The global orphan drugs market size reached USD 237.3 Billion in 2024. Looking forward, IMARC Group expects the market to reach USD 582.5 Billion by 2033, exhibiting a growth rate (CAGR) of 9.97% during 2025-2033.
Orphan drugs refer to pharmaceutical products used for the diagnosis, prevention and treatment of rare medical disorders. These drugs are manufactured to meet a specific public health requirement and usually have a limited market as they are produced for a small group of patients. They are effective against numerous oncological, metabolic, hematologic, immunologic, infectious and neurological diseases. Medical ailments, such as lymphoma, leukemia, cystic fibrosis, glioma, pancreatic cancer, ovarian cancer, multiple myeloma and renal cell carcinoma, are often life-threatening, chronic, progressive, degenerative and disabling and require specific treatment alternatives that are effective for their particular symptoms.
Orphan Drugs Market Trends:
The increasing prevalence of cancer and other rare genetic disorders is one of the key factors driving the growth of the market. Pharmaceutical manufacturers are developing innovative orphan drugs to provide personalized therapy to patients. The market is also being driven by increasing awareness among the masses regarding the benefits of orphan drugs. The emergence of new pharmaceutical drug manufacturers and the implementation of favorable government policies to inhibit the spread of contagious diseases are also contributing to the market growth. Additionally, various product innovations, such as the development of biological orphan drugs, are acting as other growth-inducing factors. These drugs can treat diseases, such as cancer, and reverse the damage caused to the stem cells, which is facilitating their overall demand significantly. Other factors, including improvements in the healthcare infrastructure, along with extensive research and development (R&D) activities, are expected to drive the market further.
Key Market Segmentation:
IMARC Group provides an analysis of the key trends in each sub-segment of the global orphan drugs market report, along with forecasts at the global, regional and country levels from 2025-2033. Our report has categorized the market based on drug type, disease type, phase, top selling drugs and distribution channel.
Breakup by Drug Type:
- Biological
- Non-Biological
- Oncology
- Hematology
- Neurology
- Cardiovascular
- Others
- Phase I
- Phase II
- Phase III
- Phase IV
- Revlimid
- Rituxan
- Copaxone
- Opdivo
- Keytruda
- Imbruvica
- Avonex
- Sensipar
- Soliris
- Others
- Hospital Pharmacies
- Retail Pharmacies
- Online Stores
- Others
- North America
- United States
- Canada
- Asia Pacific
- China
- Japan
- India
- South Korea
- Australia
- Indonesia
- Others
- Europe
- Germany
- France
- United Kingdom
- Italy
- Spain
- Russia
- Others
- Latin America
- Brazil
- Mexico
- Others
- Middle East and Africa
The report has also analysed the competitive landscape of the market with some of the key players being AbbVie Inc., Alexion Pharmaceuticals Inc., Amgen Inc., Biogen Inc., Bristol-Myers Squibb Company, F. Hoffmann-La Roche AG (Roche Holding AG), Jazz Pharmaceuticals Plc, Johnson & Johnson, Merck & Co. Inc., Novartis AG, Pfizer Inc., Sanofi S.A., Takeda Pharmaceutical Company Limited and Teva Pharmaceutical Industries Ltd.
Key Questions Answered in This Report
1.What was the size of the global orphan drugs market in 2024?
2.What is the expected growth rate of the global orphan drugs market during 2025-2033?
3.What are the key factors driving the global orphan drugs market?
4.What has been the impact of COVID-19 on the global orphan drugs market?
5.What is the breakup of the global orphan drugs market based on the drug type?
6.What is the breakup of the global orphan drugs market based on the disease type?
7.What is the breakup of the global orphan drugs market based on top selling drugs?
8.What is the breakup of the global orphan drugs market based on the distribution channel?
9.What are the key regions in the global orphan drugs market?
10.Who are the key players/companies in the global orphan drugs market?
