Orphan Drugs Market Size, Share & Industry Analysis, By Therapy Area (Oncology, Hematology, Neurology, Endocrinology, Cardiovascular, Respiratory, Immunotherapy, Infectious Diseases, and Others), By Drug Type (Biologics and Non-Biologics), By Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Online Pharmacy, and Others), and Regional Forecast, 2026-2034

Last Updated: January 26, 2026 | Format: PDF | Report ID: FBI100088

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Table of Content

Introduction
1. Research Scope
2. Market Segmentation
3. Research Methodology
4. Definitions and Assumptions
Executive Summary
Market Dynamics
1. Market Drivers
2. Market Restraints
3. Market Opportunities
4. Market Trends
Key Insights
1. Prevalence of Rare Diseases, By Key Countries/Regions, 2025
2. Pipeline Analysis, By Key Players
3. New Product Launches
4. Key Industry Developments: Mergers, Acquisitions & Partnerships
5. Technological Developments
6. Impact of COVID-19 on the Global Orphan Drugs Market
Global Orphan Drugs Market Analysis, Insights and Forecast, 2021-2034
1. Market Analysis, Insights and Forecast – By Therapy Area
  1. Oncology
  2. Hematology
  3. Neurology
  4. Endocrinology
  5. Cardiovascular
  6. Respiratory
  7. Immunotherapy
  8. Infectious Diseases
  9. Others
2. Market Analysis, Insights and Forecast – By Drug Type
  1. Biologics
  2. Non-Biologics
3. Market Analysis, Insights and Forecast – By Distribution Channel
  1. Hospital Pharmacies
  2. Retail Pharmacies
  3. Online Pharmacies
  4. Others
4. Market Analysis, Insights and Forecast – By Region
  1. North America
  2. Europe
  3. Asia Pacific
  4. Rest of the World
North America Orphan Drugs Market Analysis, Insights and Forecast, 2021-2034
1. Market Analysis, Insights and Forecast – By Therapy Area
  1. Oncology
  2. Hematology
  3. Neurology
  4. Endocrinology
  5. Cardiovascular
  6. Respiratory
  7. Immunotherapy
  8. Infectious Diseases
  9. Others
2. Market Analysis, Insights and Forecast – By Drug Type
  1. Biologics
  2. Non-Biologics
3. Market Analysis, Insights and Forecast – By Distribution Channel
  1. Hospital Pharmacies
  2. Retail Pharmacies
  3. Online Pharmacies
  4. Others
4. Market Analysis, Insights and Forecast – By Country
  1. U.S.
  2. Canada
Europe Orphan Drugs Market Analysis, Insights and Forecast, 2021-2034
1. Market Analysis, Insights and Forecast – By Therapy Area
  1. Oncology
  2. Hematology
  3. Neurology
  4. Endocrinology
  5. Cardiovascular
  6. Respiratory
  7. Immunotherapy
  8. Infectious Diseases
  9. Others
2. Market Analysis, Insights and Forecast – By Drug Type
  1. Biologics
  2. Non-Biologics
3. Market Analysis, Insights and Forecast – By Distribution Channel
  1. Hospital Pharmacies
  2. Retail Pharmacies
  3. Online Pharmacies
  4. Others
4. Market Analysis, Insights and Forecast – By Country/ Sub-Region
  1. U.K.
  2. Germany
  3. France
  4. Italy
  5. Spain
  6. Scandinavia
  7. Rest of Europe
Asia Pacific Orphan Drugs Market Analysis, Insights and Forecast, 2021-2034
1. Market Analysis, Insights and Forecast – By Therapy Area
  1. Oncology
  2. Hematology
  3. Neurology
  4. Endocrinology
  5. Cardiovascular
  6. Respiratory
  7. Immunotherapy
  8. Infectious Diseases
  9. Others
2. Market Analysis, Insights and Forecast – By Drug Type
  1. Biologics
  2. Non-Biologics
3. Market Analysis, Insights and Forecast – By Distribution Channel
  1. Hospital Pharmacies
  2. Retail Pharmacies
  3. Online Pharmacies
  4. Others
4. Market Analysis, Insights and Forecast – By Country/ Sub-Region
  1. Japan
  2. China
  3. India
  4. Australia
  5. Southeast Asia
  6. Rest of Asia Pacific
Rest of the World Orphan Drugs Market Analysis, Insights and Forecast, 2021-2034
1. Market Analysis, Insights and Forecast – By Therapy Area
  1. Oncology
  2. Hematology
  3. Neurology
  4. Endocrinology
  5. Cardiovascular
  6. Respiratory
  7. Immunotherapy
  8. Infectious Diseases
  9. Others
2. Market Analysis, Insights and Forecast – By Drug Type
  1. Biologics
  2. Non-Biologics
3. Market Analysis, Insights and Forecast – By Distribution Channel
  1. Hospital Pharmacies
  2. Retail Pharmacies
  3. Online Pharmacies
  4. Others
Competitive Analysis
1. Global Market Share Analysis (2025)
2. Company Profiles
  1. Amgen Inc. (U.S.)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  2. Bayer AG (Germany)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  3. F. Hoffmann-La Roche Ltd (Switzerland)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  4. Alexion Pharmaceuticals Inc. (U.S.)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  5. Novo Nordisk A/S (Denmark)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  6. Novartis AG (Switzerland)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  7. Bristol-Myers Squibb Company (U.S.)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  8. AstraZeneca (U.K.)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  9. DAIICHI SANKYO COMPANY, LIMITED (Japan)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  10. GlaxoSmithKline plc (U.K.)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)

