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

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

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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 gaps1 Table of Contents1 Table of Contents 31.1 List of Tables 61.2 List of Figures 82 Orphan Disease Therapeutics Market to 2018 – Introduction 102.1 Orphan Drug Definitions 122.1.1 The US Orphan Drug Criteria 122.1.2 Europe Orphan Drug Criteria 122.1.3 Japanese Orphan Drug Criteria 122.1.4 Special Cases of Orphan Drug Exclusivity 122.1.5 Comparison of Orphan Drug Designations 122.2 GBI Research Report Guidance 133 Orphan Disease Therapeutics Market to 2018 – Market Characterization 143.1 Market Forecasts 143.2 Treatment Usage Patterns 153.2.1 Disease Population 163.2.2 Treatment Seeking Population 163.2.3 Diagnosis Population 173.2.4 Prescription Population 174 Orphan Disease Therapeutics Market to 2010 – Global Fabry’s Disease Therapeutics Market 184.1 Disease Overview 184.2 Market Overview 184.3 Revenue by Geographical Segmentation 194.4 Annual Cost of Treatment 204.5 Treatment Usage Patterns 214.5.1 Disease Population 224.5.2 Treatment Seeking Population 224.5.3 Diagnosis Population 224.5.4 Prescription Population 224.6 Marketed Product Analysis 234.6.1 Fabrazyme 234.6.2 Replagal 244.7 Drug Pipeline Analysis 254.7.1 Pipeline by Phase 255 Orphan Disease Therapeutics Market to 2018 – Global Pompe Disease Therapeutics Market 265.1 Disease Overview 265.2 Market Overview 265.3 Revenue by Geographical Segmentation 285.4 Annual Cost of Treatment 295.5 Treatment Usage Patterns 305.5.1 Disease Population 315.5.2 Treatment Seeking Population 315.5.3 Diagnosis Population 315.5.4 Prescription Population 325.6 Marketed Product Analysis 325.6.1 Myozyme/Lumizyme 325.7 Drug Pipeline Analysis 345.7.1 Pipeline by Phase 346 Orphan Disease Therapeutics Market to 2018 – Global Mucopolysaccharidosis VI Disease Therapeutics Market 356.1 Disease Overview 356.2 Market Overview 356.3 Revenue by Geographical Segmentation 376.4 Annual Cost of Treatment 386.5 Treatment Usage Patterns 396.5.1 Disease Population 406.5.2 Treatment Seeking Population 406.5.3 Diagnosis Population 406.5.4 Prescription Population 406.6 Marketed Product Analysis 416.6.1 Naglazyme 416.7 Drug Pipeline Analysis 417 Orphan Disease Therapeutics Market to 2018 – Global Idiopathic Thrombocytopenic Purpura Disease Therapeutics Market 427.1 Disease Overview 427.2 Market Overview 437.3 Revenue by Geographical Segmentation 457.4 Annual Cost of Treatment 467.5 Treatment Usage Patterns 477.6 Marketed Product Analysis 497.7 Drug Pipeline Analysis 507.7.1 Pipeline by Phase 518 Orphan Disease Therapeutics Market to 2018 – Global Huntington’s Disease Therapeutics Market 528.1 Disease Overview 528.2 Market Overview 538.3 Revenue by Geographical Segmentation 548.4 Annual Cost of Treatment 558.5 Treatment Usage Patterns 568.5.1 Disease Population 578.5.2 Treatment Seeking Population 578.5.3 Diagnosis Population 578.5.4 Prescription Population 578.6 Marketed Product Analysis 578.6.1 Xenazine 578.7 Drug Pipeline Analysis 588.7.1 Pipeline by Phase 589 Orphan Disease Therapeutics Market to 2018 – Global Ovarian Cancer Therapeutics Market 609.1 Disease Overview 609.2 Market Overview 609.3 Revenue by Geographical Segmentation 629.4 Average Cost