Non-communicable Disease Prevention: Best Buys, Wasted Buys and Contestable Buys E-Book

Rachel A. Archer

• Herausgeber: Open Book Publishers
• Kategorie: Fachliteratur
• Sprache: Englisch
• Veröffentlichungsjahr: 2019

Beschreibung

Non-communicable diseases (NCDs) are the leading cause of death worldwide, contributing to over 73% of all deaths annually. Each day NCDs cause more than 100,000 deaths, 80% of which occur in low- and middle-income countries. NCDs, however, are largely preventable, and a great deal of technical knowledge exists about how to prevent and manage them. Why, then, have we, as a global community, not been more successful at reducing this NCD burden? Does a universal problem not have a universal solution?


Created by an international consortium of experts, this informative and accessible book provides practical guidelines, key learning points, and dynamic, real-world case studies to aid NCD program managers, policy officers and decision-makers in low- and middle-income countries, so that they can assess interventions for the prevention and control of NCDs.

The book was commissioned by the Prince Mahidol Award Conference (PMAC), an annual international conference centred on policy of global significance related to public health.  Non-communicable Disease Prevention: Best Buys, Wasted Buys and Contestable Buys emphasises the importance of context in NCD control and prevention, arguing that the success of an intervention lies in an ability to respond to local needs and environments. The book comprises ten chapters, which collectively explore the reasons behind, and strategies for, preventing and managing the NCD burden. It spans key themes such as political economy, the transferability of economic evidence, the role of cross-sectoral policies, the importance of deliberative processes, and health technology assessment.

This book is written for the benefit of the global health community, and is primarily targeted at those individuals who are involved in NCD programs. This book will also be of interest to NCD champions, policy advocates, and educators spearheading the movement for increased visiblity of NCDs.
 

Leseprobe

Non-Communicable Disease Prevention

Best Buys, Wasted Buys and Contestable Buys

Non-Communicable Disease Prevention

Best Buys, Wasted Buys and Contestable Buys

Edited by Wanrudee Isaranuwatchai, Rachel A. Archer, Yot Teerawattananon and Anthony J. Culyer

https://www.openbookpublishers.com

© 2019 Wanrudee Isaranuwatchai, Rachel A. Archer, Yot Teerawattananon and Anthony J. Culyer. Copyright of individual chapters is maintained by the chapters’ authors.

This work is licensed under a Creative Commons Attribution 4.0 International license (CC BY 4.0). This license allows you to share, copy, distribute and transmit the text; to adapt the text and to make commercial use of the text providing attribution is made to the authors (but not in any way that suggests that they endorse you or your use of the work). Attribution should include the following information:

Wanrudee Isaranuwatchai, Rachel A. Archer, Yot Teerawattananon and Anthony J. Culyer, Non-Communicable Disease Prevention: Best Buys, Wasted Buys and Contestable Buys. Cambridge, UK: Open Book Publishers, 2019, https://doi.org/10.11647/OBP.0195

In order to access detailed and updated information on the license, please visit, https://doi.org/10.11647/OBP.0195#copyright

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All external links were active at the time of publication unless otherwise stated and have been archived via the Internet Archive Wayback Machine at https://archive.org/web

Digital Appendices associated with this volume are available at https://hdl.handle.net/20.500.12434/09617d51

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ISBN Paperback: 978-1-78374-863-1

ISBN Hardback: 978-1-78374-864-8

ISBN Digital (PDF): 978-1-78374-865-5

ISBN Digital ebook (epub): 978-1-78374-866-2

ISBN Digital ebook (mobi): 978-1-78374-867-9

ISBN XML: 978-1-78374-868-6

DOI: 10.11647/OBP.0195

Cover image: Health check in N’Tossoni, near Koutiala, Mali. Photo by Ewien van Bergeijk (2014), CC-BY-SA 4.0.

Cover design: Anna Gatti.

Contents

Forewords

vii

Acknowledgements

xv

Notes on Contributors

xix

1.

Introduction

1

Wanrudee Isaranuwatchai, Rachel A. Archer and Anthony J. Culyer

1.1

Non-Communicable Disease

1

1.2

Best, Wasted and Contestable Buys

5

1.3

Definitions and Central Ideas

6

Box 1.1 Definitions of Basic Terminology Used in Economic Evaluation

7

1.4

The Cost-Effectiveness Plane

8

1.5

The Story of This Book

12

1.6

The Project and Its Output

13

1.7

The Project Journey

13

1.8

Target Audience

15

2.

Non-Communicable Diseases, NCD Program Managers and the Politics of Progress

17

Sumithra Krishnamurthy Reddiar and Jesse B. Bump

2.1

Background

17

2.2

Methods for Interviews and Analysis

19

2.3

Institutions: NCD Managers, NCD Units and Ministries of Health

21

2.4

Interests: Stakeholders and Power

26

2.5

Ideas: Evidence, Knowledge and Values

28

2.6

Discussion

33

2.7

Limitations

38

2.8

Conclusions and Recommendations

38

3.

Framework for Implementing Best Buys and Avoiding Wasted Buys

41

Yot Teerawattananon, Alia Luz, Manushi Sharma and Waranya Rattanavipapong

3.1

Consideration One

42

3.2

Consideration Two

44

3.3

Consideration Three

45

3.4

Consideration Four

46

3.5

Consideration Five

47

3.6

The SEED Tool in Practice

48

4.

Best Buys

51

Tazeem Bhatia, Arisa Shichijo and Ryota Nakamura

4.1

Introduction

51

4.1.1

Background

51

4.1.2

What This Chapter Offers

54

4.2

Determining Important Contextual Factors in NCD Prevention

55

4.3

Policymaking Challenges and Cost-Effectiveness Data

60

Investigating Case Studies

61

Case Study 4.4.1 Cardiovascular screening in Sri Lanka

61

Case Study 4.4.2 Prevention and control of cervical cancer in Cambodia

63

Case Study 4.4.3 Sugar-Sweetened Beverage (SSB) taxes

64

4.5

Discussion

66

4.6

Conclusion

68

5.

Wasted Buys

71

Yot Teerawattananon, Manushi Sharma, Alia Luz, Waranya Rattanavipapong and Adam G. Elshaug

5.1

Introduction

71

5.1.1

What Are ‘Wasted Buys’?

72

5.1.2

The ‘Area of Uncertainty’

74

5.2

Exploring Wasted Buys in Low- and Middle-Income Countries (LMICs)

75

A. Cochrane Collaboration Database

75

B. The Global Health Cost-Effectiveness Analysis (GH CEA) Registry

76

C. Disease Control Priorities (DCP)

76

Case Study 5.2.1 Mass-media campaigns for prevention of NCDs

77

Case Study 5.2.2 Cost-effectiveness of medical primary prevention strategies to reduce the risk of cardiovascular disease (CVD) in Tanzania

79

Case Study 5.2.3 Diabetes screening in Thailand and Indonesia

80

Case Study 5.2.4 Drug testing in Bhutan

82

5.3

Common Features of Wasted Buys

84

5.3.1

The Fallacy that Prevention Interventions Are Always Best Buys

85

5.3.2

One Size Seldom Fits All in International Guidelines

85

5.3.3

Policy-Based Evidence Versus Evidence-Based Policy

86

5.3.4

Selective Implementation of Interventions

86

5.3.5

Low CE Threshold Used for Decision-Making

87

5.4

Recommendations

88

6.

