世界の移植片対宿主病治療薬市場...市場調査レポートについてご紹介

1 Table of Contents
1 Table of Contents 12
1.1 List of Tables 18
1.2 List of Figures 22
2 Introduction 23
2.1 Catalyst 23
2.2 Related Reports 24
2.3 Upcoming Related Reports 24
3 Disease Overview 25
3.1 Etiology and Pathophysiology 25
3.1.1 Etiology 25
3.1.2 Pathophysiology 27
3.1.3 Classification and Prognosis 28
3.2 Symptoms 30
4 Epidemiology 32
4.1 Disease Background 32
4.2 Risk Factors and Comorbidities 33
4.3 Global Trends 35
4.4 Forecast Methodology 36
4.4.1 Sources Used 38
4.4.2 Sources Not Used 44
4.4.3 Diagnosed Incident Cases of HSCTs 45
4.4.4 Diagnosed Incident Cases of Autologous HSCTs 47
4.4.5 Diagnosed Incident Cases of Allogeneic HSCTs 48
4.4.6 Diagnosed Incident Cases of aGVHD 49
4.4.7 Four-Year Diagnosed Prevalent Cases of aGVHD 49
4.4.8 Diagnosed Incident Cases of cGVHD 50
4.4.9 Five-Year Diagnosed Prevalent Cases of cGVHD 50
4.5 Epidemiological Forecast for GVHD (2013-2023) 51
4.5.1 Diagnosed Incident Cases of HSCTs 51
4.5.2 Diagnosed Incident Cases of Autologous HSCTs 53
4.5.3 Diagnosed Incident Cases of Allogeneic HSCTs 54
4.5.4 Diagnosed Incident Cases of aGVHD 56
4.5.5 Four-Year Diagnosed Prevalent Cases of aGVHD 58
4.5.6 Diagnosed Incident Cases of cGVHD 60
4.5.7 Five-Year Diagnosed Prevalent Cases of cGVHD 61
4.6 Discussion 63
4.6.1 Epidemiological Forecast Insight 63
4.6.2 Limitations of the Analysis 64
4.6.3 Strengths of the Analysis 65
5 Current Treatment Options 66
5.1 Overview 66
5.2 Product Profiles – Major Therapies 68
5.2.1 Methylprednisolone (numerous brand and generic names) 68
5.2.2 Cyclosporine (numerous brands and generic names) 72
5.2.3 Anti-Thymocyte Globulin (ATG) Therapies 77
5.3 Product Profiles – Off-Label Therapies 82
5.3.1 Biologics (Tumor Necrosis Factor (TNF)α Inhibitors, Interleukin-2 Receptor α (CD25) Inhibitors, Co-Stimulatory Blockers and Other Biologics) 82
5.3.2 Small Immunosuppressive Molecules (mTOR Inhibitors, Solid Organ Transplant Therapies, and Anti-Neoplastic Therapies) 101
6 Unmet Need and Opportunity 116
6.1 Overview 116
6.2 Lack of Randomized Clinical Trials 119
6.2.1 Unmet Need 119
6.2.2 Gap Analysis 119
6.2.3 Opportunity 120
6.3 No Consensus Regarding Clinical Trial Endpoints 120
6.3.1 Unmet Need 120
6.3.2 Gap Analysis 120
6.3.3 Opportunity 121
6.4 Optimization of Prophylactic Measures 121
6.4.1 Unmet Need 121
6.4.2 Gap Analysis 122
6.4.3 Opportunity 122
6.5 Lack of Standardized Protocols for Established and Off-Label Therapies 123
6.5.1 Unmet Need 123
6.5.2 Gap Analysis 123
6.5.3 Opportunity 124
6.6 Improved Treatment Outcomes for Patients Who Develop Toxicities 124
6.6.1 Unmet Need 124
6.6.2 Gap Analysis 125
6.6.3 Opportunity 125
6.7 Desire for Treatments that Exhibit Longer-Lasting Efficacy Profiles 125
6.7.1 Unmet Need 125
6.7.2 Gap Analysis 126
6.7.3 Opportunity 127
6.8 Management of SR-aGVHD Patients 127
6.8.1 Unmet Need 127
6.8.2 Gap Analysis 128
6.8.3 Opportunity 128
6.9 Management of Sclerotic cGVHD Patients 128
6.9.1 Unmet Need 128
