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INTRODUCTION TO
MARKET ACCESS FOR
PHARMACEUTICALS
INTRODUCTION TO
MARKET ACCESS FOR
PHARMACEUTICALS
Mondher Toumi
Boca Raton London New York
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Taylor & Francis Group, an informa business
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Contents
Foreword ....................................................................................................................................................................................xix
Acknowledgments ......................................................................................................................................................................xxi
Author ......................................................................................................................................................................................xxiii
Common Abbreviations ............................................................................................................................................................ xxv
Introduction .............................................................................................................................................................................xxvii
Chapter 1 Health as a Good ..................................................................................................................................................... 1

1.1 Welfare Economics and Health .................................................................................................................... 1
1.2 Health Care: A Mixed and Collective Good ................................................................................................ 1
1.3 Equity, Health, and Health Care................................................................................................................... 2
1.4 Uncertainty Related to the Demand and Results ......................................................................................... 2
1.5 Physician’s Expected Behavior ..................................................................................................................... 2
1.6 Supply Condition .......................................................................................................................................... 2
1.7 Discussion ..................................................................................................................................................... 3
References ............................................................................................................................................................... 3
Chapter 2 Decision-Making in Public Health.......................................................................................................................... 5

2.1 Public Health Definition ............................................................................................................................... 5
2.1.1 Example of the Influence of Air Pollution on Public Health .......................................................... 5
2.2 Decision-Making in Public Health ............................................................................................................... 5
2.2.1 Organization of Public Health......................................................................................................... 5
2.2.2 Funding Health Care and Public Health ......................................................................................... 5
2.2.3 Criteria Used for Priority-Setting and Decision-Making ................................................................ 6
2.2.4 Health Inequalities .......................................................................................................................... 7
2.2.5 Monitoring and Evaluation of Public Health Policies ..................................................................... 7
2.3 Decision-Making on the Reimbursement ..................................................................................................... 8
2.3.1 Accountability for Reasonableness ................................................................................................. 8
2.3.2 Transparency ................................................................................................................................... 9
2.3.3 Participation .................................................................................................................................... 9
2.4 Methods of Incorporating Societal Preferences into Decision-Making ....................................................... 9
References ............................................................................................................................................................. 10
Suggested Reading ................................................................................................................................................ 11
Chapter 3 Definitions and Concepts ...................................................................................................................................... 13

3.1 Origin of the Market Access Term ............................................................................................................. 13
3.1.1 Market Access for Goods .............................................................................................................. 13
3.1.1.1 Tariff Measures.............................................................................................................. 13
3.1.1.2 Nontariff Barriers/Measures ......................................................................................... 13
3.1.2 Application to Health Care............................................................................................................ 13
3.1.2.1 Tariff Barriers on Pharmaceuticals ............................................................................... 13
3.1.2.2 Nontariff Barriers on Pharmaceuticals.......................................................................... 14
3.1.2.3 Health Care Market Specificities ................................................................................... 14
3.2 Market Access Key Concepts ..................................................................................................................... 16
3.2.1 What Is Value? .............................................................................................................................. 16
3.2.2 What Is Access? ............................................................................................................................ 17
3.3 Market Access Definition ........................................................................................................................... 17
3.3.1 Market Access and the Structure of Health Care System ............................................................. 19
3.3.1.1 Publicly Funded Health Care Systems .......................................................................... 19
3.3.1.2 Mixed or Private Health Care Systems ......................................................................... 20
3.3.1.3 Centralized and Regional Market Access ..................................................................... 20
3.4 Cultural Specificities of Market Access.................................................................................................... 20
v
vi Contents
3.5 Market Access for Payers ......................................................................................................................... 21

3.5.1 Payers Employ Market Access Tools to Control Drug Expenditure ........................................... 21
3.5.2 How to Identify Payers?.............................................................................................................. 23
3.5.3 How Payers Assess Value? .......................................................................................................... 23
3.5.4 How HTA Evaluation Is Translated into P&R Conditions? ....................................................... 25
3.6 Market Access for Industry....................................................................................................................... 25
3.6.1 A New Paradigm ......................................................................................................................... 25
3.6.2 Organization in the Pharmaceutical Industry.............................................................................. 25
3.6.3 Objective of Market Access Activities ........................................................................................ 26
Chapter 4 HTA Decision Analysis Framework ...................................................................................................................... 27

4.1 Introduction .............................................................................................................................................. 27
4.2 History ...................................................................................................................................................... 27
4.3 HTA Processes and Decision Analysis Frameworks ................................................................................ 27
4.3.1 National HTA Bodies and Main Assessment Outcomes............................................................. 27
4.3.2 Decisions Impacted by the Assessed Outcomes ......................................................................... 29
4.3.3 HTA Core Model® EUnetHTA ................................................................................................... 29
4.3.4 Evaluation Criteria and Processes for HTA and Pricing ............................................................. 29
4.3.4.1 HTA ............................................................................................................................. 29
4.3.4.2 Pricing ......................................................................................................................... 30
4.3.4.3 Market Access Agreements ......................................................................................... 30
4.3.5 Value Assessment Frameworks ................................................................................................... 31
4.4 France ....................................................................................................................................................... 31
4.4.1 SMR ............................................................................................................................................ 31
4.4.2 ASMR ......................................................................................................................................... 31
4.4.3 Efficiency Notice ........................................................................................................................ 32
4.5 England, Ireland, and Wales—Health Technology Appraisal................................................................... 32
4.6 Scotland—New Product Assessment ........................................................................................................ 33
4.7 Germany—Additional Benefit and Cost-Benefit ...................................................................................... 33
4.8 Sweden—Marginal Benefit and Cost-Effectiveness ................................................................................. 33
4.9 Italy—Degree of Innovation and Cost-Effectiveness ............................................................................... 34
4.10 Spain—Reimbursement and Pricing Recommendation ........................................................................... 34
Chapter 5 Early HTA Advice ................................................................................................................................................. 37

5.1 Overview of the Early HTA Advice Pathways ......................................................................................... 37
5.1.1 HTA-EMA Parallel Scientific Advice ......................................................................................... 37
5.1.2 MultiHTA Advice ....................................................................................................................... 37
5.1.3 EUnetHTA Pilot Assessment of Relative Effectiveness ............................................................. 37
5.1.4 Adaptive Pathway ....................................................................................................................... 37
5.1.5 Priority Medicines Scheme ........................................................................................................ 37
5.2 National Early HTA Advice Programs ..................................................................................................... 37
5.2.1 France ......................................................................................................................................... 37
5.2.1.1 Questions to Focus on ................................................................................................. 42
5.2.1.2 Process......................................................................................................................... 42
5.2.1.3 Content of the Dossier................................................................................................. 42
5.2.2 The United Kingdom .................................................................................................................. 42
5.2.2.1 Questions to Focus on ................................................................................................. 42
5.2.2.2 Process......................................................................................................................... 43
5.2.2.3 Content of the Dossier................................................................................................. 43
5.2.2.4 Light Scientific Advice................................................................................................ 43
5.2.3 Germany...................................................................................................................................... 44
Contents vii
5.3 Strategic Considerations ............................................................................................................................. 44

5.3.1 Multidisciplinary Approach .......................................................................................................... 44
5.3.2 Briefing Book Is the Cornerstone of Early HTA Advice .............................................................. 44
5.3.3 How to Choose the Right Option for Early HTA Advice ............................................................. 44
5.3.4 Types of Risk for the Company When Considering Early HTA Advice ...................................... 45
5.3.4.1 Target Population Related Risk ..................................................................................... 45
5.3.4.2 Development Plan Related Risk .................................................................................... 45
5.4 Conclusions................................................................................................................................................. 46
References ............................................................................................................................................................. 46
Chapter 6 Overview of Market Access Agreements.............................................................................................................. 47

