Table Of contents
1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Orphan Disease Therapeutics Market to 2018: Executive Summary
2.1 The Global Orphan Disease Therapeutics Market is Forecast to Grow Due to the Increasing Number of Orphan Drug Approvals
2.2 Genzyme: Non-Core Business Out-Licensing Model to Offset Manufacturing Losses
2.3 High Unmet Needs Boosts Research and Development in Orphan Disease Therapeutics Market
3 Orphan Disease Therapeutics Market to 2018: Introduction
3.1 Orphan Drug Definitions
3.1.1 The US Orphan Drug Criteria
3.1.2 Europe Orphan Drug Criteria
3.1.3 Japanese Orphan Drug Criteria
3.1.4 Special Cases of Orphan Drug Exclusivity
3.1.5 Comparison of Orphan Drug Designations
3.2 GBI Research Report Guidance
4 Orphan Disease Therapeutics Market to 2018: Market Characterization
4.1 Market Forecasts
4.2 Treatment Usage Patterns
4.2.1 Disease Population
4.2.2 Treatment Seeking Population
4.2.3 Diagnosis Population
4.2.4 Prescription Population
5 Orphan Disease Therapeutics Market to 2010: Global Fabry Disease Therapeutics Market
5.1 Disease Overview
5.2 Market Overview
5.3 Revenue by Geographical Segmentation
5.4 Annual Cost of Treatment
5.5 Treatment Usage Patterns
5.5.1 Disease Population
5.5.2 Treatment Seeking Population
5.5.3 Diagnosis Population
5.5.4 Prescription Population
5.6 Marketed Product Analysis
5.6.1 Fabrazyme
5.6.2 Replagal
5.7 Drug Pipeline Analysis
5.7.1 Pipeline by Phase
6 Orphan Disease Therapeutics Market to 2018: Global Pompe Disease Therapeutics Market
6.1 Disease Overview
6.2 Market Overview
6.3 Revenue by Geographical Segmentation
6.4 Annual Cost of Treatment
6.5 Treatment Usage Patterns
6.5.1 Disease Population
6.5.2 Treatment Seeking Population
6.5.3 Diagnosis Population
6.5.4 Prescription Population
6.6 Marketed Product Analysis
6.6.1 Myozyme/Lumizyme
6.7 Drug Pipeline Analysis
6.7.1 Pipeline by Phase
7 Orphan Disease Therapeutics Market to 2018: Global Mucopolysaccharidosis VI Disease Therapeutics Market
7.1 Disease Overview
7.2 Market Overview
7.3 Revenue by Geographical Segmentation
7.4 Annual Cost of Treatment
7.5 Treatment Usage Patterns
7.5.1 Disease Population
7.5.2 Treatment Seeking Population
7.5.3 Diagnosis Population
7.5.4 Prescription Population
7.6 Marketed Product Analysis
7.6.1 Naglazyme
7.7 Drug Pipeline Analysis
8 Orphan Disease Therapeutics Market to 2018: Global Idiopathic Thrombocytopenic Purpura Disease Therapeutics Market
8.1 Disease Overview
8.2 Market Overview
8.3 Revenue by Geographical Segmentation
8.4 Annual Cost of Treatment
8.5 Treatment Usage Patterns
8.6 Marketed Product Analysis
8.7 Drug Pipeline Analysis
8.7.1 Pipeline by Phase
9 Orphan Disease Therapeutics Market to 2018: Global Huntington’s Disease Therapeutics Market
9.1 Disease Overview
9.2 Market Overview
9.3 Revenue by Geographical Segmentation
9.4 Annual Cost of Treatment
9.5 Treatment Usage Patterns
9.5.1 Disease Population
9.5.2 Treatment Seeking Population
9.5.3 Diagnosis Population
9.5.4 Prescription Population
9.6 Marketed Product Analysis
9.7 Drug Pipeline Analysis
9.7.1 Pipeline by Phase
10 Orphan Disease Therapeutics Market to 2018: Global Ovarian Cancer Therapeutics Market
10.1 Disease Overview
10.2 Market Overview
10.3 Revenue by Geographical Segmentation
10.4 Average Cost of Therapy
10.5 Treatment Usage Patterns
10.5.1 Disease Population
10.5.2 Treatment Seeking Population
10.5.3 Diagnosis Population
10.5.4 Prescription Population
10.6 Marketed Product Analysis
10.7 Drug Pipeline Analysis
10.7.1 Pipeline by Phase
11 Orphan Disease Therapeutics Market to 2018: The US Orphan Disease Therapeutics Market
