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Global Orphan Diseases Market

Orphan Diseases Market - By Type: Genetic Disorders, Rare Cancers, Rare Neurological Diseases, Others. By Treatment: Gene Therapy, Monoclonal Antibodies, Enzyme Replacement Therapy, Others. By End-user: Hospitals, Clinics, Research Institutes. By Region: North America, Europe, Asia-Pacific, Latin America, Middle East & Africa.
Report Code
CVMI2501185
Published
March 3, 2025
Last Updated
March 3, 2025
Coverage
Healthcare • Healthcare
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Market Snapshot

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Market Size (latest)
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Forecast window: —
Forecast Period
2024–2031
Base years: 2019–2023
Coverage Depth
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Segments • Regions • Players

Insights

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Key Players

  • AbbVie Inc.
  • Alexion Pharmaceuticals Inc.
  • Amgen Inc.
  • Biogen Inc.
  • Bristol-Myers Squibb Company
  • F. Hoffmann-La Roche AG
  • Jazz Pharmaceuticals Plc
  • Johnson & Johnson
  • Merck & Co. Inc.
  • Novartis AG
  • Pfizer Inc.
  • Sanofi S.A.
  • Takeda Pharmaceutical Company Limited
  • Teva Pharmaceutical Industries Ltd.
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Table Of Contents

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Global Orphan Diseases Market Report