6.1 Background................................................................................................................................................. 47
6.2 Rationale behind MAAs............................................................................................................................. 47
6.3 Different Definitions and Taxonomies of MAAs ....................................................................................... 47
6.3.1 Different Definitions ..................................................................................................................... 47
6.3.2 A Possible Definition ..................................................................................................................... 49
6.3.3 Different Taxonomies .................................................................................................................... 49
6.3.4 Simplified Taxonomy .................................................................................................................... 49
6.3.4.1 Commercial Agreement................................................................................................. 51
6.3.4.2 Payment for Performance .............................................................................................. 51
6.3.4.3 Coverage with Evidence Development .......................................................................... 52
6.4 Payers’ and Manufacturers’ Motivations to Implement MAAs ................................................................. 53
6.4.1 The Increasingly Cost-Sensitive Environment .............................................................................. 53
6.4.2 The Uncertainty Related to Drug’s Performance .......................................................................... 53
6.4.3 The “Trust Crisis” ......................................................................................................................... 54
6.5 International Comparison of MAA Health Policies ................................................................................... 54
6.5.1 MAAs across Countries ................................................................................................................ 54
6.5.2 Some Countries Are More Resistant Than Others........................................................................ 56
6.5.3 Regional MAAs Growth in Europe .............................................................................................. 56
6.5.3.1 Formularies in Italy Are Subject to Regional Influence ................................................ 57
6.5.3.2 Regional MAA in Sweden ............................................................................................. 58
6.6 Best Practice of MAAs ............................................................................................................................... 59
6.6.1 The Rationale behind MAAs ........................................................................................................ 59
6.6.1.1 When Should MAAs Be Considered? ........................................................................... 59
6.6.2 The Implementation Process ......................................................................................................... 60
6.6.2.1 Requirements for Implementing MAAs ........................................................................ 60
6.6.2.2 Challenges in MAAs Implementation ........................................................................... 60
6.6.3 Evaluation ...................................................................................................................................... 61
6.7 Impact of MAAs on Product Uptake ......................................................................................................... 61
6.7.1 Example of MS MAA in the United Kingdom: Impact on Sales ................................................. 61
6.7.2 Example of Bevacizumab’s Uptake in Metastatic Colorectal Cancer across
EU Countries ................................................................................................................................. 62
6.7.3 Etanercept MAA in Germany ....................................................................................................... 63
6.8 Some Specific Case Studies........................................................................................................................ 63
6.8.1 Example of CED in the United Kingdom: Use of β-Interferons and Glatiramere for the
Treatment of MS in the United Kingdom ..................................................................................... 63
6.8.1.1 Performance of MAAs .................................................................................................. 64
6.8.2 Examples of CEDs in France ........................................................................................................ 64
6.8.3 Examples of CEDs in Sweden....................................................................................................... 65
6.8.4 Example of MAAs in Italy ............................................................................................................ 65
6.8.5 Financing of MAA Drugs ............................................................................................................. 66
viii Contents
6.9 Overview of MAA Trends in Other Countries ......................................................................................... 66

6.9.1 MAA Is a Growing Phenomenon in Various Countries ............................................................ 66
6.9.2 MAAs in Australia .................................................................................................................... 66
6.9.3 MAAs in Latin America ............................................................................................................ 67
6.9.4 India: A Different MA Pathway ................................................................................................ 67
6.9.5 MA in South Korea ................................................................................................................... 67
6.10 Perspectives .............................................................................................................................................. 68
6.10.1 MAA Is a Growing Trend and Is Shifting toward Conditional Access ..................................... 68
6.10.2 Challenges with MAAs ............................................................................................................. 68
6.11 Conclusion: MAA, a Temporary Solution? .............................................................................................. 68
6.11.1 A Paradigm Shift in the Pharmaceutical Industry ..................................................................... 69
6.11.2 From a Decision Point to a Decision Window .......................................................................... 69
6.11.3 Which MAAs in the Future? ..................................................................................................... 69
References ............................................................................................................................................................. 69
Chapter 7 External Reference Pricing.................................................................................................................................... 71

7.1 Definition of External Reference Pricing ................................................................................................. 71
7.2 ERP in Europe .......................................................................................................................................... 71
7.2.1 National Legal Framework ........................................................................................................ 71
7.2.2 Scope of ERP ............................................................................................................................ 72
7.2.3 Composition of the Country Basket .......................................................................................... 72
7.2.4 Price Calculation and Selection of Reference Products ............................................................ 72
7.3 ERP Processes in Non-European Countries ............................................................................................. 73
7.3.1 Australia .................................................................................................................................... 73
7.3.2 Canada ....................................................................................................................................... 73
7.3.3 Japan .......................................................................................................................................... 73
7.3.4 South Korea ............................................................................................................................... 73
7.3.5 Mexico ....................................................................................................................................... 74
7.3.6 New Zealand.............................................................................................................................. 74
7.3.7 Turkey........................................................................................................................................ 74
7.4 Concerns Related to ERP ......................................................................................................................... 74
7.4.1 Potential Consequences of ERP ................................................................................................ 74
7.4.1.1 Patient Access to Medicine ........................................................................................ 75
7.4.1.2 Affordability .............................................................................................................. 75
7.4.1.3 Industry Revenue and Sustainability ......................................................................... 75
7.5 VBP and ERP ........................................................................................................................................... 76
7.5.1 ERP as an Alternative to VBP .................................................................................................. 76
7.5.2 ERP and VBP Combination ...................................................................................................... 76
References ............................................................................................................................................................. 76
Chapter 8 Gap between Payers and Regulators ..................................................................................................................... 79

8.1 Introduction .............................................................................................................................................. 79
8.2 Uncertainty versus Risk ........................................................................................................................... 79
8.3 Payers versus Regulators .......................................................................................................................... 79
8.4 Sources of Uncertainty ............................................................................................................................. 80
8.4.1 Regulators ................................................................................................................................. 80
8.4.2 HTA Bodies/Payers ................................................................................................................... 80
8.5 Risk Management of Drug Value Uncertainty—HTA/Payer Level ........................................................ 80
8.5.1 Population.................................................................................................................................. 80
8.5.2 Comparator................................................................................................................................ 80
8.5.3 Design........................................................................................................................................ 81
8.5.4 Outcome .................................................................................................................................... 81
8.5.5 Indirect Comparison ................................................................................................................. 82
Contents ix
8.6 Risk Management Tools ........................................................................................................................... 82

8.6.1 Regulators .................................................................................................................................. 82
8.6.2 HTA Bodies and Payers............................................................................................................. 84
8.7 Type of Studies Requested by HTA Bodies/Payers to Reduce the Uncertainty....................................... 85
8.8 Case Studies of Gaps between Regulatory and HTA/Payer Approval ..................................................... 86
8.8.1 Drugs General Characteristics and Approval History ............................................................... 86
8.8.2 Regulatory versus Payers’ Evaluations ..................................................................................... 86
8.8.3 Discussion of Case Studies ....................................................................................................... 89
8.9 Conclusions .............................................................................................................................................. 90
References ............................................................................................................................................................. 90
Chapter 9 Early Access Programs ......................................................................................................................................... 91

9.1 Overview .................................................................................................................................................. 91
9.2 Types of EAPs: Nominative and Cohort .................................................................................................. 92
9.3 Global EAP Trends .................................................................................................................................. 92
9.4 Key Success Factors and EAP Management............................................................................................ 92
References ............................................................................................................................................................. 96
Chapter 10 Market Access of Orphan Drugs........................................................................................................................... 97