of Therapy 639.5 Treatment Usage Patterns 649.5.1 Disease Population 659.5.2 Treatment Seeking Population 659.5.3 Diagnosis Population 659.5.4 Prescription Population 659.6 Marketed Product Analysis 669.6.1 Altretamine (hexalen) 669.6.2 Yondelis 669.6.3 Gemzar (gemcitabine) 669.7 Drug Pipeline Analysis 679.7.1 Pipeline by Phase 6710 Orphan Disease Therapeutics Market to 2018 – The US Orphan Disease Therapeutics Market 7410.1 Market Overview 7410.2 Market Forecasts 7410.3 Treatment Usage Patterns 7610.3.1 Disease Population 7710.3.2 Treatment Seeking Population 7710.3.3 Diagnosis Population 7710.3.4 Prescription Population 7711 Orphan Disease Therapeutics Market to 2018 – Europe Orphan Disease Therapeutics Market 7811.1 Market Overview 7811.2 Market Forecasts 7811.3 Treatment Usage Patterns 8011.3.1 Disease Population 8111.3.2 Treatment Seeking Population 8111.3.3 Diagnosis Population 8111.3.4 Prescription Population 8112 Orphan Disease Therapeutics Market to 2018 – Japan Orphan Disease Therapeutics Market 8212.1 Market Overview 8212.2 Market Forecasts 8212.3 Treatment Usage Patterns 8312.3.1 Disease Population 8412.3.2 Treatment Seeking Population 8412.3.3 Diagnosis Population 8512.3.4 Prescription Population 8513 Orphan Disease Therapeutics Market to 2018 – Competitive Landscape 8613.1 Overview 8613.2 Competitor Profiling 8613.2.1 Genzyme/Sanofi 8613.2.2 Shire 8713.2.3 BioMarin 8814 Orphan Disease Therapeutics Market to 2018 – Strategic Consolidations 9014.1 Mergers and Acquisitions 9014.1.1 Sanofi-aventis Completes Acquisition of Genzyme 9014.1.2 GlaxoSmithKline Enters into Licensing Agreement with Amicus Therapeutics for Amigal 9114.1.3 Amicus Therapeutics Terminates Licensing Agreement with Shire 9115 Orphan Disease Therapeutics Market to 2018 – Orphan Drug Strategies and Future Outlook 9315.1 Indication Expansion Important for the Orphan Drugs Market 9315.2 Smaller Companies Find Small Clinical Trials Appealing 9315.3 Identifying Unique Disease Subsets 9315.4 Monopoly on a Particular Indication 9315.5 Genzyme Non-Core Business Out-Licensing Model to Offset Manufacturing Losses 9315.6 Innovative Risk-sharing Agreements have Emerged as Tools for Providing Market Access 9315.7 Excessive Pricing of Orphan Drugs by the Pharma Companies Demands Measures for Greater Price Control 9415.7.1 Excessive Prices of Orphan Drugs Increase Financial Pressures on Governments 9415.8 Exploiting Orphan Drug Act; Maximizing Revenue 9415.8.1 Ovation’s Indocin and Questcor’s H.P. Acthar Gel 9515.9 Pricing an Orphan Drug is Critical to Revenue Generation 9615.9.1 Price Control Measures 9615.10 Global Approach for Orphan Drug Development 9715.11 Introducing Sales Ceiling and Flexible Market Exclusivity 9715.11.1 Price Revisions 9715.11.2 GlaxoSmithKline’s Retrovir (zidovudine) 9815.11.3 Amgen’s Epogen (erythropoietin) 9815.11.4 Price Control Measures in Orphan Drugs 9916 Orphan Disease Therapeutics Market to 2018 – Appendix 10016.1 Market Definitions 10016.2 Abbreviations 10016.3 Sources 10116.4 Research Methodology 10216.4.1 Coverage 10216.4.2 Secondary Research 10216.4.3 Primary Research 10216.4.4 Epidemiology-Based Forecasting 10316.5 Contact Us 10616.6 Disclaimer 106.

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Source: marketwatch.com

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