Closing
Evidence Gaps in
Clinical Prevention

Alexis Wojtowicz, Kathleen Stratton, and Tracy A. Lieu, Editors

Committee on Addressing Evidence Gaps in
Clinical Prevention Recommendations

Board on Population Health and Public Health Practice

Health and Medicine Division

A Consensus Study Report of

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International Standard Book Number-13: 978-0-309-26957-5
International Standard Book Number-10: 0-309-26957-1
Digital Object Identifier: https://doi.org/10.17226/26351
Library of Congress Catalog Number: 2021951344

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Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2022. Closing evidence gaps in clinical prevention. Washington, DC: The National Academies Press. https://doi.org/10.17226/26351.

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COMMITTEE ON ADDRESSING EVIDENCE GAPS IN CLINICAL PREVENTION RECOMMENDATIONS

TRACY A. LIEU (Chair), Director of the Division of Research, Kaiser Permanente Northern California

CATHY J. BRADLEY, Professor and Associate Dean for Research, Colorado School of Public Health; Deputy Director, University of Colorado Cancer Center

THEODORE G. GANIATS, Professor Emeritus, Department of Family Medicine and Public Health, University of California, San Diego, School of Medicine

KENG-YEN HUANG, Associate Professor, Departments of Population Health and Child & Adolescent Psychiatry, New York University Grossman School of Medicine

PREETI GOKAL KOCHAR, Technical Information Specialist, National Library of Medicine, National Institutes of Health

ANN E. KURTH, Dean and Linda Koch Lorimer Professor of Nursing, Yale School of Nursing; Professor of Epidemiology of Microbial Diseases, Yale School of Public Health

TIANJING LI, Associate Professor, Department of Ophthalmology, University of Colorado School of Medicine

K. M. VENKAT NARAYAN, Director, Emory Global Diabetes Research Center; Ruth and O.C. Hubert Chair of Global Health and Professor of Epidemiology, Rollins School of Public Health; Professor of Medicine, Emory University School of Medicine

WANDA K. NICHOLSON, Professor, Department of General Obstetrics & Gynecology and Director, Diabetes and Obesity Core Center for Women’s Health Research, University of North Carolina School of Medicine

ELIZABETH O. OFILI, Professor of Medicine, Morehouse School of Medicine; Chief Medical Officer, Morehouse Choice Accountable Care Organization

NICOLAAS P. PRONK, President and Chief Science Officer, HealthPartners Institute

ROBERT B. WALLACE, Irene Ensminger Stecher Professor Emeritus of Epidemiology and Internal Medicine, University of Iowa College of Public Health

Study Staff

KATHLEEN R. STRATTON, Study Director

ALEXIS WOJTOWICZ, Associate Program Officer

AYSHIA COLETRANE, Senior Program Assistant

REBECCA MORGAN, Senior Librarian

MISRAK DABI, Finance Business Partner

ROSE MARIE MARTINEZ, Senior Board Director

Science Writer

ROBERT POOL

Reviewers

This Consensus Study Report 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 report as sound as possible and to ensure that it meets the institutional standards for quality, 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 thank the following individuals for their review of this report:

Although the reviewers listed above provided many constructive comments and suggestions, they were not asked to endorse the content of the report nor did they see the final draft before its release. The review of this report was overseen by BOBBIE BERKOWITZ, Columbia University, and JACK EBELER, Health Policy Alternatives, Inc. They were responsible for making certain that an independent examination of this report 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 authoring committee and the National Academies.

Preface

Preventing suffering and loss of life through early identification and treatment of individuals at risk is a central purpose of clinical care. For decades, primary care clinicians and others have relied on recommendations of the U.S. Preventive Services Task Force (USPSTF) in these efforts. The USPSTF’s careful reliance on evidence from rigorous studies to inform its statements has often highlighted topics and questions for which the evidence is insufficient to make a definitive recommendation. In an ideal world, when an insufficient evidence statement is rendered, research sponsors and researchers should quickly initiate high-quality studies needed to fill the most important evidence gaps. However, in some cases the lack of a widely agreed-on framework for describing and categorizing such evidence gaps has hampered efficient communication and progress.

The present study focused on developing a taxonomy to characterize evidence gaps in clinical prevention, and identifying research strategies and funding approaches to enable sponsors and researchers to more effectively fill these gaps. This work was sponsored by leaders in the National Institutes of Health (NIH) and the Agency for Healthcare Research and Quality (AHRQ), which supports the USPSTF. We are grateful to David Murray and Carrie Klabunde of the Office of Disease Prevention, NIH, and to Arlene Bierman and Amanda Borsky of the Center for Evidence and Practice Improvement, AHRQ, for their guidance and support. We also wish to acknowledge the important contributions of Susan Curry, who was instrumental in launching this effort.