11.1 Market Overview
11.2 Market Forecasts
11.3 Treatment Usage Patterns
11.3.1 Disease Population
11.3.2 Treatment Seeking Population
11.3.3 Diagnosis Population
11.3.4 Prescription Population
12 Orphan Disease Therapeutics Market to 2018: Europe Orphan Disease Therapeutics Market
12.1 Market Overview
12.2 Market Forecasts
12.3 Treatment Usage Patterns
12.3.1 Disease Population
12.3.2 Treatment Seeking Population
12.3.3 Diagnosis Population
12.3.4 Prescription Population
13 Orphan Disease Therapeutics Market to 2018: Japan Orphan Disease Therapeutics Market
13.1 Market Overview
13.2 Market Forecasts
13.3 Treatment Usage Patterns
13.3.1 Disease Population
13.3.2 Treatment Seeking Population
13.3.3 Diagnosis Population
13.3.4 Prescription Population
14 Orphan Disease Therapeutics Market to 2018: Competitive Landscape
14.1 Overview
14.2 Competitor Profiling
14.2.1 Genzyme/Sanofi
14.2.2 Shire
14.2.3 BioMarin
15 Orphan Disease Therapeutics Market to 2018: Strategic Consolidations
15.1 Mergers and Acquisitions
15.1.1 Sanofi-aventis Completes Acquisition of Genzyme
15.1.2 GlaxoSmithKline Enters into Licensing Agreement with Amicus Therapeutics for Amigal
15.1.3 Amicus Therapeutics Terminates Licensing Agreement with Shire
16 Orphan Disease Therapeutics Market to 2018: Orphan Drug Strategies and Future Outlook
16.1.1 Indication Expansion Important for the Orphan Drugs Market
16.1.2 Smaller Companies Find Small Clinical Trials Appealing
16.1.3 Identifying Unique Disease Subsets
16.1.4 Monopoly on a Particular Indication
16.1.5 Genzyme Non-Core Business Out-Licensing Model to Offset Manufacturing Losses
16.1.6 Innovative Risk-sharing Agreements have Emerged as Tools for Providing Market Access
16.1.7 Excessive Pricing of Orphan Drugs by the Pharma Companies Demands Measures for Greater Price Control
16.1.8 Exploiting Orphan Drug Act; Maximizing Revenue
16.1.9 Pricing an Orphan Drug is Critical to Revenue Generation
16.1.10 Global Approach for Orphan Drug Development
16.1.11 Introducing Sales Ceiling and Flexible Market Exclusivity
17 Orphan Disease Therapeutics Market to 2018: Appendix
17.1 Market Definitions
17.2 Abbreviations
17.3 Research Methodology
4.1.1 Coverage
4.1.2 Secondary Research
4.1.3 Primary Research
17.4 Therapeutic Landscape
17.4.2 Market Size by Geography
17.5 Geographical Landscape
17.6 Pipeline Analysis
17.7 Competitive Landscape
17.7.1 Expert Panel Validation
17.8 Contact Us
17.9 Disclaimer
17.10 Sources
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
Table 2: Orphan Disease Therapeutics Market, Definitions of and Incentives for Orphan Drugs, 2011
Table 3: Orphan Disease Therapeutics Market, Global, Revenue ($bn), 2004–2010
Table 4: Orphan Disease Therapeutics Market, Global, Revenue ($bn), 2010–2018
Table 5: Orphan Disease Therapeutics Market, Fabry Disease, Revenue ($m), 2004–2010
Table 6: Orphan Disease Therapeutics Market, Fabry Disease, Revenue ($m), 2010–2018
Table 7: Orphan Disease Therapeutics Market, Fabry Disease, Global, Revenue by Country ($m), 2004–2010
Table 8: Orphan Disease Therapeutics Market, Fabry Disease, Global, Revenue by Country ($m), 2010–2018
Table 9: Orphan Disease Therapeutics Market, Fabry Disease, Global, Treatment Usage Patterns (‘000), 2004–2010
Table 10: Orphan Disease Therapeutics Market, Fabry Disease, Global, Treatment Usage Patterns (‘000), 2010–2018
Table 11: Orphan Disease Therapeutics Market, Fabry Disease, Global, 2011
Table 12: Orphan Disease Therapeutics Market, Pompe Disease, Revenue ($m), 2004–2010
Table 13: Orphan Disease Therapeutics Market, Pompe Disease, Revenue ($m), 2010–2018
