New Health Technologies Managing Access, Value and Sustainability (eBook)

Foreword 5
Acknowledgements 7
Table of contents 9
Acronyms and abbreviations 13
Executive summary 15
Steer investments in biomedical research and development (R& D) and prepare for upcoming technologies in the health sector
Adapt policies to regulate market entry of new technologies 16
Use health technology assessment, coverage and pricing policies to encourage value-for-money 16
Harness the potential of health data while managing risks appropriately 17
Chapter 1.New health technologies: Managing access, value and sustainability 19
Introduction 20
Box 1.1. Health technology – a basic taxonomy 20
Figure 1.1. Health technology – a basic taxonomy 20
1. Impact of health technologies on health and health spending: Lessons from the past 21
2. Promises and challenges of new and emerging technologies 23
2.1. New types of technologies challenge regulatory pathways 23
Box 1.2. Precision medicine: some definitions 24
2.2. The proliferation of high-cost medicines questions current pricing models 26
Box 1.3. What is wrong with new treatments for hepatitis C? 28
2.3. Health care systems struggle to “pay for value” 28
3. Appropriate diffusion and funding of value-adding technologies 30
3.1. Co-operative horizon scanning can be used to better prepare for new technologies 30
Box 1.4. Why are we not getting the technology we need? The case of AMR and dementia 30
3.2. Quick access to promising technologies for unmet needs can be provided while still protecting patients 32
3.3. A lifecycle approach for Health Technology Assessment can be adopted to inform coverage and funding decisions 35
Figure 1.2. Lifecycle framework for successful integration of health technologies in health care systems 37
3.4. Solutions are needed to manage access to and budget for high-cost medicines 37
3.5. Information infrastructure and governance can be constructed to realise health technology potential 38
Box 1.5. The electronic health record 40
Figure 1.3. OECD health data governance framework 41
Conclusion 41
Notes 42
References 43
Chapter 2.The past and potential future impact of new health technology 45
Introduction 46
Box 2.1. Value in health care 46
1. The past impact of technology on health, expenditure and value 48
1.1. Medical technology has had a profound impact on human health and welfare 49
1.2. Health technology contributed to health care expenditure growth 52
Figure 2.1. Per cent of GDP spent on health care in selected countries, 1970-2015 52
Figure 2.2. Technology and the drivers of health care expenditure growth 55
1.3. The value of medical technology has declined over time 55
Figure 2.3. Longitudinal trends in the costs per year of life gained in four age groups in the United States 56
Figure 2.4. Cost per life year gained for the 45-year-old cohort in Cutler et al. (2006) using undiscounted and discounted future life years 57
Figure 2.5. Changes in survival of AMI patients and in Medicare expenditure by US hospital referral region, 1986-2002 58
1.4. Health technology’s value can be enhanced with better policy and practice 60
Table 2.1. The value framework for health technology 60
Figure 2.6. Theoretical value functions of technology types A, B and C 62
2. Challenges and opportunities of accelerating technology development 62
Box 2.2. Identifying future technologies with potentially high impact on health care systems 63
2.1. Precision medicine enables treatments tailored to individual patients 63
2.2. Combination products increasingly blur the line between drug and device technology 64
2.3. Mobile health is developing rapidly but has yet to attain mainstream adoption 65
Figure 2.7. mHealth’s potential uses 66
2.4. Wearable devices and sensors with digital tools may complement traditional diagnostics 67
2.5. Additive manufacturing to permit “3D printing” of devices and potentially of transplant organs 68
Box 2.3. Analysing potential impacts of 3D bioprinting on health care delivery 69
3. Preparation for and promotion of high-value technology in health care systems 70
3.1. Existing early awareness and alert programmes vary in objectives, authority and methods 70
Box 2.4. Examples of early awareness and alert activity across OECD countries 70
Box 2.5. Technology foresight study use by OECD health care systems 72
3.2. Limited evaluation of horizon scanning in health care systems demonstrates areas for improvement 73
3.3. Reinforced international co-operation could increase capacity, efficiency and impact 74