10.1 Definitions of Orphan Drugs ................................................................................................................... 97
10.1.1 US Definition............................................................................................................................. 97
10.1.2 EU Definition ............................................................................................................................ 97
10.1.3 Japan .......................................................................................................................................... 97
10.1.4 South Korea ............................................................................................................................... 97
10.2 The Legal Frameworks for Licensing and Assessment of Orphan Drugs and Development
Incentives ................................................................................................................................... 97
10.2.1 The European Union ................................................................................................................. 97
10.2.2 France ........................................................................................................................................ 98
10.2.2.1 Compassionate Use .................................................................................................. 98
10.2.2.2 Development Incentives ........................................................................................... 98
10.2.3 Germany .................................................................................................................................... 99
10.2.4 Spain .......................................................................................................................................... 99
10.2.5 Italy.......................................................................................................................................... 100
10.2.5.1 Compassionate Use ................................................................................................ 100
10.2.5.2 Development Incentives ......................................................................................... 100
10.2.6 The United Kingdom............................................................................................................... 100
10.2.6.1 Scotland.................................................................................................................. 100
10.2.6.2 England and Wales ................................................................................................. 100
10.2.7 Asia...........................................................................................................................................101
10.2.7.1 Japan........................................................................................................................101
10.2.7.2 South Korea.............................................................................................................101
10.3 The Pricing Process of Orphan Drugs ....................................................................................................101
10.3.1 France ...................................................................................................................................... 101
10.3.2 Germany .................................................................................................................................. 102
10.3.3 Italy.......................................................................................................................................... 102
10.3.4 Spain ........................................................................................................................................ 102
10.3.5 The United Kingdom............................................................................................................... 102
10.3.6 Japan ........................................................................................................................................ 102
10.3.7 South Korea ............................................................................................................................. 103
x Contents
10.4 Comparison of Prices of Orphan Drugs ............................................................................................... 103

10.5 The HTA Framework for Orphan Drugs and Ultra-Orphan Drugs ..................................................... 103
10.6 The Concept of Ethics and Equity for Orphan Drugs .......................................................................... 103
10.7 Potential Alternative Methods for HTA and Pricing of Orphan Drugs ............................................... 103
10.8 The Issues with Prices of Orphan Drugs ............................................................................................. 104
10.9 Future Perspectives............................................................................................................................... 104
10.10 Conclusion ............................................................................................................................................ 105
References ........................................................................................................................................................... 105
Chapter 11 Market Access of Vaccines in Developed Countries ........................................................................................... 107

11.1 Introduction .......................................................................................................................................... 107
11.1.1 Definition and Classifications .............................................................................................. 107
11.1.2 Preventive Vaccines.............................................................................................................. 107
11.1.3 Therapeutic Vaccines ........................................................................................................... 107
11.2 Vaccines’ Specifics ............................................................................................................................... 108
11.2.1 Development ........................................................................................................................ 108
11.2.2 Safety ................................................................................................................................... 108
11.2.3 Benefits and Cost-Effectiveness .......................................................................................... 108
11.2.3.1 Humanistic Benefits............................................................................................ 108
11.2.3.2 Economic Benefit ............................................................................................... 108
11.2.3.3 Cost-Effectiveness .............................................................................................. 109
11.2.4 Market Typology .................................................................................................................. 109
11.3 Overview of Vaccines’ Market Access in Developed Countries .......................................................... 109
11.3.1 Overview of NITAGs’ Processes ......................................................................................... 109
11.3.1.1 NITAGs’ Members ............................................................................................. 109
11.3.1.2 Operations........................................................................................................... 109
11.3.1.3 Decision Criteria ................................................................................................. 109
11.3.2 Implementation of NITAGs’ Recommendations ................................................................. 110
11.3.3 Time to Market .................................................................................................................... 110
11.4 Overview of Vaccines’ Market Access in a Selection of European Countries and the United States .... 110
11.4.1 Austria.................................................................................................................................. 110
11.4.2 Denmark .............................................................................................................................. 110
11.4.3 France .................................................................................................................................. 111
11.4.4 Germany............................................................................................................................... 111
11.4.5 Italy ...................................................................................................................................... 111
11.4.6 The Netherlands ................................................................................................................... 112
11.4.7 Spain .................................................................................................................................... 112
11.4.8 Sweden ................................................................................................................................. 112
11.4.9 The United Kingdom ........................................................................................................... 113
11.4.10 The United States................................................................................................................. 114
References ........................................................................................................................................................... 115
Chapter 12 France .................................................................................................................................................................. 117

12.1 Stakeholders ......................................................................................................................................... 117
12.1.1 Names of National Pricing and Reimbursement Decision Makers ..................................... 117
12.1.2 Names of National Health Technology Assessment Agencies ............................................ 117
12.1.3 Names of Other Key Stakeholders (Regional/Local Level) ................................................ 117
12.2 Pricing and Reimbursement Policies .................................................................................................... 117
12.2.1 Overview of the System ....................................................................................................... 117
12.2.2 Reimbursement Process ....................................................................................................... 118
12.2.3 Pricing Process..................................................................................................................... 118
12.3 Time to Market ..................................................................................................................................... 118
12.4 Price Regulations .................................................................................................................................. 119
12.4.1 Pricing Policy Following the Marketing Authorization ....................................................... 119
12.4.2 External Reference Pricing ...................................................................................................119
Contents xi
12.4.3 Internal Reference Pricing ..................................................................................................... 119

12.4.4 Price Control at Ex-Factory Price Level ................................................................................ 119
12.4.5 Price Control at Wholesale Level .......................................................................................... 119
12.4.6 Price Control at Pharmacy Retail Level................................................................................. 119
12.4.7 Mandatory Price Reduction on Brand Price after Generic/Biosimilar Entry ........................ 119
12.5 Reimbursement Specificities .................................................................................................................. 119
12.6 Characteristics of Public Tendering ....................................................................................................... 119
12.7 Expenditure Controls (Supply Side) ...................................................................................................... 120
12.7.1 Discounts/Rebates.................................................................................................................. 120
12.7.2 Clawback ............................................................................................................................... 120
12.7.3 Payback .................................................................................................................................. 120
12.7.4 Price-Volume Agreements ..................................................................................................... 120
12.7.5 Other Market Access Agreements.......................................................................................... 120
12.7.6 Price Freezes and Cuts ........................................................................................................... 120
12.8 Policies Targeted at Wholesalers, Pharmacists, Physicians, and Patients .............................................. 120
12.8.1 Wholesaler and Pharmacy Mark-Up...................................................................................... 120
12.8.2 Generic Substitution .............................................................................................................. 121
12.8.3 INN Prescribing ..................................................................................................................... 121
12.8.4 Prescription Guidelines .......................................................................................................... 121
12.8.5 Monitoring of Prescribing Behavior ...................................................................................... 121
12.8.6 Pharmaceutical Budgets Defined for Physicians ................................................................... 121
12.8.7 Prescription Quotas ................................................................................................................ 121
12.8.8 Financial Incentives for Physicians ....................................................................................... 121
12.8.9 Financial Incentives for Pharmacists ..................................................................................... 121
12.8.10 Copayment for Patients.......................................................................................................... 121
References ........................................................................................................................................................... 121
Further Reading ................................................................................................................................................... 122
Chapter 13 Germany .............................................................................................................................................................. 123

13.1 Stakeholders ........................................................................................................................................... 123
13.1.1 Names of National Pricing and Reimbursement Decision Makers ....................................... 123
13.1.2 Names of National Health Technology Assessment Agencies .............................................. 123
13.1.3 Names of Other Key Stakeholders (Regional/Local Level) .................................................. 123
13.2 Pricing and Reimbursement Policies ..................................................................................................... 123
13.2.1 Overview of the System ......................................................................................................... 123
13.2.2 Reimbursement Process ......................................................................................................... 123
13.2.3 Pricing Process....................................................................................................................... 124
13.3 Time to Market ....................................................................................................................................... 124
13.4 Price Regulations ................................................................................................................................... 124
13.4.1 Pricing Policy Following the Marketing Authorization ......................................................... 124
13.4.2 External Reference Pricing .................................................................................................... 124
13.5 Reimbursement Specifics ....................................................................................................................... 125
13.6.1 Expenditure Controls Discounts/Rebates .............................................................................. 125
13.6.2 Clawback ............................................................................................................................... 125
13.6.3 Payback .................................................................................................................................. 125
xii Contents
13.7 Policies Targeted at Wholesalers, Pharmacists, Physicians, and Patients ............................................. 125
13.7.1 Wholesaler Mark-Up............................................................................................................. 125
13.7.2 Pharmacy Mark-Up ............................................................................................................... 125
13.7.3 Generic Substitution.............................................................................................................. 125
13.7.4 International Nonproprietary-Name Prescribing .................................................................. 125
13.7.5 Prescription Guidelines ......................................................................................................... 126
13.7.6 Monitoring of Prescribing Behavior ..................................................................................... 126
13.7.7 Pharmaceutical Budgets Defined for Physicians .................................................................. 126
13.7.8 Prescription Quotas ............................................................................................................... 126
13.7.10 Financial Incentives for Pharmacists .................................................................................... 126
13.7.11 Copayment for Patients ......................................................................................................... 126
References ........................................................................................................................................................... 126
Chapter 14 Italy...................................................................................................................................................................... 127

