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Global Rare Disease Drug Market Research Report 2025
Published Date: August 2025
|
Report Code: QYRE-Auto-30P9346
Home | Market Reports | Health| Pharmacy
Global Rare Disease Drug Market Insights and Forecast to 2028
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Global Rare Disease Drug Market Research Report 2025

Code: QYRE-Auto-30P9346
Report
August 2025
Pages:109
QYResearch
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DESCRIPTION
TABLE OF CONTENT
TABLES & FIGURES
FEATURED COMPANIES
Novartis
Bristol Myers Squibb Company
Celgene Corporation
F Hoffmann La Roche Ltd
Pfizer Inc
Sanofi
Alexion Pharmaceuticals Inc
Eli Lilly and Company
Novo Nordisk A S
Astrazeneca plc
Eisai Co Ltd
Daiichi Sankyo Company Limited
Bayer
GlaxoSmithKline PLC
Merck& Co. Inc
Johnson & Johnson
Biogen
Takeda
Amgen Inc
Deciphera
Atara Biotherapeutics
ProQR

Rare Disease Drug Market Size

The global pharmaceutical market is 1475 billion USD in 2022, growing at a CAGR of 5% during the next six years.

Rare Disease Drug Market

Rare Disease Drug Market

The global market for Rare Disease Drug was valued at US$ million in the year 2024 and is projected to reach a revised size of US$ million by 2031, growing at a CAGR of %during the forecast period.
The pharmaceutical market includes chemical drugs and biological drugs. For biologics is expected to 381 billion USD in 2022. In comparison, the chemical drug market is estimated to increase from 1005 billion in 2018 to 1094 billion U.S. dollars in 2022. The pharmaceutical market factors such as increasing demand for healthcare, technological advancements, and the rising prevalence of chronic diseases, increase in funding from private & government organizations for development of pharmaceutical manufacturing segments and rise in R&D activities for drugs. However, the industry also faces challenges such as stringent regulations, high costs of research and development, and patent expirations. Companies need to continuously innovate and adapt to these challenges to stay competitive in the market and ensure their products reach patients in need. Additionally, the COVID-19 pandemic has highlighted the importance of vaccine development and supply chain management, further emphasizing the need for pharmaceutical companies to be agile and responsive to emerging public health needs.

Report Scope

This report aims to provide a comprehensive presentation of the global market for Rare Disease Drug, with both quantitative and qualitative analysis, to help readers develop business/growth strategies, assess the market competitive situation, analyze their position in the current marketplace, and make informed business decisions regarding Rare Disease Drug.
The Rare Disease Drug market size, estimations, and forecasts are provided in terms of sales volume (K Doses) and revenue ($ millions), considering 2024 as the base year, with history and forecast data for the period from 2020 to 2031. This report segments the global Rare Disease Drug market comprehensively. Regional market sizes, concerning products by Type, by Application, and by players, are also provided.
For a more in-depth understanding of the market, the report provides profiles of the competitive landscape, key competitors, and their respective market ranks. The report also discusses technological trends and new product developments.
The report will help the Rare Disease Drug manufacturers, new entrants, and industry chain related companies in this market with information on the revenues, sales volume, and average price for the overall market and the sub-segments across the different segments, by company, by Type, by Application, and by regions.

Scope of Rare Disease Drug Market Report

Report Metric Details
Report Name Rare Disease Drug Market
CAGR 5%
Segment by Type
  • Above 1‰
  • 0.65‰～1‰
  • Below 0.1‰
Segment by Application
  • Hospital Pharmacies
  • Speciality Pharmacies
  • Retail pharmacies
  • Others
Consumption by Region
  • North America (United States, Canada)
  • Europe (Germany, France, UK, Italy, Russia)
  • Asia-Pacific (China, Japan, South Korea, Taiwan)
  • Southeast Asia (India)
  • Latin America (Mexico, Brazil)
By Company Novartis AG, Bristol-Myers Squibb Company, Celgene Corporation, F. Hoffmann-La Roche Ltd., Pfizer, Inc., Sanofi S.A., Alexion Pharmaceuticals, Inc., Eli Lilly and Company, Novo Nordisk A/S, AstraZeneca, Eisai Co., Ltd., Daiichi Sankyo Company Limited, Bayer AG, GlaxoSmithKline, Merck & Co., Inc., Johnson & Johnson, Biogen, Inc., Takeda, Amgen, Inc., Deciphera, Atara Biotherapeutics, ProQR
Forecast units USD million in value
Report coverage Revenue and volume forecast, company share, competitive landscape, growth factors and trends

Core Chapters

  • Chapter 1: Introduces the report scope of the report, executive summary of different market segments (by region, product type, application, etc), including the market size of each market segment, future development potential, and so on. It offers a high-level view of the current state of the market and its likely evolution in the short to mid-term, and long term.
  • Chapter 2: Detailed analysis of Rare Disease Drug manufacturers competitive landscape, price, sales and revenue market share, latest development plan, merger, and acquisition information, etc.
  • Chapter 3: Sales, revenue of Rare Disease Drug in regional level and country level. It provides a quantitative analysis of the market size and development potential of each region and its main countries and introduces the market development, future development prospects, market space, and market size of each country in the world.
  • Chapter 4: Provides the analysis of various market segments by Type, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different market segments.
  • Chapter 5: Provides the analysis of various market segments by Application, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different downstream markets.
  • Chapter 6: Provides profiles of key players, introducing the basic situation of the main companies in the market in detail, including product sales, revenue, price, gross margin, product introduction, recent development, etc.
  • Chapter 7: Analysis of industrial chain, including the upstream and downstream of the industry.
  • Chapter 8: Introduces the market dynamics, latest developments of the market, the driving factors and restrictive factors of the market, the challenges and risks faced by manufacturers in the industry, and the analysis of relevant policies in the industry.
  • Chapter 9: The main points and conclusions of the report. 

