Orphan Drugs Market by Product and Geography - Forecast and Analysis 2021-2025

  • Published: Jan 2021
  • Pages: 120
  • SKU: IRTNTR46586

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The orphan drugs market size has the potential to grow by USD 138.70 billion during 2021-2025, and the market’s growth momentum will accelerate at a CAGR of 14.56%. 

This report provides a detailed analysis of the market by product (biologics and non-biologics), geography (North America, Europe, Asia, and ROW), and key vendors. 

Market Overview


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Market Competitive Analysis

The report analyzes the market’s competitive landscape and offers information on several market vendors, including: 


  • AbbVie Inc.
  • Alnylam Pharmaceuticals Inc.
  • bluebird bio Inc.
  • Bristol-Myers Squibb Co.
  • CSL Ltd.
  • F. Hoffmann-La Roche Ltd.
  • Johnson & Johnson
  • Novartis AG
  • Pfizer Inc.
  • Takeda Pharmaceutical Co. Ltd.


The orphan drugs market is fragmented and the vendors are deploying growth strategies such as M&A to compete in the market. Click here to uncover other successful business strategies deployed by the vendors. 

The market players also significantly leverage external market drivers such as rising incidence of rare diseases to achieve growth opportunities. However, factors such as delay in diagnosis will challenge the growth of the market participants. To make the most of the opportunities and recover from post COVID-19 impact, market vendors should focus more on the growth prospects in the fast-growing segments, while maintaining their positions in the slow-growing segments.

Download a free sample of the orphan drugs market forecast report for insights on complete key vendor profiles. The profiles include information on the production, sustainability, and prospects of the leading companies. 

This orphan drugs market analysis report also provides detailed information on the upcoming trends and challenges that will influence market growth. This will help companies create strategies to make the most of their future growth opportunities. 

Orphan Drugs Market: Key Drivers and Trends

  • Orphan drugs are specifically used to treat rare diseases, and the prevalence of such health conditions has a direct influence on the sales of orphan drugs.
  •  It has been observed that the incidence of rare health conditions, including inherited genetic disorders, rare types of cancer, tropical infectious diseases, and neurodegenerative ailments, is increasing. 
  • This will directly influence the demand for orphan drugs, propelling the growth of the market. 
  • Moreover, the market is recording advanced research on orphan drugs to cater to the unmet treatment need. 
  • Vendors are leveraging monopolistic practice laws to charge monopoly prices on orphan drugs for an extended period. These factors will augment the sales of vendors over the forecast period.
  • Recent potential advances in therapeutics such as immunotherapies and cell and gene therapies are enabling progress in the development of improved orphan drugs.
  • Most of the development is focused on targeted and combination therapies as they are proven to have a higher efficacy rate. 
  • Most of the orphan drugs are available as combination and target therapies. 
  • The majority of vendors are formulating advanced combination therapies that can reverse the immunosuppressive mechanism. Immune checkpoint inhibitors (ICI) have demonstrated proven efficacy in a variety of oncology indications. 
  • The efficacy and durability of such drugs will have a positive influence on market growth. Therefore, the orphan drugs market forecast highlights the emergence of combination therapies and novel targets as one of the key market trends. 


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Orphan Drugs Market: Segmentation by Geography


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41% of the market’s growth will originate from North America during the forecast period. The US is one of the key markets for orphan drugs in North America. Market growth in this region will be slower than the growth of the market in Asia and ROW.

New drug approvals is one of the prime factors that will facilitate the orphan drugs market growth in North America over the forecast period. To garner further competitive intelligence and regional opportunities in store for vendors, view our sample report. 

Orphan Drugs Market: Segmentation by Product


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The high target affinity and specificity toward specific human antigenic targets of biologics, the presence of advanced drug development technology platforms, and the rise in approval of new biologics are expected to boost the growth of the market segment.

Fetch actionable market insights on post COVID-19 impact on each segment. This report provides an accurate prediction of the contribution of all the segments to the growth of the orphan drugs market size.   