Global Orphan Diseases Market Report

    1. Global Orphan Diseases Market Research Report
    1. 1.1 Study Objectives
    2. 1.2 Global Orphan Diseases Market - Overview
    3. 1.3 Reason to Read This Report
    4. 1.4 Methodology and Forecast Analysis
    2. Global Orphan Diseases Market Research Report - Preface
    1. 2.1 Global Orphan Diseases Market Research Report – Detailed Scope and Definitions
      1. 2.1.1 By Type
      2. 2.1.2 By Treatment
      3. 2.1.3 By End-user
      4. 2.1.4 By Region
    3. Global Orphan Diseases Market Dynamics
    1. 3.1. Drivers - Macro-Economic Based, Supply Side, and Demand Side Drivers
    2. 3.2. Restraints – By Type, By Treatment, By End-user, By Country
    3. 3.3. Opportunities – By Type, By Treatment, By End-user, By Country
    4. 3.4. Trends – By Type, By Treatment, By End-user, By Country
    5. 3.5. PEST Analysis
    6. 3.6. Porters Five Rule Analysis
    7. 3.7. Company’s Share Analysis (CSA) by Region or By Country
    8. 3.8. Global Orphan Diseases Market Research Report – DROTs Impact Analysis
    4. Global Orphan Diseases Market Research Report, Historic Data 2019 - 2023 and Forecast Analysis Data 2024 - 2031
    1. 4.1. Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
    2. 4.2. Annual Market Trend Assessment – Year-on-Year (YoY) Growth Analysis (%)
    3. 4.3. Incremental Market Value/Volume Opportunity between 2019 - 2023 and 2024 - 2031
    4. 4.4. Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    5. Global Orphan Diseases Market, By Type, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
    1. 5.1 Genetic Disorders
      1. 5.1.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 5.1.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 5.1.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 5.1.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    2. 5.2 Rare Cancers
      1. 5.2.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 5.2.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 5.2.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 5.2.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    3. 5.3 Rare Neurological Diseases
      1. 5.3.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 5.3.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 5.3.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 5.3.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    4. 5.4 Others
      1. 5.4.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 5.4.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 5.4.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 5.4.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    6. Global Orphan Diseases Market, By Treatment, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
    1. 6.1 Gene Therapy
      1. 6.1.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 6.1.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 6.1.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 6.1.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    2. 6.2 Monoclonal Antibodies
      1. 6.2.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 6.2.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 6.2.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 6.2.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    3. 6.3 Enzyme Replacement Therapy
      1. 6.3.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 6.3.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 6.3.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 6.3.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    4. 6.4 Others
      1. 6.4.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 6.4.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 6.4.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 6.4.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    7. Global Orphan Diseases Market, By End-user, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
    1. 7.1 Hospitals
      1. 7.1.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 7.1.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 7.1.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 7.1.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    2. 7.2 Clinics
      1. 7.2.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 7.2.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 7.2.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 7.2.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    3. 7.3 Research Institutes.
      1. 7.3.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 7.3.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 7.3.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 7.3.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    8. Global Orphan Diseases Market Forecast, By Region, 2019 - 2023 and 2024 - 2031 (Market Value, In USD Mn)
    1. 8.1 North America
      1. 8.1.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 8.1.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 8.1.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 8.1.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    2. 8.2 Europe
      1. 8.2.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 8.2.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 8.2.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 8.2.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    3. 8.3 Asia-Pacific
      1. 8.3.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 8.3.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 8.3.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 8.3.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    4. 8.4 Latin America
      1. 8.4.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 8.4.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 8.4.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 8.4.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
    5. 8.5 Middle East & Africa
      1. 8.5.1 Market Performance Review & Future Outlook: Assessing 2019 - 2023 and Predicting 2024 - 2031 Trends (USD Millions)
      2. 8.5.2 Annual Market Trend Assessment – Yearly Growth Observation (Y-O-Y)(%)
      3. 8.5.3 Incremental Market Value/Volume Opportunity between 2019 - 2023 and From 2024 to 2031
      4. 8.5.4 Market Shares Analysis in Years - 2019, 2023, 2024 and 2031
      6. 8.6 Global Orphan Diseases Market - Opportunity Analysis Index, By Type, By Treatment, By End-user, and Region, 2024 - 2031
    9. North America Global Orphan Diseases Market Analysis, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
      9.1 By Type Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 9.1.1 Genetic Disorders
      2. 9.1.2 Rare Cancers
      3. 9.1.3 Rare Neurological Diseases
      4. 9.1.4 Others
      9.2 By Treatment Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 9.2.1 Gene Therapy
      2. 9.2.2 Monoclonal Antibodies