14.1 Stakeholders ........................................................................................................................................... 127
14.1.1 Names of National Pricing and Reimbursement Decision Makers....................................... 127
14.1.2 Names of National Health Technology Assessment Agencies.............................................. 127
14.1.3 Other Key Stakeholders at National Level ............................................................................ 127
14.1.4 Regional and Local Stakeholders .......................................................................................... 127
14.2 Pricing and Reimbursement of Pharmaceuticals in Italy ....................................................................... 127
14.2.1 Overview of the System ........................................................................................................ 127
14.2.2 The Process ........................................................................................................................... 128
14.2.3 Reimbursement Classes ........................................................................................................ 128
14.3 Time to Market Access for Drugs .......................................................................................................... 128
14.4 Price Regulation ..................................................................................................................................... 129
14.4.1 Pricing Policy Following the Marketing Authorization ........................................................ 129
14.4.2 External Reference Pricing ................................................................................................... 129
14.4.3 Internal Reference Pricing .................................................................................................... 129
14.4.4 Price Control (at Ex-Factory, Wholesale, and Pharmacy Retail) .......................................... 129
14.4.5 Mandatory Price Reduction on Brand Price after Generic/Biosimilar Entry........................ 129
14.5 Cost-Containment Policies ..................................................................................................................... 129
14.5.1 Prescription Guidelines ......................................................................................................... 129
14.5.2 Discounts/Rebates ................................................................................................................. 129
14.5.3 Payback ................................................................................................................................. 129
14.5.4 Price-Volume Agreements..................................................................................................... 129
14.5.5 Other Market Access Agreements ......................................................................................... 129
14.5.6 Monitoring of Prescribing Behavior ..................................................................................... 130
14.5.7 Public Tenders ....................................................................................................................... 130
14.5.8 Generic Substitution.............................................................................................................. 130
14.5.9 Copayment for Patients ......................................................................................................... 130
14.6 Policies Targeted at Wholesalers, Pharmacists ...................................................................................... 130
14.6.1 Wholesaler Mark-Up............................................................................................................. 130
14.6.2 Pharmacy Mark-Up ............................................................................................................... 130
References ........................................................................................................................................................... 130
Chapter 15 Spain .................................................................................................................................................................... 131

15.1 Stakeholders ........................................................................................................................................... 131
15.1.1 Names of National Pricing and Reimbursement Decision Makers....................................... 131
15.1.2 Names of National Health Technology Assessment Agencies.............................................. 131
15.2.1 Overview of the System ........................................................................................................ 131
15.2.2 Reimbursement Process ........................................................................................................ 131
15.2.3 Pricing Process ...................................................................................................................... 132
Contents xiii
15.3 Time to Market ....................................................................................................................................... 132

15.4 Price Regulations .................................................................................................................................... 132
15.4.1 Pricing Policy Following the Marketing Authorization.......................................................... 132
15.4.2 External Reference Pricing ..................................................................................................... 132
15.4.3 Internal Reference Pricing ...................................................................................................... 132
15.4.4 Price Control at Ex-Factory Price Level................................................................................. 132
15.4.5 Price Control at Wholesale Level ........................................................................................... 132
15.4.6 Price Control at Pharmacy Retail Level ................................................................................. 132
15.4.7 Mandatory Price Reduction on Brand Price after Generic/Biosimilar Entry ......................... 132
15.6 Characteristics of Public Tendering ........................................................................................................ 132
15.7 Expenditure Controls .............................................................................................................................. 133
15.7.1 Discounts/Rebates .................................................................................................................. 133
15.7.2 Clawback ................................................................................................................................ 133
15.7.3 Payback................................................................................................................................... 133
15.7.4 Price-Volume Agreements ...................................................................................................... 133
15.8.1 Wholesaler Mark-Up .............................................................................................................. 133
15.8.2 Pharmacy Mark-Up ................................................................................................................ 133
15.8.3 Generic Substitution ............................................................................................................... 133
15.8.4 International Nonproprietary Name (INN) Prescribing ........................................................ 133
15.8.6 Monitoring of Prescribing Behavior ....................................................................................... 133
15.8.7 Pharmaceutical Budgets Defined for Physicians .................................................................... 133
15.8.9 Financial Incentives for Physicians ........................................................................................ 134
15.8.10 Financial Incentives for Pharmacists ...................................................................................... 134
15.8.11 Copayment for Patients........................................................................................................... 134
References ........................................................................................................................................................... 134
Chapter 16 Sweden ................................................................................................................................................................ 135

16.1 Stakeholders ........................................................................................................................................... 135
16.1.1 National Pricing and Reimbursement Decision Makers......................................................... 135
16.1.2 National Health Technology Assessment Agencies................................................................ 135
16.1.3 Other Key Stakeholders .......................................................................................................... 135
16.2.2 Reimbursement Process.......................................................................................................... 136
16.2.3 Pricing Process ....................................................................................................................... 136
16.2.4 Pharmaco-Economic Assessment ........................................................................................... 136
16.3 Time to Market ....................................................................................................................................... 137
16.4.1 Pricing Policy Following the Marketing Authorization.......................................................... 137
16.4.2 External Reference Pricing ..................................................................................................... 137
16.4.3 Internal Reference Pricing ...................................................................................................... 137
16.4.4 Price Control at Ex-Factory Price Level................................................................................. 137
16.4.5 Price Control at Wholesale Level ........................................................................................... 137
16.4.6 Price Control at Pharmacy Retail Level ................................................................................. 137
16.4.7 Mandatory Price Reduction on Brand Price after Generic/Biosimilar Entry ......................... 137
16.6 Characteristics of Public Tendering........................................................................................................ 138
16.7.1 Discounts/Rebates .................................................................................................................. 138
xiv Contents
16.7.2 Clawback ................................................................................................................................ 138

16.7.3 Payback .................................................................................................................................. 138
16.8.1 Wholesaler Mark-Up ............................................................................................................. 138
16.8.2 Pharmacy Mark-Up................................................................................................................ 138
16.8.4 International Nonproprietary Name (INN) Prescribing ......................................................... 138
References ........................................................................................................................................................... 139
Chapter 17 United Kingdom ...................................................................................................................................................141

17.1 Stakeholders ............................................................................................................................................141
17.1.1 National Pricing and Reimbursement Decision Makers ........................................................ 141
17.1.2 National Health Technology Assessment (HTA) Agencies ................................................... 141
17.2 Pricing and Reimbursement Policies .......................................................................................................141
17.2.3 Pricing Process....................................................................................................................... 142
17.3 Time to Market ....................................................................................................................................... 142
17.4.2 Reference Pricing................................................................................................................... 142
17.4.3 Price Control .......................................................................................................................... 142
17.5 Reimbursement Specifics ........................................................................................................................143
17.6.1 Applied to Hospital Care ....................................................................................................... 143
17.6.2 Applied to Ambulatory Care .................................................................................................. 143
17.7 Expenditure Controls (Supply Side)........................................................................................................143
17.7.2 Clawback ............................................................................................................................... 143
17.7.3 Payback .................................................................................................................................. 143
17.8.2 Pharmacy Mark-Up................................................................................................................ 144
References ........................................................................................................................................................... 145
Contents xv
Chapter 18 Belgium ................................................................................................................................................................147