Assessing the Transferability of Economic Evaluations: A Decision Framework

91

David D. Kim, Rachel L. Bacon and Peter J. Neumann

6.1

Introduction

91

6.2

Review of the Literature

95

6.3

A Decision Framework for Identifying Locally-Relevant Best and Wasted Buys

97

6.3.1

Background

97

6.3.2

A Decision Framework and a Transferability Assessment Checklist

98

Step 1: Initial Assessment of Study Design

102

A. Study Perspective

102

B. Intervention and its Comparator(s)

103

C. Time Horizon

103

D. Discounting

104

E. Study Quality

104

Step 2: Data Transferability Assessment

105

A. Baseline Risk (Disease Profile)

106

B. Treatment Effects (Clinical Information)

106

C. Unit Costs/Prices

106

D. Resource Utilization

107

E. Health-State Preference Weight

107

6.4

Worked Example: Assessing Transferability of Best Buy Interventions for Diabetes Prevention and Management in Kenya

109

6.4.1

Background and Rationale

109

6.4.2

Evaluator’s Guideline on Economic Evaluation

110

6.4.3

Transferability Assessment Process

111

6.4.4

Transferability Assessment Results

114

6.5

Using the Impact Inventory

116

6.6

Conclusion and Next Steps

117

7.

Finding the Best Evidence

119

Thunyarat Anothaisintawee

7.1

Determining the Impact of Behavior Change on NCDs Through Research

119

Case Study 7.1.1 Knowledge growth: A case study of low-calorie sweeteners

119

7.2

Types of Study Design

120

7.3

Quality Assessment of Studies/Evidence

121

7.4

Types of Evidence Synthesis

123

Case Study 7.4.1 Efficacy of lifestyle interventions and effect of lifestyle factors on the risk of type 2 diabetes mellitus, cardiovascular diseases and hypertension: An umbrella review

125

7.5

Role of Environmental Interventions in Changing Health Behavior

126

7.6

Conclusion

128

8.

Cross-Sectoral Policies to Address Non-Communicable Diseases

129

Melitta Jakab and Peter C. Smith

8.1

Introduction

129

8.2

Why Are Cross-Sectoral Policies So Challenging?

132

8.3

Analytic Framework

134

8.4

Institutional Requirements

137

8.5

Types of Cross-Sectoral Policies

140

Case Study 8.5.1 The public catering decree in Hungary

141

Case Study 8.5.2 Employing people with disabilities in Croatia

142

8.6

Conclusions

144

8.7

Analytical Appendix

145

9.

Deliberative Processes in Decisions about Best Buys, Wasted Buys and Contestable Buys: Uncertainty and Credibility

147

Kalipso Chalkidou and Anthony J. Culyer

9.1

Introduction

147

9.2

Criteria, Opportunity Costs and Social Value Judgments: A Role for Deliberation

148

9.3

Deliberation Contrasted with Algorithms

150

9.4

Evidence

152

Box 9.1 Categories of Evidence

152

9.5

Uncertainty

159

9.6

Credibility

159

9.7

Some Characteristics of Deliberative Processes

163

Case Study 9.7.1 The (then) National Institute for Clinical Excellence (England and Wales)

164

9.8

Conclusions

168

10.

Summing Up

171

Wanrudee Isaranuwatchai, Rachel A. Archer and Anthony J. Culyer

Glossary of Abbreviations

175

List of Illustrations and Tables

179

Online Appendixes: https://hdl.handle.net/20.500.12434/09617d51

To those who are making a difference, and those who would like to make a difference in our healthcare system.

Forewords

The ultimate purpose of the Prince Mahidol Award Foundation under Royal Patronage, according to my interpretation, is to pursue the ideology of Prince Mahidol of Songkla in serving the benefit of mankind. The Foundation has three activities: the Prince Mahidol Award, the PMAC (Prince Mahidol Award Conference) and the Prince Mahidol Award Youth Program. The theme of PMAC 2019 was ‘The Political Economy of NCDs: A Whole of Society Approach’. The idea to publish this book, Non-communicable Disease Prevention: Best Buys, Wasted Buys and Contestable Buys, was proposed during the PMAC 2019 preparation meeting and has been partially funded by PMAC.

PMAC is proud to have supported the development and dissemination of this book. In fact, it is more than just a book. We hope that it will be a collective learning tool for NCD managers and stakeholders, together with health economists or health intervention and technology assessment specialists. The ultimate goal of the learning process is ‘good health at reasonable cost’ with emphasis on NCDs. Taking a broader perspective, this learning process aims to help strengthen universal health coverage (UHC) schemes.

NCDs and their root causes are very complex; addressing or preventing them is even more complex. A policy or intervention which is thought to be Best Buy can turn out to be Wasted Buy. Even when there is evidence of high cost-effectiveness in one country, when the intervention is transferred directly to another country, it can become a Wasted Buy.

This is a book of evidence management and utilization in NCD prevention, which can be applied to the development of health systems as a whole. The key proposal is the SEED Tool (Systematic thinking for Evidence-based and Efficient Decision-making). To me, it is a framework or conceptualization tool that can handle complex situations. The decision-making process proposed is not linear, but a learning loop to guide deliberation. The book helps the target audience scrutinize evidence, mainly cost-effectiveness analyses, to be applied in local contexts with involvement.

PMAC is proud to present this high-quality commissioned work. We hope it will help to change the paradigm from communicable-disease-oriented health systems to more NCD-oriented systems, which is a much more complex paradigm.

Prof. Vicharn Panich

Chairperson of PMAC International Organizing Committee

Since 2007, the Prince Mahidol Award Conference (PMAC) has been organized as an annual international conference focusing on policy of global significance related to public health. For over a decade, PMAC has provided opportunities for debate, discussion and deliberation on priority global health policy and systems, and it has contributed to the exchange of knowledge and experience on global health between participants from across the world. In 2018, PMAC initiated commissioned work to provide a body of evidence to facilitate the sharing of experience at country level and among country and regional networks, in order to influence the implementation of global health and/or national policies and to enhance PMAC’s capacities to deliver its knowledge and experiences to a wider spectrum of people.

Non-Communicable Disease Prevention: Best Buys, Wasted Buys and Contestable Buys is the first PMAC-commissioned work. This book provides evidence-informed insights to help understand which non-communicable disease (NCD) interventions work and which don’t, so that program managers, policy officers and decision-makers in low- and middle-income countries (LMICs) can assess and implement interventions for the prevention and control of NCDs. It is a gold mine of very informative, easy to read and extremely helpful guidelines for those who wish to implement or reassess their strategies for preventing the NCD burden in their settings.

Non-Communicable Disease Prevention will augment PMAC’s contribution in terms of changing health policy and improving health systems in different settings in relation to NCDs. It will continue the momentum of the PMAC 2019 theme on ‘The Political Economy of NCDs: A Whole of Society Approach’, enable the contributions from PMAC to reach a wider audience and sustain PMAC work into the future. This sharing of real-world case studies, practical guidelines and key learning points will truly benefit all relevant stakeholders and the global health community and help accelerate the global progress in NCD prevention and control.