- 1 Preface
- 2 Scope and Methodology
- 2.1 Objectives of the Study
- 2.2 Stakeholders
- 2.3 Data Sources
- 2.3.1 Primary Sources
- 2.3.2 Secondary Sources
- 2.4 Market Estimation
- 2.4.1 Bottom-Up Approach
- 2.4.2 Top-Down Approach
- 2.5 Forecasting Methodology
- 3 Executive Summary
- 4 Introduction
- 4.1 Overview
- 4.2 Key Industry Trends
- 5 Global Orphan Drugs Market
- 5.1 Market Overview
- 5.2 Market Performance
- 5.3 Impact of COVID-19
- 5.4 Market Forecast
- 6 Market Breakup by Drug Type
- 6.1 Biological
- 6.1.1 Market Trends
- 6.1.2 Market Forecast
- 6.2 Non-Biological
- 6.2.1 Market Trends
- 6.2.2 Market Forecast
- 7 Market Breakup by Disease Type
- 7.1 Oncology
- 7.1.1 Market Trends
- 7.1.2 Market Forecast
- 7.2 Hematology
- 7.2.1 Market Trends
- 7.2.2 Market Forecast
- 7.3 Neurology
- 7.3.1 Market Trends
- 7.3.2 Market Forecast
- 7.4 Cardiovascular
- 7.4.1 Market Trends
- 7.4.2 Market Forecast
- 7.5 Others
- 7.5.1 Market Trends
- 7.5.2 Market Forecast
- 8 Market Breakup by Phase
- 8.1 Phase I
- 8.1.1 Market Trends
- 8.1.2 Market Forecast
- 8.2 Phase II
- 8.2.1 Market Trends
- 8.2.2 Market Forecast
- 8.3 Phase III
- 8.3.1 Market Trends
- 8.3.2 Market Forecast
- 8.4 Phase IV
- 8.4.1 Market Trends
- 8.4.2 Market Forecast
- 9 Market Breakup by Top Selling Drugs
- 9.1 Revlimid
- 9.1.1 Market Trends
- 9.1.2 Market Forecast
- 9.2 Rituxan
- 9.2.1 Market Trends
- 9.2.2 Market Forecast
- 9.3 Copaxone
- 9.3.1 Market Trends
- 9.3.2 Market Forecast
- 9.4 Opdivo
- 9.4.1 Market Trends
- 9.4.2 Market Forecast
- 9.5 Keytruda
- 9.5.1 Market Trends
- 9.5.2 Market Forecast
- 9.6 Imbruvica
- 9.6.1 Market Trends
- 9.6.2 Market Forecast
- 9.7 Avonex
- 9.7.1 Market Trends
- 9.7.2 Market Forecast
- 9.8 Sensipar
- 9.8.1 Market Trends
- 9.8.2 Market Forecast
- 9.9 Soliris
- 9.9.1 Market Trends
- 9.9.2 Market Forecast
- 9.10 Others
- 9.10.1 Market Trends
- 9.10.2 Market Forecast
- 10 Market Breakup by Distribution Channel
- 10.1 Hospital Pharmacies
- 10.1.1 Market Trends
- 10.1.2 Market Forecast
- 10.2 Retail Pharmacies
- 10.2.1 Market Trends
- 10.2.2 Market Forecast
- 10.3 Online Stores
- 10.3.1 Market Trends
- 10.3.2 Market Forecast
- 10.4 Others
- 10.4.1 Market Trends
- 10.4.2 Market Forecast
- 11 Market Breakup by Region
- 11.1 North America
- 11.1.1 United States
- 11.1.1.1 Market Trends
- 11.1.1.2 Market Forecast
- 11.1.2 Canada
- 11.1.2.1 Market Trends
- 11.1.2.2 Market Forecast
- 11.2 Asia Pacific
- 11.2.1 China
- 11.2.1.1 Market Trends
- 11.2.1.2 Market Forecast
- 11.2.2 Japan
- 11.2.2.1 Market Trends
- 11.2.2.2 Market Forecast
- 11.2.3 India
- 11.2.3.1 Market Trends
- 11.2.3.2 Market Forecast
- 11.2.4 South Korea
- 11.2.4.1 Market Trends
- 11.2.4.2 Market Forecast
- 11.2.5 Australia
- 11.2.5.1 Market Trends
- 11.2.5.2 Market Forecast
- 11.2.6 Indonesia
- 11.2.6.1 Market Trends
- 11.2.6.2 Market Forecast
- 11.2.7 Others
- 11.2.7.1 Market Trends
- 11.2.7.2 Market Forecast
- 11.3 Europe
- 11.3.1 Germany
- 11.3.1.1 Market Trends
- 11.3.1.2 Market Forecast
- 11.3.2 France
- 11.3.2.1 Market Trends
- 11.3.2.2 Market Forecast
- 11.3.3 United Kingdom
- 11.3.3.1 Market Trends