Conclusion 75
Notes 76
References 77
Chapter 3.Innovation, access and value in pharmaceuticals 83
Introduction 84
1. Current trends in pharmaceutical markets 84
1.1. The pharmaceutical industry pipeline is rich but some unmet medical needs require further investment 84
Box 3.1. Recommendations of the United Kingdom AMR Review to encourage innovation and development of new technologies 86
1.2. Many new medicines approved are therapies targeting small populations 88
Figure 3.1. Number of new active substances approved by six regulatory authorities, approval years 2006-15 88
Figure 3.2. Number of orphan drug designations/approvals in the United States and the European Union, 2000-15 89
Figure 3.3. Original FDA approval for oncology, stratified by personalised medicine status, 2006-15 90
Figure 3.4. Original FDA approval for oncology, stratified by orphan status, 2006-15 91
1.3. Pharmaceutical spending is increasingly skewed towards expensive medicines 92
Figure 3.5. Median monthly costs of cancer drugs at FDA approval in the United States, 1965-2015 92
Box 3.2. Cross-country variations in list prices of expensive medicines 93
Figure 3.6. Price per life year gained versus FDA approval date for oncology products, 1995-2013 94
2. Recent policy initiatives to provide faster access to pharmaceutical treatments 96
2.1. Regulators are under pressure to speed up the regulatory process 96
Figure 3.7. New Active Substance median approval time for six regulatory authorities, 2006-15 97
2.2. Payers struggle to assess benefits and pay for value 99
Table 3.1. Use of HTA to make coverage and pricing decisions for pharmaceuticals in OECD countries 100
Table 3.2. Transparent value framework proposed for orphan drugs in European countries 102
2.3. Managed entry agreements are used to address uncertainty on effectiveness or contain costs 102
3. Exploring new policy options to ensure sustainable access to innovation 105
3.1. In some market segments, negotiation powers of purchasers and sellers need to be rebalanced 105
3.2. A re-assessment of orphan legislation would be welcome 106
3.3. Imagining new ways to steer and finance pharmaceutical R& D
Box 3.3. Future scenarios about drug development and drug pricing 107
Conclusion 108
Notes 109
References 110
Annex 3.A1.OECD country policies to boost innovation for orphan diseases 114
Table 3.A1.1. Policies to encourage development of orphan drugs in OECD countries 114
Annex 3.A2.Coverage and funding of medicines in OECD countries 115
Chapter 4.Ensuring timely and affordable access to medical devices 119
Introduction 120
Box 4.1. Definition of medical device 120
1. Regulating medical devices 122
1.1. Safety, performance and effectiveness are key concepts for medical device regulation 122
Table 4.1. Risk categories and evidentiary requirements for medical devices in the United States and Europe 123
1.2. Managing uncertainty may require viewing regulation as a cycle 125
Figure 4.1. Illustration of the regulatory cycle 126
1.3. Health care systems should explore opportunities for streamlining the regulatory cycle 127
1.4. mHealth presents an emerging challenge to regulators 128
2. Coverage and funding of medical devices 131
2.1. HTA often informs coverage decisions 132
Table 4.2. Countries using HTA to make coverage decisions or to set reimbursement level or price for new medical devices 132
Box 4.2. MaRS EXCITE Program 134
Box 4.3. Examples of Coverage with Evidence Development schemes in OECD countries 135
2.2. Funding mechanisms are crucial to encourage appropriate use of medical devices 137
Table 4.3. Paying for medical devices covered in health care systems 138
Table 4.4. Frequency of updates, time lags and number of groupings for hospital care payment systems in selected countries 139
Table 4.5. Examples of additional payments for new technologies 140
Figure 4.2. Technology diffusion and reinvention in the US context 143
2.3. Coverage and funding of mHealth is an emerging challenge 145
Box 4.4. Telehealth 146
3. Institutional requirements for effective regulation, coverage and funding of medical devices 147
3.1. Information infrastructure provides a necessary platform 147
3.2. Governance provides context for approval and funding decisions 150
Conclusion 152
Figure 4.3. A regulatory and funding framework for medical devices and their use 153
Notes 154
References 155
Chapter 5.Achieving the promise of precision medicine 161
Introduction 162
Box 5.1. OECD country initiatives in precision medicine 162