Table 14: Orphan Disease Therapeutics Market, Pompe Disease, Revenue by Country ($m), 2004–2010
Table 15: Orphan Disease Therapeutics Market, Pompe, Revenue by Country ($m), 2010–2018
Table 16: Orphan Disease Therapeutics Market, Pompe Disease, Annual Cost of Treatment ($), 2004–2010
Table 17: Orphan Disease Therapeutics Market, Pompe Disease, Annual Cost of Treatment ($), 2004–2010
Table 18: Orphan Disease Therapeutics Market, Pompe Disease, Global, Treatment Usage Patterns, 2004–2010
Table 19: Orphan Disease Therapeutics Market, Pompe Disease, Global, Treatment Usage Patterns (‘000), 2010–2018
Table 20: Orphan Disease Therapeutics Market, Pompe Disease, Global, Pipeline, 2011
Table 21: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Revenue ($m), 2004–2010
Table 22: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Revenue ($m), 2010–2018
Table 23: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Revenue by Country ($m), 2004–2010
Table 24: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Revenue by Country ($m), 2010–2018
Table 25: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Annual Cost of Treatment ($), 2004–2010
Table 26: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Annual Cost of Treatment ($), 2010–2018
Table 27: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Global, Treatment Usage Patterns, ‘000, 2004–2010
Table 28: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Global, Treatment Usage Patterns (‘000), 2010–2018
Table 29: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue ($m), 2004–2010
Table 30: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue ($m), 2010–2018
Table 31: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue by Country ($m), 2004–2010
Table 32: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue by Country ($m), 2010–2018
Table 33: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Annual Cost of Treatment ($), 2004–2010
Table 34: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Annual Cost of Treatment ($), 2010–2018
Table 35: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Treatment Usage Patterns (‘000), 2004–2010
Table 36: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Treatment Usage Patterns (‘000), 2010–2018
Table 37: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Pipeline Molecules by Phase, (%), 2011
Table 38: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Pre-clinical Pipeline Molecules, 2011
Table 39: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Phase I Pipeline Molecules, 2011
Table 40: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Phase II Pipeline Molecules, 2011
Table 41: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Phase III Pipeline Molecules, 2011
Table 42: Orphan Disease Therapeutics Market, Huntington’s Disease, Global, Revenue ($m), 2004–2010
Table 43: Orphan Disease Therapeutics Market, Huntington’s Disease, Global, Revenue ($m), 2010–2018
Table 44: Orphan Disease Therapeutics Market, Huntington’s Disease, Revenue by Country ($m), 2004–2010
Table 45: Orphan Disease Therapeutics Market, Huntington’s Disease, Revenue by Country ($m), 2010–2018
Table 46: Orphan Disease Therapeutics Market, Huntington’s Disease, Annual Cost of Treatment ($), 2004–2010
Table 47: Orphan Disease Therapeutics Market, Huntington’s Disease, Annual Cost of Treatment ($), 2010–2018
Table 48: Orphan Disease Therapeutics Market, Huntington’s Disease, Global Treatment Usage Pattern (‘000), 2004–2010
Table 49: Orphan Disease Therapeutics Market, Huntington’s Disease, Global Treatment Usage Pattern (‘000), 2010–2018
