Published : Feb-2017
No. of Pages : 243
1 Table of Contents 8
1.1 List of Tables 13
1.2 List of Figures 16
2 Introduction 18
2.1 Catalyst 18
2.2 Related Reports 18
2.3 Upcoming Related Reports 19
3 Disease Overview 20
3.1 Etiology and Pathophysiology 20
3.1.1 Etiology 20
3.1.2 Pathophysiology 22
3.2 Phases and Severity of ITP 24
3.3 Symptoms 25
3.4 Prognosis 26
3.5 Quality of Life 27
4 Epidemiology 29
4.1 Disease Background 29
4.2 Risk Factors and Comorbidities 29
4.3 Global Trends 31
4.3.1 US 33
4.3.2 5EU 34
4.3.3 Japan 35
4.4 Forecast Methodology 35
4.4.1 Sources Used Tables 36
4.4.2 Forecast Assumptions and Methods 39
4.4.3 Sources Not Used 47
4.5 Epidemiological Forecast for ITP (2015-2025) 48
4.5.1 Diagnosed Prevalent Cases of ITP 48
4.5.2 Age-Specific Diagnosed Prevalent Cases of ITP 52
4.5.3 Sex-Specific Diagnosed Prevalent Cases of ITP 56
4.5.4 Age-Standardized Diagnosed Prevalence of ITP 60
4.5.5 Diagnosed Incident Cases of ITP 63
4.5.6 Age-Specific Diagnosed Incident Cases of ITP 67
4.5.7 Sex-Specific Diagnosed Incident Cases of ITP 71
4.5.8 Age-Standardized Diagnosed Incidence of ITP 75
4.6 Discussion 77
4.6.1 Epidemiological Forecast Insight 77
4.6.2 Limitations of the Analysis 79
4.6.3 Strengths of the Analysis 80
5 Current Treatment Options 81
5.1 Overview 81
5.2 Diagnosis and Treatment 81
5.2.1 Diagnosis 82
5.2.2 Treatment Guidelines and Leading Prescribed Drugs 85
5.3 Clinical Practice 86
5.3.1 Decision on the Initiation of Treatment 86
5.3.2 First-Line Treatment 89
5.3.3 Second-Line Therapy 96
5.4 Product Profiles - Thrombopoietin Receptor Agonists 108
5.4.1 Nplate (Romiplostim) 108
5.4.2 Promacta/Revolade (Eltrombopag) 123
6 Unmet Needs Assessment and Opportunity Analysis 139
6.1 Overview 139
6.2 Safer and More Efficacious First-Line Therapy 139
6.2.1 Unmet Need 139
6.2.2 Gap Analysis 140
6.2.3 Opportunity 141
6.3 High Treatment Cost of Thrombopoietin Receptor Agonists 141
6.3.1 Unmet Need 141
6.3.2 Gap Analysis 142
6.3.3 Opportunity 143
6.4 Second-Line Therapeutic Options that Offer Long-Term Remission 143
6.4.1 Unmet Need 143
6.4.2 Gap Analysis 144
6.4.3 Opportunity 145
6.5 New Treatment for Multiple Drug Refractory Patients 146
6.5.1 Unmet Need 146
6.5.2 Gap Analysis 146
6.5.3 Opportunity 147
6.6 Difficulties in Clinical Trial Patient Recruitment 147
6.6.1 Unmet Need 147
6.6.2 Gap Analysis 148
6.6.3 Opportunity 149
7 Research and Development Strategies 150
7.1 Overview 150
7.1.1 Enlarging Eligible Patient Pool Through Label Expansion to Pediatric Patients 150
7.1.2 Targeting New Mechanisms of Action to Increase Competitiveness 151
7.2 Clinical Trial Design 151
7.2.1 Historical Lack of Consistency in Patient Selection Criteria 153
7.2.2 Inconsistent Timeframe for Endpoint Measurement and Study Duration 154
7.2.3 Selection of Suitable Comparator 155
7.2.4 Other Clinical Trial Requirements 155
8 Pipeline Assessment 157
8.1 Overview 157
8.2 Promising Drugs in Clinical Development 158
8.2.1 Avatrombopag 158
8.2.2 Fostamatinib (R-788) 165
8.3 Innovative Early-Stage Approaches 173
8.3.1 Overview 173
8.3.2 CD40/CD40L Antagonists 175
8.3.3 Anti-CD20 Monoclonal Antibodies 176
8.3.4 Thrombopoietin Receptor Agonists 177
8.3.5 Syk Inhibitors 178
8.3.6 Other Approaches 178
8.4 Biosimilars 179
8.5 Other Drugs in Development 180
9 Pipeline Valuation Analysis 181
9.1 Clinical Benchmark of Key Pipeline Drugs 181
9.2 Commercial Benchmark of Key Pipeline Drugs 183
9.3 Competitive Assessment 184
9.4 Top-Line 10-Year Forecast 185
9.4.1 US 191
9.4.2 5EU 193
9.4.3 Japan 195
10 Appendix 197
10.1 Bibliography 197
10.2 Abbreviations 218
10.3 Methodology 223
10.4 Forecasting Methodology 223
10.4.1 Diagnosed Immune Thrombocytopenia Patients 223
10.4.2 Percent Drug-Treated Patients 224
10.4.3 Drugs Included in Each Therapeutic Class 224
10.4.4 Launch and Patent Expiry Dates 225
10.4.5 General Pricing Assumptions 226