18.1 Stakeholders ............................................................................................................................................147
18.1.3 Names of Other Key Stakeholders (Regional/Local Level) ...................................................147
18.2 Pricing and Reimbursement Policies Overview ......................................................................................147
18.2.3 Pricing Process ...................................................................................................................... 148
18.3 Time to Market ....................................................................................................................................... 148
18.4.1 Pricing Policy Following the Marketing Authorization......................................................... 149
18.4.2 External Reference Pricing ................................................................................................... 149
18.4.7 Mandatory Price Reduction on Brand Price after Generic/Biosimilar Entry ....................... 149
18.5.1 Characteristics of Public Tendering ....................................................................................... 149
18.5.2 Expenditure Controls Discounts/Rebates .............................................................................. 149
18.5.3 Clawback ............................................................................................................................... 149
18.5.4 Payback .................................................................................................................................. 149
18.5.7 Price Freezes and Cuts........................................................................................................... 150
18.6.2 Pharmacy Mark-Up................................................................................................................ 150
18.6.4 International Nonproprietary Name Prescribing ................................................................... 150
18.6.12 Special Funding Procedure for Individual Patients ............................................................... 151
References ........................................................................................................................................................... 151
Chapter 19 The United States ................................................................................................................................................ 153

19.1 Stakeholders ........................................................................................................................................... 153
19.2 Overview of Pricing and Reimbursement Policies ................................................................................ 153
19.2.3 Pricing Process ...................................................................................................................... 154
19.3 Time to Market ....................................................................................................................................... 154
xvi Contents

19.7.2 Clawback ............................................................................................................................... 155
19.7.3 Payback .................................................................................................................................. 155
19.8.2 Pharmacy Mark-Up................................................................................................................ 155
References ........................................................................................................................................................... 156
Chapter 20 Japan .................................................................................................................................................................... 159

20.1 Stakeholders ........................................................................................................................................... 159
20.1.1 National Pricing and Reimbursement Decision Makers ........................................................ 159
20.1.2 National Health Technology Assessment Agencies ............................................................... 159
20.1.3 Other Key Stakeholders (Regional/Local Level) .................................................................. 159
20.2 Overview of Pricing and Reimbursement Policies ................................................................................ 159
20.2.3 Pricing Process ...................................................................................................................... 159
20.3 Time to Market ....................................................................................................................................... 159
20.4 Price Regulations.................................................................................................................................... 159
20.4.1 Pricing Policy Following the Marketing Authorization......................................................... 159
20.4.7 Mandatory Price Reduction on Brand Price after Generic/Biosimilar Entry ....................... 160
20.7 Expenditure Controls.............................................................................................................................. 160
20.7.2 Clawback ............................................................................................................................... 160
20.7.3 Payback .................................................................................................................................. 160
20.7.5 Other Market Access Agreements ......................................................................................... 160
20.7.6 Price Freezes and Cuts .......................................................................................................... 160
Contents xvii
References ............................................................................................................................................................161
Chapter 21 China ................................................................................................................................................................... 163

21.1 Stakeholders ........................................................................................................................................... 163
21.2.3 Pricing Process....................................................................................................................... 163
21.3 Time to Market ....................................................................................................................................... 164
21.4.7 Mandatory Price Reduction of Price of On-Patent Drugs after Generic/Biosimilar Entry ........... 164
21.7.2 Clawback ............................................................................................................................... 164
21.7.3 Payback .................................................................................................................................. 164
21.8.11 Changes in the Pricing Mechanism ....................................................................................... 165
References ........................................................................................................................................................... 166
Epilogue ................................................................................................................................................................................... 167