Prof. Churnrurtai Kanchanachitra

PMAC Secretariat

Demographic change, like climate change, proceeds slowly. National populations — starting from very different positions — exhibit a steady, usually predictable, but always slow increase in the number of individuals at older ages. The risks of stroke, heart disease, cancers and chronic respiratory illness increase sharply with age. Thus demography drives increases in incidence and mortality from these conditions. These changes — like the consequences of climate change — often remain below the threshold of visibility. Until they don’t. Almost all middle-income countries (and many low-income ones) have crossed a threshold where the major non-communicable diseases (NCDs) have become highly salient in public discourse and, more practically, in the budgetary demands on health systems. Yet only recently — in many countries — have the medical, public health and public policy communities begun to assess critically how best to respond to the inexorable rise in NCDs.

Thailand achieved unusually early success in reducing child mortality and infectious disease mortality more generally with one consequence being the aging of its population and concomitant rise in NCDs. Also unusually, Thailand invested early and substantially to create the analytic capacity to identify and develop approaches to NCD prevention and management. The Thai Health Ministry’s Health Intervention and Technology Assessment Programs (HITAP), as well as closely associated efforts at Mahidol University, have provided world leadership in developing and applying techniques of economic evaluation to help ensure that public money spent on health buys the greatest possible reduction in premature mortality and morbidity. This timely volume — Non-Communicable Disease Prevention: Best Buys, Wasted Buys and Contestable Buys — brings to a global audience a distillation of much of HITAP’s experience. An international editorial team was formed to match authors to topics. Then a broadly inclusive and iterative process of chapter development, described in Chapter 1, led to a volume that will become required reading for two important audiences: one concerned with implementation of strategies for NCD control and, significantly, the community of economists and others seeking an up-to-date account of how best to apply economic methods in practice.

Three important characteristics contribute to making this book an unusually informative resource. First, the volume results from an extensive international collaboration of individuals and institutions. This collaboration enriches the book’s content and facilitates communication with diverse audiences. Second and closely related, the volume relies heavily on case studies to convey its main message — a total of fifty-eight case studies from thirty countries. The case studies ground the lessons of the book in operational experience and should prove of particular salience to NCD program managers, an audience the volume particularly tries to reach. Finally, this book develops and present a practical guide to the assessment of intervention attractiveness — the ‘Systemic thinking for Evidence-based and Efficient Decision-making (SEED)’ tool. SEED provides a valuable framework both for the book itself and for its application in practice.

It is not my purpose in this brief forword to overview this rich a volume. Nonetheless, I would like to touch on three points that resonated with my own experience working on the Disease Control Priorities (DCP) Project and, in particular, on issues that various iterations of DCP have had to deal with over many years.1 One concerns the quality and transferability of evidence. A second persistent issue concerns economic evaluation when an intervention has significant non-health consequences (what the volume’s authors call cross-sectoral intervention). Third, the volume’s title points to consideration of Wasted Buys as well as Best Buys, a topic too often neglected in the literature. On each of these issues the authors provide valuable insights.

Two of the volume’s chapters discuss evidence: one focuses on the synthesis of evidence of varying degrees of quality; and another on transferring findings from one setting or population to another and perhaps to very different settings or populations. The volume concurs in the general observation that randomized-controlled trials (RCTs) provide the highest quality evidence, but it is equally insistent in pointing out that an RCT must be supplemented by judgement about transferability if the results are to be applied outside of the original setting. ‘Hard’ evidence can become soft very quickly and the DCP approach has been to acknowledge the ever-present need for informed judgement about the relevance and transferability of evidence. The explicit objective of the DCP’s approach has been to balance concerns about accepting that an intervention is attractive when it is not — unfortunately the dominant concern of the medical community — with apprehension about rejecting an intervention that might be appropriate. The approach advocated in this book provides a welcome, systematic approach to facilitate judgement in this necessary search for balance. This approach can be applied equally to two other areas where judgement is required: evaluating the effect of a combined intervention (multi-drug approaches to secondary prevention of vascular disease, for example, when trials have been undertaken only on single-drug regimens); and deciding whether two different interventions (two health promotion campaigns or two anti-hypertensive drugs, for examples) can be viewed as essentially the same in terms of efficacy.

Reducing behavioral and environmental risk often involves action outside the health sector. Issuing and enforcing controls on air quality, for example, could result in significant reductions in mortality in many cities. Health ministries lack money and mandates to issue such regulations and, even if they did, there are likely to be significant benefits that derive from such investments that are unrelated to health. An economic evaluation of air quality regulation that relies on a standard incremental cost-effectiveness assessment — cost per death averted, say — will fail to capture all relevant benefits. On the other hand, an economic evaluation from the perspective of an energy ministry may simply neglect to consider health benefits. This book includes a thoughtful chapter on how to approach this problem within a cost-effectiveness framework. DCP authors assessing cross-sectoral intervention have tended either to report ‘dashboards’ of outcomes, without aggregation into a single figure of merit, or to use monetary metrics within a benefit-cost analysis (BCA) framework. I don’t see an approach that is obviously best (although I lean toward BCAs). This book very much contributes to the thinking on this topic and national experience with the methods explained here will, over time, provide insight into what is practical and useful.

Separating economic evaluation from advocacy can prove difficult. Groups that work on immunization (or any other interventions you can name) often do so from a laudable commitment to the value of what they are doing. Likewise, because of their interest, these groups often commission or participate in economic evaluations. It requires no conscious bias to have results lean toward the favorable. Thus, this book’s explicit argument for the importance of also considering Wasted Buys is very much to be welcomed. My own experience in DCP was that asking authors to identify interventions of low priority met with little success. Most DCP authors — there were important exceptions — simply avoided doing this. It appears that the authors of this book experienced similar problems. They report that of the fifty-eight case studies received, forty-seven were of Best Buys, seven were Contestable Buys and only four were Wasted Buys. This simply underscores this volume’s contribution to generating sustained and serious consideration of what not to do (or to do only later). The inclusion of Wasted Buys in the title of the book and in the analysis sends a good message.

******

Analysts often neglect the political economy of implementation. One could argue that there is a natural division of labor between analysis and the politics of implementation (and in the past I have so argued). This book takes the perspective that considerations of political economy need inclusion from the outset. It is reasonable to predict that this explicit approach will combine with the book’s analytic strength to give it enduring value.

Prof. Dean T. Jamison

Institute for Global Health Sciences University of California, San Francisco

1 Dean T. Jamison et al., Disease Control Priorities: Improving Health and Reducing Poverty (Washington, DC: World Bank, 2017), 3rd edition, IX.

Acknowledgements

First, we would like to thank our funders for their generous support and for making our vision for this project a reality: the Prince Mahidol Award Foundation (PMAF), the Thai Health Promotion Foundation (THF) and the international Decision Support Initiative (iDSI).