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Orphan Drugs Market: Key Highlights of the Report for 2021-2025

  • CAGR of the market during the forecast period 2021-2025
  • Detailed information on factors that will drive orphan drugs market growth during the next five years
  • Precise estimation of the orphan drugs market size and its contribution to the parent market
  • Accurate predictions on upcoming trends and changes in consumer behavior
  • The growth of the orphan drugs industry across North America, Europe, Asia, and ROW
  • A thorough analysis of the market’s competitive landscape and detailed information on vendors
  • Comprehensive details of factors that will challenge the growth of orphan drugs market vendors

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Executive Summar

    Market Landscape

    • Market ecosystem
    • Value chain analysis

    Market Sizing

    • Market definition
    • Market segment analysis
    • Market size 2020
    • Market outlook: Forecast for 2020 - 2025

    Five Forces Analysis

    • Five forces summary
    • Bargaining power of buyers
    • Bargaining power of suppliers
    • Threat of new entrants
    • Threat of substitutes
    • Threat of rivalry
    • Market condition

    Market Segmentation by Product

    • Market segments
    • Comparison by Product
    • Biologics - Market size and forecast 2020-2025
    • Non-biologics - Market size and forecast 2020-2025
    • Market opportunity by Product

    Customer landscape

      Geographic Landscape

      • Geographic segmentation
      • Geographic comparison
      • North America - Market size and forecast 2020-2025
      • Europe - Market size and forecast 2020-2025
      • Asia - Market size and forecast 2020-2025
      • ROW - Market size and forecast 2020-2025
      • Key leading countries
      • Market opportunity by geography
      • Market drivers
      • Market challenges
      • Market trends

      Vendor Landscape

      • Vendor landscape
      • Landscape disruption
      • Competitive Landscape

      Vendor Analysis

      • Vendors covered
      • Market positioning of vendors
      • AbbVie Inc.
      • Alnylam Pharmaceuticals Inc.
      • bluebird bio Inc.
      • Bristol-Myers Squibb Co.
      • CSL Ltd.
      • F. Hoffmann-La Roche Ltd.
      • Johnson & Johnson
      • Novartis AG
      • Pfizer Inc.
      • Takeda Pharmaceutical Co. Ltd.


      • Scope of the report
      • Currency conversion rates for US$
      • Research methodology
      • List of abbreviations

      Research Framework

      Technavio presents a detailed picture of the market by way of study, synthesis, and summation of data from multiple sources. The analysts have presented the various facets of the market with a particular focus on identifying the key industry influencers. The data thus presented is comprehensive, reliable, and the result of extensive research, both primary and secondary.


      Primary sources

      • Manufacturers and suppliers
      • Channel partners
      • Industry experts
      • Strategic decision makers

      Secondary sources

      • Industry journals and periodicals
      • Government data
      • Financial reports of key industry players
      • Historical data
      • Press releases

      TechnavioDATA ANALYSIS

      Data Synthesis

      • Collation of data
      • Estimation of key figures
      • Analysis of derived insights

      Data Validation

      • Triangulation with data models
      • Reference against proprietary databases
      • Corroboration with industry experts

      TechnavioREPORT WRITING


      • Market drivers
      • Market challenges
      • Market trends
      • Five forces analysis


      • Market size and forecast
      • Market segmentation
      • Geographical insights
      • Competitive landscape
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      Orphan Drugs market growth will increase by $138.70 billion during 2020-2025.
      The orphan drugs market is expected to grow at a CAGR of 14.56% during 2020-2025.
      Technavio has segmented the orphan drugs market by product (Biologics and Non-biologics) and geographic (North America, Europe, Asia, and ROW).
      AbbVie Inc., Alnylam Pharmaceuticals Inc., bluebird bio Inc., Bristol-Myers Squibb Co., CSL Ltd., F. Hoffmann-La Roche Ltd., Johnson & Johnson, Novartis AG, Pfizer Inc., Takeda Pharmaceutical Co. Ltd. are a few of the key vendors in the orphan drugs market.
      North America will register the highest growth rate of 40.79% among the other regions. Therefore, the orphan drugs market in North America is expected to garner significant business opportunities for the vendors during the forecast period.
      The key factors driving the orphan drugs market growth are:
      • Robust pipeline and recent drug approvals
      • Strategic focus on alliances, partnerships, and M&A
      The orphan drugs market vendors should focus on grabbing business opportunities from the biologics segment as it accounted for the largest market share in the base year.
      • What are the key global market and the regional market share?
      • What are the revenue-generating key market segments?
      • What are the key factors driving and challenging this market’s growth?
      • Who are the key market vendors and their growth strategies?
      • What are the latest trends influencing the growth of this market?
      • What are the variables influencing the market growth in the primary regions?
      • What are the factors influencing the growth of the parent market?
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