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

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

Orphan Disease Therapeutics Market to 2018 - Improved Understanding of Rare Diseases' Heterogeneity and Novel New Clinical Trial Designs to Foster Innovation - GBI Research Reports
Orphan Disease Therapeutics Market to 2018 - Improved Understanding of Rare Diseases' Heterogeneity and Novel New Clinical Trial Designs to Foster Innovation
Published Apr 30, 2012
106 pages — Published Apr 30, 2012
Price US$ 3,500.00  |  Buy this Report Now

About This Report

  
Abstract:

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 Researchs team of industry experts. The report provides an in-depth analysis of Fabry, Pompe, Mucopolysaccharidosis VI, Idiopathic Thrombocytopenic Purpura, Huntingtons 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, Huntingtons 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 Researchs 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, Huntingtons 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

  
Source:
Document ID
GBIHC192MR
Industry
Format:
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Sections

TitleStarting PageNumber of Pages
Table of Contents37
  List of Tables62
  List of Figures82
Orphan Disease Therapeutics Market to 2018 - Introduction104
  Orphan Drug Definitions121
    The US Orphan Drug Criteria121
    Europe Orphan Drug Criteria121
    Japanese Orphan Drug Criteria121
    Special Cases of Orphan Drug Exclusivity121
    Comparison of Orphan Drug Designations121
  GBI Research Report Guidance131
Orphan Disease Therapeutics Market to 2018 - Market Characterization144
  Market Forecasts141
  Treatment Usage Patterns151
    Disease Population161
    Treatment Seeking Population161
    Diagnosis Population171
    Prescription Population171
Orphan Disease Therapeutics Market to 2010 - Global Fabry s Disease Therapeutics Market188
  Disease Overview181
  Market Overview181
  Revenue by Geographical Segmentation191
  Annual Cost of Treatment201
  Treatment Usage Patterns211
    Disease Population221
    Treatment Seeking Population221
    Diagnosis Population221
    Prescription Population221
  Marketed Product Analysis231
    Fabrazyme231
      Overview231
      Mechanism of Action231
      Safety Issues231
      Efficacy Details231
    Replagal241
      Overview241
      Mechanism of Action241
      Safety Issues241
      Efficacy Details241
  Drug Pipeline Analysis251
    Pipeline by Phase251
Orphan Disease Therapeutics Market to 2018 - Global Pompe Disease Therapeutics Market269
  Disease Overview261
  Market Overview262
  Revenue by Geographical Segmentation281
  Annual Cost of Treatment291
  Treatment Usage Patterns301
    Disease Population311
    Treatment Seeking Population311
    Diagnosis Population311
    Prescription Population321
  Marketed Product Analysis321
    Myozyme/Lumizyme321
      Overview321
      Mechanism of Action321
      Safety Issues321
      Efficacy Details331
  Drug Pipeline Analysis341
    Pipeline by Phase341
Orphan Disease Therapeutics Market to 2018 - Global Mucopolysaccharidosis VI Disease Therapeutics Market357
  Disease Overview351
  Market Overview352
  Revenue by Geographical Segmentation371
  Annual Cost of Treatment381
  Treatment Usage Patterns391
    Disease Population401
    Treatment Seeking Population401
    Diagnosis Population401
    Prescription Population401
  Marketed Product Analysis411
    Naglazyme411
      Overview411
      Mechanism of Action411
      Safety Issues411
      Efficacy Details411
  Drug Pipeline Analysis411
Orphan Disease Therapeutics Market to 2018 - Global Idiopathic Thrombocytopenic Purpura Disease Therapeutics Market4210
  Disease Overview421
  Market Overview432
  Revenue by Geographical Segmentation451
  Annual Cost of Treatment461
  Treatment Usage Patterns471
    Disease Population481
    Treatment Seeking Population481
    Diagnosed Population481
    Prescription Population481
  Marketed Product Analysis491
    Nplate (romiplostim)491
    Promacta/Revolade (eltrombopag)491
    Rhophylac (Rh0 (D) Immune Globulin (Human))491
  Drug Pipeline Analysis501
    Pipeline by Phase511
Orphan Disease Therapeutics Market to 2018 - Global Huntington s Disease Therapeutics Market528
  Disease Overview521
  Market Overview531
  Revenue by Geographical Segmentation541
  Annual Cost of Treatment551
  Treatment Usage Patterns561
    Disease Population571
    Treatment Seeking Population571
    Diagnosis Population571
    Prescription Population571
