Principles and Practice of Emergency Research Response

ISBN-13: 9783031484070 / Angielski

Elizabeth S. Higgs; Robert A. Sorenson; Mosoka P. Fallah

Spis treści

Foreword: Bill Gates

Editors’ Preface

Tips for Learners

Abbreviations and Acronyms

Section I. Pandemic Preparedness and Research Response: A Necessary New Field

Section Editor Elizabeth S. Higgs

1. Introduction 

Anthony S. Fauci, Tedros Adhanom Ghebreyesus 

2 .Clinical Research on Infectious Diseases: An Overview

Gerald T. Keusch, Keith McAdam

3. Guiding Principles for Emergency Research Response 

Elizabeth S. Higgs

Section II.  Norms for Emergency Research Response

Section Editor Robert A. Sorenson

4. Ethics of Pandemic Research

Maxwell J. Smith

4.1. In Practice: Vaccine Efficacy and Safety Testing – an Ethical Case for Individual Randomization

Nir Eyal, Marc Lipsitch

4.2. In Practice: Research Ethics Committee Review in Public Health Emergencies

Koneti Rao

5. Health Emergency Research amid Global Inequities: Some Considerations for Researchers

Dirceu Greco 

6. Meeting Regulatory Criteria and Seeking Licensure: Medicines Development before and during Public Health Emergencies 

Marco Cavaleri, Emer Cooke, Mimi Darko, Marion Gruber

7. Research, Sample, and Data Sharing during Outbreaks, Pandemics, and Beyond 

Robert Fraser Terry, Katherine Littler 

Section III. Preparedness for Emergency Research Response

Section Editor Robert A. Sorenson

8. Building Biomedical Research Capacity in Low- and Middle-Income Countries: Why It Matters and Some of the Barriers to Success

Nahid Bhadelia, Vasee Moorthy, Robert A. Sorenson, Soumya Swaminathan

9. Laboratory Needs for Research Response 

Lisa E. Hensley, John D. Klena, Jason T. DeBoer, Joel M. Montgomery, Placide Mbala, Melissa E. Moses, Katie J. Knapek, Gene G. Olinger

10. Understanding How and Where Pathogens Emerge: Preparedness and Response for Zoonotic Diseases

Andrew Clements, Ian H. Mendenhall, Daniel Schar

11. Accelerating Diagnostic Innovation for Pandemic Control

C. Taylor Gilliland, William Heetderks, Krishna Juluru, Anthony Kirilusha, Tiffani Lash, Todd Merchak, Felicia Qashu, Douglas Sheeley, Mark Snyder, Andrew Weitz, Michael Wolfson, Bruce Tromberg

12. Vaccine Candidates for Novel Pathogens

Karin Bok

12.1. In Focus: Novel Manufacturing Platforms for Pandemic Preparedness and Emergency Response 

Karin Bok

13. Accelerating Vaccine Development: The 100 Days Mission

Aishani Aatresh, Richard Hatchett, Nicole Lurie

14. Accelerating Development of Therapeutics for Preparedness, Response, and a More Secure World 

Elizabeth S. Higgs

14.1. In Practice: The RECOVERY Trial

Peter Horby

Section IV.  Research Response

Section Editor Mosoka Fallah

15. ACTIV: A U.S. Public-Private Partnership Responds to COVID-19

David Wholley, Francis Collins, Paul Stoffels

15.1. In Practice: Leveraging an Integrated National Health System for Research Response: UK NIHR Respiratory Translational Research Collaboration

Nick Lemoine, Patrick Chinnery, Charlotte Taylor

16. Challenges for Emergency Research Interventions in Fragile, Weak, and Failed Nation States

Rebecca Katz, Alexandra L. Phelan, Cyrus Shahpar

16.1. In Practice: Responding to an Infectious Disease Outbreak amid a Humanitarian Emergency

Elizabeth Ross, Carol Han, Emily Rasinski

17. Integrating Clinical Research into Ebola Response: Liberia Case Study

Mosoka Fallah

17.1. In Practice: Integration of Clinical Research and Patient Care in the DRC PALM Ebola Therapeutics Trial

Richard Kojan

18. Good Participatory Practice. Social Mobilization, Communications, and Community Engagement 

Robert A. Sorenson, Yvette Delph, Barthalamew Wilson, Mosoka Fallah, Elizabeth S. Higgs  

18.1. In Practice: Building Community Engagement for Clinical Research Response

Michele Andrasik, Gail Broder, Linda Oseso, Patricia Segura, Kagisho Baepanye, Luciana Kamel, Nelson Michael

