Orphan Disease Therapeutics Market to 2018 - Improved Understanding of Rare Diseases' Heterogeneity and Novel New Clinical Trial Designs to Foster Innovation



Summary



GBI Research, the leading business intelligence provider, has released its latest research “Orphan Disease Therapeutics Market to 2018 - Improved Understanding of Rare Diseases' Heterogeneity and Novel New Clinical Trial Designs to Foster Innovation”, which provides insights into the orphan disease therapeutics market until 2018. The report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GBI Research’s team of industry experts. The report provides an in-depth analysis of Fabry, Pompe, Mucopolysaccharidosis VI, Idiopathic Thrombocytopenic Purpura, Huntington’s Disease and Ovarian cancer. The report examines the global orphan diseases therapeutics treatment usage patterns. It includes the geographical distribution of Fabry, Pompe, Mucopolysaccharidosis VI, Idiopathic Thrombocytopenic Purpura, Huntington’s Disease and Ovarian cancer markets across the US, the top five countries of Europe and in Japan. The report provides competitive benchmarking for the leading companies and also analyzes the mergers, acquisitions and licensing agreements that shape the global markets.

GBI Research’s analysis shows that the overall global orphan disease therapeutics market is expected to grow at a significant compound annual growth rate (CAGR) of 13.1% from $2.3 billion 2010 to $6 billion in 2018 in the US, the top five countries of Europe and Japan. Increasing awareness of the disease and drugs among patients and physicians, patent protection and exclusivity of Nplate and Promacta for ITP market, anticipated launch of new molecules such as ACR-16, AMR-101 and HD-02 for the treatment of HD and the approval of Avastin for the treatment of advanced ovarian cancer in Europe will drive the global orphan disease therapeutics market in the forecast period.



Scope


- Annualized market data for the orphan diseases therapeutics market from 2004 to 2010, forecast forward to 2018.
- Analysis of the leading therapeutic segments. These include Fabry Disease, Pompe Disease and Mucopolysaccharidosis VI, Idiopathic Thrombocytopenic Purpura, Huntington’s disease and Ovarian Cancer.
- Analysis of the orphan diseases therapeutics market in the leading geographies of the world, which include the US, the UK, Germany, France, Italy, Spain, and Japan
- Market characterization of the orphan diseases therapeutics market including market size, annual cost of therapy, and treatment usage patterns
- Key drivers and barriers that have a significant impact on the market
- Coverage of pipeline molecules in various phases of drug development
- Competitive benchmarking of leading companies. The key companies studied in this report are Genzyme Corporation, Shire Plc and Biomarin.
- Key M&A activities, licensing agreements, that have taken place between 2010 and 2011 in the global orphan diseases therapeutics market.



Reasons to buy


- Align product portfolio to the markets with high growth potential
- Develop market-entry and market expansion strategies by identifying the leading therapeutic segments and geographic markets poised for strong growth.
- Reinforce R&D pipelines by identifying new target mechanisms which can produce first in class molecules with more efficiency and better safety
- Develop key strategic initiatives by understanding key focus areas of leading companies
- Exploit in-licensing and out-licensing opportunities by identifying products that could fill portfolio gaps