  Marketed Product Analysis571
    Xenazine571
  Drug Pipeline Analysis581
    Pipeline by Phase582
Orphan Disease Therapeutics Market to 2018 - Global Ovarian Cancer Therapeutics Market6014
  Disease Overview601
  Market Overview602
  Revenue by Geographical Segmentation621
  Average Cost of Therapy631
  Treatment Usage Patterns641
    Disease Population651
    Treatment Seeking Population651
    Diagnosis Population651
    Prescription Population651
  Marketed Product Analysis661
    Altretamine (hexalen)661
    Yondelis661
    Gemzar (gemcitabine)661
  Drug Pipeline Analysis671
    Pipeline by Phase677
Orphan Disease Therapeutics Market to 2018 - The US Orphan Disease Therapeutics Market744
  Market Overview741
  Market Forecasts742
  Treatment Usage Patterns761
    Disease Population771
    Treatment Seeking Population771
    Diagnosis Population771
    Prescription Population771
Orphan Disease Therapeutics Market to 2018 - Europe Orphan Disease Therapeutics Market784
  Market Overview781
  Market Forecasts782
  Treatment Usage Patterns801
    Disease Population811
    Treatment Seeking Population811
    Diagnosis Population811
    Prescription Population811
Orphan Disease Therapeutics Market to 2018 - Japan Orphan Disease Therapeutics Market824
  Market Overview821
  Market Forecasts821
  Treatment Usage Patterns831
    Disease Population841
    Treatment Seeking Population841
    Diagnosis Population851
    Prescription Population851
Orphan Disease Therapeutics Market to 2018 - Competitive Landscape864
  Overview861
  Competitor Profiling861
    Genzyme/Sanofi861
      Overview861
      SWOT Analysis871
    Shire871
      Overview871
      SWOT Analysis881
    BioMarin881
      SWOT Analysis891
Orphan Disease Therapeutics Market to 2018 - Strategic Consolidations903
  Mergers and Acquisitions901
    Sanofi-aventis Completes Acquisition of Genzyme901
      Deal Summary901
      Deal Rationale901
    GlaxoSmithKline Enters into Licensing Agreement with Amicus Therapeutics for Amigal911
    Amicus Therapeutics Terminates Licensing Agreement with Shire912
Orphan Disease Therapeutics Market to 2018 - Orphan Drug Strategies and Future Outlook937
  Indication Expansion Important for the Orphan Drugs Market931
  Smaller Companies Find Small Clinical Trials Appealing931
  Identifying Unique Disease Subsets931
  Monopoly on a Particular Indication931
  Genzyme Non-Core Business Out-Licensing Model to Offset Manufacturing Losses931
  Innovative Risk-sharing Agreements have Emerged as Tools for Providing Market Access931
  Excessive Pricing of Orphan Drugs by the Pharma Companies Demands Measures for Greater Price Control941
    Excessive Prices of Orphan Drugs Increase Financial Pressures on Governments941
  Exploiting Orphan Drug Act; Maximizing Revenue941
    Ovation s Indocin and Questcor s H.P. Acthar Gel951
  Pricing an Orphan Drug is Critical to Revenue Generation961
    Price Control Measures961
  Global Approach for Orphan Drug Development971
  Introducing Sales Ceiling and Flexible Market Exclusivity971
    Price Revisions971
    GlaxoSmithKline s Retrovir (zidovudine)981
    Amgen s Epogen (erythropoietin)981
    Price Control Measures in Orphan Drugs991
Orphan Disease Therapeutics Market to 2018 - Appendix1007
  Market Definitions1001
  Abbreviations1001
  Sources1011
  Research Methodology1024
    Coverage1021
    Secondary Research1021
    Primary Research1021
    Epidemiology-Based Forecasting1033
  Contact Us1061
  Disclaimer1061

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Cite this Report

  
MLA:
GBI Research Reports. "Orphan Disease Therapeutics Market to 2018 - Improved Understanding of Rare Diseases' Heterogeneity and Novel New Clinical Trial Designs to Foster Innovation" Apr 30, 2012. Alacra Store. May 07, 2025. <http://www.alacrastore.com/storecontent/GBI-Research-Reports/Orphan-Disease-Therapeutics-Market-to-2018-Improved-Understanding-of-Rare-Diseases-Heterogeneity-and-Novel-New-Clinical-Trial-Designs-to-Foster-Innovation-2115-408>
  
APA:
GBI Research Reports. (2012). Orphan Disease Therapeutics Market to 2018 - Improved Understanding of Rare Diseases' Heterogeneity and Novel New Clinical Trial Designs to Foster Innovation Apr 30, 2012. New York, NY: Alacra Store. Retrieved May 07, 2025 from <http://www.alacrastore.com/storecontent/GBI-Research-Reports/Orphan-Disease-Therapeutics-Market-to-2018-Improved-Understanding-of-Rare-Diseases-Heterogeneity-and-Novel-New-Clinical-Trial-Designs-to-Foster-Innovation-2115-408>
  
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