18.2. In Practice: Adapting Social Analytics for Research Response

Rhys O’Neill, David Cyprian, Elizabeth S. Higgs

19. Understanding and Reporting the Natural History of an Infectious Disease

Ian Crozier

20. Turning Research Results into Clinical Practice Guidelines in Public Health Emergencies

Donna M. Jacobsen, Henry Masur, Michael S. Saag, Paul A. Volberding

Section V. Methodology for Research Response

Section Editor Peter G. Smith

21. Epidemiologic Research in the Setting of Outbreak Response

Arthur Reingold

21.1. In Focus: The Impact and Mechanisms of Superspreading

Bjarke Frost Nielsen, Kim Sneppen, Lone Simonsen

22. Vaccine Trial Designs

Rebecca Kahn, Sofia S. Villar, Natalie E.  Dean, Marc Lipsitch

22.1. In Focus: Ring Trial Design

Natalie E. Dean, Ira M. Longini, Jr.

23. Data and Safety Monitoring of Clinical Trials During Public Health Emergencies

Michael A. Proschan, Birgit Grund

23.1. In Practice: Monitoring the PALM Ebola Therapeutics Study in the Democratic Republic of the Congo

Michael A. Proschan, David L. DeMets

24. Mathematical Modeling for Emergency Response: Using Models to Inform and Direct Response Priorities and Shape the Research Agenda

Bradford  Greening, Jr., Martin I. Meltzer

25. Models in the COVID-19 Pandemic

Natsuko Imai, Marc Baguelin, Neil M. Ferguson

25.1. Case Study: Modeling Fractional-Dose Emergency Vaccination Campaigns for Yellow Fever

Joseph T. Wu, Corey M. Peak

26. Social Science Evidence for Outbreak and Pandemic Response: Rapid Research and Analytics for Public Health Emergencies

Nina Gobat, Simone Carter, Ruth Kutalek, Sabina Faiz Rashid, Shelley Lees, Julienne Ngoundoung Anoko

Section VI. Governance, Institutions, and Partnerships

Section Editor Nicole Lurie

27.1. Global Framework for Research Preparedness and Response

Nicole Lurie, Gerald T. Keusch

28. Financing Emergency Research Response during Infectious Disease Outbreaks: Lessons from the World Bank and Other International Financial Institutions

Muhammad Pate, Sulzhan Bali

29. International Collaboration to Advance Research Preparedness and Response

Amanda Rojek, Gail Carson 

Case studies: Kenneth Ballie, Lennie Derde, Alice Norton, Lina Moses  

30. Organizational Partnerships for Preparedness and Response to Emerging and Re-emerging Infectious Diseases 

Yazdan Yazdanpanah, Claire Madelaine, Nicolas Pulik, Yves Souteyrand

30.1. In Focus: Research and Medical Humanitarian NGOs

Rebecca F. Grais, Emmanuel Baron

30.2. In Practice: Building and Maintaining Preparedness for a Rapid Research Response in Indonesia

Chuen-Yen Lau, Louis Grue, Aaron Neil, Muhammad Karyana

Section VII. Research Operations

Section Editor Laura McNay

31. Operational Recommendations for Streamlining Emergency Research Responses to Pandemics  

Gregg Larson, Rachel Harrigan, Laura McNay

32. Launching a Clinical Research Operation

Gregg Larson, Laura McNay

32.1. In Practice: Clinical Research Communications during an Outbreak: Media Outreach Supporting Liberia-U.S. Research Partnership 

Laurie K. Doepel, Hassan Kiawu

32.2. In Focus: Clinical Trial Insurance and Indemnification

Gregg Larson

33. Ethical Review of Research during an Emergency Response 

Jerome F. Pierson

33.1. In Practice: Ethical Review During Emergencies: The Liberian Experience

Fatorma Bolay, Robert A. Sorenson

33.2. In Practice: Independent Monitoring of Emergency Response Clinical Trials 

Susan Vogel, Jerome F. Pierson

33.3. In Practice: Capacity Building for Research Ethics Review in Low- and Middle-Income Countries

Barbara Sina, John Tierney

34. Information and Communications Technology to Support Research in Low-Resource Settings

Mike Galcik, David Parrish

35. Data Management in Emergency Response Research

Harry van Loen, Moses Badio, Yven Van Herrewege

36. Safety and Pharmacovigilance in Emergency Research Response

Marc Teitelbaum, Negin Atri, Kelly Cahill

37. Supply and Logistics for Clinical Research in Low-Resource Settings

Beth Baseler, Calvin Proffitt, Jen Sandrus, Jonathan Marchand, Eric Stavale 

38. Pharmaceutical Management

Matthew Carl Kirchoff

39. Cold Chain and Electrical Power for Emergency Research Response

Daniel J. Littlefield

40. Selecting and Opening a Clinical Research Site in a Low-Resource Setting

Olivier Tshiani Mbaya, Wissedi Sio Njoh, Kevin Barrett, Mary Smolskis, Alejandra Miranda, Nikki Gettinger 