10.4.6 Individual Drug Assumptions 227
10.4.7 Generic Erosion 233
10.4.8 Pricing of Pipeline Agents 233
10.5 Primary Research - KOLs Interviewed for this Report 235
10.6 Primary Research - Prescriber Survey 237
10.7 About the Authors 238
10.7.1 Analyst 238
10.7.2 Therapy Area Directors 238
10.7.3 Epidemiologists 239
10.7.4 Epidemiology Reviewer 240
10.7.5 Director of Epidemiology 240
10.7.6 Global Director of Therapy Analysis and Epidemiology 241
10.8 About GlobalData 242
10.9 Disclaimer 242
1.1 List of Tables
Table 1: Secondary Causes of ITP 23
Table 2: Phases and Severity of ITP 26
Table 3: Symptoms of ITP 27
Table 4: Risk Factors and Comorbidities for ITP 31
Table 5: 7MM, Sources for Diagnosed Prevalence of Primary ITP 38
Table 6: 7MM, Sources for Diagnosed Prevalence of Secondary ITP 39
Table 7: 7MM, Sources for Diagnosed Incidence of Primary ITP 39
Table 8: 7MM, Sources for Diagnosed Incidence of Secondary ITP 40
Table 9: 7MM, Sources Not Used in the Epidemiological Analysis of ITP 49
Table 10: 7MM, Diagnosed Prevalent Cases of Primary ITP, All Ages, Men and Women, Select Years, 2015-2025 50
Table 11: 6MM, Diagnosed Prevalent Cases of Secondary ITP, All Ages, Men and Women, Select Years, 2015-2025 52
Table 12: 7MM, Age-Specific Diagnosed Prevalent Cases of Primary ITP, Men and Women, N (Row %), 2015 54
Table 13: 6MM, Age-Specific Diagnosed Prevalent Cases of Secondary ITP, Men and Women, N (Row %), 2015 56
Table 14: 7MM, Sex-Specific Diagnosed Prevalent Cases of Primary ITP, All Ages, N (Row %), 2015 58
Table 15: 6MM, Sex-Specific Diagnosed Prevalent Cases of Secondary ITP, All Ages, N, 2015 60
Table 16: 7MM, Diagnosed Incident Cases of Primary ITP, All Ages, Men and Women, Select Years, 2015-2025 65
Table 17: 6MM, Diagnosed Incident Cases of Secondary ITP, All Ages, Men and Women, Select Years, 2015-2025 67
Table 18: 7MM, Age-Specific Diagnosed Incident Cases of Primary ITP, Men and Women, N (Row %), 2015 69
Table 19: 6MM, Age-Specific Diagnosed Incident Cases of Secondary ITP, Men and Women, N (Row %), 2015 71
Table 20: 7MM, Sex-Specific Diagnosed Incident Cases of Primary ITP, All Ages, N (Row %), 2015 73
Table 21: 6MM, Sex-Specific Diagnosed Incident Cases of Secondary ITP, All Ages, N (Row %), 2015 75
Table 22: Non-Immunologic Causes of Thrombocytopenia 84
Table 23: Clinical Guidelines for ITP 86
Table 24: Leading Treatments for ITP, 2016 87
Table 25: Corticosteroid Therapy for First-Line ITP Treatment 92
Table 26: Efficacy of Rituxan in the Second-Line Setting 106
Table 27: Other Second-Line Options for ITP Treatment 109
Table 28: Product Profile - Nplate 110
Table 29: Efficacy of Nplate in Pivotal Trials 116
Table 30: Long-Term Efficacy of Nplate in Open-Label Studies 118
Table 31: Efficacy of Nplate Compared with SOC in Non-Splenectomized Patients 119
Table 32: Efficacy of Nplate in the NCT01444417 Trial 120
Table 33: Summary of AEs from the Two Pivotal Trials of Nplate 121
Table 34: Summary of AEs in the NCT01444417 Trial 123
Table 35: Nplate SWOT Analysis, 2016 124
Table 36: Product Profile - Promacta 125
Table 37: Efficacy of Promacta in the NCT00102739 Trial 130
Table 38: Efficacy of Promacta in the RAISE Trial 131
Table 39: Efficacy of Promacta in the PETIT Trial 133
Table 40: Age-Stratified Efficacy of Promacta in the PETIT Trial 133
Table 41: Efficacy of Promacta in the PETIT2 Trial 134
Table 42: Age-Stratified Efficacy of Promacta in the PETIT2 Trial 135
Table 43: Summary of AEs from the Pivotal Trials of Promacta in Adults 137
Table 44: Summary of AEs from the Pivotal Trials of Promacta in Children 138
Table 45: Promacta SWOT Analysis, 2016 139
Table 46: Unmet Need and Opportunity in ITP, 2016 140
Table 47: Phase III Pipeline Clinical Trial Design for ITP 153
Table 48: Key Late-Stage Pipeline Agents for ITP, 2016 159
Table 49: Product Profile - Avatrombopag 160