Table Of Contents

1 Table of Contents
1 Table of Contents 3
1.1 List of Tables 6
1.2 List of Figures 8
2 Orphan Disease Therapeutics Market to 2018 - Introduction 10
2.1 Orphan Drug Definitions 12
2.1.1 The US Orphan Drug Criteria 12
2.1.2 Europe Orphan Drug Criteria 12
2.1.3 Japanese Orphan Drug Criteria 12
2.1.4 Special Cases of Orphan Drug Exclusivity 12
2.1.5 Comparison of Orphan Drug Designations 12
2.2 GBI Research Report Guidance 13
3 Orphan Disease Therapeutics Market to 2018 - Market Characterization 14
3.1 Market Forecasts 14
3.2 Treatment Usage Patterns 15
3.2.1 Disease Population 16
3.2.2 Treatment Seeking Population 16
3.2.3 Diagnosis Population 17
3.2.4 Prescription Population 17
4 Orphan Disease Therapeutics Market to 2010 - Global Fabry’s Disease Therapeutics Market 18
4.1 Disease Overview 18
4.2 Market Overview 18
4.3 Revenue by Geographical Segmentation 19
4.4 Annual Cost of Treatment 20
4.5 Treatment Usage Patterns 21
4.5.1 Disease Population 22
4.5.2 Treatment Seeking Population 22
4.5.3 Diagnosis Population 22
4.5.4 Prescription Population 22
4.6 Marketed Product Analysis 23
4.6.1 Fabrazyme 23
4.6.2 Replagal 24
4.7 Drug Pipeline Analysis 25
4.7.1 Pipeline by Phase 25
5 Orphan Disease Therapeutics Market to 2018 - Global Pompe Disease Therapeutics Market 26
5.1 Disease Overview 26
5.2 Market Overview 26
5.3 Revenue by Geographical Segmentation 28
5.4 Annual Cost of Treatment 29
5.5 Treatment Usage Patterns 30
5.5.1 Disease Population 31
5.5.2 Treatment Seeking Population 31
5.5.3 Diagnosis Population 31
5.5.4 Prescription Population 32
5.6 Marketed Product Analysis 32
5.6.1 Myozyme/Lumizyme 32
5.7 Drug Pipeline Analysis 34
5.7.1 Pipeline by Phase 34
6 Orphan Disease Therapeutics Market to 2018 - Global Mucopolysaccharidosis VI Disease Therapeutics Market 35
6.1 Disease Overview 35
6.2 Market Overview 35
6.3 Revenue by Geographical Segmentation 37
6.4 Annual Cost of Treatment 38
6.5 Treatment Usage Patterns 39
6.5.1 Disease Population 40
6.5.2 Treatment Seeking Population 40
6.5.3 Diagnosis Population 40
6.5.4 Prescription Population 40
6.6 Marketed Product Analysis 41
6.6.1 Naglazyme 41
6.7 Drug Pipeline Analysis 41
7 Orphan Disease Therapeutics Market to 2018 - Global Idiopathic Thrombocytopenic Purpura Disease Therapeutics Market 42
7.1 Disease Overview 42
7.2 Market Overview 43
7.3 Revenue by Geographical Segmentation 45
7.4 Annual Cost of Treatment 46
7.5 Treatment Usage Patterns 47
7.6 Marketed Product Analysis 49
7.7 Drug Pipeline Analysis 50
7.7.1 Pipeline by Phase 51
8 Orphan Disease Therapeutics Market to 2018 - Global Huntington’s Disease Therapeutics Market 52
8.1 Disease Overview 52
8.2 Market Overview 53
8.3 Revenue by Geographical Segmentation 54
8.4 Annual Cost of Treatment 55
8.5 Treatment Usage Patterns 56
8.5.1 Disease Population 57
8.5.2 Treatment Seeking Population 57
8.5.3 Diagnosis Population 57
8.5.4 Prescription Population 57
8.6 Marketed Product Analysis 57
8.6.1 Xenazine 57
8.7 Drug Pipeline Analysis 58
8.7.1 Pipeline by Phase 58
9 Orphan Disease Therapeutics Market to 2018 - Global Ovarian Cancer Therapeutics Market 60
9.1 Disease Overview 60
9.2 Market Overview 60
9.3 Revenue by Geographical Segmentation 62
9.4 Average Cost of Therapy 63