Index ......................................................................................................................................................................................... 169
Foreword
With this original contribution to health economics, Professor the corresponding clinical science has evolved to point where
Mondher Toumi has done us all a great service by defining genetic and other biomarkers can predict individual response
and illustrating the role and utility of the term, and the con- to therapy. Manufacturers, academics, consultants, and even
cept of, market access. Make no mistake about it; there are government all compete in this market to differentiate the
markets for terminology and ideas, and therefore competition value that their concepts can provide.
among them. In this book, he explains the concept of market Most readers of this book and even most health econo-
access and makes a convincing case for its prevalence and use mists will not be familiar with the original use of the term
in health economics and for its popularity among pharmaceu- market access, as explained well by Professor Toumi, in the
tical manufacturers. field of international trade by the World Trade Organization.
The use of market access as an analytical term is relatively Clearly, thinking internationally, that is, with nation states
a new development in health economics. Based on Google competing to serve their own citizens first, world economic
Ngram, the use of the term health economics in books came history has been replete with all sorts of barriers—both tar-
into use after 1970: Its annual use grew dramatically between iff and nontariff—to trade. He defines market access as “…
1970 and 1978, leveled off during the period of 1978–1983, the ability for a drug to achieve through a health insurance
then grew strongly until 2003, and has leveled off again from system a reimbursed price and a favorable recommendation
2003 to 2006. Interestingly, the overall Ngram pattern for for prescription.” For a manufacturer interested in selling its
market access is similar but growth is steadier until a recent products in as many national markets as possible, the term
plateau, and interestingly, it is the more commonly found market access means overcoming those barriers to get one’s
phrase. But a quick search of PubMed keywords for the pub- product on the market in a particular country. For innova-
lished scientific literature finds some 15,000 articles using tive pharmaceuticals, the substantial barriers are market
health economics as a keyword versus only about 600 arti- authorization, price and reimbursement negotiations, and
cles using market access as a keyword. On the other hand, a controls on utilization. Of course, the concept can also apply
simple English-only search for health economics yields more to specific insurance market segments in a complex and frag-
than four million Google hits, whereas market access yields mented public and private insurance market such as the one
more than seven million. But “market access” “health” yields that the United States has.
only about two million hits. These patterns will make sense Professor Toumi and his colleagues have surveyed phar-
as I recount below Professor Toumi’s explanation of the origin maceutical companies and found that they organize their
of the term market access. activities in this area in a variety of ways. Most staff work-
Health economics emerged as a subfield of economics in the ing in this area were originally trained in fields such as
early 1970s, when university-level courses began to be taught. decision sciences, pharmaceutical administration, health
Professor Toumi cites Professor Kenneth Arrow’s seminal economics, epidemiology, biostatistics, psychometrics,
1963 article on “Uncertainty and the Welfare Economics of clinical pharmacology, and business administration. But
Health Care.” It identified the unique characteristics of medi- with the complementarity of these fields being fairly obvi-
cal care and health as economic goods. Owing to uncertainty ous, especially when confronted with a range of regulatory,
of various kinds, these markets can fail to perform optimally, health technology assessments, and pricing and reimburse-
that is, achieve Pareto efficiency, and many of the regulatory ment barriers, companies have often grouped some of these
interventions that are evident can be explained as an evolu- activities, particularly at a global level, under the umbrella
tionary response—both from the public and private sectors— of market access. Having worked in a global health econom-
to these market failures. ics group in a large company, and having taught in a PhD
Professor Toumi is also a leading thinker in the subfield program to train interested researchers with the capability to
of health economics called pharmacoeconomics. We have work in these company units, I have seen a gradual evolu-
also witnessed the tension of terminology within this subfield. tion in the recognition that staff working in these groups need
Taking an economics perspective, one could argue that there basic training in all of these areas and an increased under-
is indeed a demand and supply for new terms, concepts, and standing of the market access barriers in these markets: so
ideas. In addition, it can be a bit of a battlefield: Consider, our curriculum continues to evolve in this direction. To my
for example, the general concept of personalized medicine, knowledge, Professor Toumi is the pioneer here—the first to
which has variously been called stratified medicine, tailored create an academic department that explicitly offers degrees
treatment, individualized therapy, and, mostly recently, preci- in market access.
sion medicine. Proponents of each would argue that these are More than 200 years ago, Adam Smith pointed out in his
imperfect synonyms and that each has some unique aspect book The Wealth of Nations that “division of labor is lim-
that is valuable. These words can have power and influence ited by the extent of the market.” Professor Toumi is proba-
as well: President Barack Obama launched a “Precision bly ahead of the curve here, but it is important to recognize
Medicine Initiative” in January 2015, reflecting a belief that that academic markets are competitive and will be somewhat
xix
xx Foreword
resistant to change, even if it only means renaming the inte- Although I have emphasized the novelty of Professor
grated, multidisciplinary training they are already doing under Toumi’s case for the unifying concept of market access,
a new label. It is difficult to think of a better term than market readers of this book will also be astounded by the breadth
access that captures the essence of these activities. Even in my and depth of his knowledge and understanding of the real-
own 12 years working in the industry, our unit evolved from world institutions affecting market access in each of the
pharmacoeconomics to health economics (to consider diagnos- individual chapters for nine major pharmaceutical markets:
tics and devices), and to economics (to add pricing and reim- France, Germany, Italy, Spain, Sweden, the United Kingdom,
bursement considerations). And subsequently this has evolved Belgium, the United States, Japan, and China. In addition,
to payer strategy (to focus on strategic thinking to overcome these themes are extended to the related specific issues, such
market access barriers). The conceptual innovation of grouping as the market for vaccines, orphan drugs, external reference
these activities under market access is understandably appeal- pricing, early advice, early access, and market access agree-
ing, and it is essentially facing a market test at the moment. ments. I would encourage you to read this book, and learn and
I have considerable doubt about whether and when this enjoy contemplating these important insights and connections
might reach a tipping point resulting in market access becom- that Professor Toumi is sharing with us.
ing the most widely used term for these activities. But I have
no doubt that Professor Toumi has done all of us a great ser- Lou Garrison
vice by exploring and clarifying the utility of this concept, Pharmaceutical Outcomes Research & Policy Program
and identifying its relevance to a wide range of phenomena Department of Pharmacy
such as health technology assessment, market access agree- Departments of Global Health and Health Services
ments, value propositions, and value-based pricing. University of Washington
Acknowledgments
I thank everyone who helped me complete this book Thanks to Pr. Lou Garrison and Lieven Annemans for your
by providing review, input, comments, and continuous preface and postface.
encouragement. Dear all, without your effective support,
To my family for their continous support!
I would have not been able to bring my work to a success-
ful completion. Un clin d’œil à la Commune de Paris
Very special thanks to Szymon Jarosławski and Cécile
J’aimerai toujours le temps des cerises
Rémuzat for your continuous review and input along this
C’est de ce temps-là que je garde au coeur,
book’s development.
Une plaie ouverte.
Thanks to Samuel Aballéa, Lieven Annemans, Charles
Et Dame Fortune en m’étant offerte
Sabourin, Anna D’Ausilio, Emna El Hammi, Jaime Espin,
Ne pourra jamais calmer ma douleur...
Clément François, Shunya Ikeda, Patrycja Jaros, Åsa Kornfeld,
J’amerai toujours le temps des cerises
Stefan Lhachimi, Abayomi Odeyemi, Florence Baron Papillon,
Et le souvenir que je garde au coeur.
Steve Sherman, Marion Thivolet, Keith Tolley, and Katherine
Eve Young for your review. Le temps des Cerises de Jean Baptiste Clément
xxi
Author
Mondher Toumi, MD, MSc (biostatistics), MSc (biological Lyon, France. In the same year, he was appointed director
sciences, option pharmacology), PhD (economic sciences) of the chair of public health and market access. He launched
is a professor of public health at Aix-Marseille University, the first European University Diploma of Market Access
Marseille, France. After working for 12 years as research (EMAUD), an international course already followed by
manager in the Department of Pharmacology at the University approximately 400 students. In addition, he recently created
of Marseille, he joined the Public Health Department in 1993. the Market Access Society to promote education, research,
From 1995, he pursued a career in the pharmaceutical indus- and scientific activities at the interface of market access,
try for 13 years. health technology assessment (HTA), public health, and
Mondher Toumi was appointed global vice president health economic assessment. He is the editor-in-chief of the
at Lundbeck A/S in charge of health economics, outcome Journal of Market Access and Health Policy (JMAHP), which
research, pricing, market access, epidemiology, risk manage- was recently granted PubMed indexation.
ment, governmental affairs, and competitive intelligence. In Toumi is also a visiting professor at Beijing University
2008, he founded Creativ-Ceutical, an international consult- (Third Hospital), China. He is a recognized expert in health
ing firm dedicated to support health industries and authori- economics and an authority on market access and risk man-
ties in strategic decision-making. In February 2009, he was agement. He has authored more than 250 scientific publica-
appointed professor in the Department of Decision Sciences tions and communications, and has contributed to several
and Health Policies at Claude Bernard University Lyon 1, books.
xxiii
Common Abbreviations
ACIP Advisory Committee on Immunization Practices DCE Discrete choice experiment
AEMPS Agencia Española del Medicamentos y Productos DDD Daily defined dose
Sanitarios/The Spanish Agency for Medicines DERP Drug effectiveness review project
and Health DGFPS Dirección General de Farmacia y Productos
AETS Agencia de Evaluacion de Tecnologias Sanitarias/ Sanitarios/The Directorate General of Pharmacy
The National Health Technology Assessment and Health Products
Agency DH Department of Health
AFR Annual financial return DIMDI Deutsches Institut für Medizinische Dokumen-
AGES Austrian Agency for Health and Food Safety tation und Information/The German Institute of
AHRQ Agency for Healthcare Research and Quality Medical Documentation and Information
AIFA Agenzia Italiana del Farmaco/Italian Medicines DMA Danish Health and Medicines Authority
Agency DPO Drug Pricing Organization
AMNOG Arzneimittelmarktneuordnungsgesetz/The Act of DRG Disease-related group
the Reform of the Market for Medicinal Products DT Drug tariff
AMP Average manufacturer price EBA Early benefit assessment
ASL Azienda Sanitaria Locale/Local Health Unit EMA European Medicines Agency
ASMR Amélioration du Service Médical Rendu EU European Union
(improvement in actual benefit) FDA Food and Drug Administration
ASNM Agence nationale de sécurité du médicament et FUL Federal upper limit
des produits de santé G-BA Gemeinsamer Bundesausschuss/The Federal
ASP Average sale price Joint Committee
AWP Average wholesale price GBS Guillain–Barré syndrome
BfArM Bundesinstitut für Arzneimittel und GKV-SV Gesetzliche Krankenversicherung-Spitzenverband/
Medizinprodukte/The Federal Institute for The National Association of Statutory Health
Drugs and Medical Devices Insurance Funds
BLA Biologics License Application GU Gazzetta Ufficiale/The Official Journal of the
BMG Bundesministerium für Gesundheit/The Federal Italian Republic
Ministry of Health HAS Haute Autorité de Santé/French National Authority
CA Commercial agreement for Health
CBER Center for Biologics Evaluation and Research HC Health Council
CDC Centers for Disease Control and Prevention HEOR Health Economics and Outcomes Research
CE Cost-effectiveness HHS Department of Health and Human Services
CED Coverage with evidence development HPV Human papilloma virus
CEPS Economic Committee of Health Products HTA Health technology assessment
CHIP Children’s Health Insurance Programme ICER Incremental cost-effectiveness ratio
CIPM Comisión Interministerial de Precios de los INN International nonproprietary name
Medicamentos/The Interministerial Commission IQWiG Institut für Qualität und Wirtschaftlichkeit im
for Pricing of Medicinal Products Gesundheitswese/The Institute for Quality and
CISNS Consejo Interterritorial del Sistema Nacional de Efficiency in Healthcare
Salud/Spanish Interterritorial Council ITR Index Thérapeutique Relatif
CMS Centers for Medicare and Medicaid Services JCVI Joint Committee on Vaccination and Immunisation
CPR Comitato Comitato Prezzi e Rimborso/Pricing LFN Läkemedelsförmånsnämnden/Pharmaceutical
and Reimbursement Committee Benefits Board
CST Comitato Scientifico e Tecnico/Scientific-Technical MA Market access
Commission MAA Market access agreement
CT Commission de la Transparence MAC Maximum allowable cost
CTC Conditional treatment continuation MAH Marketing Authorization Holder
CTV Technical Committee on Vaccinations MAu Marketing authorization
CVZ College voor Zorgverzekeringen/The National MCDA Multicriteria decision analysis
Health Care Institute MHLW Ministry of Health, Labour and Welfare
DAHTA Deutsche Agentur für Health Technology MHRA Medicines and Healthcare Products Regulatory
Assessment/The German Agency of Health Agency
Technology Assessment MoH Ministry of Health
xxv
xxvi Common Abbreviations
MOHRSS The Ministry of Human Resources and Social QALY Quality adjusted life year
Security RCT Randomized Controlled Trial
MPA Läkemedelsverket/The Medical Products Agency R&D Research and development
MS Multiple sclerosis RP Reference pricing
MSSSI Ministerio de Sanidad, Servicios Sociales e RSA Risk sharing agreement
Igualdad/The Ministry of Health, Social Services SBU Statens beredning för medicinsk och social
and Equality utvärdering/Swedish Agency for Health Technology
MTA Multiple technology assessment Assessment and Assessment of Social Services
NDRC The National Development and Reform SGB Sozialgesetzbuch/ Social Code Book
Commission SGCMPS Subdirección General de Calidad de Medicamentos
NHFPC The National Health and Family Planning y Productos Sanitarios/The General Subdirectorate
Commission of Quality of Medicines and Health Products
NHI National Health Insurance SHI Statutory Health Insurance
NHS National Health Service SMC Scottish Medicines Consortium
NICE National Institute for Health and Care Excellence SMR Service Médical Rendu/Actual Medical Benefit
NIP National Immunization Program SNS Sistema Nacional de la Salud/The National Health
NITAG National Immunization Technical Advisory Group System
NVC National Vaccination Calendar SOP (Farmaci) Senza Obbligo di Prescrizione/
OECD The Organisation for Economic Co-operation Nonprescription pharmaceuticals with advertis-
and Development ing prohibition
OsMED Osservatorio Nazionale sull’impiego dei STA Single technology assessment
Medicinali/National Observatory on the Use of STIKO Standing Vaccination Committee (Ständigen
Pharmaceuticals Impfkommission)
OTC Over-the-counter TC Transparency Committee
P&R Pricing and reimbursement TFR Tarif forfaitaire de responsabilité
P4P Payment for performance TLV Tandvårds- och läkemedelsförmånsverket/The
PAS Patient access scheme Dental and Pharmaceutical Benefits Agency
PBAC Pharmaceutical Benefits Advisory Committee TVs Therapeutic vaccines
PBMSHG Pharmacy Benefits Management Strategic UNCAM Union nationale des caisses d’assurance maladie
Healthcare USP United States Pharmacopeia
PEC Department of Defense Pharmacoeconomic VA Veterans Affairs
Center VBP Value based pricing
PEI Paul Ehrlich Institute VENICE Vaccine European New Integrated Collaboration
PFN Prontuario Farmaceutico Nazionale/The National Effort
Pharmaceutical Formulary WAC Wholesale acquisition cost
POM Prescription-only medicines ZIN Zorginstituut Nederland/National Healthcare
PPRS Pharmaceutical Price Regulation Scheme Institute
PV Preventive vaccine
Introduction
Inadequate market access has become the primary reason for • Draw a mapping of market access stakeholders
commercial failure of any new pharmaceutical launch. It is • Present basic knowledge in decision sciences applied
often referred to as the fourth hurdle in reaching the patients, to public health
following the need to evidence the traditional three hurdles of • Review the latest regulations and guidelines in main
efficacy, safety, and quality in order to obtain the regulatory global markets, including Europe, United States, and
marketing authorization. Asia
Nowadays, market access has become the primary driver • Review novel pharmaceutical policies such as risk-
for global income of any new drug in development. Without a sharing, managed entry agreements, early entry,
convincing strategy in place to evidence and communicate the early dialogue, vaccine, and orphan drugs market
product value to decision makers, the drug will fail to reach access policies
its intended commercial value. This requires understanding • Discuss the best practices in market access across
the key decision drivers. case studies
Market access is a complex process with as many defi-
nitions as individuals. Even prestigious publications may
KEY LEARNINGS
contain definitions that are questionable, inappropriate, or
inaccurate. This book attempts to harmonize and unify defini- • Being able to understand the grounds for a market
tions, concepts, and history related to the market access field. access strategy with a multidisciplinary approach
The approach presented in this book is situated at a crossroads • Identify the stakeholders and develop a market
of multiple disciplines that form an integral part of a success- access plan that matches their expectations
ful approach to market access. • Understand specificities of some geographies and
This book is based on an accredited course in this area some types of pharmaceutical products
out of the European Market Access University Diploma • Anticipate the future paradigm changes in market
(EMAUD) affiliated with the Market Access Society, France, access
and the Aix-Marseille University, Marseille, France.
WHO SHOULD READ?
OBJECTIVES OF THE BOOK
This book is intended for students and for industry and author-
• Present an advanced understanding of the market ity professionals in the fields of pharmaceuticals, medical bio-
access environment, concepts, and principles technology, and public health.
xxvii
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answered by [238]a “burning, as in hell-fire, of priestly shams and
lies,” and not one out of twenty French monasteries escaped the fury
of the avengers. Our Protestant clergymen see their temple walls
cracked by a breach of ever-multiplying schisms, and can prop their
prestige only by more and more humiliating concessions, and in
every intelligent community have to purchase popularity by rank
heresies against the dogmas of their predecessors. Here and there
the orthodox tenets of the New Testament have survived the
progress of rationalism, but haunt the shade, like specters scenting
the morning air, and momentarily expecting the summons that shall
banish them to the realms of their native night.
[Contents]
E.—REFORM.
When the harbinger of day dispels the specters of darkness, half-