      3. 9.2.3 Enzyme Replacement Therapy
      4. 9.2.4 Others
      9.3 By End-user Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 9.3.1 Hospitals
      2. 9.3.2 Clinics
      3. 9.3.3 Research Institutes.
      9.4 Country Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 9.4.1 United States
      2. 9.4.2 Canada
      9.5 North America Global Orphan Diseases Market – Opportunity Analysis Index, By Type, By Treatment, By End-user, and Country, 2024 - 2031
      9.6 Regional Trends Analysis
      9.7 North America Global Orphan Diseases Market Research Report - Company Profiles
      1. 9.7.1 Company 1 (United States)
      2. 9.7.2 Company 2 (Canada)
      3. 9.7.3 Company 3 (Canada)
    10. Europe Global Orphan Diseases Market Analysis, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
      10.1 By Type Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 10.1.1 Genetic Disorders
      2. 10.1.2 Rare Cancers
      3. 10.1.3 Rare Neurological Diseases
      4. 10.1.4 Others
      10.2 By Treatment Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 10.2.1 Gene Therapy
      2. 10.2.2 Monoclonal Antibodies
      3. 10.2.3 Enzyme Replacement Therapy
      4. 10.2.4 Others
      10.3 By End-user Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 10.3.1 Hospitals
      2. 10.3.2 Clinics
      3. 10.3.3 Research Institutes.
      10.4 Country Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 10.4.1 Germany
      2. 10.4.2 United Kingdom
      3. 10.4.3 France
      4. 10.4.4 Spain
      5. 10.4.5 Italy
      6. 10.4.6 Russia
      7. 10.4.7 Netherlands
      8. 10.4.8 Poland
      9. 10.4.9 Rest of Europe
      10.5 Europe Global Orphan Diseases Market – Opportunity Analysis Index, By Type, By Treatment, By End-user, and Country, 2024 - 2031
      10.6 Regional Trends Analysis
      10.7 Europe Global Orphan Diseases Market Research Report - Company Profiles
      1. 10.7.1 Company 1 (Germany)
      2. 10.7.2 Company 2 (United Kingdom)
      3. 10.7.3 Company 3 (United Kingdom)
    11. Asia-Pacific Global Orphan Diseases Market Analysis, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
      11.1 By Type Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 11.1.1 Genetic Disorders
      2. 11.1.2 Rare Cancers
      3. 11.1.3 Rare Neurological Diseases
      4. 11.1.4 Others
      11.2 By Treatment Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 11.2.1 Gene Therapy
      2. 11.2.2 Monoclonal Antibodies
      3. 11.2.3 Enzyme Replacement Therapy
      4. 11.2.4 Others
      11.3 By End-user Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 11.3.1 Hospitals
      2. 11.3.2 Clinics
      3. 11.3.3 Research Institutes.
      11.4 Country Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 11.4.1 China
      2. 11.4.2 Japan
      3. 11.4.3 India
      4. 11.4.4 South Korea
      5. 11.4.5 Australia & NZ
      6. 11.4.6 ASEAN
      7. 11.4.7 Rest of Asia-Pacific
      11.5 Asia-Pacific Global Orphan Diseases Market – Opportunity Analysis Index, By Type, By Treatment, By End-user, and Country, 2024 - 2031
      11.6 Regional Trends Analysis
      11.7 Asia-Pacific Global Orphan Diseases Market Research Report - Company Profiles
      1. 11.7.1 Company 1 (China)
      2. 11.7.2 Company 2 (Japan)
      3. 11.7.3 Company 3 (Japan)
    12. Latin America Global Orphan Diseases Market Analysis, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
      12.1 By Type Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 12.1.1 Genetic Disorders
      2. 12.1.2 Rare Cancers
      3. 12.1.3 Rare Neurological Diseases
      4. 12.1.4 Others
      12.2 By Treatment Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 12.2.1 Gene Therapy
      2. 12.2.2 Monoclonal Antibodies
      3. 12.2.3 Enzyme Replacement Therapy
      4. 12.2.4 Others
      12.3 By End-user Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 12.3.1 Hospitals
      2. 12.3.2 Clinics
      3. 12.3.3 Research Institutes.
      12.4 Country Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 12.4.1 Brazil
      2. 12.4.2 Mexico
      3. 12.4.3 Argentina
      4. 12.4.4 Peru
      5. 12.4.5 Colombia
      6. 12.4.6 Rest of Latin America
      12.5 Latin America Global Orphan Diseases Market – Opportunity Analysis Index, By Type, By Treatment, By End-user, and Country, 2024 - 2031
      12.6 Regional Trends Analysis
      12.7 Latin America Global Orphan Diseases Market Research Report - Company Profiles
      1. 12.7.1 Company 1 (Brazil)
      2. 12.7.2 Company 2 (Mexico)
      3. 12.7.3 Company 3 (Mexico)
    13. Middle East & Africa Global Orphan Diseases Market Analysis, 2019 - 2023 and Forecast, 2024 - 2031 (Market Value, In USD Mn)
      13.1 By Type Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 13.1.1 Genetic Disorders
      2. 13.1.2 Rare Cancers
      3. 13.1.3 Rare Neurological Diseases
      4. 13.1.4 Others
      13.2 By Treatment Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 13.2.1 Gene Therapy
      2. 13.2.2 Monoclonal Antibodies
      3. 13.2.3 Enzyme Replacement Therapy
      4. 13.2.4 Others
      13.3 By End-user Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 13.3.1 Hospitals
      2. 13.3.2 Clinics
      3. 13.3.3 Research Institutes.
      13.4 Country Analysis 2019 - 2023 and Forecast, 2024 - 2031 by Market Assessment (USD Mn), Yearly Growth Rate (%), and Market Presence (%)
      1. 13.4.1 Saudi Arabia
      2. 13.4.2 UAE
      3. 13.4.3 South Africa
      4. 13.4.4 Egypt
      5. 13.4.5 Israel
      6. 13.4.6 Rest of Middle East and Africa
      13.5 Middle East & Africa Global Orphan Diseases Market – Opportunity Analysis Index, By Type, By Treatment, By End-user, and Country, 2024 - 2031
      13.6 Regional Trends Analysis
      13.7 Middle East & Africa Global Orphan Diseases Market Research Report - Company Profiles
      1. 13.7.1 Company 1 (Saudi Arabia)
      2. 13.7.2 Company 2 (UAE)
      3. 13.7.3 Company 3 (UAE)
    14. Competition Landscape
      14.1 Strategic Dashboard of Top Market Players
      14.2 Company Profiles (Introduction, Financial Assessments, Portfolio of Offerings, Milestones and Achievements, Strategic Initiative, and SWOT Analysis)
      1. 14.2.1 AbbVie Inc.
      2. 14.2.2 Alexion Pharmaceuticals Inc.
      3. 14.2.3 Amgen Inc.
      4. 14.2.4 Biogen Inc.
      5. 14.2.5 Bristol-Myers Squibb Company
      6. 14.2.6 F. Hoffmann-La Roche AG
      7. 14.2.7 Jazz Pharmaceuticals Plc
      8. 14.2.8 Johnson & Johnson
      9. 14.2.9 Merck & Co. Inc.
      10. 14.2.10 Novartis AG
      11. 14.2.11 Pfizer Inc.
      12. 14.2.12 Sanofi S.A.
      13. 14.2.13 Takeda Pharmaceutical Company Limited
      14. 14.2.14 Teva Pharmaceutical Industries Ltd.
    15. Data Collection Method and Research Approach
    16. Principal Presumptions and Acronyms

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