40.1. In Practice: Improving Patient Care in the Field: The CUBE Isolation Unit

Richard Kojan

41. Management of Security for Clinical Trials During Emergencies 

Billy Sivahera Muyisa, Eric Barte de Sainte Fare, Jamila Aboulhab

42. Locally Hired Staff for Clinical Research Sites in Low-Resource Settings

Beth Baseler, Mary Smolskis, Jestina Doe-Anderson, Melvin Johnson, Wissedi Njoh, Sara Albert, Chris Worthington

O autorze

Dr. Elizabeth S. Higgs, MD, MIA, DTMH, is a global health science advisor, clinical scientist, and pandemic preparedness research response policy expert working in the Division of Clinical Research at the U.S. National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH). Throughout her career, Dr. Higgs has contributed to critical health emergencies, including H1N1, Ebola outbreaks in West Africa and the DRC, the 2018 Nipah outbreak in Kerala, India, mpox, and SARS-CoV-2, demonstrating that regulatory-level clinical trials during health emergencies are possible and contribute to medical countermeasures that accelerate the end of outbreaks. 

Known for global health diplomacy while fostering interagency and multilateral collaborations, she actively engages in strategic global health security initiatives and policy groups, making impactful contributions to key policy documents such as the World Bank's Money and Microbes, the G7 Clinical Trials Charter, and the U.S. National Biosecurity Strategy and Global Health Security Strategy.

Her passions include nurturing sustainable clinical research capacity in low- and middle-income countries and promoting cooperative global clinical trial networks. With a foundation in internal medicine and infectious diseases, Dr. Higgs holds a doctorate in medicine, a master’s degree in international affairs, an interdisciplinary bachelor's degree in bioethics, and a Diploma in Tropical Medicine and Hygiene. Through her commitment to response research, she is dedicated to safeguarding and enhancing the well-being of people worldwide in the face of health emergencies caused by emerging infectious disease.

Robert A. Sorenson has worked with the NIAID Division of Clinical Research on infectious disease emergency response policy, especially urgent clinical research response to emerging pathogens, since 2016, after having worked on global health policy issues at the U.S. Department of State since 2009. 

Mr. Sorenson was a Foreign and Civil Service Officer at State for 33 years. After 2001, when family circumstances curtailed his overseas career, he primarily served in the Bureau of Oceans, Environment, and Science, including terms as Deputy Director of the Offices of International Health and Biodefense, Environmental Policy, and Ecology and Terrestrial Conservation. His overseas experience, starting in 1986, included political, consular, environmental, and deputy chief of mission positions in Manila, Moscow, Osaka, Skopje, Tashkent, and Tirana, as well as several temporary duty assignments in crisis spots. He retired from State on New Year’s Eve, 2017 and has since been a contractor with NIAID. 

As managing editor of PPERR, Mr. Sorenson revived and updated his professional editorial experience, which dated to 1981-1986. He was a National Fellow at the Hoover Institution in 1999-2000 and has an MA in Russian literature from Cornell University and a BA from St. Olaf College

Nicole Lurie, M.D., M.S.P.H., is Executive Director for Preparedness and Response at the Coalition for Epidemic Preparedness Innovations (CEPI) and Director of CEPI-US. She is also a Senior Lecturer at Harvard Medical School and Professor of Medicine at George Washington University School of Medicine.  

She served an eight-year term as Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services from 2009 to 2017. In that role she led the HHS response to numerous public health emergencies, ranging from infectious disease to natural and man-made disasters, and is responsible for many innovations in emergency preparedness and response.  

Prior to federal service, she was the Paul O'Neill Professor of Policy Analysis at RAND, and a Professor of Medicine and Public Health at the University of Minnesota. Her research has spanned access to and quality of care, health system redesign, health equity, mental health, public health, and preparedness. She is the recipient of numerous awards and is a member of the National Academy of Medicine. She continues to practice medicine in a community clinic in Washington DC.

Peter Smith is Professor of Tropical Epidemiology at the London School of Hygiene &Tropical Medicine (LSHTM). 

