The global orphan drugs market size is calculated at USD 216.55 billion in 2025 and is predicted to surpass around USD 610.24 billion by 2034, representing a healthy CAGR of 12.20% between 2025 and 2034. The North America orphan drugs market size is calculated at USD 84.92 billion in 2024 and is expected to grow at a fastest CAGR of 12.32% during the forecast year. The market sizing and forecasts are revenue-based (USD Million/Billion), with 2024 as the base year.
1.1. Research Objective
1.2. Scope of the Study
1.3. Definition
2.1. Research Approach
2.2. Data Sources
2.3. Assumptions & Limitations
3.1. Market Snapshot
4.1. Introduction
4.2. Market Classification and Scope
4.3. Industry Value Chain Analysis
4.3.1. Raw Material Procurement Analysis
4.3.2. Sales and Distribution Channel Analysis
4.3.3. Downstream Buyer Analysis
5.1. COVID-19 Landscape: Orphan Drugs Industry Impact
5.2. COVID 19 - Impact Assessment for the Industry
5.3. COVID 19 Impact: Global Major Government Policy
5.4. Market Trends and Opportunities in the COVID-19 Landscape
6.1. Market Dynamics
6.1.1. Market Drivers
6.1.2. Market Restraints
6.1.3. Market Opportunities
6.2. Porter’s Five Forces Analysis
6.2.1. Bargaining power of suppliers
6.2.2. Bargaining power of buyers
6.2.3. Threat of substitute
6.2.4. Threat of new entrants
6.2.5. Degree of competition
7.1.1. Company Market Share/Positioning Analysis
7.1.2. Key Strategies Adopted by Players
7.1.3. Vendor Landscape
7.1.3.1. List of Suppliers
7.1.3.2. List of Buyers
8.1. Orphan Drugs Market, by Drug Type
8.1.1 Biologicals
8.1.1.1. Market Revenue and Forecast
8.1.2. Non-Biologicals
8.1.2.1. Market Revenue and Forecast
9.1. Orphan Drugs Market, by Therapy
9.1.1. Oncology
9.1.1.1. Market Revenue and Forecast
9.1.2. Haematology
9.1.2.1. Market Revenue and Forecast
9.1.3. Neurology
9.1.3.1. Market Revenue and Forecast
9.1.4. Infectious Diseases
9.1.4.1. Market Revenue and Forecast
9.1.5. Metabolic Disorders
9.1.5.1. Market Revenue and Forecast
9.1.6. Endocrinology
9.1.6.1. Market Revenue and Forecast
9.1.7. Immunology
9.1.7.1. Market Revenue and Forecast
9.1.8. Others
9.1.8.1. Market Revenue and Forecast
10.1. Orphan Drugs Market, by Distribution Channel
10.1.1. Hospital Pharmacies
10.1.1.1. Market Revenue and Forecast
10.1.2. Retail Pharmacies
10.1.2.1. Market Revenue and Forecast
10.1.3. Online Pharmacies
10.1.3.1. Market Revenue and Forecast
10.1.4. Others
10.1.4.1. Market Revenue and Forecast
11.1. North America
11.1.1. Market Revenue and Forecast, by Drug Type
11.1.2. Market Revenue and Forecast, by Therapy
11.1.3. Market Revenue and Forecast, by Distribution Channel
11.1.4. U.S.
11.1.4.1. Market Revenue and Forecast, by Drug Type
11.1.4.2. Market Revenue and Forecast, by Therapy
11.1.4.3. Market Revenue and Forecast, by Distribution Channel
11.1.5. Rest of North America
11.1.5.1. Market Revenue and Forecast, by Drug Type
11.1.5.2. Market Revenue and Forecast, by Therapy
11.1.5.3. Market Revenue and Forecast, by Distribution Channel
11.2. Europe
11.2.1. Market Revenue and Forecast, by Drug Type
11.2.2. Market Revenue and Forecast, by Therapy
11.2.3. Market Revenue and Forecast, by Distribution Channel
11.2.4. UK
11.2.4.1. Market Revenue and Forecast, by Drug Type
11.2.4.2. Market Revenue and Forecast, by Therapy
11.2.4.3. Market Revenue and Forecast, by Distribution Channel
11.2.5. Germany
11.2.5.1. Market Revenue and Forecast, by Drug Type
11.2.5.2. Market Revenue and Forecast, by Therapy
11.2.5.3. Market Revenue and Forecast, by Distribution Channel
11.2.6. France
11.2.6.1. Market Revenue and Forecast, by Drug Type
11.2.6.2. Market Revenue and Forecast, by Therapy
11.2.6.3. Market Revenue and Forecast, by Distribution Channel
11.2.7. Rest of Europe
11.2.7.1. Market Revenue and Forecast, by Drug Type
11.2.7.2. Market Revenue and Forecast, by Therapy