Introduction
1. Research Scope
2. Market Segmentation
3. Research Methodology
4. Definitions and Assumptions
Executive Summary
Market Dynamics
1. Market Drivers
2. Market Restraints
3. Market Opportunities
4. Market Trends
Key Insights
1. Prevalence of Rare Diseases, By Key Countries/Regions, 2025
2. Pipeline Analysis, By Key Players
3. New Product Launches
4. Key Industry Developments: Mergers, Acquisitions & Partnerships
5. Technological Developments
6. Impact of COVID-19 on the Global Orphan Drugs Market
Global Orphan Drugs Market Analysis, Insights and Forecast, 2021-2034
1. Market Analysis, Insights and Forecast – By Therapy Area
  1. Oncology
  2. Hematology
  3. Neurology
  4. Endocrinology
  5. Cardiovascular
  6. Respiratory
  7. Immunotherapy
  8. Infectious Diseases
  9. Others
2. Market Analysis, Insights and Forecast – By Drug Type
  1. Biologics
  2. Non-Biologics
3. Market Analysis, Insights and Forecast – By Distribution Channel
  1. Hospital Pharmacies
  2. Retail Pharmacies
  3. Online Pharmacies
  4. Others
4. Market Analysis, Insights and Forecast – By Region
  1. North America
  2. Europe
  3. Asia Pacific
  4. Rest of the World
North America Orphan Drugs Market Analysis, Insights and Forecast, 2021-2034
1. Market Analysis, Insights and Forecast – By Therapy Area
  1. Oncology
  2. Hematology
  3. Neurology
  4. Endocrinology
  5. Cardiovascular
  6. Respiratory
  7. Immunotherapy
  8. Infectious Diseases
  9. Others
2. Market Analysis, Insights and Forecast – By Drug Type
  1. Biologics
  2. Non-Biologics
3. Market Analysis, Insights and Forecast – By Distribution Channel
  1. Hospital Pharmacies
  2. Retail Pharmacies
  3. Online Pharmacies
  4. Others
4. Market Analysis, Insights and Forecast – By Country
  1. U.S.
  2. Canada
Europe Orphan Drugs Market Analysis, Insights and Forecast, 2021-2034
1. Market Analysis, Insights and Forecast – By Therapy Area
  1. Oncology
  2. Hematology
  3. Neurology
  4. Endocrinology
  5. Cardiovascular
  6. Respiratory
  7. Immunotherapy
  8. Infectious Diseases
  9. Others
2. Market Analysis, Insights and Forecast – By Drug Type
  1. Biologics
  2. Non-Biologics
3. Market Analysis, Insights and Forecast – By Distribution Channel
  1. Hospital Pharmacies
  2. Retail Pharmacies
  3. Online Pharmacies
  4. Others
4. Market Analysis, Insights and Forecast – By Country/ Sub-Region
  1. U.K.
  2. Germany
  3. France
  4. Italy
  5. Spain
  6. Scandinavia
  7. Rest of Europe
Asia Pacific Orphan Drugs Market Analysis, Insights and Forecast, 2021-2034
1. Market Analysis, Insights and Forecast – By Therapy Area
  1. Oncology
  2. Hematology
  3. Neurology
  4. Endocrinology
  5. Cardiovascular
  6. Respiratory
  7. Immunotherapy
  8. Infectious Diseases
  9. Others
2. Market Analysis, Insights and Forecast – By Drug Type
  1. Biologics
  2. Non-Biologics
3. Market Analysis, Insights and Forecast – By Distribution Channel
  1. Hospital Pharmacies
  2. Retail Pharmacies
  3. Online Pharmacies
  4. Others
4. Market Analysis, Insights and Forecast – By Country/ Sub-Region
  1. Japan
  2. China
  3. India
  4. Australia
  5. Southeast Asia
  6. Rest of Asia Pacific
Rest of the World Orphan Drugs Market Analysis, Insights and Forecast, 2021-2034
1. Market Analysis, Insights and Forecast – By Therapy Area
  1. Oncology
  2. Hematology
  3. Neurology
  4. Endocrinology
  5. Cardiovascular
  6. Respiratory
  7. Immunotherapy
  8. Infectious Diseases
  9. Others
2. Market Analysis, Insights and Forecast – By Drug Type
  1. Biologics
  2. Non-Biologics
3. Market Analysis, Insights and Forecast – By Distribution Channel
  1. Hospital Pharmacies
  2. Retail Pharmacies
  3. Online Pharmacies
  4. Others
Competitive Analysis
1. Global Market Share Analysis (2025)
2. Company Profiles
  1. Amgen Inc. (U.S.)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  2. Bayer AG (Germany)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  3. F. Hoffmann-La Roche Ltd (Switzerland)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  4. Alexion Pharmaceuticals Inc. (U.S.)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  5. Novo Nordisk A/S (Denmark)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  6. Novartis AG (Switzerland)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  7. Bristol-Myers Squibb Company (U.S.)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  8. AstraZeneca (U.K.)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  9. DAIICHI SANKYO COMPANY, LIMITED (Japan)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)
  10. GlaxoSmithKline plc (U.K.)
    1. Overview
    2. Products & Services
    3. SWOT Analysis
    4. Recent Developments
    5. Strategies
    6. Financials (Based on Availability)

