Acute Myeloid Leukemia (AML)：市場分析及び予測（～2017）...市場調査レポートについてご紹介

1 Table of Contents
1 Table of Contents 9
1.1 List of Tables 14
1.2 List of Figures 17
2 Introduction 18
2.1 Catalyst 18
2.2 Upcoming Related Reports 18
3 Disease Overview 19
3.1 Etiology and Pathophysiology 19
3.1.1 Etiology 19
3.1.2 Pathophysiology 20
3.1.3 Classification 21
3.1.4 Cytogenetic and Molecular Abnormalities 23
3.1.5 Prognosis 30
3.1.6 Quality of Life 30
3.2 Symptoms 31
4 Epidemiology 32
4.1 Risk Factors and Comorbidities 33
4.1.1 Increased age is associated with increased risk and worsened prognosis 34
4.1.2 Smoking increases risk of M2 AML 35
4.1.3 APL is more common among Spanish and Italian origins 36
4.1.4 Chemotherapy and radiation therapy increases risk of AML 36
4.1.5 Benzene increases risk of AML 37
4.2 Global Trends 38
4.2.1 Incidence 38
4.2.2 Survival and Prevalence 40
4.2.3 Subtypes and Mutations 41
4.2.4 Risk Groups 43
4.3 Forecast Methodology 46
4.3.1 Sources Used 47
4.3.2 Forecast Assumptions and Methods 53
4.3.3 Sources Not Used 57
4.4 Epidemiological Forecast of Acute Myeloid Leukemia (2012–2022) 58
4.4.1 Incident Cases of AML 58
4.4.2 Incident Cases of AML by Age 59
4.4.3 Incident Cases of AML by Sex 61
4.4.4 Age-Standardized Incidence of AML 63
4.4.5 Incident Cases of AML Subtypes 64
4.4.6 Incident Cases of AML Subtypes by Age 68
4.4.7 Incident Cases of AML with Mutations in the FLT3 Gene 69
4.4.8 Incident Cases of AML by Risk Group Classifications 71
4.4.9 Five-Year Prevalent Cases of AML 75
4.4.10 Five-Year Prevalent Cases of AML by Age 78
4.4.11 Five-Year Prevalent Cases of APL and MDS/Therapy-Related AML 79
4.4.12 Five-Year Prevalent Cases of APL and MDS/Therapy-Related AML by Age 81
4.5 Discussion 83
4.5.1 Epidemiological Forecast Insight 83
4.5.2 Limitations of the Analysis 84
4.5.3 Strengths of the Analysis 84
5 Disease Management 85
5.1 Diagnosis 85
5.2 Response Criteria 86
5.3 General Treatment Overview 87
5.3.1 Younger AML Patients 89
5.3.2 Older AML Patients 91
5.4 Hematopoietic Stem Cell Transplant 93
5.5 Monitoring for Residual Disease 95
5.6 APL 96
6 Current Treatment Options 98
6.1 Overview 98
6.2 Product Profiles – Major Brands 101
6.2.1 Cytarabine 101
6.2.2 Dacogen (decitabine) 105
6.2.3 Vidaza (azacitidine) 110
6.2.4 Other Therapeutic Agents 115
7 Unmet Needs Assessment and Opportunity Analysis 117
7.1 Overview 117
7.2 Unmet Needs Analysis 118
7.2.1 Unmet Need: Therapies that Prolong the OS of AML Patients 118
7.2.2 Unmet Need: The Elimination of Residual Disease 120
7.2.3 Unmet Need: Therapies that Achieve Durable CR in AML Patients 121
7.2.4 Unmet Need: Safety 121
7.2.5 Unmet Need: Maintenance Therapies 122
7.2.6 Unmet Need: Guidelines Directing the Optimum Course of Therapy 123
7.2.7 Unmet Need: Therapies with More Convenient Dosing Regimens 123
7.2.8 Unmet Need: Lower Cost of Therapy 124
7.3 Opportunity Analysis 124
7.3.1 Opportunity: Targeting Minimal Residual Disease (Leukemic Stem Cells) 124
7.3.2 Opportunity: Targeting Driver Molecular Mutations with Combination Therapy 125
7.3.3 Opportunity: Development of Immunotherapies for AML 126
7.3.4 Opportunity: Differentiation Agents 126
7.3.5 Opportunity: Development of Oral Maintenance Therapies 127
8 R&D Strategies 128
8.1 Overview 128
8.1.1 Targeting Multiple Patient Segments 128
8.1.2 Developing Novel Drugs in Combination with Established Therapies 129
8.1.3 Targeting Elderly AML Patients 130
8.1.4 Investigating New Targets Involved in the Pathogenesis of AML 130
8.1.5 Alliances with Academic Groups 131
8.2 Clinical Trial Design 133
8.2.1 Selection of the Appropriate Efficacy Endpoints to Support Regulatory Approval 133
8.2.2 Selection of Active Comparator 136
8.2.3 Randomization and Stratification of Patients 137
8.2.4 Current Clinical Trial Design 139
8.2.5 Future Trends in Clinical Trial Design 141
9 Pipeline Assessment 144
9.1 Overview 144
9.2 Promising Drugs in Clinical Development 145
9.2.1 CC-486 (oral azacitidine) 146
9.2.2 CPX-351 149
9.2.3 Midostaurin 154
9.2.4 Quizartinib 158
9.2.5 Sapacitabine 164
9.2.6 Volasertib 168
9.2.7 Vosaroxin 172
9.3 Innovative Early-stage Approaches 176
9.3.1 Biologic-Targeted Therapy 177
9.3.2 Immunotherapies 179
9.3.3 Epigenetic Approaches 181
10 Pipeline Valuation Analysis 183
10.1 Clinical Benchmark of Key Pipeline Drugs 184
10.2 Commercial Benchmark of Key Pipeline Drugs 187
10.3 Competitive Assessment 189
10.4 Top Line Five-Year Forecast 190
10.4.1 US 192
10.4.2 5EU 194
11 Appendix 197
11.1 Bibliography 197
11.2 Abbreviations 215
11.3 Methodology 217
11.4 Forecasting Methodology 217
11.4.1 Diagnosed AML Patients 217
11.4.2 Percent Drug-treated Patients 218
11.4.3 Drugs Included in Each Therapeutic Class 218
11.4.4 Launch and Patent Expiry Dates 218
11.4.5 General Pricing Assumptions 219
11.4.6 Individual Drug Assumptions 220
11.4.7 Generic Erosion 223
11.4.8 Pricing of Pipeline Agents 223
11.5 Physicians and Specialists Included in this Study 224
11.6 About the Authors 226
11.6.1 Authors 226
11.6.2 Epidemiologist 227
11.6.3 Global Head of Healthcare 227
11.7 About GlobalData 228
11.8 Disclaimer 228