9.5 Treatment Usage Patterns 64
9.5.1 Disease Population 65
9.5.2 Treatment Seeking Population 65
9.5.3 Diagnosis Population 65
9.5.4 Prescription Population 65
9.6 Marketed Product Analysis 66
9.6.1 Altretamine (hexalen) 66
9.6.2 Yondelis 66
9.6.3 Gemzar (gemcitabine) 66
9.7 Drug Pipeline Analysis 67
9.7.1 Pipeline by Phase 67
10 Orphan Disease Therapeutics Market to 2018 - The US Orphan Disease Therapeutics Market 74
10.1 Market Overview 74
10.2 Market Forecasts 74
10.3 Treatment Usage Patterns 76
10.3.1 Disease Population 77
10.3.2 Treatment Seeking Population 77
10.3.3 Diagnosis Population 77
10.3.4 Prescription Population 77
11 Orphan Disease Therapeutics Market to 2018 - Europe Orphan Disease Therapeutics Market 78
11.1 Market Overview 78
11.2 Market Forecasts 78
11.3 Treatment Usage Patterns 80
11.3.1 Disease Population 81
11.3.2 Treatment Seeking Population 81
11.3.3 Diagnosis Population 81
11.3.4 Prescription Population 81
12 Orphan Disease Therapeutics Market to 2018 - Japan Orphan Disease Therapeutics Market 82
12.1 Market Overview 82
12.2 Market Forecasts 82
12.3 Treatment Usage Patterns 83
12.3.1 Disease Population 84
12.3.2 Treatment Seeking Population 84
12.3.3 Diagnosis Population 85
12.3.4 Prescription Population 85
13 Orphan Disease Therapeutics Market to 2018 - Competitive Landscape 86
13.1 Overview 86
13.2 Competitor Profiling 86
13.2.1 Genzyme/Sanofi 86
13.2.2 Shire 87
13.2.3 BioMarin 88
14 Orphan Disease Therapeutics Market to 2018 - Strategic Consolidations 90
14.1 Mergers and Acquisitions 90
14.1.1 Sanofi-aventis Completes Acquisition of Genzyme 90
14.1.2 GlaxoSmithKline Enters into Licensing Agreement with Amicus Therapeutics for Amigal 91
14.1.3 Amicus Therapeutics Terminates Licensing Agreement with Shire 91
15 Orphan Disease Therapeutics Market to 2018 - Orphan Drug Strategies and Future Outlook 93
15.1 Indication Expansion Important for the Orphan Drugs Market 93
15.2 Smaller Companies Find Small Clinical Trials Appealing 93
15.3 Identifying Unique Disease Subsets 93
15.4 Monopoly on a Particular Indication 93
15.5 Genzyme Non-Core Business Out-Licensing Model to Offset Manufacturing Losses 93
15.6 Innovative Risk-sharing Agreements have Emerged as Tools for Providing Market Access 93
15.7 Excessive Pricing of Orphan Drugs by the Pharma Companies Demands Measures for Greater Price Control 94
15.7.1 Excessive Prices of Orphan Drugs Increase Financial Pressures on Governments 94
15.8 Exploiting Orphan Drug Act; Maximizing Revenue 94
15.8.1 Ovation’s Indocin and Questcor’s H.P. Acthar Gel 95
15.9 Pricing an Orphan Drug is Critical to Revenue Generation 96
15.9.1 Price Control Measures 96
15.10 Global Approach for Orphan Drug Development 97
15.11 Introducing Sales Ceiling and Flexible Market Exclusivity 97
15.11.1 Price Revisions 97
15.11.2 GlaxoSmithKline’s Retrovir (zidovudine) 98
15.11.3 Amgen’s Epogen (erythropoietin) 98
15.11.4 Price Control Measures in Orphan Drugs 99
16 Orphan Disease Therapeutics Market to 2018 - Appendix 100
16.1 Market Definitions 100
16.2 Abbreviations 100
16.3 Sources 101
16.4 Research Methodology 102
16.4.1 Coverage 102
16.4.2 Secondary Research 102
16.4.3 Primary Research 102
16.4.4 Epidemiology-Based Forecasting 103
16.5 Contact Us 106
16.6 Disclaimer 106

