Global Orphan Drug Market (Types, Application, Geography)

Size, Share, Global Trends, Company Profiles, Demand, Insights, Analysis, Research, Report, Opportunities, Segmentation and Forecast, 2012 - 2020
Category : Pharmaceuticals
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Orphan drugs are type of pharmaceutical drugs that are used in the treatment of rare genetic diseases or rare medical conditions. The global pharmaceutical drugs market was valued at approximately $962.1 billion in 2012, which is the parent market for orphan drugs. Receiving approvals for commercialization of orphan drugs is quite easy in developed countries such as U.S. and European countries due to favorable government policies. The major driving factor for this market is increase in investment from pharmaceutical companies that are suffering from loss in terms of revenue from their conventional products. Due to favorable government policies, the companies that manufacture orphan drugs have monopolized the market. Government policies such as providing reimbursement for orphan drug discovery and development is driving the growth of orphan drug market. FDA and European Commission are involved in providing special type of protocol to companies that are experiencing commercial barriers such as product competition and supply chain barriers. These protocols from government promote the application of orphan drug in rare disease treatments.
The major restraint for the orphan drug market is the competition from generic pharmaceutical drug market to gain competitive advantage. For instance, development of orphan drug Abelcel from Sigma-tau pharmaceutical Inc. for the treatment of fungal infection has been challenged by conventional anti-fungal drugs. The global orphan drug market growth has been hampered by lack of investment in development of drugs. 
The global orphan drug market is challenged by existing pharmaceutical drug market, especially in rare genetic diseases. Developing countries are not at par with developed countries due high cost and un-availability of orphan drugs, and that is hindering commercialization. However, prevalence for rare genetic diseases in developing countries such as China, Africa, India etc, is attracting global pharmaceutical companies to invest in orphan drug production. These companies are investing in development and commercialization of cost effective drugs to generate monopoly in orphan drug market. This report provide an in depth intelligence over key developmental strategies adopted by top companies involved in production of orphan drugs. The report provides market intelligence on the revenue share of each company mentioned in the company profiles.
Key companies included in this report are Acorda Therapeutics, Aegerion Pharmaceuticals, Genzyme, Glaxosmithkline, Novartis Pharmaceuticals, Novo Nordisk, Roche, Sanofi, Seattle Genetics and Pfizer.


  • Key developmental strategies adopted by top market players engaged in orphan drugs business are discussed so that companies involved in development of orphan drugs can get competitive intelligence of their competitors
  • The market estimations of product types, applications and geographic segment is derived from current market scenario and based on expected market trends
  • Market attractiveness analysis has been included for various product segments, applications and geographic regions with detailed analysis of factors responsible for rapid growth of the market segments
  • Market dynamics of global orphan drug market is dealt in the report with impact analysis. 
  • Value chain analysis, Porter’s five force model, top investment pockets (GE9 Cell Matrix) are analyzed and presented in detail in the report so that decision makers receive clear picture of the orphan market


  • Non-biological orphan drugs
  • Biological orphan drugs


  • Blood-related disorders and cystic fibrosis
  • Neurology
  • Oncology nephrology and infectious diseases
  • Nephrology and infectious diseases
  • Others


  • North America
  • Europe
  • Asia-Pacific
  • RoW


  • Pharmaceutical companies
  • Academic Institutes
  • Research Institutes
  • Government Authorities


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