Table 50: Orphan Disease Therapeutics Market, Huntington’s Disease, Product Pipeline, 2011
Table 51: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Revenue, ($m), 2004–2010
Table 52: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Revenue, ($m), 2010–2018
Table 53: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Geographical Segmentation, ($m), 2004–2010
Table 54: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Geographical Segmentation, ($m), 2010–2018
Table 55: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Annual Cost of Treatment, ($), 2004–2010
Table 56: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Annual Cost of Treatment, ($), 2010–2018
Table 57: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Treatment Usage Pattern, (‘000), 2004–2010
Table 58: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Treatment Usage Pattern, (‘000), 2010–2018
Table 59: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline, Filed, 2011
Table 60: Orphan Disease Therapeutics Market, Ovarian Cancer Therapeutics Market, Global, Product Pipeline, Phase III, 2011
Table 61: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline, Phase II, 2011
Table 62: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline, Phase I, 2011
Table 63: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline, Pre-clinical Stage, 2011
Table 64: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline, Discovery Stage, 2011
Table 65: Orphan Disease Therapeutics Market, The US, Revenue ($bn), 2004–2010
Table 66: Orphan Disease Therapeutics Market, The US, Revenue ($bn), 2010–2018
Table 67: Orphan Disease Therapeutics Market, Japan, Revenue ($bn), 2004–2010
Table 68: Orphan Disease Therapeutics Market, Japan, Revenue ($bn), 2010–2018
Table 69: Orphan Disease Therapeutics Market, Global, Comparison of Therapy Areas under Drug Development and Research, 2011
Table 70: Orphan Disease Therapeutics Market, Major Deals in Orphan Diseases – 2010–2011
List Of Figures
Figure 1: Orphan Disease Therapeutics Market, Global, Revenue ($bn), 2004–2018
Figure 2: Orphan Disease Therapeutics Market, Introduction, Number of Orphan Drugs Approved and Number of Drugs Assigned Orphan Drug Status, 1983-2010
Figure 3: Orphan Disease Therapeutics Market, Global, Revenue ($bn), 2004–2018
Figure 4: Orphan Disease Therapeutics Market, Global, Treatment Usage Patterns, ‘000, 2004–2018
Figure 5: Orphan Disease Therapeutics Market, Global, Treatment Usage Patterns, ‘000, 2004–2010
Figure 6: Orphan Disease Therapeutics Market, Global, Treatment Usage Patterns, ‘000, 2010–2018
Figure 7: Orphan Disease Therapeutics Market, Fabry Disease, Revenue ($m), 2004–2018
Figure 8: Orphan Disease Therapeutics Market, Fabry Disease, Global, Revenue by Country ($m), 2004–2018
Figure 9: Orphan Disease Therapeutics Market, Fabry Disease, Annual Cost of Treatment ($), 2004–2018
Figure 10: Orphan Disease Therapeutics Market, Fabry Disease, Annual Cost of Treatment ($), 2004–2010
Figure 11: Orphan Disease Therapeutics Market, Fabry, Annual Cost of Treatment ($), 2010–2018
Figure 12: Orphan Disease Therapeutics Market, Fabry Disease, Global, Treatment Usage Patterns (‘000), 2004–2018
Figure 13: Orphan Disease Therapeutics Market, Pompe Disease, Revenue ($m), 2004–2018
Figure 14: Orphan Disease Therapeutics Market, Pompe Disease, Revenue ($m), 2004–2018
Figure 15: Orphan Disease Therapeutics Market, Pompe Disease, Annual Cost of Treatment ($), 2004–2018
Figure 16: Orphan Disease Therapeutics Market, Pompe Disease, Global, Treatment Usage Pattern, 2004–2018
Figure 17: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Revenue ($m), 2004–2018
