クッシング症候群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 14

2 Introduction 16
2.1 Catalyst 16
2.2 Related Reports 17
2.3 Upcoming Related Reports 17

3 Disease Overview 18
3.1 Etiology and Pathophysiology 19
3.2 Prognosis 21
3.3 Quality of Life 22
3.4 Symptoms 23
3.5 Diagnosis 25

4 Epidemiology 30
4.1 Disease Background 30
4.2 Risk Factors and Comorbidities 31
4.3 Global Trends 32
4.3.1 US 32
4.3.2 5EU 32
4.4 Forecast Methodology 33
4.4.1 Sources Used 36
4.4.2 Sources Not Used 38
4.4.3 Forecast Assumptions and Methods – Prevalent Cases 39
4.4.4 Forecast Assumptions and Methods – Immediate TSS Outcomes 40
4.5 Epidemiological Forecast for Cushing’s Syndrome (2013-2023) 40
4.5.1 Cushing’s Syndrome 40
4.5.2 Cushing’s Disease 46
4.5.3 Ectopic Adrenocorticotropic Hormone Cushing’s Syndrome 51
4.5.4 Adrenal Adenoma Cushing’s Syndrome 57
4.5.5 Adrenal Carcinoma Cushing’s Syndrome 62
4.5.6 Age-Standardized Diagnosed Prevalence Rates 67
4.5.7 Immediate Transsphenoidal Adenomectomy Surgery Outcomes 69
4.6 Discussion 70
4.6.1 Epidemiological Forecast Insight 70
4.6.2 Limitations of the Analysis 71
4.6.3 Strengths of the Analysis 72

5 Current Treatment Options 73
5.1 Overview 73
5.2 Treatment Approach 74
5.3 Product Profiles – Major Brands 78
5.3.1 Signifor (pasireotide) and Signifor LAR (pasireotide) 78
5.3.2 Korlym (mifepristone) 87
5.3.3 Ketoconazole and Ketoconazole HRA (ketoconazole) 96
5.3.4 Metopirone (metyrapone) 103
5.3.5 Lysodren (mitotane) 109

6 Unmet Needs Assessment and Oppportunity Analysis 114
6.1 Overview 114
6.2 Unmet Needs Analysis 115
6.2.1 Unmet Need: Effective Drugs Directed against the Underlying Cause of Cushing’s Disease 115
6.2.2 Unmet Need: Few Drugs – Especially Those for Severe Cushing’s Syndrome – Possess Favorable Safety and Efficacy 116
6.2.3 Unmet Need: Physician Awareness of Cushing’s Syndrome Symptomatology 118
6.2.4 Unmet Need: Accelerated Referral Systems to Specialists in Secondary and Tertiary Care Settings 119
6.2.5 Unmet Need: Uniform Guidelines Unambiguously Defining Treatment and Remission 121
6.3 Opportunity Analysis 121
6.3.1 Opportunity: Development of Improved Adrenocorticotropic Hormone Modulator Drugs 121
6.3.2 Opportunity: Reformulation of the Steroidogenic Inhibitors into Longer-Acting Release Treatments 122
6.3.3 Opportunity: Development of Universally Accepted Tests and Measures for Better Diagnosis and Monitoring of Cushing’s Syndrome Activity and Treatment Response 122
6.3.4 Opportunity: Prospective, Randomized Trials for Off-Label Cushing’s Syndrome Agents 123
6.3.5 Opportunity: Gaining Understanding of the Pathophysiology of Cushing’s Syndrome 124

7 Research and Development Strategies 125
7.1 Overview 125
7.1.1 Inhibitors of Cortisol Synthesis in the Adrenal Glands 125
7.1.2 Licensing Agreements, Partnerships, and Alliances 126
7.1.3 Secondary Indications 127
7.2 Clinical Trial Design 128
7.2.1 Selecting Suitable Comparator Arms 128
7.2.2 Evaluating and Determining Appropriate Clinical Trial Endpoints 129
7.2.3 Accurately Representing the Patient Population in Clinical Trials 131

8 Pipeline Assessment 135
8.1 Overview 135
8.2 Promising Drugs in Clinical Development 135
8.2.1 NormoCort (COR-003) 136
8.2.2 LCI699 141
8.3 Innovative Early-Stage Approaches 146
8.3.1 Somatoprim (DG3173) 147
8.3.2 Anti-Sense Technology and RNA Interference 147
8.3.3 Retinoic Acid 148
8.3.4 Other Early-Stage Approaches and Non-Pharmacotherapies 149

9 Pipeline Valuation Analysis 151
9.1 Clinical Benchmark of Key Pipeline Drugs 151
9.2 Commercial Benchmark of Key Pipeline Drugs 153
9.3 Competitive Assessment 154
9.4 Top-Line Five-Year Forecast 156
9.4.1 US 158
9.4.2 5EU 159

10 Appendix 161
10.1 Bibliography 161
10.2 Abbreviations 178
10.3 Methodology 180
10.4 Forecasting Methodology 180
10.4.1 Diagnosed Cushing’s Syndrome Patients 180
10.4.2 Percent Drug-Treated Patients 181
10.4.3 Drugs Included in Each Therapeutic Class 181
10.4.4 Launch and Patent Expiry Dates 182
10.4.5 General Pricing Assumptions 183
10.4.6 Individual Drug Assumptions 183
10.4.7 Pricing of Pipeline agents 187
10.5 Physicians and Specialists Included in This Study 189
10.6 About the Authors 190
10.6.1 Analyst 190
10.6.2 Therapy Director 190
10.6.3 Epidemiologist 191
10.6.4 Global Head of Healthcare 192
10.7 About GlobalData 193
10.8 Disclaimer 193