1.1 List of Tables

Table 1: Orphan Disease Therapeutics Market, Introduction, Number of Orphan Drugs Approved and Number of Drugs Assigned Orphan Drug Status, 1983-2010 11
Table 2: Orphan Disease Therapeutics Market, Definitions of and Incentives for Orphan Drugs, 2011 12
Table 3: Orphan Disease Therapeutics Market, Global, Revenue ($bn), 2004-2010 14
Table 4: Orphan Disease Therapeutics Market, Global, Revenue ($bn), 2010-2018 15
Table 5: Orphan Disease Therapeutics Market, Fabry’s Disease, Revenue ($m), 2004-2010 19
Table 6: Orphan Disease Therapeutics Market, Fabry’s Disease, Revenue ($m), 2010-2018 19
Table 7: Orphan Disease Therapeutics Market, Fabry’s Disease, Global, Revenue by Country ($m), 2004-2010 19
Table 8: Orphan Disease Therapeutics Market, Fabry’s Disease, Global, Revenue by Country ($m), 2010-2018 20
Table 9: Orphan Disease Therapeutics Market, Fabry’s Disease, Global, Treatment Usage Patterns (‘000), 2004-2010 22
Table 10: Orphan Disease Therapeutics Market, Fabry’s Disease, Global, Treatment Usage Patterns (‘000), 2010-2018 22
Table 11: Orphan Disease Therapeutics Market, Fabry’s Disease, Global, 2011 25
Table 12: Orphan Disease Therapeutics Market, Pompe Disease, Revenue ($m), 2004-2010 27
Table 13: Orphan Disease Therapeutics Market, Pompe Disease, Revenue ($m), 2010-2018 27
Table 14: Orphan Disease Therapeutics Market, Pompe Disease, Revenue by Country ($m), 2004-2010 28
Table 15: Orphan Disease Therapeutics Market, Pompe, Revenue by Country ($m), 2010-2018 28
Table 16: Orphan Disease Therapeutics Market, Pompe Disease, Annual Cost of Treatment ($), 2004-2010 29
Table 17: Orphan Disease Therapeutics Market, Pompe Disease, Annual Cost of Treatment ($), 2004-2010 30
Table 18: Orphan Disease Therapeutics Market, Pompe Disease, Global, Treatment Usage Patterns, 2004-2010 31
Table 19: Orphan Disease Therapeutics Market, Pompe Disease, Global, Treatment Usage Patterns (‘000), 2010-2018 31
Table 20: Orphan Disease Therapeutics Market, Pompe Disease, Global, Pipeline, 2011 34
Table 21: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Revenue ($m), 2004-2010 36
Table 22: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Revenue ($m), 2010-2018 36
Table 23: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Revenue by Country ($m), 2004-2010 37
Table 24: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Revenue by Country ($m), 2010-2018 37
Table 25: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Annual Cost of Treatment ($), 2004-2010 38
Table 26: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Annual Cost of Treatment ($), 2010-2018 38
Table 27: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Global, Treatment Usage Patterns, ‘000, 2004-2010 39
Table 28: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Global, Treatment Usage Patterns (‘000), 2010-2018 40
Table 29: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue ($m), 2004-2010 44
Table 30: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue ($m), 2010-2018 44
Table 31: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue by Country ($m), 2004-2010 45
Table 32: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue by Country ($m), 2010-2018 45
Table 33: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Annual Cost of Treatment ($), 2004-2010 46
Table 34: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Annual Cost of Treatment ($), 2010-2018 46
Table 35: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Treatment Usage Patterns (‘000), 2004-2010 47
Table 36: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Treatment Usage Patterns (‘000), 2010-2018 48
Table 37: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Pipeline Molecules by Phase, (%), 2011 50
Table 38: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Pre-clinical Pipeline Molecules, 2011 51