Read Less

List of Figures

Figure 1: Global Orphan Drugs Market Revenue Breakdown (USD billion, %) By Region, 2025 & 2034

Figure 2: Global Orphan Drugs Market Value Share (%), By Therapy Area, 2025 & 2034

Figure 3: Global Orphan Drugs Market Value Share (%), By Drug Type, 2025 & 2034

Figure 4: Global Orphan Drugs Market Value Share (%), By Distribution Channels, 2025 & 2034

Figure 5: Global Orphan Drugs Market Value (USD billion), By Region, 2025 & 2034

Figure 6: North America Orphan Drugs Market Value (USD billion), By Therapy Area, 2025 & 2034

Figure 7: North America Orphan Drugs Market Value Share (%), By Therapy Area, 2025

Figure 8: North America Orphan Drugs Market Value (USD billion), By Drug Type, 2025 & 2034

Figure 9: North America Orphan Drugs Market Value Share (%), By Drug Type, 2025

Figure 10: North America Orphan Drugs Market Value (USD billion), By Distribution Channels, 2025 & 2034

Figure 11: North America Orphan Drugs Market Value Share (%), By Distribution Channels, 2025

Figure 12: North America Orphan Drugs Market Value (USD billion), By Country, 2025 & 2034

Figure 13: North America Orphan Drugs Market Value Share (%), By Country, 2025

Figure 14: Europe Orphan Drugs Market Value (USD billion), By Therapy Area, 2025 & 2034

Figure 15: Europe Orphan Drugs Market Value Share (%), By Therapy Area, 2025

Figure 16: Europe Orphan Drugs Market Value (USD billion), By Drug Type, 2025 & 2034

Figure 17: Europe Orphan Drugs Market Value Share (%), By Drug Type, 2025

Figure 18: Europe Orphan Drugs Market Value (USD billion), By Distribution Channels, 2025 & 2034

Figure 19: Europe Orphan Drugs Market Value Share (%), By Distribution Channels, 2025

Figure 20: Europe Orphan Drugs Market Value (USD billion), By Country/ Sub-region, 2025 & 2034

