Orphan Drugs Market by Product and Geography - Global Forecast and Analysis 2019-2023

Orphan Drugs Market by Product and Geography - Global Forecast and Analysis 2019-2023

Global Orphan Drugs Market: About this market

Technavio’s orphan drugs market analysis considers sales from both biologics and non-biologics. Our analysis also considers the sales of orphan drugs in Asia, Europe, North America, and ROW. In 2018, the biologics segment had a significant market share, and this trend is expected to continue over the forecast period. Factors such as high efficacy will play a significant role in the biologics segment to maintain its market position. Also, our global orphan drugs market report looks at factors such as robust pipeline and recent drug approvals, rising incidence of the rare disease, availability of incentives for orphan drug development. However, uncertainties in the delay in diagnosis, risks associated with the clinical development of novel orphan drugs and challenges associated with pricing and reimbursements for orphan drugs may hamper the growth of the orphan drugs industry over the forecast period.

Global Orphan Drugs Market: Overview

Rising incidence of rare diseases

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. Rare diseases include inherited genetic disorders, rare types of cancer, tropical infectious diseases, and neurodegenerative diseases. The prevalence and distribution of rare diseases are skewed. Although some rare diseases are inherited, others are a product of harmful environmental factors. This will lead to the expansion of the global orphan drugs market at a CAGR of almost 12% during the forecast period.

The emergence of combination therapies and novel targets

The potential advances in several areas such as immunotherapies and CGT are rekindling interest and enabling progress in the development of orphan drugs. These advances have been made in target selection, and combination therapy approaches for effectively reversing the immunosuppressive mechanisms, thereby enhancing the durability of the therapeutics. This development is expected to have a positive impact on the overall market growth.

For the detailed list of factors that will drive the global orphan drugs market during the forecast period 2019-2023, click here.

Competitive Landscape

With the presence of several major players, the global orphan drugs market is moderately fragmented. Technavio’s robust vendor analysis is designed to help clients improve their market position, and in line with this, this report provides a detailed analysis of several leading orphan drugs manufacturers, that include Bristol-Myers Squibb Co., F. Hoffmann-La Roche Ltd., Johnson & Johnson Services Inc., Novartis AG, Takeda Pharmaceutical Co. Ltd.

Also, the orphan drugs market analysis report includes information on upcoming trends and challenges that will influence market growth. This is to help companies strategize and leverage on all forthcoming growth opportunities."