Table 50: Efficacy of Avatrombopag in the Amendment 02 Trial 163
Table 51: Efficacy of Avatrombopag in the NCT00441090 Trial 163
Table 52: Summary of AEs from Two Phase II Trials of Avatrombopag 165
Table 53: Avatrombopag SWOT Analysis, 2016 166
Table 54: Product Profile - Fostamatinib 167
Table 55: Efficacy of Fostamatinib in ITP 170
Table 56: Safety of Fostamatinib in ITP 172
Table 57: Fostamatinib SWOT Analysis, 2016 174
Table 58: Early-Stage Clinical and Preclinical ITP Pipeline 175
Table 59: Discontinued or Inactive ITP Pipeline 176
Table 60: Clinical Benchmark of Key Pipeline Drugs - ITP 182
Table 61: Commercial Benchmark of Key Pipeline Drugs - ITP 184
Table 62: Top-Line Sales Forecasts ($M) for ITP, 2015-2025 187
Table 63: Key Events Impacting Sales for ITP, 2015-2025 191
Table 64: ITP Market - Global Drivers and Barriers, 2015?2025 191
Table 65: Key Historical and Projected Launch Dates for ITP Therapeutics 226
Table 66: Key Historical and Projected Patent Expiry Dates for ITP Therapeutics 226
Table 67: Average Adult Body Weight and Surface Area Across the 7MM 228
Table 68: High-Prescribing Physicians (non-KOLs) Surveyed, By Country 238
1.2 List of Figures
Figure 1: Diagnosed Prevalence Rate for ITP in the 7MM for All Ages, Men and Women, Cases per 100,000 Population, 2015 33
Figure 2: Diagnosed Incidence Rate for ITP in the 7MM for All Ages, Men and Women, Cases per 100,000 Population, 2015 34
Figure 3: Case Flow Map 37
Figure 4: 7MM, Diagnosed Prevalent Cases of Primary ITP Cases, All Ages, Men and Women, N, Select Years, 2015-2025 51
Figure 5: 6MM, Diagnosed Prevalent Cases of Secondary ITP Cases, All Ages, Men and Women, N, Select Years, 2015-2025 53
Figure 6: 7MM, Age-Specific Diagnosed Prevalent Cases of Primary ITP, Men and Women, N, 2015 55
Figure 7: 6MM, Age-Specific Diagnosed Prevalent Cases of Secondary ITP, Men and Women, N, 2015 57
Figure 8: 7MM, Sex-Specific Diagnosed Prevalent Cases of Primary ITP, All Ages, N, 2015 59
Figure 9: 6MM, Sex-Specific Diagnosed Prevalent Cases of Secondary ITP, All Ages, N, 2015 61
Figure 10: 7MM, Age-Standardized Diagnosed Prevalence of Primary ITP, All Ages, Cases per 100,000 population, 2015 62
Figure 11: 6MM, Age-Standardized Diagnosed Prevalence of Secondary ITP, All Ages, Cases per 100,000 population, 2015 63
Figure 12: 7MM, Diagnosed Incident Cases of Primary ITP, All Ages, Men and Women, N, Select Years, 2015-2025 65
Figure 13: 6MM, Diagnosed Incident Cases of Secondary ITP, All Ages, Men and Women, N, Select Years, 2015-2025 67
Figure 14: 7MM, Age-Specific Diagnosed Incident Cases of Primary ITP, Men and Women, N, 2015 70
Figure 15: 6MM, Age-Specific Diagnosed Incident Cases of Secondary ITP, Men and Women, N, 2015 72
Figure 16: 7MM, Sex-Specific Diagnosed Incident Cases of Primary ITP, All Ages, N, 2015 74
Figure 17: 6MM, Sex-Specific Diagnosed Incident Cases of Secondary ITP, All Ages, N, 2015 76
Figure 18: 7MM, Age-Standardized Diagnosed Incidence of Primary ITP, All Ages, Cases per 100,000 Population, 2015 77
Figure 19: 6MM, Age-Standardized Diagnosed Incidence of Secondary ITP, All Ages, Cases per 100,000 Population, 2015 78
Figure 20: Nplate's Development in ITP 113
Figure 21: Development of Promacta in ITP 127
Figure 22: ITP - Phase III Pipeline, 2016 159
Figure 23: Avatrombopag's Development in ITP 162
Figure 24: Fostamatinib's Development in ITP 169
Figure 25: Competitive Assessment of Pipeline Agents in ITP, 2015-2025 186
Figure 26: Top-Line Sales for ITP by Region, 2015 and 2025 188
Figure 27: Top-Line Sales for ITP by Region, 2015-2025 189
Figure 28: Global Sales for ITP by Drug Class, 2015 and 2025 190
Figure 29: Sales for ITP by Drug Class in the US, 2015 and 2025 193
Figure 30: Sales for ITP by Drug Class in the 5EU, 2015 and 2025 195
Figure 31: Sales for ITP by Drug Class in the Japan, 2015 and 2025 197