The editors gratefully acknowledge the contribution of all the authors of this work, for without their diligence, commitment and expertise, this book would not have been possible. We specifically thank the following: Dr. Adam Elshaug (University of Sydney, Australia), Ms. Alia Luz (Health Intervention and Technology Assessment Program (HITAP), Thailand), Ms. Arisa Shichijo (Hitotsubashi University, Japan), Dr. David Kim (Tufts Medical Center, United States), Dr. Jesse Boardman Bump (Harvard University, United States), Dr. Kalipso Chalkidou (Center for Global Development in Europe, United Kingdom), Ms. Manushi Sharma (HITAP, Thailand), Dr. Melitta Zsuzsanna Jakab (World Health Organization, Spain), Dr. Peter Neumann (Tufts Medical Center, United States), Dr. Peter Smith (Imperial College London, United Kingdom), Ms. Rachel Bacon (Tufts Medical Center, United States), Dr. Ryota Nakamura (Hitotsubashi University, Japan), Ms. Sumithra Krishnamurthy Reddiar (Harvard University, United States), Dr. Tazeem Bhatia (Public Health England, United Kingdom), Dr. Thunyarat Anothaisintawee (Faculty of Medicine, Ramathibodi Hospital, Thailand) and Ms. Waranya Rattanavipapong (HITAP, Thailand). We would also like to thank the following individuals who have supported the writing of chapters. Thanks to Dr. Olaa Mohamed-Ahmed (Public Health England, United Kingdom) for their contribution to the appendices for Chapter 4 and thanks to Assistant Professor Kanokporn Sukhato, Dr. Kridsada Chareonrungrueangchai and Dr. Keerati Wongkawinwoot (all from Ramathibodi Hospital, Thailand) for working on the study selection and data extraction of the umbrella review in Chapter 7.

We are especially grateful to Ms. Benjarin Santatiwongchai and Ms. Jirata Tienphati and rest of the HITAP communication team for their tireless support in the communication and presentation of this work. We also deeply appreciate all the efforts of the HITAP staff who have helped in the coordination and administration of the project.

Our external reviewers, whom we thank for their support in advancing our work, were: Dr. Amanda Glassman (Center for Global Development, United States), Dr. Shankar Prinja (PGIMER, India), Ms. Ursula Giedion (International Health Policy Consultant, Switzerland), Dr. Edwine Barasa (KEMRI-Wellcome Trust, Kenya), Dr. Arian Hatefi (University of California San Francisco, United States), Dr. Justin Parkhurst (London School of Economics, United Kingdom), Dr. Bundit Sornpaisarn (University of Toronto, Canada), Dr. Sara Bennett (Johns Hopkins Bloomberg School of Public Health, United States), Dr. Jeremy Addison Lauer (World Health Organization, Switzerland) and Ms. Priya Kanayson (NCD Alliance, United States).

Our thanks also to those listed below for reviewing our preliminary findings and also providing invaluable feedback:

Dr. Anita Jain (The BMJ, India)

Dr. Bundit Sornpaisarn (University of Toronto, Canada)

Dr. Chanuantong Tanasugarn (Mahidol University, Thailand)

Dr. Douglas Webb (United Nations Development Program, United States)

Ms. Emily Kobayashi (Clinton Health Access Initiative, United States)

Dr. Kanchan Mukherjee (Tata Institute of Social Sciences, India)

Dr. Karen Hofman (PRICELESS SA, South Africa)

Dr. Kelvin Tan (Ministry of Health, Singapore)

Ms. Milin Sakornsin (Thai Health Promotion Foundation, Thailand)

Dr. Myo Paing (World Health Organization, Myanmar)

Dr. Naomi Hamada (Ministry of Health and Medical Services, Fiji)

Mr. Pempa (Health Technology Assessment Program, Bhutan)

Ms. Saudamini Dabak (HITAP, Thailand)

Dr. Suchita Bhattacharyya (University of Liverpool, United Kingdom)

Dr. Sumudu Karunaratna (Ministry of Health, Sri Lanka)

Dr. Tea Collins (World Health Organization, Switzerland)

The authors of Chapter 2 and the project team gratefully acknowledge the contributions of their interviewees.

Finally, we owe a sincere thanks to the contributors who submitted case studies of their experiences with Best, Wasted and Contestable Buys in their local settings, in particular to Mr. Pempa, Dr. Rohan Jayasuriya, Dr. Sumudu Karunaratne and Dr. Amala de Silva, whose case studies were chosen as special features. We also acknowledge Dr. Koum Kanal, Dr. Karen Hofman, Mr. Gavin Surgey et al., Dr. Cristóbal Cuadrado and Ms. Frances Claire Onagan, whose case studies were discussed in the book. Thanks is also given to Ms. Aparna Ananthakrishnan from HITAP for proofreading this book.

Notes on Contributors

Thunyarat Anothaisintawee, MD., Ph.D., is a Family Physician. She holds a Ph.D. in Clinical Epidemiology and has worked as a faculty staff member at the Department of Family Medicine, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Thailand. She is an expert in systematic reviews and meta-analysis and published several papers about the association between sleep factors and risk of developing diabetes mellitus in international medical journals. Currently, she is conducting the Prediabetes cohort study in Thailand. This cohort aims to investigate the association between sleep factors, eating habits, level of physical activity, genetic factors and risk of developing diabetes mellitus and chronic kidney disease in prediabetes people in Thailand.

Rachel A. Archer, M.P.H.,is a Project Associate at the Health Intervention and Technology Assessment Program (HITAP). Her work focuses on health system strengthening and supporting evidence-informed health policy making in low- and middle-income countries (LMICs). She is the focal point at HITAP for the Total Systems Effectiveness (TSE) project, an approach to strengthen vaccine decision-making in LMICs, and she currently leads the PMAC Commissioned Work project. Rachel has also supported capacity-building activities for Indonesia, Kenya and The Philippines. Rachel holds a Master’s degree in Public Health from the University of Sheffield and a Bachelor of Arts in International Development from the University of Leeds. Whilst studying, she interned with various non-profit organizations across East and West Africa. For her Master’s thesis, Rachel collaborated with a non-profit to investigate the trend towards teenage pregnancy in Luwero District, Uganda, through an intersectional framework. She was awarded the Carpenter Prize for Best Dissertation.

Rachel L. Bacon, M.P.H., is the Project Manager for global health initiatives at the Center for the Evaluation of Value and Risk in Health (CEVR) at the Institute for Clinical Research and Health Policy Studies (ICRHPS) at Tufts Medical Center. Rachel is a public health professional with knowledge and applied experience in global health, reproductive health, health economics, health systems strengthening, population health management, clinical business management and quality improvement. She has a strong cross-cultural work history, with consulting experience developed internationally within the United States, Sub-Saharan Africa, Europe and the Asia Pacific. She is a member of the Consortium of Universities for Global Health (CUGH), the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the Institute for Health Care Improvement (IHI). She is also a trained labor and delivery birth doula with the Doula Organization of North America (DONA). Rachel holds a Master’s of Public Health from Boston University and a Bachelor of Arts in Anthropology from the University of New Hampshire.