Figure 21: Europe Orphan Drugs Market Value Share (%), By Country/ Sub-region, 2025

Figure 22: Asia Pacific Orphan Drugs Market Value (USD billion), By Therapy Area, 2025 & 2034

Figure 23: Asia Pacific Orphan Drugs Market Value Share (%), By Therapy Area, 2025

Figure 24: Asia Pacific Orphan Drugs Market Value (USD billion), By Drug Type, 2025 & 2034

Figure 25: Asia Pacific Orphan Drugs Market Value Share (%), By Drug Type, 2025

Figure 26: Asia Pacific Orphan Drugs Market Value (USD billion), By Distribution Channels, 2025 & 2034

Figure 27: Asia Pacific Orphan Drugs Market Value Share (%), By Distribution Channels, 2025

Figure 28: Asia Pacific Orphan Drugs Market Value (USD billion), By Country/ Sub-region, 2025 & 2034

Figure 29: Asia Pacific Orphan Drugs Market Value Share (%), By Country/ Sub-region, 2025

Figure 30: Rest of the World Orphan Drugs Market Value (USD billion), By Therapy Area, 2025 & 2034

Figure 31: Rest of the World Orphan Drugs Market Value Share (%), By Therapy Area, 2025

Figure 32: Rest of the World Orphan Drugs Market Value (USD billion), By Drug Type, 2025 & 2034

Figure 33: Rest of the World Orphan Drugs Market Value Share (%), By Drug Type, 2025

Figure 34: Rest of the World Orphan Drugs Market Value (USD billion), By Distribution Channels, 2025 & 2034

Figure 35: Rest of the World Orphan Drugs Market Value Share (%), By Distribution Channels, 2025

Figure 36: Global Orphan Drugs Market Share (%), By Company, 2025

List of Tables

Table 1: Global Orphan Drugs Market Revenue (USD billion) Forecast, By Therapy Area, 2021–2034

Table 2: Global Orphan Drugs Market Revenue (USD billion) Forecast, By Drug Type, 2021–2034

Table 3: Global Orphan Drugs Market Revenue (USD billion) Forecast, By Distribution Channels, 2021–2034

Table 4: Global Orphan Drugs Market Revenue (USD billion) Forecast, By Region, 2021-2034

Table 5: North America Orphan Drugs Market Revenue (USD billion) Forecast, By Therapy Area, 2021-2034

Table 6: North America Orphan Drugs Market Revenue (USD billion) Forecast, By Drug Type, 2021-2034

Table 7: North America Orphan Drugs Market Revenue (USD billion) Forecast, By Distribution Channels, 2021-2034

Table 8: North America Orphan Drugs Market Revenue (USD billion) Forecast, By Country, 2021-2034

Table 9: Europe Orphan Drugs Market Revenue (USD billion) Forecast, By Therapy Area, 2021-2034

Table 10: Europe Orphan Drugs Market Revenue (USD billion) Forecast, By Drug Type, 2021-2034

Table 11: Europe Orphan Drugs Market Revenue (USD billion) Forecast, By Distribution Channels, 2021-2034

Table 12: Europe Orphan Drugs Market Revenue (USD billion) Forecast, By Country/ Sub-region, 2021-2034

Table 13: Asia Pacific Orphan Drugs Market Revenue (USD billion) Forecast, By Therapy Area, 2021-2034

Table 14: Asia Pacific Orphan Drugs Market Revenue (USD billion) Forecast, By Drug Type, 2021-2034

Table 15: Asia Pacific Orphan Drugs Market Revenue (USD billion) Forecast, By Distribution Channels, 2021-2034

Table 16: Asia Pacific Orphan Drugs Market Revenue (USD billion) Forecast, By Country/ Sub-region, 2021-2034

Table 17: Rest of the World Orphan Drugs Market Revenue (USD billion) Forecast, By Therapy Area, 2021-2034

Table 18: Rest of the World Orphan Drugs Market Revenue (USD billion) Forecast, By Drug Type, 2021-2034

Table 19: Rest of the World Orphan Drugs Market Revenue (USD billion) Forecast, By Distribution Channels, 2021-2034

STUDY PERIOD: 2021-2034
BASE YEAR: 2025
HISTORICAL DATA: 2021-2024
NO OF PAGES: 135

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