Global Orphan Drugs Market 2023-2027

Global Orphan Drugs Market 2023-2027

Technavio has been monitoring the orphan drugs market and is forecast to grow by $170.8 bn during 2022-2027, accelerating at a CAGR of 14.02% during the forecast period. Our report on the orphan drugs market provides a holistic analysis, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis covering around 25 vendors.

The report offers an up-to-date analysis regarding the current market scenario, the latest trends and drivers, and the overall market environment. The market is driven by robust pipeline and recent drug approvals, rising incidence of rare diseases, and availability of incentives for orphan drug development.

Technavio's orphan drugs market is segmented as below:

By Distribution Channel

  • Hospital pharmacy
  • Specialty pharmacy
  • Retail pharmacy
  • Others
By Product
  • Biologics
  • Non-biologics
By Geographical Landscape
  • North America
  • Europe
  • Asia
  • Rest of World (ROW)
This study identifies the strategic focus on alliances, partnerships, and M&A as one of the prime reasons driving the orphan drugs market growth during the next few years. Also, the emergence of combination therapies and novel targets and rising demand for genetic tests and protein biomarkers will lead to sizable demand in the market.

Technavio presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters. Our report on the orphan drugs market covers the following areas:
  • Orphan drugs market sizing
  • Orphan drugs market forecast
  • Orphan drugs market industry analysis
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 market vendors that include AbbVie Inc., Alnylam Pharmaceuticals Inc., Astellas Pharma Inc., AstraZeneca Plc, bluebird bio Inc., Bristol Myers Squibb Co., CSL Ltd., F. Hoffmann La Roche Ltd., Gilead Sciences Inc., GlaxoSmithKline Plc, Ipsen Pharma, Johnson and Johnson Services Inc., Merck and Co. Inc., Novartis AG, Novo Nordisk AS, Pfizer Inc., Ractigen Therapeutics, Sanofi SA, Takeda Pharmaceutical Co. Ltd., and Vertex Pharmaceuticals Inc. 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 all forthcoming growth opportunities.

The study was conducted using an objective combination of primary and secondary information including inputs from key participants in the industry. The report contains a comprehensive market and vendor landscape in addition to an analysis of the key vendors.

Technavio presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters such as profit, pricing, competition, and promotions. It presents various market facets by identifying the key industry influencers. The data presented is comprehensive, reliable, and a result of extensive research - both primary and secondary. Technavio's market research reports provide a complete competitive landscape and an in-depth vendor selection methodology and analysis using qualitative and quantitative research to forecast accurate market growth.