Tazeem Bhatia, MPhil., MD., MRCGP,is a Public Health and Primary Care physician with twenty years’ experience of medical and public health practice in England, Myanmar, Afghanistan, Tajikistan and India. She has extensive expertise in the public health approach and tackling the wider determinants of health; community engagement and primary care; Universal Health Coverage (UHC) and models of social protection; and communicable and non-communicable disease (NCD) systems in high and low-income settings. She has conducted national level service and impact evaluations and sector wide health needs assessments, influencing senior leaders at a strategic level. She has worked in diverse environments and resource settings, from NGOs and think tanks, to Local Government, the NHS and UK Civil Service. Tazeem currently leads Public Health England’s global engagement on non-communicable disease with a focus on obesity. This includes advocating through evidence generation for action on the upstream social determinants of health.

Jesse B. Bump, M.P.H, Ph.D., is Executive Director of the Takemi Program in International Health and Lecturer on Global Health Policy in the Department of Global Health and Population at the Harvard T. H. Chan School of Public Health. He leads the global health field of study in the Master of Public Health degree and teaches on the political economy of global health. His research focuses on the intellectual ecology of global health, examining the historical, political and economic forces that are among the most fundamental determinants of ill health, and the most significant contextual factors that shape institutions and the approaches they embrace. This work addresses major themes in global health history and in the political economy of global health to analyze these macro forces and develop strategies for navigating solutions within them.

Kalipso Chalkidou, MD., Ph.D.,is the Director of Global Health Policy and a Senior Fellow at the Center for Global Development, based in London and a Professor of Practice in Global Health at Imperial College London. Her work concentrates on helping governments build technical and institutional capacity for using evidence to inform health policy as they move towards Universal Healthcare Coverage. She is interested in how local information, local expertise and local institutions can drive scientific and legitimate healthcare resource allocation decisions. She has been involved in the Chinese rural health reforms and in national health reform projects in Colombia, Turkey and the Middle East, working with the World Bank, the Pan American Health Organization (PAHO), the Department for International Development (DFID) and the Inter-American Development Bank (IDB), as well as national governments. Between 2007 and 2008, she spent a year at the Johns Hopkins School of Public Health, as a Harkness fellow in Health Policy and Practice, studying how comparative effectiveness research can inform policy and US government drug pricing policies.

Kalipso led the establishment of NICE International, which she ran for eight years, and, more recently, of the international Decision Support Initiative (iDSI) which she directs and which is a multi-million, multi-country network working towards better health around the world through evidence-informed spending in healthcare in low to middle income countries. IDSI is funded by the Bill and Melinda Gates Foundation, the UK’s Department for International Development and the Rockefeller Foundation and is currently involved in national reform projects in China, India, Vietnam, Ghana, Indonesia and South Africa working together with key organizations such as the Thai Health Intervention and Technology Assessment Program (HITAP), the US Center for Global Development and PRICELESS, at Wits University in South Africa.

Anthony J. Culyer, Ph.D.,is Emeritus Professor of Economics at the University of York (England), Senior Fellow at the Institute of Health Policy, Management and Evaluation at the University of Toronto (Canada) and Visiting Professor at Imperial College London. He is Chair of the Board of the international Decision Support Initiative (iDSI). He was the founding Organizer of the Health Economists’ Study Group. For thirty-three years he was the founding Co-Editor, with Joe Newhouse at Harvard, of Journal of Health Economics. He was founding Vice Chair of the National Institute for Health and Care Excellence (NICE) until 2003. He is Editor-in-Chief of the online Encyclopaedia of Health Economics. For many years he was chair of the Department of Economics & Related Studies at York and, for six of them, was also deputy vice-chancellor. He has published widely, mostly in health economics.

He is a Founding Fellow of the Academy of Medical Sciences, an Honorary Fellow of the Royal College of Physicians of London and an Honorary Member of the Finnish Society for Health Economics (2013). He holds an honorary doctorate from the Stockholm School of Economics and is a Commander of the British Empire (CBE). He has been a member or chaired many policy committees and boards in the UK and Canada including authoring the 1994 reforms of NHS Research and Development and being a director of the Canadian Agency for Drugs and Technologies in Health (CADTH).

David D. Kim, Ph.D., is an Assistant Professor of Medicine at Tufts University School of Medicine and a Program Director of the CEA Registry at the Center for the Evaluation of Value and Risk in Health (CEVR) at the Institute for Clinical Research and Health Policy Studies (ICRHPS) at Tufts Medical Center. As a health economist, he has been passionate about generating the best available economic evidence to inform health care decisions and public health policies through mathematical modeling. His primary research focuses on developing disease simulation models; improving methodology in economic evaluation and research prioritization; understanding health and economic consequences of health policies; and examining access to and utilization of cost-effective health interventions. He has developed several disease models for hepatitis C, alcohol use disorders, diabetes, cancer and cardiovascular diseases. Also, as a lead author of the worked example included in the Second Edition of Cost-Effectiveness in Health and Medicine, he conducted a cost-effectiveness analysis to reflect the comprehensive guidelines and recommendations. David received his doctorate in Health Economics at the University of Washington and his Master’s degree in Biostatistics from the University of Michigan.

Adam Elshaug, M.P.H., Ph.D., is a researcher specializing in the calculation of low-value care and a policy advisor on approaches to reducing waste to optimize value in health care. He is Professor of Health Policy and Co-Director of the Menzies Centre for Health Policy (MCHP) at The University of Sydney, Australia and is a Visiting Fellow with the USC-Brookings Schaeffer Initiative for Health Policy at The Brookings Institution in the USA. Professor Elshaug has numerous committee and Board appointments, including as a Ministerial appointee to the (Australian) Medicare Benefits Schedule (MBS) Review Taskforce. This is a five-year process to review Australia’s entire Medicare fee-for-service system utilizing Health Technology Assessment (HTA) principles and processes. Professor Elshaug was a 2010–2011 Commonwealth Fund Harkness Fellow based at the US Agency for Healthcare Research and Quality (AHRQ). From mid-2011 to mid-2013, he served as the National Health and Medical Research Council (NHMRC) Sidney Sax Fellow in Harvard Medical School’s Department of Health Care Policy. He is the recipient of numerous research awards and has published over 130 technical reports and peer review articles with first-author publications in journals such as The New England Journal of Medicine, BMJ and Journal of the American Medical Association. Professor Elshaug was Co-Lead of 2017 ‘Right Care’ Series of papers in The Lancet.

Wanrudee Isaranuwatchai, Ph.D.,is a Senior Researcher at the Health Intervention and Technology Assessment Program (a part of the Ministry of Public Health) in Bangkok, Thailand, a Director at the Centre for Excellence in Economic Analysis Research of St. Michael’s Hospital and a Senior Health Economist at the Canadian Centre for Applied Research in Cancer Control in Canada. She is also an Assistant Professor at the Institute of Health Policy, Management and Evaluation, University of Toronto. Her research focuses on how to apply economic evaluation in the real world setting as well as how to advance methods in economic evaluation. She has experience conducting economic evaluations using person-level data and decision modelling. She has collaborated with researchers and decision-makers in various areas to help communicate the value of health initiatives using economic evidence. Dr. Isaranuwatchai is dedicated to promoting the use of evidence in healthcare decision making.