6.9.2 Gap Analysis 129
6.9.3 Opportunity 129
6.10 Improved Prognosis for Patients with Lung Involvement 130
6.10.1 Unmet Need 130
6.10.2 Gap Analysis 130
6.10.3 Opportunity 131
7 Research and Development Strategies 132
7.1 Overview 132
7.1.1 Improvement of Prophylactic Regimens 132
7.1.2 Inhibition of T-Cell Proliferation and Activation 133
7.1.3 Adopting Treatments from Hematologic Indications 134
7.1.4 Targeting Niche Subgroups Within GVHD 135
7.2 Current Clinical Trial Design 136
7.2.1 Current Trial Designs are Dependent on the Stage Within the Treatment Algorithm That a Product is Targeting 136
7.2.2 Lack of Consensus on Clinical Endpoints in Current Trial Designs 137
7.2.3 Patient Exclusion Issues in Current Trial Designs 137
7.2.4 Current Trial Design of Key Pipeline Products 137
7.3 Future Clinical Trial Design 139
7.3.1 Future Trial Designs Need to Incorporate Randomization 139
7.3.2 Future Trial Designs Need to Incorporate Conditioning Regimens and Hematological Patients 140
7.3.3 Companies Behind Off-Label Therapies Should Strategize to Conduct Randomized Studies 140
7.3.4 Design of Early-Phase Clinical Trials for Cellular and Gene Therapy Products Accommodates 2014 Guidance from the FDA 140
8 Pipeline Assessment 141
8.1 Overview 141
8.2 Promising Drugs in Clinical Development 142
8.2.1 Leukotac (inolimomab) 142
8.2.2 Begedina (BT 5/9) 147
8.2.3 Budenofalk (budesonide) 150
8.2.4 ATG-Fresenius (EZ-2053) 153
8.2.5 Prochymal (remestemcel-L) 156
8.2.6 MultiStem (modified mesenchymal stem cells) 161
8.2.7 Uvadex (Extracorporeal Photophoresis) 164
8.2.8 orBec (beclomethasone dipropionate) 168
8.3 Innovative Early-Stage Approaches 171
8.3.1 Targeting Regulatory T Cells 174
8.3.2 Mesenchymal Stem Cell Therapies 175
8.3.3 Immunomodulatory Cell Surface Receptor Inhibitors 176
8.3.4 IL-6 Inhibitors 177
9 Pipeline and Off-Label Valuation Analysis 178
9.1 Clinical Benchmarking of Key Pipeline and Off-Label Drugs 178
9.1.1 GVHD Prophylaxis 178
9.1.2 Acute GVHD 181
9.1.3 Chronic GVHD 185
9.2 Commercial Benchmarking of Key Pipeline and Off-Label Drugs 186
9.2.1 GVHD Prophylaxis 187
9.2.2 Acute GVHD 189
9.2.3 Chronic GVHD 191
9.3 Competitive Assessment 193
9.3.1 GVHD Prophylaxis 193
9.3.2 Acute GVHD 194
9.3.3 Chronic GVHD 196
9.4 Top-Line Five-Year Forecast 198
9.4.1 US 203
9.4.2 5EU 207
10 Appendix 211
10.1 Bibliography 211
10.2 Abbreviations 230
10.3 Methodology 236
10.4 Forecasting Methodology 236
10.4.1 Diagnosed GVHD Patients 236
10.4.2 Percent Drug-Treated Patients 237
10.4.3 Drugs Included in Each Therapeutic Class 237
10.4.4 Launch and Patent Expiry Dates 238
10.4.5 General Pricing Assumptions 238
10.4.6 Individual Drug Assumptions 239
10.4.7 Generic Erosion 246
10.4.8 Pricing of Pipeline Agents 246
10.5 Physicians and Specialists Included in This Study 247
10.6 About the Authors 249
10.6.1 Author 249
10.6.2 Reviewer 249
10.6.3 Epidemiologist 250
10.6.4 Global Head of Healthcare 250
10.7 About GlobalData 251
10.8 Disclaimer 251