ファブリー病治療薬の世界市場機会分析...市場調査レポートについてご紹介

1 Table of Contents
1 Table of Contents 8
1.1 List of Tables 12
1.2 List of Figures 13
2 Introduction 14
2.1 Catalyst 14
2.2 Related Reports 14
3 Disease Overview 15
3.1 Etiology and Pathophysiology 15
3.1.1 Etiology 15
3.1.2 Pathophysiology 15
3.2 Symptoms 18
4 Epidemiology 19
4.1 Disease Background 19
4.2 Risk Factors and Comorbidities/Manifestations 20
4.3 Global Trends 20
4.3.1 US 20
4.3.2 5EU 20
4.3.3 Japan 21
4.4 Forecast Methodology 21
4.4.1 Sources Used 22
4.4.2 Sources Not Used 23
4.4.3 Forecast Assumptions and Methods – Diagnosed Prevalent Cases 24
4.5 Epidemiological Forecast for Fabry Disease (2014-2024) 26
4.5.1 Diagnosed Prevalent Cases of Fabry Disease 26
4.5.2 Age-Specific Diagnosed Prevalent Cases of Fabry Disease 27
4.5.3 Sex-Specific Diagnosed Prevalent Cases of Fabry Disease 29
4.6 Discussion 31
4.6.1 Epidemiological Forecast Insight 31
4.6.2 Limitations of the Analysis 32
4.6.3 Strengths of the Analysis 32
5 Current Treatment Options 34
5.1 Overview 34
5.2 Product Profiles 40
5.2.1 Fabrazyme (Agalsidase Beta) 40
5.2.2 Replagal (Agalsidase Alfa) 50
6 Unmet Needs Assessment and Opportunity Analysis 57
6.1 Overview 57
6.2 Earlier Fabry Disease Diagnosis 57
6.2.1 Unmet Need 57
6.2.2 Gap Analysis 59
6.2.3 Opportunity 61
6.3 Fabry Disease Treatments with Improved Efficacy 62
6.3.1 Unmet Need 62
6.3.2 Gap Analysis 64
6.3.3 Opportunity 65
6.4 Lower Cost of Fabry Treatments 66
6.4.1 Unmet Need 66
6.4.2 Gap Analysis 67
6.4.3 Opportunity 69
6.5 Widespread Availability of Home-Based Infusion to Improve Compliance 69
6.5.1 Unmet Need 69
6.5.2 Gap Analysis 70
6.5.3 Opportunity 72
7 Research and Development Strategies 73
7.1 Overview 73
7.1.1 Chaperone Therapies and Their Drug Combinations 73
7.1.2 Substrate Reduction Therapies 75
7.2 Clinical Trial Design 77
7.2.1 Efficacy Endpoints 77
7.2.2 Clinical Trial Treatment Periods 78
7.2.3 Challenges in Fabry Disease Clinical Trials 79
8 Pipeline Assessment 82
8.1 Overview 82
8.2 Promising Drugs in Clinical Development 83
8.2.1 Migalastat 83
8.3 Innovative Early-Stage Approaches 98
8.4 Biosimilars 99
9 Pipeline Valuation Analysis 103
9.1 Clinical Benchmark of Key Pipeline Drugs 103
9.2 Commercial Benchmark of Key Pipeline Drugs 104
9.3 Competitive Assessment 104
9.4 Top-Line 10-Year Forecast 107
9.4.1 US 110
9.4.2 5EU 110
9.4.3 Japan 111
9.4.4 Drivers and Barriers 113
10 Appendix 114
10.1 Bibliography 114
10.2 Abbreviations 121
10.3 Methodology 123
10.4 Forecast Methodology 123
10.4.1 Percent Diagnosed Patients 123
10.4.2 Percent Drug-Treated Patients 124
10.4.3 Drugs Included in Each Therapeutic Class 124
10.4.4 Launch Dates 124
10.4.5 General Pricing Assumptions 124
10.4.6 Individual Drug Assumptions 125
10.5 Physicians and Specialists Included in This Study 127
10.6 About the Authors 129
10.6.1 Analyst 129
10.6.2 Therapy Area Director 129
10.6.3 Epidemiologist 130
10.6.4 Global Director of Therapy Analysis and Epidemiology 130
10.6.5 Global Head of Healthcare 131
10.7 About GlobalData 132
10.8 Disclaimer 132