  • Executive Summary
    • Market overview
      • Table Executive Summary – Chart on Market Overview
      • Table Executive Summary – Data Table on Market Overview
      • Table Executive Summary – Chart on Global Market Characteristics
      • Table Executive Summary – Chart on Market by Geography
      • Table Executive Summary – Chart on Market Segmentation by Distribution Channel
      • Table Executive Summary – Chart on Market Segmentation by Product
      • Table Executive Summary – Chart on Incremental Growth
      • Table Executive Summary – Data Table on Incremental Growth
      • Table Executive Summary – Chart on Vendor Market Positioning
  • Market Landscape
    • Market ecosystem
      • Table Parent market
      • Table Market Characteristics
  • Market Sizing
    • Market definition
      • Table Offerings of vendors included in the market definition
    • Market segment analysis
      • Table Market segments
    • Market size 2022
    • Market outlook: Forecast for 2022-2027
      • Table Chart on Global - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Global - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Global Market: Year-over-year growth 2022-2027 (%)
      • Table Data Table on Global Market: Year-over-year growth 2022-2027 (%)
  • Historic Market Size
    • Global orphan drugs market 2017 - 2021
      • Table Historic Market Size – Data table on global orphan drugs market 2017 - 2021 ($ billion)
    • Distributed Channel Segment Analysis 2017 - 2021
      • Table Historic Market Size – Distributed Channel Segment 2017 - 2021 ($ billion)
    • Product Segment Analysis 2017 - 2021
      • Table Historic Market Size – Product Segment 2017 - 2021 ($ billion)
    • Geography Segment Analysis 2017 - 2021
      • Table Historic Market Size – Geography Segment 2017 - 2021 ($ billion)
    • Country Segment Analysis 2017 - 2021
      • Table Historic Market Size – Country Segment 2017 - 2021 ($ billion)
  • Five Forces Analysis
    • Five forces summary
      • Table Five forces analysis - Comparison between 2022 and 2027
    • Bargaining power of buyers
      • Table Chart on Bargaining power of buyers – Impact of key factors 2022 and 2027
    • Bargaining power of suppliers
      • Table Bargaining power of suppliers – Impact of key factors in 2022 and 2027
    • Threat of new entrants
      • Table Threat of new entrants – Impact of key factors in 2022 and 2027
    • Threat of substitutes
      • Table Threat of substitutes – Impact of key factors in 2022 and 2027
    • Threat of rivalry
      • Table Threat of rivalry – Impact of key factors in 2022 and 2027
    • Market condition
      • Table Chart on Market condition - Five forces 2022 and 2027
  • Market Segmentation by Distribution Channel
    • Market segments
      • Table Chart on Distribution Channel - Market share 2022-2027 (%)
      • Table Data Table on Distribution Channel - Market share 2022-2027 (%)
    • Comparison by Distribution Channel
      • Table Chart on Comparison by Distribution Channel
      • Table Data Table on Comparison by Distribution Channel
    • Hospital pharmacy - Market size and forecast 2022-2027
      • Table Chart on Hospital pharmacy - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Hospital pharmacy - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Hospital pharmacy - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Hospital pharmacy - Year-over-year growth 2022-2027 (%)
    • Specialty pharmacy - Market size and forecast 2022- 2027
      • Table Chart on Specialty pharmacy - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Specialty pharmacy - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Specialty pharmacy - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Specialty pharmacy - Year-over-year growth 2022-2027 (%)
    • Retail pharmacy - Market size and forecast 2022-2027
      • Table Chart on Retail pharmacy - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Retail pharmacy - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Retail pharmacy - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Retail pharmacy - Year-over-year growth 2022-2027 (%)
    • Others - Market size and forecast 2022-2027
      • Table Chart on Others - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Others - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Others - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Others - Year-over-year growth 2022-2027 (%)
    • Market opportunity by Distribution Channel
      • Table Market opportunity by Distribution Channel ($ billion)
      • Table Data Table on Market opportunity by Distribution Channel ($ billion)
  • Market Segmentation by Product
    • Market segments
      • Table Chart on Product - Market share 2022-2027 (%)
      • Table Data Table on Product - Market share 2022-2027 (%)
    • Comparison by Product
      • Table Chart on Comparison by Product
      • Table Data Table on Comparison by Product
    • Biologics - Market size and forecast 2022-2027
      • Table Chart on Biologics - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Biologics - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Biologics - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Biologics - Year-over-year growth 2022-2027 (%)
    • Non-biologics - Market size and forecast 2022-2027
      • Table Chart on Non-biologics - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Non-biologics - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Non-biologics - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Non-biologics - Year-over-year growth 2022-2027 (%)
    • Market opportunity by Product
      • Table Market opportunity by Product ($ billion)
      • Table Data Table on Market opportunity by Product ($ billion)
  • Customer Landscape
    • Customer landscape overview
      • Table Analysis of price sensitivity, lifecycle, customer purchase basket, adoption rates, and purchase criteria
  • Geographic Landscape
    • Geographic segmentation
      • Table Chart on Market share by geography 2022-2027 (%)
      • Table Data Table on Market share by geography 2022-2027 (%)
    • Geographic comparison
      • Table Chart on Geographic comparison
      • Table Data Table on Geographic comparison
    • North America - Market size and forecast 2022-2027
      • Table Chart on North America - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on North America - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on North America - Year-over-year growth 2022-2027 (%)
      • Table Data Table on North America - Year-over-year growth 2022-2027 (%)
    • Europe - Market size and forecast 2022-2027
      • Table Chart on Europe - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Europe - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Europe - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Europe - Year-over-year growth 2022-2027 (%)
    • Asia - Market size and forecast 2022-2027
      • Table Chart on Asia - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Asia - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Asia - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Asia - Year-over-year growth 2022-2027 (%)
    • Rest of World (ROW) - Market size and forecast 2022- 2027
