vaccines should—and, just as importantly, should not—be used. The committee has also offered ideas for future enhancements for SMART Vaccines as well as for ideas for expanded uses and considerations and possibilities for the future. SMART Vaccines will need to secure ongoing feedback and input from potential users so the software can ultimately perform up to its maxi- mum capacity. The committee invites suggestions for further improve- ments to enhance this software tool. Our committee was highly diverse with a broad range of experiences and expertise. With a combination of ideas and intellect, the committee greatly enhanced the SMART Vaccines tool. We envision this tool serving as a living guide, one that will gain greater utility over time through con- tinuous learning and improvements. The creation of SMART Vaccines is unique to the Institute of Medi- cine (IOM), and it may also be ushering in a new era for the National Acad- emies. There are multiple users and stakeholders who could benefit from SMART Vaccines 1.0 and they include decision makers in all realms of vac- cine development and delivery in the public, private, and nongovernmental enterprises. Their involvement and input is necessary to further enhance the utility and functionality of SMART Vaccines. More importantly, the committee believes that the development of a data warehouse to support SMART Vaccines will be crucial for the successful application of this tool. On behalf of the committee I would like to recognize and thank a number of individuals whose expertise, time, and counsel helped to develop SMART Vaccines and to produce this report. We were indeed for- tunate to have a very talented, diligent, and especially hard-working IOM project staff. The committee gratefully acknowledges our study director Guru Madhavan, whose knowledge, organizational skills, and commitment to our work and to this topic were truly outstanding. We also appreciate the exceptional contributions of our research associate Kinpritma Sangha, and we recognize Angela Martin for her administrative support. We are indebted to Rose Marie Martinez, senior director of the Board on Population Health and Public Health Practice, whose experience, guidance, and intellect proved invaluable. We wish to thank Patrick Kelley, senior director of the Board on Global Health; Clyde Behney, interim exec- utive officer of the IOM; and Marc Gold, the associate general counsel for the National Academy of Sciences for their continued advice to the com-

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Preface xiii mittee. For their terrific editorial and other assistance, the committee also thanks Anton Bandy, Katharine Bothner, Patrick Burke, Laura DeStefano, Megan Ellinger, Chelsea Frakes, Dev Mani, Abbey Meltzer, Robert Pool, and Jessica Rasmussen. We were especially well served by Scott Levin of Johns Hopkins Uni- versity whose help with modeling was critical to the committee’s vision for SMART Vaccines. The committee’s software development process was also greatly informed by the feedback of prototype evaluators Jon Andrus, Mark Feinberg, David Heymann, Tyler Martin, Simon Mercer, Paul Radspinner, and John Spika. We also thank the commentators at the committee’s public forum and elsewhere as well as the expert reviewers, whose rigorous and thoughtful input helped to improve the committee’s products substantially. A final note of thanks goes to the National Vaccine Program Office of the Department of Health and Human Services for their enthusiastic com- mitment to this project, for their encouragement, and for their sponsorship. Lonnie King, Chair

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Contents DISCLAIMER......................................................................................................xvii SUMMARY.................................................................................................................1 1 INTRODUCTION: NEW VACCINES AND SMART VACCINES....................................................................................................13 Study Context and Scope, 17 Study Process and Feedback from Stakeholders, 19 2 REFINEMENTS TO THE SMART VACCINES MODEL............. 25 A Brief Review of the Modeling Framework, 25 Setting Ranges for Attributes: A Heuristic Process, 28 Weights and Ranks: Attention to the Ranges of the Attributes, 28 Comparison of SMART Scores, 31 3 DATA SYNTHESIS, SOFTWARE REDESIGN, AND EVALUATION............................................................................................. 33 Selection of Vaccine Candidates, 33 Data Sourcing and Quality, 35 Development of a Test Model to Inform Software Redesign, 36 Interface Redesign for SMART Vaccines 1.0, 38 Representative Use Case Scenarios, 47 Users’ Evaluation of the Prototype, 51 4 OBSERVATIONS AND LOOKING FORWARD................................61 Guiding Principle and Strategy, 61 A Vision for the Future, 62 Future Steps, 63 Enhanced Applications of SMART Vaccines, 66 Next Phase, 69 xv

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xvi Contents REFERENCES.........................................................................................................71 APPENDIXES A Computational Modeling for SMART Vaccines.................................... 73 B Candidate Disease Profiles......................................................................... 85 C Data Sources and Methodology for SMART Vaccines........................99 D Verification and Analyses of the SMART Vaccines Computational Model................................................................................ 111 E Stakeholder Speakers................................................................................ 125 F Biographical Information......................................................................... 127

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Disclaimer This report describes SMART Vaccines—Strategic Multi- Attribute Ranking Tool for Vaccines—an early-stage prototype software application based on multi-attribute utility theory. This report does not provide a ranking of vaccine priorities. It describes the committee’s modeling strategy and assumptions in order to demonstrate a proof of concept. The SMART Vaccines software application is intended to serve only as a decision-support tool. Specific decisions about pri- orities should not be made solely on the basis of SMART Vaccines. The examples that appear in this report are limited to comparing hypothetical vaccines only. The National Academy of Sciences and the Institute of Medi- cine do not warrant the completeness of the model, the accuracy of the software in development, or the reliability of any data pre- sented in this report. September 2013 xvii

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