Melitta Jakab, M.Sc., Ph.D., is a senior health economist at the WHO Barcelona Office for Health Systems Strengthening. She has twenty years of experience in health system strengthening, health financing, policy analysis and education in global health. Her work includes advising WHO Member States on health financing policy design and implementation, in particular in Moldova, Kazakhstan, Kyrgyzstan, Tajikistan, Turkey, Ukraine and Uzbekistan. She has been leading a multidisciplinary work program on the Health System Response to NCDs. She has been co-director of the Barcelona Courses on Health Systems Strengthening and Health Financing. She is co-editor of Health Systems Respond to NCDs: Time for Ambition (Jakab, Farrington, Borgermans, Mantingh, WHO Regional Office for Europe 2018) and of Implementing Health Financing Reform: Lessons from Countries in Transition (Kutzin, Cashin and Jakab, European Observatory, 2010). She has a PhD from Harvard University and M.Sc. in Health Policy for the Harvard School of Public Health.

Sumithra Krishnamurthy, M.P.H.,has a particular interest in the political and social implications of NCDs for vulnerable populations, with an emphasis on access to services. Sumithra holds an M.P.H. in Global Health from the Harvard T. H. Chan School of Public Health and received her Bachelor’s degree in International Development from the University of Sussex in the UK. Her current research focuses on the political economy of NCDs from a global perspective. Sumithra has previously served at the United Nations Entity for Gender Equality and the Empowerment of Women (UN Women) as well as various civil society organizations in support of human rights and health in the UK, Mexico and Rwanda. She has also supported national health systems strengthening projects through consultancies in Mexico and Burkina Faso.

Alia Luz, M.Sc.,works as a Project Associate with the management and research team of the international unit at Health Intervention and Technology Assessment Program (HITAP). She provides technical support in international and local economic evaluation projects, as well as administrative coordination for the organization’s regional and country projects. Her portfolio of work includes management of HITAP projects in the Philippines, as well as the Guide to Economic Analysis and Research (GEAR) online resource. In 2018, she received her Masters of Science (M.Sc.) in Health Policy, Planning and Financing (HPPF) from both the London School of Hygiene and Tropical Medicine (LSHTM) and the London School of Economics (LSE). Alia graduated from Bryn Mawr College in 2013 with a degree in economics. Post-graduation, she worked in Liberia on renewable energy economics for a project funded by the United States Agency for International Development (USAID).

Ryota Nakamura, M.A., Ph.D., is an Associate Professor based in the Hitotsubashi Institute for Advanced Study (HIAS), Hitotsubashi University. He also serves as a Visiting Associate Professor at the Institute of Statistical Mathematics. He is an applied microeconomist specializing in health. He holds a B.A. and an M.A. in Economics from Kyoto University and a Ph.D. in Economics from the University of York in the UK. Prior to joining Hitotsubashi University in 2016, he held positions at the University of East Anglia and the University of York. His research interests include empirical and theoretical investigations of health-related behavior, as well as of healthcare systems to inform national and international public health policies, using a wide range of research methods including micro-econometric analysis of observational data e.g., impact evaluation), economic experiment, modelling and evidence synthesis.

Peter J. Neumann, Sc.D., is Director of the Center for the Evaluation of Value and Risk in Health (CEVR) at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center and Professor of Medicine at Tufts University School of Medicine. He is the Founder and Director of the Cost-Effectiveness Analysis Registry. Dr. Neumann has written widely on clinical and economic evidence and on regulatory and reimbursement issues. He served as co-chair of the 2nd Panel on Cost-Effectiveness in Health and Medicine. He is the author or co-author of over 250 papers in medical literature, the author of Using Cost-Effectiveness Analysis to Improve Health Care (Oxford University Press, 2005) and co-editor of Cost-Effectiveness in Health and Medicine, 2nd Edition (Oxford University Press, 2016). Dr. Neumann has served as President of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). He is a member of the editorial advisory board of Health Affairs and the health policy advisory board for the Congressional Budget Office. He has held several policy positions in Washington, including Special Assistant to the Administrator at the Health Care Financing Administration. He received his doctorate in health policy and management from Harvard University.

Waranya Rattanavipapong, M.Sc., joined Health Intervention and Technology Assessment Program (HITAP) in February 2010. She gained her Master’s degree in Health Economics and Decision Modelling from the University of Sheffield in 2014. She has strong expertise in health economic evaluations and has been involved in several research projects to support the Thai government as well as the public agencies in Bhutan, Indonesia, India and Vietnam.

Manushi Sharma, M.B.A.,is an International Cooperation Officer at the Health Intervention and Technology Assessment Program (HITAP). She is a pharmacist by training with a Master’s in Business Management (M.B.A.). Previously, she worked with the Public Health Foundation of India with the health-economics and financing group. In the past, as a part of HITAP international unit (HIU), she has managed the iDSI Indonesia workstream. Currently, she is leading the monitoring and evaluation for all projects under HIU along with networking activities.

Arisa Shichijo, M.P.P., joined the project as a chapter team member. She received a B.A. in Law and Political science from Kyoto University and she is a second-year Master’s student at the School of International and Public Policy Hitotsubashi University. Her main field of research is health economics with a special focus on the process of policymaking and implementations in health-related areas and empirical analysis to inform public health interventions. She also completed a one-year exchange at McGill University, with a focus on Health Policy. She is now a Research Assistant at the Hitotsubashi Institute for Advanced Study (HIAS).

Peter C. Smith, Ph.D., is Emeritus Professor of Health Policy at Imperial College London and Honorary Professor of Health Economics, University of York. He is a mathematics graduate from the University of Oxford, with previous appointments at the University of Cambridge and the University of York, where he was Director of the Centre for Health Economics. His main research interest is in the economics of health, and his recent work has focused mainly on the financing and efficiency of health systems in low- and middle-income countries. Peter has published over 150 academic articles and twelve books, and has advised many governments and international agencies, including the World Health Organization, the International Monetary Fund, the Global Fund, the World Bank, the European Commission and the Organization for Economic Cooperation and Development.

Yot Teerawattananon, MD., Ph.D.,is the founding leader of the Health Intervention and Technology Assessment Program (HITAP), which is a semi-autonomous research institute of Thailand’s Ministry of Public Health. The works of HITAP have been used to inform policy decisions regarding the adoption of medicines, medical devices, health promotion and disease prevention programmes under the Universal Health Coverage Scheme and the national pharmaceutical reimbursement list, the National List of Essential Medicines. Recently, he joins the National University of Singapore as a visiting professor at Saw Swee Hock School of Public Health as well as is the Executive Board of the international Decision Support Initiative (iDSI). He has published more than 140 peer-reviewed journal articles and provided technical support on HTA capacity building in Asia and Africa. He is also one of the founders of HTAsiaLink, a regional network comprising governmental health technology assessment agencies in the Asia and Pacific region.