      • Table Chart on Rest of World (ROW) - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Rest of World (ROW) - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Rest of World (ROW) - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Rest of World (ROW) - Year-over-year growth 2022-2027 (%)
    • US - Market size and forecast 2022-2027
      • Table Chart on US - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on US - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on US - Year-over-year growth 2022-2027 (%)
      • Table Data Table on US - Year-over-year growth 2022-2027 (%)
    • UK - Market size and forecast 2022-2027
      • Table Chart on UK - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on UK - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on UK - Year-over-year growth 2022-2027 (%)
      • Table Data Table on UK - Year-over-year growth 2022-2027 (%)
    • Canada - Market size and forecast 2022-2027
      • Table Chart on Canada - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Canada - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Canada - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Canada - Year-over-year growth 2022-2027 (%)
    • China - Market size and forecast 2022-2027
      • Table Chart on China - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on China - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on China - Year-over-year growth 2022-2027 (%)
      • Table Data Table on China - Year-over-year growth 2022-2027 (%)
    • Germany - Market size and forecast 2022-2027
      • Table Chart on Germany - Market size and forecast 2022-2027 ($ billion)
      • Table Data Table on Germany - Market size and forecast 2022-2027 ($ billion)
      • Table Chart on Germany - Year-over-year growth 2022-2027 (%)
      • Table Data Table on Germany - Year-over-year growth 2022-2027 (%)
    • Market opportunity by geography
      • Table Market opportunity by geography ($ billion)
      • Table Data Tables on Market opportunity by geography ($ billion)
  • Drivers, Challenges, and Trends
    • Market drivers
      • Robust pipeline and recent drug approvals
      • Rising incidence of rare diseases
      • Availability of incentives for orphan drug development
    • Market challenges
      • Delay in diagnosis
      • Risks associated with clinical development of novel orphan drugs
      • Challenges associated with pricing and reimbursements for orphan drugs
    • Impact of drivers and challenges
      • Table Impact of drivers and challenges in 2022 and 2027
    • Market trends
      • Strategic focus on alliances, partnerships, and M and A
      • Emergence of combination therapies and novel targets
      • Rising demand for genetic tests and protein biomarkers
  • Vendor Landscape
    • Overview
    • Vendor landscape
      • Table Overview on Criticality of inputs and Factors of differentiation
    • Landscape disruption
      • Table Overview on factors of disruption
    • Industry risks
      • Table Impact of key risks on business
  • Vendor Analysis
    • Vendors covered
      • Table Vendors covered
    • Market positioning of vendors
      • Table Matrix on vendor position and classification
    • AbbVie Inc.
      • Table AbbVie Inc. - Overview
      • Table AbbVie Inc. - Product / Service
      • Table AbbVie Inc. - Key news
      • Table AbbVie Inc. - Key offerings
    • Alnylam Pharmaceuticals Inc.
      • Table Alnylam Pharmaceuticals Inc. - Overview
      • Table Alnylam Pharmaceuticals Inc. - Product / Service
      • Table Alnylam Pharmaceuticals Inc. - Key news
      • Table Alnylam Pharmaceuticals Inc. - Key offerings
    • Astellas Pharma Inc.
      • Table Astellas Pharma Inc. - Overview
      • Table Astellas Pharma Inc. - Product / Service
      • Table Astellas Pharma Inc. - Key news
      • Table Astellas Pharma Inc. - Key offerings
    • AstraZeneca Plc
      • Table AstraZeneca Plc - Overview
      • Table AstraZeneca Plc - Product / Service
      • Table AstraZeneca Plc - Key news
      • Table AstraZeneca Plc - Key offerings
    • bluebird bio Inc.
      • Table bluebird bio Inc. - Overview
      • Table bluebird bio Inc. - Product / Service
      • Table bluebird bio Inc. - Key offerings
    • Bristol Myers Squibb Co.
      • Table Bristol Myers Squibb Co. - Overview
      • Table Bristol Myers Squibb Co. - Product / Service
      • Table Bristol Myers Squibb Co. - Key news
      • Table Bristol Myers Squibb Co. - Key offerings
    • CSL Ltd.
      • Table CSL Ltd. - Overview
      • Table CSL Ltd. - Business segments
      • Table CSL Ltd. - Key news
      • Table CSL Ltd. - Key offerings
      • Table CSL Ltd. - Segment focus
    • F. Hoffmann La Roche Ltd.
      • Table F. Hoffmann La Roche Ltd. - Overview
      • Table F. Hoffmann La Roche Ltd. - Business segments
      • Table F. Hoffmann La Roche Ltd. - Key news
      • Table F. Hoffmann La Roche Ltd. - Key offerings
      • Table F. Hoffmann La Roche Ltd. - Segment focus
    • Gilead Sciences Inc.
      • Table Gilead Sciences Inc. - Overview
      • Table Gilead Sciences Inc. - Product / Service
      • Table Gilead Sciences Inc. - Key news
      • Table Gilead Sciences Inc. - Key offerings
    • GlaxoSmithKline Plc
      • Table GlaxoSmithKline Plc - Overview
      • Table GlaxoSmithKline Plc - Business segments
      • Table GlaxoSmithKline Plc - Key news
      • Table GlaxoSmithKline Plc - Key offerings
      • Table GlaxoSmithKline Plc - Segment focus
    • Ipsen Pharma
      • Table Ipsen Pharma - Overview
      • Table Ipsen Pharma - Business segments
      • Table Ipsen Pharma - Key offerings
      • Table Ipsen Pharma - Segment focus
    • Johnson and Johnson Services Inc.
      • Table Johnson and Johnson Services Inc. - Overview
      • Table Johnson and Johnson Services Inc. - Business segments
      • Table Johnson and Johnson Services Inc. - Key news
      • Table Johnson and Johnson Services Inc. - Key offerings
      • Table Johnson and Johnson Services Inc. - Segment focus
    • Merck and Co. Inc.
      • Table Merck and Co. Inc. - Overview
      • Table Merck and Co. Inc. - Business segments
      • Table Merck and Co. Inc. - Key news
      • Table Merck and Co. Inc. - Key offerings
      • Table Merck and Co. Inc. - Segment focus
    • Novartis AG
      • Table Novartis AG - Overview
      • Table Novartis AG - Business segments
      • Table Novartis AG - Key offerings
      • Table Novartis AG - Segment focus
    • Ractigen Therapeutics
      • Table Ractigen Therapeutics - Overview
      • Table Ractigen Therapeutics - Product / Service
      • Table Ractigen Therapeutics - Key offerings
  • Appendix
    • Scope of the report
      • Market definition
      • Objectives
      • Notes and caveats
    • Inclusions and exclusions checklist
      • Table Inclusions checklist
      • Table Exclusions checklist
    • Currency conversion rates for US$
      • Table Currency conversion rates for US$
    • Research methodology
      • Table Research methodology
      • Table Validation techniques employed for market sizing
      • Table Information sources
    • List of abbreviations
      • Table List of abbreviations

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