11.2.7.3. Market Revenue and Forecast, by Distribution Channel
11.3. APAC
11.3.1. Market Revenue and Forecast, by Drug Type
11.3.2. Market Revenue and Forecast, by Therapy
11.3.3. Market Revenue and Forecast, by Distribution Channel
11.3.4. India
11.3.4.1. Market Revenue and Forecast, by Drug Type
11.3.4.2. Market Revenue and Forecast, by Therapy
11.3.4.3. Market Revenue and Forecast, by Distribution Channel
11.3.5. China
11.3.5.1. Market Revenue and Forecast, by Drug Type
11.3.5.2. Market Revenue and Forecast, by Therapy
11.3.5.3. Market Revenue and Forecast, by Distribution Channel
11.3.6. Japan
11.3.6.1. Market Revenue and Forecast, by Drug Type
11.3.6.2. Market Revenue and Forecast, by Therapy
11.3.6.3. Market Revenue and Forecast, by Distribution Channel
11.3.7. Rest of APAC
11.3.7.1. Market Revenue and Forecast, by Drug Type
11.3.7.2. Market Revenue and Forecast, by Therapy
11.3.7.3. Market Revenue and Forecast, by Distribution Channel
11.4. MEA
11.4.1. Market Revenue and Forecast, by Drug Type
11.4.2. Market Revenue and Forecast, by Therapy
11.4.3. Market Revenue and Forecast, by Distribution Channel
11.4.4. GCC
11.4.4.1. Market Revenue and Forecast, by Drug Type
11.4.4.2. Market Revenue and Forecast, by Therapy
11.4.4.3. Market Revenue and Forecast, by Distribution Channel
11.4.5. North Africa
11.4.5.1. Market Revenue and Forecast, by Drug Type
11.4.5.2. Market Revenue and Forecast, by Therapy
11.4.5.3. Market Revenue and Forecast, by Distribution Channel
11.4.6. South Africa
11.4.6.1. Market Revenue and Forecast, by Drug Type
11.4.6.2. Market Revenue and Forecast, by Therapy
11.4.6.3. Market Revenue and Forecast, by Distribution Channel
11.4.7. Rest of MEA
11.4.7.1. Market Revenue and Forecast, by Drug Type
11.4.7.2. Market Revenue and Forecast, by Therapy
11.4.7.3. Market Revenue and Forecast, by Distribution Channel
11.5. Latin America
11.5.1. Market Revenue and Forecast, by Drug Type
11.5.2. Market Revenue and Forecast, by Therapy
11.5.3. Market Revenue and Forecast, by Distribution Channel
11.5.4. Brazil
11.5.4.1. Market Revenue and Forecast, by Drug Type
11.5.4.2. Market Revenue and Forecast, by Therapy
11.5.4.3. Market Revenue and Forecast, by Distribution Channel
11.5.5. Rest of LATAM
11.5.5.1. Market Revenue and Forecast, by Drug Type
11.5.5.2. Market Revenue and Forecast, by Therapy
11.5.5.3. Market Revenue and Forecast, by Distribution Channel
12.1. F. Hoffmann-La Roche Ltd
12.1.1. Company Overview
12.1.2. Product Offerings
12.1.3. Financial Performance
12.1.4. Recent Initiatives
12.2. Celgene Corporation
12.2.1. Company Overview
12.2.2. Product Offerings
12.2.3. Financial Performance
12.2.4. Recent Initiatives
12.3. Merk & Co., Inc.
12.3.1. Company Overview
12.3.2. Product Offerings
12.3.3. Financial Performance
12.3.4. Recent Initiatives
12.4. Novartis AG
12.4.1. Company Overview
12.4.2. Product Offerings
12.4.3. Financial Performance
12.4.4. Recent Initiatives
12.5. Sanofi
12.5.1. Company Overview
12.5.2. Product Offerings
12.5.3. Financial Performance
12.5.4. Recent Initiatives
12.6. Takeda Pharmaceutical Company Limited
12.6.1. Company Overview
12.6.2. Product Offerings
12.6.3. Financial Performance
12.6.4. Recent Initiatives
12.7. AstraZeneca
12.7.1. Company Overview
12.7.2. Product Offerings
12.7.3. Financial Performance
12.7.4. Recent Initiatives
12.8. Alexion Pharmaceuticals Inc.
12.8.1. Company Overview
12.8.2. Product Offerings
12.8.3. Financial Performance
12.8.4. Recent Initiatives
12.9. Eli Lilly and Company
12.9.1. Company Overview
12.9.2. Product Offerings
12.9.3. Financial Performance
12.9.4. Recent Initiatives
12.10. Amgen Inc
12.10.1. Company Overview
12.10.2. Product Offerings
12.10.3. Financial Performance
12.10.4. Recent Initiatives
13.1. Primary Research
13.2. Secondary Research
13.3. Assumptions
14.1. About Us
13.2. Glossary of Terms
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