Figure 18: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Global, Revenue ($m), 2004–2018
Figure 19: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Global, Annual Cost of Treatment ($), 2004–2018
Figure 20: Orphan Disease Therapeutics Market, Mucopolysaccharidosis VI, Global, Treatment Usage Pattern, 2004–2018
Figure 21: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue ($m), 2004–2018
Figure 22: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Revenue by Country ($m), 2004–2018
Figure 23: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Annual Cost of Treatment ($), 2004–2018
Figure 24: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Treatment Usage Patterns (‘000), 2004–2018
Figure 25: Orphan Disease Therapeutics Market, Idiopathic Thrombocytopenic Purpura, Global, Pipeline Molecules by Phase, (%), 2011
Figure 26: Orphan Disease Therapeutics Market, Huntington’s Disease, Global, Revenue ($m), 2004–2018
Figure 27: Orphan Disease Therapeutics Market, Huntington’s Disease, Revenue by Country ($m), 2004–2018
Figure 28: Orphan Disease Therapeutics Market, Huntington’s Disease, Annual Cost of Treatment ($), 2004–2018
Figure 29: Orphan Disease Therapeutics Market, Huntington’s Disease, Global Treatment Usage Pattern (‘000), 2004–2018
Figure 30: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Revenue, ($m), 2004–2018
Figure 31: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Geographical Segmentation, ($m), 2004–2018
Figure 32: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Annual Cost of Treatment, ($), 2004–2018
Figure 33: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Treatment Usage Pattern, (‘000), 2004–2018
Figure 34: Orphan Disease Therapeutics Market, Ovarian Cancer, Global, Product Pipeline by Phase, (%), 2011
Figure 35: Orphan Disease Therapeutics Market, The US, Revenue ($bn), 2004–2018
Figure 36: Orphan Disease Therapeutics Market, The US, Treatment Usage Patterns, ‘000, 2004–2018
Figure 37: Orphan Disease Therapeutics Market, The US, Treatment Usage Patterns, ‘000, 2004–2010
Figure 38: Orphan Disease Therapeutics Market, The US, Treatment Usage Patterns, ‘000, 2010–2018
Figure 39: Orphan Disease Therapeutics Market, Europe, Revenue ($bn), 2004–2018
Figure 40: Orphan Disease Therapeutics Market, Europe, Revenue, 2004–2010
Figure 41: Orphan Disease Therapeutics Market, Europe, Revenue ($bn), 2010–2018
Figure 42: Orphan Therapeutics Market, Europe, Treatment Usage Patterns, 2004–2018
Figure 43: Orphan Therapeutics Market, Europe, Treatment Usage Patterns, 2004–2010
Figure 44: Orphan Therapeutics Market, Europe, Treatment Usage Patterns, 2010–2018
Figure 45: Orphan Disease Therapeutics Market, Japan, Revenue ($bn), 2004–2018
Figure 46: Orphan Disease Therapeutics Market, Japan, Treatment Usage Patterns, 2004–2018
Figure 47: Orphan Disease Therapeutics Market, Japan, Treatment Usage Patterns, 2004–2010
Figure 48: Orphan Disease Therapeutics Market, Japan, Treatment Usage Patterns, 2010–2018
Figure 49: Orphan Disease Therapeutics Market, Global, Sales Revenue of Major Orphan Drugs, 2010
Figure 50: Orphan Disease Therapeutics Market, Global, Genzyme, SWOT Analysis, 2011
Figure 51: Orphan Disease Therapeutics Market, Global, Shire, SWOT Analysis, 2011
Figure 52: Orphan Disease Therapeutics Market, Global, Biomarin Pharmaceuticals, SWOT Analysis, 2011
Figure 53: Orphan Disease Therapeutics Market, Global, Pricing of an Orphan Drug, 2011
Figure 54: Orphan Disease Therapeutics Market, Global, Price Control Measures on Orphan Drugs
Figure 55: GBI Research Market Forecasting Model