1. Precision medicine in today’s practice and associated challenges 164
Box 5.2. Biomarkers, diagnostic and genomic tests 164
1.1. Companies have incentives to develop personalised medicines 165
1.2. Regulatory challenges must be overcome 166
1.3. Clinical and economic assessment of biomarker tests and personalised medicines raises a number of issues 168
Box 5.3. The cost-effectiveness of testing for epidermal growth factor receptor mutations in patients with non-small-cell lung cancer 169
1.4. Personalised medicine is sometimes cost-effective 170
Box 5.4. OECD case studies on precision medicines 171
1.5. Coverage and funding of personalised and precision medicines are often complex 172
Table 5.1. Funding/reimbursement of diagnostic tests in selected OECD countries 172
2. Emerging trends in precision medicine 172
2.1. Next-generation sequencing methods are increasingly used 173
Box 5.5. Recommendations of the Canadian College of Medical Geneticists regarding the use of genome-wide sequencing of germline DNA in the context of diagnosis of monogenic diseases 173
Figure 5.1. Recent and projected number of oncology patients diagnosed using molecular testing in France, 2014-20 174
2.2. The move is towards more efficient research and quicker integration in clinical practice 176
Box 5.6. Examples of initiatives to promote data sharing 177
Conclusion 179
Notes 180
References 182
Annex 5.A1.Sample of products selected for the 2015 OECD case study 185
Table 5.A1.1. List of medicines selected for case studies 186
Chapter 6.Digital technology: Making better use of health data 187
Introduction 188
1. Promise and opportunities for health data 189
Box 6.1. Big Data 189
Box 6.2. Examples of applying health data analytics to improve outcomes 190
1.1. Primary and secondary data use can improve patient care 192
1.2. Data can be used for better health care system management 193
1.3. Health data contribute to surveillance efforts and population health issues 194
1.4. Technological advances in data collection and analysis enable research 195
2. Challenges, risks and policy implications of using health data 196
2.1. Data interoperability and linkage must be established for maximum value 197
Figure 6.1. Extent of linkage across relevant databases in 22 OECD countries, 2013/14 198
2.2. Privacy and security are sensitive issues 199
Figure 6.2. Risks associated with the collection and use of personal health data 199
2.3. Legal and legislative challenges need to be resolved 200
2.4. Infrastructure and human capital must be reinforced to keep pace with technological advances 201
2.5. How well stakeholders are engaged can determine success 202
2.6. Costs and resourcing for digital health technology must be carefully evaluated 203
2.7. A data governance framework would promote realisation of health data benefits 204
Figure 6.3. Health data governance framework 205
3. EHR systems’ readiness to contribute to secondary uses of health data 205
Figure 6.4. Multiple uses of data within clinical electronic health record systems 206
3.1. EHR data governance readiness varies greatly among countries surveyed 207
Figure 6.5. Data governance readiness among OECD member and partner countries surveyed, 2016 207
3.2. Technical and operational factors supporting secondary uses of EHR data reveal key differences 210
Figure 6.6. Technical and operational readiness in OECD member and partner countries surveyed, 2016 211
3.3. Many countries are investing in EHRs to strengthen national health and health care monitoring and research 214
3.4. Countries’ outlooks for the future reflect their position in the current continuum of health data use readiness 215
Figure 6.7. Data governance and technical/operational readiness to develop national information from EHRs in countries surveyed, 2016 216
Conclusion 216
Notes 217
References 217
Annex 6.A1.Risk-benefit evaluation tool for decision making about the processing of personal health data 221
Table 6.A1.1. Risk-benefit evaluation tool for decision making about the processing of personal health data 221
Annex 6.A2.Key results from the 2016 HCQI study of electronic health record system development and data use 223
Table 6.A2.1. Data governance readiness to generate health information from EHRs 224
Table 6.A2.2. Technical and operational readiness to generate health information from EHRs 225
Table 6.A2.3. Projects where data from EHR systems are used to regularly monitor and report on health care quality at the health care system level 227
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