Our study committee approached this task with a sense of significance and optimism, and demonstrated intellectual generosity and graciousness throughout this multidisciplinary process. The committee and the Health and Medicine Division’s (HMD’s) staff of the National Academies developed this report and the taxonomy in an iterative manner, based on reviews of existing USPSTF recommendation statements and public meetings with experts from NIH, AHRQ, and other key organizations that sponsor research or support guideline committees, USPSTF members, and the research community. During this process we were particularly attuned to the fact that both the USPSTF and research sponsors must balance competing priorities among the many evidence gaps that could be addressed. As a former member of the USPSTF, the past chair of an NIH grant review committee, and a leader of a research department in a health care delivery system, I greatly appreciated the ability of our committee members and expert key informants to think pragmatically about how to facilitate communication and collaboration by the multiple stakeholders needed to successfully address evidence gaps.

We appreciate the education offered us by the speakers at our public meetings, including the following:

• David Atkins, M.D., M.P.H., Director, Health Services Research & Development, U.S. Department of Veterans Affairs
• Arlene Bierman, M.D., M.S., Director, Center for Evidence and Practice Improvement, AHRQ
• Amanda Borsky, Dr.P.H., M.P.P., Program Manager, USPSTF, AHRQ
• Sarabeth Broder-Fingert, M.D., M.P.H., Associate Professor of Pediatrics, Boston University School of Medicine; Co-Director, Innovation in Autism Disparities, Inclusion, Outreach, and Transition Services Lab, Boston University
• Ned Calonge, M.D., M.P.H., President and Chief Executive Officer, Colorado Trust; Associate Professor of Family Medicine, Colorado School of Medicine, and Associate Professor of Epidemiology, Colorado School of Public Health, University of Colorado Denver
• Doug Corley, M.D., Ph.D., Lead Investigator, National Cancer Institute (NCI) Population-based Research to Optimize the Screening Process Consortium; Research Scientist, Kaiser Permanente Northern California Division of Research
• Susan Curry, Ph.D., Senior Advisor for Preventive Services, NIH; Dean Emerita and Distinguished Professor of Health Management and Policy, University of Iowa

• Paul Doria-Rose, Ph.D., D.V.M., Chief, Healthcare Assessment Research Branch, Health Delivery Research Program, Division of Cancer Control & Population Sciences, NCI
• Chyke Doubeni, M.D., M.P.H., Member, USPSTF; Director, Mayo Clinic Center for Health Equity and Community Engagement Research
• Carrie Klabunde, Ph.D., Senior Advisor for Disease Prevention, Office of Disease Prevention, NIH
• Alex Krist, M.D., M.P.H., Immediate Past Chair, USPSTF; Professor of Family Medicine and Population Health, Virginia Commonwealth University
• Amy Lansky, Ph.D., M.P.H., Director, Centers for Disease Control and Prevention Community Guide
• Michael Lauer, M.D., Deputy Director, Extramural Research, NIH
• Alan Leshner, Ph.D., Chief Executive Officer Emeritus, American Association for the Advancement of Science; Executive Publisher, Science; Former Director, National Institute on Drug Abuse, NIH; Former Deputy Director and Acting Director, National Institute of Mental Health (NIMH), NIH
• Carol Mangione, M.D., M.S.P.H., FACP, Vice-Chair, USPSTF; Chief, Division of General Medicine & Health Services Research, University of California, Los Angeles (UCLA); Barbara A. Levey & Gerald S. Levey Professor of Medicine and Public Health, UCLA
• David Murray, Ph.D., Associate Director for Prevention and Director, Office of Disease Prevention, NIH
• Karen Robinson, Ph.D., M.Sc., Director, Evidence-based Practice Center, Johns Hopkins University
• Michael Silverstein, M.D., M.P.H., Member, USPSTF; Professor of Pediatrics, Director, Division of General Academic Pediatrics, and Vice Chair, Research for the Department of Pediatrics, Boston University School of Medicine
• Harold (Hal) Sox, M.D., Director, Peer Review, Patient-Centered Outcomes Research Institute
• Ann Wagner, Ph.D., National Autism Coordinator, Office of NIMH Director, NIMH, NIH

We are also grateful to Caitlin Phelps (American College of Obstetricians and Gynecologists), Joe Selby, Steve Teutsch, and Gregory Traversy (Public Health Agency of Canada) for their helpful observations and suggestions.