Table 39: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Phase I Pipeline Molecules, 2011 51
Table 40: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Phase II Pipeline Molecules, 2011 51
Table 41: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Phase III Pipeline Molecules, 2011 51
Table 42: Orphan Disease Therapeutics Market, Huntington’s Disease, Global, Revenue ($m), 2004-2010 53
Table 43: Orphan Disease Therapeutics Market, Huntington’s Disease, Global, Revenue ($m), 2010-2018 53
Table 44: Orphan Disease Therapeutics Market, Huntington’s Disease, Revenue by Country ($m), 2004-2010 54
Table 45: Orphan Disease Therapeutics Market, Huntington’s Disease, Revenue by Country ($m), 2010-2018 54
Table 46: Orphan Disease Therapeutics Market, Huntington’s Disease, Annual Cost of Treatment ($), 2004-2010 55
Table 47: Orphan Disease Therapeutics Market, Huntington’s Disease, Annual Cost of Treatment ($), 2010-2018 55
Table 48: Orphan Disease Therapeutics Market, Huntington’s Disease, Global Treatment Usage Pattern (‘000), 2004-2010 56
Table 49: Orphan Disease Therapeutics Market, Huntington’s Disease, Global Treatment Usage Pattern (‘000), 2010-2018 57
Table 50: Orphan Disease Therapeutics Market, Huntington’s Disease, Product Pipeline, 2011 58
Table 51: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Revenue, ($m), 2004-2010 61
Table 52: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Revenue, ($m), 2010-2018 61
Table 53: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Geographical Segmentation, ($m), 2004-2010 62
Table 54: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Geographical Segmentation, ($m), 2010-2018 62
Table 55: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Annual Cost of Treatment, ($), 2004-2010 63
Table 56: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Annual Cost of Treatment, ($), 2010-2018 63
Table 57: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Treatment Usage Pattern, (‘000), 2004-2010 64
Table 58: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Treatment Usage Pattern, (‘000), 2010-2018 65
Table 59: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline, Filed, 2011 68
Table 60: Orphan Disease Therapeutics Market, Ovarian Cancer Therapeutics Market, Global, Product Pipeline, Phase III, 2011 68
Table 61: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline, Phase II, 2011 69
Table 62: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline, Phase I, 2011 71
Table 63: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline, Pre-clinical Stage, 2011 72
Table 64: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline, Discovery Stage, 2011 73
Table 65: Orphan Disease Therapeutics Market, The US, Revenue ($bn), 2004-2010 75
Table 66: Orphan Disease Therapeutics Market, The US, Revenue ($bn), 2010-2018 75
Table 67: Orphan Disease Therapeutics Market, The US, Treatment Usage Patterns, ‘000, 2004-2010 76
Table 68: Orphan Disease Therapeutics Market, The US, Treatment Usage Patterns, ‘000, 2010-2018 77
Table 69: Orphan Disease Therapeutics Market, Europe, Revenue, 2004-2010 79
Table 70: Orphan Disease Therapeutics Market, Europe, Revenue ($bn), 2010-2018 79
Table 71: Orphan Therapeutics Market, Europe, Treatment Usage Patterns, 2004-2010 80
Table 72: Orphan Therapeutics Market, Europe, Treatment Usage Patterns, 2010-2018 81
Table 73: Orphan Disease Therapeutics Market, Japan, Revenue ($bn), 2004-2010 83
Table 74: Orphan Disease Therapeutics Market, Japan, Revenue ($bn), 2010-2018 83
Table 75: Orphan Disease Therapeutics Market, Japan, Treatment Usage Patterns, 2004-2010 84
Table 76:Orphan Disease Therapeutics Market, Japan, Treatment Usage Patterns, 2010-2018 84
Table 77: Orphan Disease Therapeutics Market, Global, Comparison of Therapy Areas under Drug Development and Research, 2011 91
Table 78: Orphan Disease Therapeutics Market, Major Deals in Orphan Diseases - 2010-2011 92