1. Introduction

Wanrudee Isaranuwatchai, Rachel A. Archer and Anthony J. Culyer

© W. Isaranuwatchai, R. A. Archer and A. J. Culyer, CC BY 4.0 https://doi.org/10.11647/OBP.0195.01

1.1 Non-Communicable Disease

Non-communicable diseases (NCDs) are the leading cause of death worldwide and contribute to over 73% of all deaths annually.1 Each day, NCDs cause more than 100,000 deaths; 80% of which occur in low- and middle-income countries (LMICs).2 Over the last 30 years, NCDs have replaced communicable diseases (CDs) as the cause of greatest health burden.3 This trend is evident in the risk factors for NCDs. According to the World Health Organization (WHO), obesity has tripled since 1975;4 while the International Diabetes Federation estimates that the global prevalence of diabetes, 8.8% in 2017, will increase by 48% by 2045, with an additional 204 million people living with diabetes.5 Much of this burden could be completely avoided because NCDs are largely preventable.6 Approximately 40% of all cancers and three-quarters of the incidence of heart disease, stroke and type 2 diabetes could be prevented by addressing tobacco use, unhealthy diet, physical inactivity and harmful use of alcohol. A great deal of technical knowledge exists about how to prevent and manage NCDs, such as the WHO Package of Essential NCD interventions (WHO PEN),7 the SHAKE8 (the technical package for salt reduction) and HEARTS9 (the technical package for cardiovascular disease management in primary health care) packages.

In addition to the NCD burden,10 there is an increasing demand on governments to address the health needs arising from NCDs through universal health coverage (UHC) policies, a direction that has been endorsed by the World Health Assembly11 and the United Nations General Assembly.12 NCDs are the result of various factors, for example genetic, physiological, and environmental and behavioral13 individually or in combination. They frequently require a collective response. They are not contagious, unlike communicable or infectious diseases, which can be spread, directly or indirectly, from one person to another.14 CDs, accidents and injuries also often need collective actions (such as mass vaccination or health and safety legislation) for effective treatment but can also be treated effectively on an individual basis. There are noticeable patterns in prevalence and mortality between CDs and NCDs. Figures 1.1 and 1.2 show the prevalence and mortality of CDs and NCDs in high-income countries (HICs), LMICs and around the globe.15 CDs are prevalent (~70%) in HICs compared to ~45% in LMICs (Fig. 1.1). NCDs are more prevalent in LMICs (~55%) compared to HICs (~30%). From the 37 years of data examined for Global Burden of Disease Study 2016, NCDs now dominate premature death.16 Over 80% of the world’s premature deaths are attributable to NCDs in LMICs.17 The probability of premature death from NCDs is almost four times higher in LMICs compared to HICs.18

Fig. 1.1 Prevalence of CDs and NCDs by World Bank country income categories.19

Fig. 1.2 Deaths from CDs and NCDs by World Bank country income categories.20

NCDs represent a significant burden through both an epidemiological and an economic lens.21 They affect everyone regardless of sex and age.22 The four main NCDs are cardiovascular disease, chronic respiratory disease, cancer and diabetes, which account for over 80% of NCDs deaths.23 A macroeconomic simulation model suggested a cumulative loss of USD $47 trillion over the next 2 decades due to NCDs.24 The Disease Control Priorities 3rd edition (DCP3) estimated that the number of deaths averted through prevention in LMICs could be between 2 to 4.2 million by 2030.25 Additionally, mental health problems are the leading cause of disability around the world.26 For example, approximately 800,000 people commit suicide every year and about 75% of those occur in LMICs.27 Mental health problems represent risk factors for other diseases such as cardiovascular diseases and diabetes including unintentional and intentional injury. There is significant inequity in the support (e.g., health services) for mental health around the world.

In an ideal world, it would be easy to prioritize interventions and allocate resources to have the maximum impact on health and its fair distribution, while simultaneously minimizing the risk to families of serious financial hardship from out-of-pocket payments. These are generally seen as the main concerns of cost-effectiveness analysis.Systems are, however, faced with a diversity of investment options, inescapable limits on resources, evidence that is at best sporadic, many other political, financial and social constraints, and a host of other additional28 considerations; all of which make identifying good value-for-money interventions challenging. The question naturally arises: why have we, the global community, not been more successful at reducing this NCD burden? Does a universal problem not have a universal solution? Is resource scarcity the fundamental culprit? Is cost-effectiveness really the answer? Are there better ways of using the resources that countries already have? Do countries have the essential human capital required to develop and roll out the right policies? Are there higher priorities for public spending against which the NCDs simply cannot compete? We try to answer these questions and make some suggestions for future actions in this book.

1.2 Best, Wasted and Contestable Buys

One response by the WHO to the NCD crisis was the idea of ‘Best Buys’. WHO defines Best Buys almost solely by their cost-effectiveness, that is, interventions which achieve best value for money in comparison to all comparators.29 More precisely, Best Buys for LMICs are interventions with an incremental cost-effectiveness ratio (ICER) under 100 International United States dollars per disability-adjusted life-year (DALY) averted.30

Some Wasted Buys are easy to define: they are interventions that have no beneficial effect. Others are slightly harder to specify, for they are interventions that do have a beneficial effect (with NCDs, the effect almost always lies in the more distant future) but ones that require too great a sacrifice of resources. That is, those resources would have a more beneficial effect used elsewhere on other health interventions or elsewhere in the economy. In other words, their opportunity cost is too high.

We suggest the addition of the category ‘Contestable Buys’ when there are suggestions that an intervention and its associated attributes may be a Best Buy but there is no direct evidence of cost-effectiveness in the local setting in which the intervention might be implemented. Thus, interventions in the WHO’s Best Buys list may be better classed as Contestable Buys if there is no demonstrative evidence of cost-effectiveness for the particular setting in question. The main distinction between Best and Contestable Buys is thus the availability of context-specific evidence.

1.3 Definitions and Central Ideas

Box 1.1 contains the definitions of central ideas that are used throughout the book. The reader will find these definitions to vary slightly from the many that lie elsewhere in the literature, though we are confident that any differences are minor and more questions of emphasis than of substance. We — all the authors here represented — have sought to be consistent in the way we have used these terms.

We refer frequently to ‘interventions’. This word is intended as an all-embracing term to capture a package of care over a relevant time period as applied to a particular condition or combination of morbidities. It may be restorative, maintenance or preventive. It may be provided in part from a health service program and partly from another like childcare or primary education. This is especially significant in the field of NCDs, with which this book is concerned. An intervention should not be seen as merely the purchase and use of a medicine, or any other single input, but rather as the planned or usual combination of human and physical resources required for the delivery of a service at a chosen standard. Some of these inputs may not be what we customarily think of as ‘healthcare’.

A critical starting point in determining the value of an intervention is its cost-effectiveness — if an analysis establishes empirical evidence of cost-effectiveness in the context of the location in which it is intended to be used, the intervention will be categorized as a ‘Best Buy’. If it establishes empirical evidence of its cost inefficiency, it will essentially be categorized as a ‘Wasted Buy’ or if it confers very little effectiveness, that is, the costs are not proportional to effectiveness, then it will again be categorized as a Wasted Buy.

Box 1.1 Definitions of Basic Terminology Used in Economic Evaluation

Economic evaluation is the comparative analysis of two or more alternatives in terms of their costs and outcomes. There are different types of economic evaluation, namely cost-benefit analysis, cost-minimization analysis, cost-utility analysis and cost-effectiveness analysis. They differ primarily in the measurement of consequences or outcome; however, each approach entails value judgements that should be explicitly considered in terms of their appropriateness in the decision context.