awakened sleepers often mourn the fading visions of dreamland, as
they would mourn the memories of a vanished world, till they find
that the solid earth still remains, with its mountains and forests, and
that the enjoyment of real life has but just begun. With a similar
regret the dupes of Jesuitism mourn the collapse of their creed and
lament the decline of morality, till they find that religion still remains,
with its consolations and hopes, and that the true work of redemption
has but just begun.
The reign of superstition begins to yield to a religion of reason and

humanity. The first forerunners of that religion appeared at the end of
the sixteenth century, when the philosophers of northern Europe
[239]first dared to appeal from dogma to nature, and since that revival
of common-sense the prison walls of clerical obscurantism have
been shaken by shock after shock, till daylight now enters through a
thousand fissures.
But Secularism has a positive as well as a negative mission, and

after removing the ruins of exploded idols, the champions of reform
will begin the work of reconstruction. Temples dedicated to the
religion of progress will rise from the ruins of superstition.
Communities of reformants will intrust the work of education to
chosen teachers, who will combine the functions of an instructor with
those of an exhorter. In the languages of several European nations
the word “rector” still bears that twofold significance. The ministers of
Secularism will not sacrifice physical health to mental culture. They
will be gymnasiarchs, like the Grecian pedagogues who
superintended the athletic exercises of their pupils and accompanied
them on foot journeys and hunting excursions. They will be teachers
of hygiene, laboring to secure the foundations of mental energy by
the preservation of physical vigor, and to banish diseases by the
removal of their causes. They will seek to circumscribe the power of
prejudice by the extension of knowledge. They will obviate the perils
of poverty by lessons of industry and prudence. Their doctrines will
dispense with miracles; they will make experience the test of truth,
and justice the test of integrity; they will not suppress, but
encourage, free inquiry; their war against error will employ no
weapons but those of logic. [240]
The religion of reason will limit its proper sphere to the secular
welfare of mankind, but will ask, as well as grant, the fullest freedom
of metaphysical speculation. Why should the friends of light darken
the sunshine of earth with fanatical wars for the suppression of
private theories about the mystery of the unrevealed first cause?
Why should they rage about the riddle of the veiled hereafter to
please the ordainer of the eternal law that visits such inexorable
penalties upon the neglect of the present world? Should the friends
of common sense quarrel about guesses at the solution of
unknowable secrets? We need not grudge our wonder-loving brother
the luxury of meditating on the mysteries of the unseen or the
possibilities of resurrection. Shall the soul of the dying patriarch live
only in his children? Shall it wing its way to distant stars? Shall it
linger on earth:
“Sigh in the breeze, keep silence in the cave,