1.2 List of Figures

Figure 1: Orphan Disease Therapeutics Market, Introduction, Number of Orphan Drugs Approved and Number of Drugs Assigned Orphan Drug Status, 1983-2010 10
Figure 2: Orphan Disease Therapeutics Market, Global, Revenue ($bn), 2004-2018 14
Figure 3: Orphan Disease Therapeutics Market, Global, Treatment Usage Patterns, ‘000, 2004-2018 15
Figure 4: Orphan Disease Therapeutics Market, Global, Treatment Usage Patterns, ‘000, 2004-2010 16
Figure 5: Orphan Disease Therapeutics Market, Global, Treatment Usage Patterns, ‘000, 2010-2018 16
Figure 6: Orphan Disease Therapeutics Market, Fabry’s Disease, Revenue ($m), 2004-2018 18
Figure 7: Orphan Disease Therapeutics Market, Fabry’s Disease, Global, Revenue by Country ($m), 2004-2018 19
Figure 8: Orphan Disease Therapeutics Market, Fabry’s Disease, Annual Cost of Treatment ($), 2004-2018 20
Figure 9: Orphan Disease Therapeutics Market, Fabry’s Disease, Annual Cost of Treatment ($), 2004-2010 20
Figure 10: Orphan Disease Therapeutics Market, Fabry, Annual Cost of Treatment ($), 2010-2018 21
Figure 11: Orphan Disease Therapeutics Market, Fabry’s Disease, Global, Treatment Usage Patterns (‘000), 2004-2018 21
Figure 12: Orphan Disease Therapeutics Market, Pompe Disease, Revenue ($m), 2004-2018 27
Figure 13: Orphan Disease Therapeutics Market, Pompe Disease, Revenue ($m), 2004-2018 28
Figure 14: Orphan Disease Therapeutics Market, Pompe Disease, Annual Cost of Treatment ($), 2004-2018 29
Figure 15: Orphan Disease Therapeutics Market, Pompe Disease, Global, Treatment Usage Pattern, 2004-2018 30
Figure 16: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Revenue ($m), 2004-2018 36
Figure 17: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Global, Revenue ($m), 2004-2018 37
Figure 18: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Global, Annual Cost of Treatment ($), 2004-2018 38
Figure 19: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Global, Treatment Usage Pattern, 2004-2018 39
Figure 20: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue ($m), 2004-2018 43
Figure 21: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue by Country ($m), 2004-2018 45
Figure 22: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Annual Cost of Treatment ($), 2004-2018 46
Figure 23: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Treatment Usage Patterns (‘000), 2004-2018 47
Figure 24: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Pipeline Molecules by Phase, (%), 2011 50
Figure 25: Orphan Disease Therapeutics Market, Huntington’s Disease, Global, Revenue ($m), 2004-2018 53
Figure 26: Orphan Disease Therapeutics Market, Huntington’s Disease, Revenue by Country ($m), 2004-2018 54
Figure 27: Orphan Disease Therapeutics Market, Huntington’s Disease, Annual Cost of Treatment ($), 2004-2018 55
Figure 28: Orphan Disease Therapeutics Market, Huntington’s Disease, Global Treatment Usage Pattern (‘000), 2004-2018 56
Figure 29: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Revenue, ($m), 2004-2018 61
Figure 30: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Geographical Segmentation, ($m), 2004-2018 62
Figure 31: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Annual Cost of Treatment, ($), 2004-2018 63
Figure 32: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Treatment Usage Pattern, (‘000), 2004-2018 64
Figure 33: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline by Phase, (%), 2011 67
Figure 34: Orphan Disease Therapeutics Market, The US, Revenue ($bn), 2004-2018 74
Figure 35: Orphan Disease Therapeutics Market, The US, Treatment Usage Patterns, ‘000, 2004-2018 76
Figure 36: Orphan Disease Therapeutics Market, Europe, Revenue ($bn), 2004-2018 79
Figure 37: Orphan Therapeutics Market, Europe, Treatment Usage Patterns, 2004-2018 80
Figure 38: Orphan Disease Therapeutics Market, Japan, Revenue ($bn), 2004-2018 82
Figure 39: Orphan Disease Therapeutics Market, Japan, Treatment Usage Patterns, 2004-2018 83
Figure 40: Orphan Disease Therapeutics Market, Global, Sales Revenue of Major Orphan Drugs, 2010 86
Figure 41: Orphan Disease Therapeutics Market, Global, Genzyme, SWOT Analysis, 2011 87
Figure 42: Orphan Disease Therapeutics Market, Global, Shire, SWOT Analysis, 2011 88
Figure 43: Orphan Disease Therapeutics Market, Global, Biomarin Pharmaceuticals, SWOT Analysis, 2011 89
Figure 44: Orphan Disease Therapeutics Market, Global, Pricing of an Orphan Drug, 2011 95
Figure 45: Orphan Disease Therapeutics Market, Global, Price Control Measures on Orphan Drugs 99
Figure 46: GBI Research Market Forecasting Model 105

Companies mentioned
Genzyme/Sanofi
Shire
BioMarin

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