He graduated in mathematics and statistics from City University, London and in 1965 joined the Medical Research Council's Statistical Research Unit in London. Since then, he has worked on various aspects of epidemiological and statistical research based in Edinburgh, Kampala, Oxford, Boston and Geneva. He joined LSHTM in 1979 to head the MRC International Statistics & Epidemiology Group. Research interests include large-scale intervention studies against tropical diseases, recently focussing on vaccines. 

From 1999 to 2004 Dr. Smith chaired the UK Government Spongiform Encephalopathy Advisory Committee.  He has chaired the WHO Global Advisory Committee on Vaccine Safety and the WHO Technical Expert Group on the RTS,S/AS01 malaria vaccine. He served as the Deputy Chair of the Nuffield Council on Bioethics and from 2004 to 2014 was a Governor of the Wellcome Trust. He has served on the Scientific Advisory Committees of the Coalition for Epidemic Preparedness Innovations (CEPI) and the European and Developing Countries Clinical Trials Partnership (EDCTP). He chairs the WHO SAGE Working Group on malaria vaccines and serves on the WHO SAGE Working Group on SARS-CoV-2 vaccines.

Laura Ann McNay, MS, is the Deputy Director for Operations and Management in the Division of Clinical Research, NIAID.  She started her career at NIAID in 1991 and has served in many roles at NIAID over the past 32 years.  During Ms. McNay's tenure with DCR, she has been responsible for the management and oversight of numerous small and large, multinational clinical trials.  

In addition to her role as DCR Deputy Director for Operations and Management, she serves as the DCR Project Lead for the Partnership for Research on Vaccines and Infectious Diseases in Liberia (PREVAIL). PREVAIL is a clinical research partnership between the U.S. Department of Health and Human Services (DHHS) and the Liberian Ministry of Health. 

Ms. McNay attended the University of Maryland Baltimore County where she received her bachelor’s degree in economics in 1987.  In 2002, she received her Master of Science in Management and Healthcare Administration.

Mosoka P. Fallah, PhD, MPH, MA is Program Manager of the Saving Lives and Livelihoods Initiative, Africa Centres for Disease Control and Prevention. The Initiative supports the acquisition and distribution of 65 million COVID-19 vaccine doses and has a mandate to focus on turning vaccine deliveries into vaccinations. Dr. Fallah completed his PhD in Immunology at the University of Kentucky and his MPH in Global Health/Infectious Disease Epidemiology at the Harvard T. H. Chan School of Public Health. For his work building community-level trust in the Ebola response, Dr. Fallah was named one of the Time Magazine Persons of the Year in 2014.  

Dr. Fallah was also the co-founder and Director General of the National Public Health Institute of Liberia (NPHIL), which was founded in response to the devastation of the 2014-2016 Ebola outbreak in Liberia. He has also held positions as a health security technical consultant on pandemic diagnostics with the World Bank, visiting lecturer at Harvard Medical School, and senior consulting scientist at Medical Science & Computing, LLC. He continues to serve as President and CEO of Refuge Place International (RPI), a non-governmental organization dedicated to creating a replicable health care model for economically poor and disenfranchised people residing in the urban slums and poor rural communities of Liberia.

Blurb

Robust global health security requires effective preparation for and response to emerging and re-emerging pathogens. The nascent field of emergency research response and preparedness is a crucial component in preventing and mitigating outbreaks, yet it is rife with critical scientific, ethical, and policy questions. How can local, national, and global systems collaborate across disciplines and political boundaries to protect health security? How can we conduct rigorous research in fragile or failed nation-states? What is needed for equitable distribution of vaccines and therapeutic medicines in short supply? How can we accelerate trials of vaccines and therapeutics during an emergency without compromising scientific rigor or ethical standards? How do we integrate communities and stakeholders into research preparedness and response? 

 

The editors bring decades of experience to their collaboration with renowned research scientists and health policy experts. Together they have crafted a compendium that proposes normative standards and offers practical guidance for preparedness and rapid research response. This pioneering, open access textbook presents principles and practices forged from experience in health emergencies – insights that illuminate a path forward for research response and preparedness. The goal is to stop outbreaks from becoming avoidable pandemics, and to mitigate illness, death, and social disruption if they cannot be stopped.

 

Principles and Practice of Emergency Research Response merges historical understanding with insights into possible futures. It will serve as an indispensable compass for curtailing infectious disease threats through research, global health policy changes, and research capacity improvements where they are most needed. This book will be an ongoing reference for governments, health organizations, development agencies, researchers, policy experts, ethicists, and social scientists. To facilitate focused and enhanced learning, it incorporates pedagogical tools such as abstracts, learning objectives, discussion questions, real-life examples, and learning tracks.