And glide with airy foot o’er yonder sea?”
Why should we wrangle about riddles which we cannot possibly

solve? But we might certainly have honesty enough to admit that
impossibility. Musing on the enigmas of the “land beyond the veil”
may entertain us with the visions of a dreamy hour, but should not
engross the time needed for the problems of the only world thus far
revealed.
Thus, founded on a basis of health-culture, reason, and justice, the

office of priesthood will regain its ancient prestige, and the best and
wisest of men will become ministers of Secularism by devoting their
lives to the science of happiness on earth. [241]
[Contents]
PROF. FELIX L. OSWALD’S WORKS.
THE SECRET OF THE EAST; or, The Origin of the

Christian Religion, and the Significance of its Rise
and Decline. Cloth, $1.
THE BIBLE OF NATURE; or, The Principles of

Secularism. A Contribution to the Religion of the
Future. Cloth, $1.
PHYSICAL EDUCATION; or, The Health-Laws of

Nature. Cloth, $1.
HOUSEHOLD REMEDIES; for the Prevalent

Disorders of the Human Organism. Cloth, $1.
THE POISON PROBLEM; or, The Cause and Cure of

Intemperance. Pap., 25cts; clo., 75cts.
SUMMERLAND SKETCHES; or, Rambles in the

Backwoods of Mexico and Central America.
Profusely Illustrated from Designs by H. F. Farny and H.
Faber. 8vo, cloth, $2.50.
ZOOLOGICAL SKETCHES. A Contribution to the

Outdoor Study of Natural History. 8vo, cloth, $2.
For all of the above address
THE TRUTH SEEKER CO.,

28 Lafayette Place, New York. [242]
[Contents]
WORKS OF
L. K. WASHBURN.
Sunday and the Sabbath. “A law regulating human

conduct on the Sabbath is an impertinence.” Price, 10
cents.
The False Teaching of the Christian Church. “The

Thirty-nine Articles of the Christian church are thirty-nine
poor, broken-down opinions.” Price, 10 cents.
The Foolishness of Prayer. “Think of a minister’s

praying God to kill grasshoppers, or trying to induce the
deity to undertake a crusade of one against the Colorado
beetle!” Price, 10 cents.
Followers of Jesus. Price, 10 cents.
Religious Problems. Price, 10 cents.
Spiritualism: Is It a Faith or a Fact? Price, 5 cents.
Do You Love Jesus? Price, 5 cents.
America’s Debt to Thomas Paine. Price, 5 cents.
Is Liberalism Moral? Price, 5 cents.
A Holy Superstition. Price, 5 cents.
Temperance and the Bible. Price, 5 cents.

Free Religion; or, The Religious Demand of To-day.
Price, 5 cents.
The Brute God of the Old Testament. Price, 5 cents.
The Public Schools and the Catholic Church. 5 cents.

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The Bible of Nature;

Or, The Principles of
Title: Secularism. A
Contribution to the
Religion of the Future
Felix Leopold Oswald Info
Author:
(1845–1906) https://viaf.org/viaf/16832833/
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2023-11-15 20:45:25
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date:
Language: English
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distance
13 jailors jailers 1
31 - [Deleted] 1
38 night-shaft night-shift 1
42 Golgatha Golgotha 1
44 [Not in source] . 1
75 koenig König 3/2
75 koennen können 2/1
89 Jerstaecker Gerstäcker 3/2
90 Rhus Rushd 3
97 Hartz Harz 1
98 kreuze Kreuze 1
98 Luetzen Lützen 2/1
98 Oudenarde Oudenaarde 1
102 wordly worldly 1
105 Hooglyn Hooghly 2
117 cooperation coöperation 1/0
123 intemperence intemperance 1
124 eshalten erhalten 1
152 hypocrits hypocrites 1
160 michievousness mischievousness 1
165,
179, Moriscoes Moriscos 1
189
173 groessre grössre 2/1
188 gardenlands garden-lands 1
197 Budha-Pesth Buda-Pesth 1
226 favorit favorite 1
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Introduction to Market Access for

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Introduction to MS Access 2016 v3 1 Nikos Dimitrakas

Gene and Cell Therapies-Market Access and Funding 1st

An Introduction to Cryptocurrencies The Crypto Market

Employability and Access to Training A Contribution to

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International Standard Book Number-13: 978-1-138-03218-7 (Paperback)

Chapter 1 Health as a Good ..................................................................................................................................................... 1

Chapter 2 Decision-Making in Public Health.......................................................................................................................... 5

Chapter 3 Definitions and Concepts ...................................................................................................................................... 13

3.5 Market Access for Payers ......................................................................................................................... 21

Chapter 4 HTA Decision Analysis Framework ...................................................................................................................... 27

Chapter 5 Early HTA Advice ................................................................................................................................................. 37

5.3 Strategic Considerations ............................................................................................................................. 44

Chapter 6 Overview of Market Access Agreements.............................................................................................................. 47

6.9 Overview of MAA Trends in Other Countries ......................................................................................... 66

Chapter 7 External Reference Pricing.................................................................................................................................... 71

Chapter 8 Gap between Payers and Regulators ..................................................................................................................... 79

8.6 Risk Management Tools ........................................................................................................................... 82

Chapter 9 Early Access Programs ......................................................................................................................................... 91

Chapter 10 Market Access of Orphan Drugs........................................................................................................................... 97

10.4 Comparison of Prices of Orphan Drugs ............................................................................................... 103

Chapter 11 Market Access of Vaccines in Developed Countries ........................................................................................... 107

Chapter 12 France .................................................................................................................................................................. 117

12.4.3 Internal Reference Pricing ..................................................................................................... 119

Chapter 13 Germany .............................................................................................................................................................. 123

Chapter 14 Italy...................................................................................................................................................................... 127

Chapter 15 Spain .................................................................................................................................................................... 131

15.3 Time to Market ....................................................................................................................................... 132

Chapter 16 Sweden ................................................................................................................................................................ 135

16.7.2 Clawback ................................................................................................................................ 138

Chapter 17 United Kingdom ...................................................................................................................................................141

Chapter 18 Belgium ................................................................................................................................................................147

Chapter 19 The United States ................................................................................................................................................ 153

19.4.3 Internal Reference Pricing ..................................................................................................... 154

Chapter 20 Japan .................................................................................................................................................................... 159

Chapter 21 China ................................................................................................................................................................... 163

Epilogue ................................................................................................................................................................................... 167

When the harbinger of day dispels the specters of darkness, half-

The reign of superstition begins to yield to a religion of reason and

But Secularism has a positive as well as a negative mission, and

“Sigh in the breeze, keep silence in the cave,

Why should we wrangle about riddles which we cannot possibly

Thus, founded on a basis of health-culture, reason, and justice, the

THE SECRET OF THE EAST; or, The Origin of the

THE BIBLE OF NATURE; or, The Principles of

PHYSICAL EDUCATION; or, The Health-Laws of

HOUSEHOLD REMEDIES; for the Prevalent

THE POISON PROBLEM; or, The Cause and Cure of

SUMMERLAND SKETCHES; or, Rambles in the

ZOOLOGICAL SKETCHES. A Contribution to the

For all of the above address

THE TRUTH SEEKER CO.,

Sunday and the Sabbath. “A law regulating human

The False Teaching of the Christian Church. “The