グローバルにおける嚢胞性線維症（CF症）治療薬市場：市場機会分析...市場調査レポートについてご紹介

1 Table of Contents
1 Table of Contents 7
1.1 List of Tables 11
1.2 List of Figures 13
2 Introduction 14
2.1 Catalyst 14
2.2 Related Reports 15
2.3 Upcoming Related Reports 15
3 Disease Overview 16
3.1 Etiology and Pathophysiology 16
3.1.1 Etiology 16
3.1.2 Pathophysiology 17
3.1.3 Prognosis 19
3.1.4 Quality of Life 20
3.2 Symptoms 20
4 Epidemiology 22
4.1 Risk Factors and Co-morbidities 22
4.2 Global and Historical Trends 23
4.2.1 US 23
4.2.2 France 24
4.2.3 Germany 24
4.2.4 Italy 25
4.2.5 Spain 25
4.2.6 UK 26
4.3 Forecast Methodology 26
4.3.1 Sources Used 26
4.3.2 Forecast Assumptions and Methods 29
4.3.3 Sources Not Used 32
4.4 Epidemiology Forecast 32
4.4.1 Total Prevalent Cases of Cystic Fibrosis 32
4.4.2 Total Prevalent Cases of Cystic Fibrosis Segmented by Age 34
4.4.3 Total Prevalent Cases of Cystic Fibrosis Segmented by Sex 35
4.4.4 Total Prevalent Cases of Cystic Fibrosis by Mutation Type 35
4.5 Discussion 37
4.5.1 Conclusion on Epidemiology Trends 37
4.5.2 Limitations of the Analysis 37
4.5.3 Strengths of the Analysis 38
5 Current Treatment Options 39
5.1 Overview 39
5.2 Product Profiles – Major Brands, Inhaled Antibiotics 41
5.2.1 TOBI (tobramycin) 41
5.2.2 TOBI Podhaler (tobramycin inhalation powder) 44
5.2.3 Bramitob (tobramycin) 50
5.2.4 Colistimethate Sodium (nebulized; numerous generic names) 53
5.2.5 Colobreathe (colistimethate sodium dry powder) 56
5.2.6 Cayston (aztreonam) 59
5.3 Product Profiles – Major Brands, Mucolytics 63
5.3.1 Pulmozyme (dornase alfa) 63
5.3.2 Bronchitol (mannitol) 66
5.4 Product Profiles – Major Brands, CFTR Modulators 71
5.4.1 Kalydeco (ivacaftor, VX-770) 71
5.5 Product Profiles – Major Brands, Other Therapies 77
5.5.1 Pancreatic Enzyme Replacement Therapies (PERTs) 77
6 Unmet Needs Assessment and Oppportunity Analysis 80
6.1 Overview 80
6.2 Unmet Needs Analysis 81
6.2.1 The Development of Curative Therapies 81
6.2.2 Improving Treatment of CF-related Lung Infections 82
6.2.3 Improving Airway Clearance with Mucolytic Agents 83
6.2.4 Improving Treatment Compliance 84
6.2.5 The Development of Safe Anti-inflammatory Therapies 85
6.3 Opportunity Analysis 86
6.3.1 Therapies that Target CFTR Protein Function 86
6.3.2 New Classes and Formulations of Inhaled Antibiotics 86
6.3.3 Novel Mucolytic Agents 87
6.3.4 Novel Anti-inflammatory Agents 88
7 R&D Strategies 90
7.1 Overview 90
7.1.1 Reformulation Strategies 90
7.1.2 Personalized Treatment Approach 90
7.1.3 Diverse Proof-of-Concept Research 91
7.1.4 Licensing and Alliances 92
7.2 Clinical Trial Design 92
8 Pipeline Assessment 95
8.1 Overview 95
8.2 Promising Drugs in Clinical Development 95
8.3 Promising Drugs in Clinical Development, Inhaled Antibiotics 96
8.3.1 Arikayce (amikacin) 96
8.4 Promising Drugs in Clinical Development, CFTR Modulators 101
8.4.1 Lumacaftor (VX-809)/Kalydeco (VX-770) 101
8.4.2 Ataluren (PTC124) 105
8.5 Innovative Early-Stage Approaches 109
9 Pipeline Valuation Analysis 113
9.1 Clinical Benchmark of Key Pipeline Drugs 113
9.2 Commercial Benchmark of Key Pipeline Drugs 114
9.3 Competitive Assessment 116
9.4 Top-Line Six-Year Forecast 117
9.4.1 US 119
9.4.2 5EU 120
10 Appendix 122
10.1 Bibliography 122
10.2 Abbreviations 137
10.3 Methodology 140
10.4 Forecasting Methodology 140
10.4.1 Diagnosed CF Patients 140
10.4.2 Percent Drug-treated Patients 140
10.4.3 Drugs Included in Each Therapeutic Class 141
10.4.4 Launch and Patent Expiry Dates 141
10.4.5 General Pricing Assumptions 142
10.4.6 Drug Assumptions 142
10.4.7 Generic Erosion 144
10.4.8 Pricing of Pipeline Agents 144
10.5 Physicians and Specialists Included in this Study 145
10.6 About the Authors 146
10.6.1 Analyst 146
10.6.2 Reviewer 146
10.6.3 Epidemiologist 147
10.6.4 Global Head of Healthcare 147
10.7 About GlobalData 148
10.8 Disclaimer 148