- 11.3.3.2 Market Forecast
- 11.3.4 Italy
- 11.3.4.1 Market Trends
- 11.3.4.2 Market Forecast
- 11.3.5 Spain
- 11.3.5.1 Market Trends
- 11.3.5.2 Market Forecast
- 11.3.6 Russia
- 11.3.6.1 Market Trends
- 11.3.6.2 Market Forecast
- 11.3.7 Others
- 11.3.7.1 Market Trends
- 11.3.7.2 Market Forecast
- 11.4 Latin America
- 11.4.1 Brazil
- 11.4.1.1 Market Trends
- 11.4.1.2 Market Forecast
- 11.4.2 Mexico
- 11.4.2.1 Market Trends
- 11.4.2.2 Market Forecast
- 11.4.3 Others
- 11.4.3.1 Market Trends
- 11.4.3.2 Market Forecast
- 11.5 Middle East and Africa
- 11.5.1 Market Trends
- 11.5.2 Market Breakup by Country
- 11.5.3 Market Forecast
- 12 SWOT Analysis
- 12.1 Overview
- 12.2 Strengths
- 12.3 Weaknesses
- 12.4 Opportunities
- 12.5 Threats
- 13 Value Chain Analysis
- 14 Porters Five Forces Analysis
- 14.1 Overview
- 14.2 Bargaining Power of Buyers
- 14.3 Bargaining Power of Suppliers
- 14.4 Degree of Competition
- 14.5 Threat of New Entrants
- 14.6 Threat of Substitutes
- 15 Price Analysis
- 16 Competitive Landscape
- 16.1 Market Structure
- 16.2 Key Players
- 16.3 Profiles of Key Players
- 16.3.1 AbbVie Inc.
- 16.3.1.1 Company Overview
- 16.3.1.2 Product Portfolio
- 16.3.1.3 Financials
- 16.3.1.4 SWOT Analysis
- 16.3.2 Alexion Pharmaceuticals Inc.
- 16.3.2.1 Company Overview
- 16.3.2.2 Product Portfolio
- 16.3.2.3 Financials
- 16.3.2.4 SWOT Analysis
- 16.3.3 Amgen Inc.
- 16.3.3.1 Company Overview
- 16.3.3.2 Product Portfolio
- 16.3.3.3 Financials
- 16.3.3.4 SWOT Analysis
- 16.3.4 Biogen Inc.
- 16.3.4.1 Company Overview
- 16.3.4.2 Product Portfolio
- 16.3.4.3 Financials
- 16.3.4.4 SWOT Analysis
- 16.3.5 Bristol Myers Squibb Company
- 16.3.5.1 Company Overview
- 16.3.5.2 Product Portfolio
- 16.3.5.3 Financials
- 16.3.5.4 SWOT Analysis
- 16.3.6 F. Hoffmann-La Roche AG (Roche Holding AG)
- 16.3.6.1 Company Overview
- 16.3.6.2 Product Portfolio
- 16.3.6.3 SWOT Analysis
- 16.3.7 Jazz Pharmaceuticals Plc
- 16.3.7.1 Company Overview
- 16.3.7.2 Product Portfolio
- 16.3.7.3 Financials
- 16.3.7.4 SWOT Analysis
- 16.3.8 Johnson & Johnson
- 16.3.8.1 Company Overview
- 16.3.8.2 Product Portfolio
- 16.3.8.3 Financials
- 16.3.8.4 SWOT Analysis
- 16.3.9 Merck & Co. Inc.
- 16.3.9.1 Company Overview
- 16.3.9.2 Product Portfolio
- 16.3.9.3 Financials
- 16.3.9.4 SWOT Analysis
- 16.3.10 Novartis AG
- 16.3.10.1 Company Overview
- 16.3.10.2 Product Portfolio
- 16.3.10.3 Financials
- 16.3.10.4 SWOT Analysis
- 16.3.11 Pfizer Inc.
- 16.3.11.1 Company Overview
- 16.3.11.2 Product Portfolio
- 16.3.11.3 Financials
- 16.3.11.4 SWOT Analysis
- 16.3.12 Sanofi S.A.
- 16.3.12.1 Company Overview
- 16.3.12.2 Product Portfolio
- 16.3.12.3 Financials
- 16.3.12.4 SWOT Analysis
- 16.3.13 Takeda Pharmaceutical Company Limited
- 16.3.13.1 Company Overview
- 16.3.13.2 Product Portfolio
- 16.3.13.3 Financials
- 16.3.13.4 SWOT Analysis
- 16.3.14 Teva Pharmaceutical Industries Ltd
- 16.3.14.1 Company Overview
- 16.3.14.2 Product Portfolio
- 16.3.14.3 Financials
- 16.3.14.4 SWOT Analysis
Research Assistance
Learn how to effectively navigate the market research process to help guide your organization on the journey to success.
Download eBook