Global Orphan Drugs Market by Disease Type (Oncologic Diseases, Metabolic Diseases, Hematologic and Immunologic Diseases, Infectious Diseases, Neurologic Diseases and Other Rare Diseases), Indication (Non-Hodgkin Lymphoma, Acute Myeloid Leukemia, Cystic Fibrosis, Glioma, Pancreatic Cancer, Ovarian Cancer, Multiple Myeloma, Duchenne Muscular Dystrophy, Graft vs Host Disease, Renal Cell Carcinoma and Others) - Global Opportunity Analysis and Industry Forecast, 2014-2022

  • ALL22757
  • 140 Pages
  • April 2017
  • Pharmaceuticals

The pharmaceutical agents that are administered to treat, diagnose, and prevent life-threatening diseases (rare diseases) are known as orphan drugs. The global orphan drugs market accounted for $106 billion in 2015, and is anticipated to reach $169 billion by 2022, registering a CAGR of 6.8% from 2016 to 2022.

Orphan drugs are used to treat rare (orphaned) diseases prevalent in a very small patient pool. Hence, the pharmaceutical industry takes little interest in their development. Moreover, their development requires high cost and the chances of returns on investment are comparatively less when compared to non-orphan drugs. These drugs treat different rare diseases, such as non-Hodgkin lymphoma, acute myeloid leukemia, cystic fibrosis, and others.

Conducive government regulations and market exclusivity for orphan drugs drives the global orphan drugs market. In addition, surge in prevalence of rare diseases and rise in investment in research, drug development, and innovation supplement the market growth. However, limited patient pool for clinical trials & product marketing and high treatment costs per patient impede the market growth. Furthermore, growth in novel indications for known orphan drugs and untapped emerging markets offer lucrative opportunities for the market players.

The orphan drugs market is segmented based on disease type, indication, and geography. Based on disease type, the market is divided into oncologic diseases, metabolic diseases, hematologic and immunologic diseases, infectious diseases, neurologic diseases, and other rare diseases. In the orphan drugs disease type segment, oncologic diseases occupied dominant share in 2015, and the segment is expected to maintain this trend during the forecast period. This is attributed to the array of different forms of rare cancers, such as leukemia, myeloma, angiosarcoma, and others prevalent in the patient population.

Based on indication, this market is classified into non-Hodgkin lymphoma, acute myeloid leukemia, cystic fibrosis, glioma, pancreatic cancer, ovarian cancer, multiple myeloma, Duchenne muscular dystrophy, Graft vs host disease, renal cell carcinoma, and others. The non-Hodgkin lymphoma was the highest revenue contributor in 2015, while renal cell carcinoma is anticipated to grow at the highest rate. The growth for this segment is due to increase in investment in R&D throughout the world along with surge in awareness about kidney cancer.

Geographically, this market is analyzed across North America, Europe, Asia-Pacific, and LAMEA. North America accounted for the largest market share in the orphan drugs market in 2015, and is expected to retain its dominance throughout the forecast period. This is primarily attributed to the higher buying power, availability, accessibility, and treatment options that favor the utilization of orphan drugs in this region. There is also an increase in product approvals for different pharmaceutical products designated as orphan drugs by the U.S. Food and Drug Administration (FDA), which fosters the market growth. However, Asia-Pacific is expected to emerge as a lucrative area with maximum growth potential, owing to the improvement in R&D facilities, available disposable income, and rapidly developing economic conditions.

Product development is the key strategy adopted by market players. The report provides a comprehensive analysis of the key players that operate in the global orphan drugs market.


  • AbbVie Inc.
  • Aegerion Pharmaceuticals, Inc.
  • Bristol-Myers Squibb Company
  • Celgene Corporation
  • F. Hoffmann-La Roche Ltd.  
  • GlaxoSmithKline plc
  • Johnson & Johnson.
  • Novartis AG
  • Pfizer Inc.
  • Sanofi


  • The study provides an in-depth analysis of the global orphan drugs market along with the current trends and future estimations to elucidate the imminent investment pockets.
  • The report presents quantitative analysis of the market from 2014 to 2022 to enable stakeholders to capitalize on the prevailing market opportunities.
  • Extensive analysis of the market based on indications assists in understanding the trends in the industry.
  • Key market players along with their strategies are thoroughly analyzed to understand the competitive outlook of the industry.


By Disease Type

  • Oncologic Diseases
  • Metabolic Diseases
  • Hematologic and Immunologic Diseases
  • Infectious Diseases
  • Neurologic Diseases
  • Other Rare Diseases

By Indication

  • Non-Hodgkin Lymphoma
  • Acute Myeloid Leukemia
  • Cystic Fibrosis
  • Glioma
  • Pancreatic Cancer
  • Ovarian Cancer
  • Multiple Myeloma
  • Duchenne Muscular Dystrophy
  • Graft vs Host Disease
  • Renal Cell Carcinoma
  • Others

By Geography

  • North America
    • U.S.
    • Canada
    • Mexico
  • Europe
    • Germany
    • France
    • UK
    • Italy
    • Spain
    • Rest of Europe
  • Asia-Pacific
    • Japan
    • China
    • Australia
    • India
    • South Korea
    • Taiwan
    • Rest of Asia-Pacific
    • Brazil
    • Turkey
    • Saudi Arabia
    • South Africa
    • Rest of LAMEA

Other players in the value chain include (profiles not included in the report)

  • Bayer HealthCare Pharmaceuticals Inc
  • Vertex Pharmaceuticals
  • Merck & Co., Inc.,
  • Alexion Pharmaceuticals, Inc.
  • Boehringer Ingelheim GmbH
  • Amgen Inc.
  • Biogen Idec
  • Actelion Pharmaceuticals Ltd
  • AstraZeneca plc
  • Janssen Biotech, Inc.
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