  • Executive summary
  • Scope of the report
    • Preface
    • Currency conversion rates for US$
  • Market landscape
    • Market ecosystem
      • Table Recent mega-mergers in pharmaceuticals
      • Table Global pharmaceuticals market
      • Table Segments of global pharmaceuticals market
    • Market characteristics
      • Table Market characteristics
    • Market segmentation analysis
      • Table Market segments
  • Market sizing
    • Market definition
      • Table Market definition - Inclusions and exclusions checklist
    • Market sizing 2018
      • Table Market size 2018
    • Market size and forecast 2018-2023
      • Table Global market: Size and forecast 2018-2023 ($ billions)
      • Table Global market: Year-over-year growth 2019-2023 (%)
      • Table Some of the first-in-class orphan drugs approved by the US FDA during 2017-2018
  • Five forces analysis
    • Table Five forces analysis 2018
    • Table Five forces analysis 2023
    • Bargaining power of buyers
      • Table Bargaining power of buyers
    • Bargaining power of suppliers
      • Table Bargaining power of suppliers
    • Threat of new entrants
      • Table Threat of new entrants
    • Threat of substitutes
      • Table Threat of substitutes
    • Threat of rivalry
      • Table Threat of rivalry
    • Market condition
      • Table Market condition - Five forces 2018
  • Pipeline analysis
    • Table Some of the late-stage orphan drugs
  • Market segmentation by product
    • Table Product - Market share 2018-2023 (%)
    • Comparison by product
      • Table Comparison by product
    • Biologics - Market size and forecast 2018-2023
      • Table Biologics - Market size and forecast 2018-2023 ($ billions)
      • Table Number of new or expanded indications approved for novel orphan biologics to the number of rare and nonrare diseases from the US FDA January 2013-June 2019
      • Table Promising regenerative orphan drugs pricing plans
      • Table Biologics - Year-over-year growth 2019-2023 (%)
    • Non-biologics - Market size and forecast 2018-2023
      • Table Non-biologics - Market size and forecast 2018-2023 ($ billions)
      • Table Non-biologics - Year-over-year growth 2019-2023 (%)
    • Market opportunity by product
      • Table Market opportunity by product
  • Customer landscape
    • Table Customer landscape
  • Geographic landscape
    • Geographic segmentation
      • Table Market share by geography 2018-2023 (%)
    • Geographic comparison
      • Table Geographic comparison
    • North America - Market size and forecast 2018-2023
      • Table North America - Market size and forecast 2018-2023 ($ billions)
      • Table Number of orphan drug approvals in US 2014-2018
      • Table US FDA orphan drug marketing approvals in major therapeutic areas 2008-2017
      • Table List of expensive orphan drugs launched in US
      • Table Leading vendors and their product sales in US 2018.
      • Table Status of BTD, PR, and ODD for novel drugs 2013-2017
      • Table RMAT designation requests by product type 2018
      • Table North America - Year-over-year growth 2019-2023 (%)
    • Europe - Market size and forecast 2018-2023
      • Table Europe - Market size and forecast 2018-2023 ($ billions)
      • Table Orphan drug approvals in Europe during 2010-2018
      • Table Applications for ODD in EU 2005-2018
      • Table Europe - Year-over-year growth 2019-2023 (%)
    • Asia - Market size and forecast 2018-2023
      • Table Asia - Market size and forecast 2018-2023 ($ billions)
      • Table Orphan drugs eligible for reimbursable under China's state health insurance schemes
      • Table Asia - Year-over-year growth 2019-2023 (%)
    • ROW - Market size and forecast 2018-2023
      • Table ROW - Market size and forecast 2018-2023 ($ billions)
      • Table ROW - Year-over-year growth 2019-2023 (%)
    • Key leading countries
      • Table Key leading countries
    • Market opportunity
      • Table Market opportunity
  • Decision framework
  • Drivers and challenges
    • Market drivers
      • Table List of promising drugs approved in US, Japan, and EU 2017-2019
      • Table Number of orphan drugs approved in US, EU, and Japan 2017 and 2018
      • Table Percentage of orphan and non-orphan drugs approved by the US FDA 2018
      • Table Incidence rate of rare diseases per year in US
      • Table Prevalence of rare diseases 2018 (millions)
      • Table Orphan drugs regulatory framework and incentives in key leading regions or countries
    • Market challenges
      • Table Impact of drivers and challenges
  • Market trends
    • Strategic focus on alliances, partnerships, and M&A
      • Table Several major M&A in the market
    • Emergence of combination therapies and novel targets
      • Table Expansion of combination therapies research areas of ICI approved drugs
      • Table Ongoing immunotherapy combinations studies with co-inhibitory molecules
      • Table Ongoing immunotherapy combinations studies with co-stimulatory molecules
    • Rising demand for genetic tests and protein biomarkers
  • Vendor landscape
    • Overview
      • Table Vendor landscape
    • Landscape disruption
      • Table Landscape disruption
    • Competitive scenario
  • Vendor analysis
    • Vendors covered
      • Table Vendors covered
    • Vendor classification
      • Table Vendor classification
    • Market positioning of vendors
      • Table Market positioning of vendors
    • Bristol-Myers Squibb Co.
      • Table Bristol-Myers Squibb Co. - Vendor overview
      • Table Bristol-Myers Squibb Co. - Business segments
      • Table Bristol-Myers Squibb Co. - Organizational developments
      • Table Bristol-Myers Squibb Co. - Geographic focus
      • Table Bristol-Myers Squibb Co. - Key offerings
      • Table Bristol-Myers Squibb Co. - Key customers
    • F. Hoffmann-La Roche Ltd.
      • Table F. Hoffmann-La Roche Ltd. - Vendor overview
      • Table F. Hoffmann-La Roche Ltd. - Business segments
      • Table F. Hoffmann-La Roche Ltd. - Organizational developments
      • Table F. Hoffmann-La Roche Ltd. - Geographic focus
      • Table F. Hoffmann-La Roche Ltd. - Segment focus
      • Table F. Hoffmann-La Roche Ltd. - Key offerings
      • Table F. Hoffmann-La Roche Ltd. - Key customers
    • Johnson & Johnson Services Inc.
      • Table Johnson & Johnson Services Inc. - Vendor overview
      • Table Johnson & Johnson Services Inc. - Business segments
      • Table Johnson & Johnson Services Inc. - Organizational developments
      • Table Johnson & Johnson Services Inc. - Geographic focus
      • Table Johnson & Johnson Services Inc. - Segment focus
      • Table Johnson & Johnson Services Inc. - Key offerings
      • Table Johnson & Johnson Services Inc. - Key customers
    • Novartis AG
      • Table Novartis AG - Vendor overview
      • Table Novartis AG - Business segments
      • Table Novartis AG - Organizational developments
      • Table Novartis AG - Geographic focus
      • Table Novartis AG - Segment focus
      • Table Novartis AG - Key offerings
      • Table Novartis AG - Key customers
    • Takeda Pharmaceutical Co. Ltd.
      • Table Takeda Pharmaceutical Co. Ltd. - Vendor overview
      • Table Takeda Pharmaceutical Co. Ltd. - Business segments
      • Table Takeda Pharmaceutical Co. Ltd. - Organizational developments
      • Table Takeda Pharmaceutical Co. Ltd. - Geographic focus
      • Table Takeda Pharmaceutical Co. Ltd. - Key offerings
      • Table Takeda Pharmaceutical Co. Ltd. - Key customers
  • Appendix
    • Research methodology
      • Table Validation techniques employed for market sizing
    • List of abbreviations
    • Definition of market positioning of vendors
      • Table Definition of market positioning of vendors
  • Explore Technavio

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