Cost-effectiveness analysis (CEA) is a form of economic evaluation that uses monetary units to measure/value costs and (usually) a single effect of interest that is common to the alternatives in consideration. The effect is measured in terms of clinical natural units (e.g., life-years gained). Often, cost-effectiveness analyses are interchangeable with cost-utility analysis that uses generic outcome measures such as the quality-adjusted life-year (QALY) or disability-adjusted life- year (DALY) instead of clinical natural effects. The advantage of the cost-utility analysis approach over cost-effectiveness analysis is that the former allows comparison of value in health investment between different health problems such as diabetes and mental health.

Disability-adjusted life-year (DALY) is a measure of overall disease burden, expressed as the total number of years of life lost due to ill-health, disability, or premature death. One DALY is equal to one year of healthy life lost.

Quality-adjusted life-year (QALY) is a measure of the state of health of a person or group, which is a function of the length and quality of life. One QALY is equal to one year of life in perfect health.

Incremental cost-effectiveness ratio (ICER) is calculated as the difference in cost between two possible interventions, divided by the difference in their outcomes. It is a standard measure representing marginal cost per marginal benefit from health investment.

Health Technology Assessment (HTA) is a form of evaluation that includes CEA but goes beyond its categories by including non-financial constraints and local environmental, organizational, social and political factors that may affect the costliness, effectiveness and feasibility of interventions.

Sources: adapted from Drummond et al., 2015 and Briggs et al., 2006.31

1.4 The Cost-Effectiveness Plane

Fig. 1.3 Cost-effectiveness plane.

These ideas are illustrated by what is called a Cost-Effectiveness Plane. In Fig. 1.3, the health effects of an intervention are measured in terms of lives saved, QALYs, DALYs averted, or other suitable indicators on the horizontal axis (positive effects to the right and negative ones to the left). Its relative costliness is shown by the vertical axis. A comparator intervention can be understood to be at the origin where the two axes cross, so the health gain and the cost are both relative to a comparator. The distances along the axes measure the difference between the intervention under investigation and the comparator. The slope of the dashed line labelled ‘threshold’ indicates the willingness of the payer (usually an insurer or the government) to pay for additional health (ΔC/ΔE): the ICER. This threshold is also known as the cost-effectiveness threshold or willingness-to-pay (WTP) threshold and is expected to be different for each country setting.32 That being so, what is regarded as cost-effective will also differ according to country.

The area lying in quadrant D clearly identifies a Best Buy — the intervention is both more effective and less costly than a relevant comparator. The area lying in quadrant A is clearly a Wasted Buy. D and A are areas in which an intervention dominates or is dominated by the comparator in terms of cost-effectiveness and its position relative to the threshold. This analysis is a development of the approach taken by the Institute of Medicine’s (IOM) famous book Crossing the Quality Chasm.33 In discussing efficiency as one of the six specific aims for improvement in health care, the book asserts that ‘the opposite of efficiency is waste, the use of resources without benefit to the patients a system is intended to help’.34

What of areas B and C? An intervention falling in B could be either a Best Buy or a Wasted Buy. In quadrant B, the intervention is more expensive but it is also more effective, so the question become whether the additional effectiveness is ‘worth’ the additional expense. Here, the dashed line comes into play because cost-effectiveness will depend on the maximum amount the payer is willing to spend for additional health outcomes. When the intervention is located above the dashed line, the additional or incremental cost (ΔC) exceeds the payer’s willingness to pay for the additional or incremental health (ΔE), and the intervention will be judged to be cost-ineffective by the payer and therefore a Wasted Buy. Conversely, an intervention falling below the threshold line will be deemed cost-effective and therefore a Best Buy.

Quadrant C brings up some counter-intuitive possibilities. In this quadrant, the intervention is definitely less effective than the comparator. However, it is also less costly. Again, whether it would be a Wasted Buy depends on whether the cost savings of using it sufficiently compensate for using this intervention rather than its more effective comparator. How can this be? Only if the cost savings, if realized, can be used to generate more health elsewhere. In quadrant B, the threshold line indicates the maximum willingness of the payer to pay for additional units of health. In quadrant C, the line indicates the minimum the payer is willing to accept to forgo a marginal health benefit. If the threshold genuinely indicates the payer’s judgment of value, then an intervention located below the dashed line will indicate a larger cost saving than the minimum indicated as acceptable by the dashed line. Paradoxically, then, a less effective intervention need not be a Wasted Buy — as long as it is also sufficiently cheaper than the comparator it will replace. It may even be a Best Buy!

Various techniques have been used to define thresholds. The three most popular methods35 are:

deriving the threshold from previous decisions or other jurisdictions, the willingness to pay of the payer (‘demand-side method’), orvalue of displaced services (‘supply-side method’).

The latter two have gained popularity and are the most cited approaches. However, each has limitations. The demand-side approach requires the society’s or the government’s willingness to pay (WTP) for healthcare to determine the threshold that would guide expenditures from the healthcare budget accordingly. In most cases, society’s willingness to pay will be set explicitly or implicitly by the government. The chosen threshold will inevitably be controversial, so the methods used to determine it should be well-founded, clear and transparent. Where experts are consulted, they should be of appropriate distinction and independence. Calculating an aggregate social willingness to pay by asking citizens is also fraught with difficulties and can be controversial. The WHO previously adopted the approach of the first bullet in the list above. It generated a global threshold ratio taking the form that interventions costing less than three times the average per capita income per disability-adjusted life-years (DALY) averted were considered to be cost-effective and those exceeding this level were considered to be cost-ineffective.36 Subsequently, there was an updated suggestion that the threshold could be between one to three Gross Domestic Product (GDP) per capita.37 This approach in both cases implicitly assumed that there is fixed relationship between GDP and the appropriate magnitude of expenditure on healthcare, despite this being a policy decision that can legitimately vary depending on local priorities. Context and additional considerations38 are matters that should be considered prior to the implementation of any threshold. A global threshold for all countries is an average (which may be generally too high or too low) but will rarely exactly fit the conditions in any particular country and may lead countries into committing themselves to merely Contestable Buys or, worse, to Wasted Buys.39

The threshold, though potentially useful, is not itself a decision rule. It is only a guide. There may be circumstances under which a country may rationally choose to admit interventions that have ICERs above the threshold or reject some that lie below it. For analyses that try to take account of factors other than cost-effectiveness in deciding whether an intervention is a Best Buy, one may turn to Health Technology Assessment (HTA).

Cost-saving and cost-effectiveness are not synonyms. Depending on the context, it is possible, especially in a highly resource-constrained setting, that a less expensive and slightly less effective strategy is preferable, and vice-versa; interventions that are expensive may be cost-effective if they result in significant health outcomes and the cost-effectiveness threshold is sufficiently high.

These, then, are the basic ideas around which this book is built. What initially seems clear, and even obvious, turns out to be complex, controversial and may require the tools of Health Technology Appraisal (HTA) rather than those of CEA alone.

1.5 The Story of This Book

The Prince Mahidol Award Conference (PMAC) was first convened in 2007 and has continued annually since. This global health forum honors the memory of Prince Mahidol of Songkla, who dedicated his life’s work to advancing public health and medical practice in Thailand and is respectfully regarded as the Father of Modern Medicine and Public Health of Thailand. Further information about PMAC is available via this link: https://pmaconference.mahidol.ac.th/site.