The committee’s work would not have been possible without the insightful leadership of our study director Kathleen Stratton and the thoughtful and analytic contributions of Associate Program Officer Alexis

Wojtowicz. We benefited from the excellent organizational skills of Ayshia Coletrane and the lucid writing assistance of Robert Pool as we crafted this report. We extend thanks to Rose Marie Martinez for her farsighted guidance and to the National Academies’ Research Center staff and many other staff members within HMD and the National Academies for their support.

The committee hopes this study will facilitate a common understanding of the key concepts related to evidence gaps in clinical prevention, support thoughtful discussion of priorities among multiple stakeholders, and lead to research that enhances clinical prevention in the years ahead.

Tracy A. Lieu, Chair
Committee on Addressing Evidence Gaps in Clinical Prevention Recommendations

Contents

ACRONYMS AND ABBREVIATIONS

SUMMARY

1 INTRODUCTION

Statement of Task

Committee Process

Organization of the Report

References

2 CLINICAL PRACTICE GUIDELINES AND THE U.S. PREVENTIVE SERVICES TASK FORCE

What Are Clinical Practice Guidelines?

U.S. Preventive Services Task Force

How Other Clinical Practice Guideline Developers Address Evidence Gaps

References

3 CLINICAL PREVENTION RESEARCH ENTERPRISE

National Institutes of Health

Other Clinical Prevention Research Funders

Researchers

References

4 TAXONOMY AND WORKFLOW

Committee’s Approach

Workflow

Taxonomy for Classifying Evidence Gaps

Taxonomy for Developing the Research Agenda

Examples of Applying the Taxonomy to I Statements

References

5 RECOMMENDATIONS

Using the Taxonomy

Fostering Clinical Prevention Research

Advancing the Work of the U.S. Preventive Services Task Force

References

APPENDIXES

A Topics with I Statements from the U.S. Preventive Services Task Force

B Research Methods

C Public Meeting Agendas

D Committee Member and Staff Biographies

E U.S. Preventive Services Task Force’s Reports to Congress

F U.S. Preventive Services Task Force Research Needs Statements from I Statements Issued Between 2017 and 2021

Boxes, Figures, and Tables

BOXES

1-1 Statement of Task

3-1 Setting Research Priorities

3-2 National Institutes of Health Strategic Plan for Fiscal Years 2021–2025

4-1 Clinical Prevention Evidence Gaps Taxonomy Terms

4-2 Evidence Gaps Identified by the U.S. Preventive Services Task Force for the Cognitive Impairment Screening Recommendation

FIGURES

S-1 A generic version of the analytic framework used by the U.S. Preventive Services Task Force

S-2 Workflow for using the evidence gaps taxonomy

S-3 The committee’s vision for the report and taxonomy’s role in improving clinical research by addressing evidence gaps

2-1 U.S. Preventive Services Task Force recommendations development process: a graphic overview

2-2 The relationship between the U.S. Preventive Services Task Force and the Community Preventive Services Task Force

3-1 2020 I statement survey summary

4-1 Generic analytic framework for screenings

4-2 Generic analytic framework for behavioral interventions

4-3 Analytic framework for screenings and interventions to prevent dental caries in children younger than age 5

4-4 Analytic framework for preventing HIV infection with PrEP

4-5 The workflow for identifying evidence gaps in clinical prevention with the taxonomy

4-6 The workflow with foundational evidence gaps facet

4-7 The workflow with analytic framework evidence gaps facet

4-8 The workflow with dissemination and implementation evidence gaps facet

4-9 The workflow with prioritization criteria facet

4-10 The workflow with study specifications facet

5-1 The committee vision for improving clinical prevention research

TABLES

2-1 Matrix of Assessing Certainty and Magnitude of Net Benefit Used by the U.S. Preventive Services Task Force

2-2 Types of Evidence Gaps That May Lead to an I Statement for a Topic

3-1 Frequently Used Funding Mechanisms at the National Institutes of Health

3-2 Comparison of Grants and Contracts

3-3 National Institutes of Health Activity Related to I Statements

4-1 Foundational Evidence Gaps in the Cognitive Impairment Recommendation

4-2 Analytic Framework Gaps in the Cognitive Impairment Recommendation

4-3 Dissemination and Implementation Evidence Gaps in the Cognitive Impairment Recommendation

4-4 Foundational Evidence Gaps in the Atrial Fibrillation Recommendation

4-5 Analytic Framework Evidence Gaps in the Atrial Fibrillation Recommendation

4-6 Dissemination and Implementation Evidence Gaps in the Atrial Fibrillation Recommendation

Acronyms and Abbreviations