FAQ for this report

Who are the main players in the Rare Disease Drug Market report?

Ans: The main players in the Rare Disease Drug Market are Novartis AG, Bristol-Myers Squibb Company, Celgene Corporation, F. Hoffmann-La Roche Ltd., Pfizer, Inc., Sanofi S.A., Alexion Pharmaceuticals, Inc., Eli Lilly and Company, Novo Nordisk A/S, AstraZeneca, Eisai Co., Ltd., Daiichi Sankyo Company Limited, Bayer AG, GlaxoSmithKline, Merck & Co., Inc., Johnson & Johnson, Biogen, Inc., Takeda, Amgen, Inc., Deciphera, Atara Biotherapeutics, ProQR

What are the Application segmentation covered in the Rare Disease Drug Market report?

Ans: The Applications covered in the Rare Disease Drug Market report are Hospital Pharmacies, Speciality Pharmacies, Retail pharmacies, Others

What are the Type segmentation covered in the Rare Disease Drug Market report?

Ans: The Types covered in the Rare Disease Drug Market report are Above 1‰, 0.65‰～1‰, Below 0.1‰

1 Rare Disease Drug Market Overview
1.1 Product Definition
1.2 Rare Disease Drug by Type
1.2.1 Global Rare Disease Drug Market Value Comparison by Type (2024 VS 2031)
1.2.2 Above 1‰
1.2.3 0.65‰～1‰
1.2.4 Below 0.1‰
1.3 Rare Disease Drug by Application
1.3.1 Global Rare Disease Drug Market Value by Application (2024 VS 2031)
1.3.2 Hospital Pharmacies
1.3.3 Speciality Pharmacies
1.3.4 Retail pharmacies
1.3.5 Others
1.4 Global Rare Disease Drug Market Size Estimates and Forecasts
1.4.1 Global Rare Disease Drug Revenue 2020-2031
1.4.2 Global Rare Disease Drug Sales 2020-2031
1.4.3 Global Rare Disease Drug Market Average Price (2020-2031)
1.5 Assumptions and Limitations
2 Rare Disease Drug Market Competition by Manufacturers
2.1 Global Rare Disease Drug Sales Market Share by Manufacturers (2020-2025)
2.2 Global Rare Disease Drug Revenue Market Share by Manufacturers (2020-2025)
2.3 Global Rare Disease Drug Average Price by Manufacturers (2020-2025)
2.4 Global Key Players of Rare Disease Drug, Industry Ranking, 2022 VS 2023 VS 2024
2.5 Global Key Manufacturers of Rare Disease Drug, Manufacturing Sites & Headquarters
2.6 Global Key Manufacturers of Rare Disease Drug, Product Type & Application
2.7 Global Key Manufacturers of Rare Disease Drug, Date of Enter into This Industry
2.8 Global Rare Disease Drug Market Competitive Situation and Trends
2.8.1 Global Rare Disease Drug Market Concentration Rate
2.8.2 The Global 5 and 10 Largest Rare Disease Drug Players Market Share by Revenue
2.8.3 Global Rare Disease Drug Market Share by Company Type (Tier 1, Tier 2, and Tier 3)
2.9 Manufacturers Mergers & Acquisitions, Expansion Plans
3 Global Rare Disease Drug Market Scenario by Region
3.1 Global Rare Disease Drug Market Size by Region: 2020 Versus 2024 Versus 2031
3.2 Global Rare Disease Drug Sales by Region: 2020-2031
3.2.1 Global Rare Disease Drug Sales by Region: 2020-2025
3.2.2 Global Rare Disease Drug Sales by Region: 2026-2031
3.3 Global Rare Disease Drug Revenue by Region: 2020-2031
3.3.1 Global Rare Disease Drug Revenue by Region: 2020-2025
3.3.2 Global Rare Disease Drug Revenue by Region: 2026-2031
3.4 North America Rare Disease Drug Market Facts & Figures by Country
3.4.1 North America Rare Disease Drug Market Size by Country: 2020 VS 2024 VS 2031
3.4.2 North America Rare Disease Drug Sales by Country (2020-2031)
3.4.3 North America Rare Disease Drug Revenue by Country (2020-2031)
3.4.4 U.S.
3.4.5 Canada
3.5 Europe Rare Disease Drug Market Facts & Figures by Country
3.5.1 Europe Rare Disease Drug Market Size by Country: 2020 VS 2024 VS 2031
3.5.2 Europe Rare Disease Drug Sales by Country (2020-2031)
3.5.3 Europe Rare Disease Drug Revenue by Country (2020-2031)
3.5.4 Germany
3.5.5 France
3.5.6 U.K.
3.5.7 Italy
3.5.8 Russia
3.6 Asia Pacific Rare Disease Drug Market Facts & Figures by Region
3.6.1 Asia Pacific Rare Disease Drug Market Size by Region: 2020 VS 2024 VS 2031
3.6.2 Asia Pacific Rare Disease Drug Sales by Region (2020-2031)
3.6.3 Asia Pacific Rare Disease Drug Revenue by Region (2020-2031)
3.6.4 China
3.6.5 Japan
3.6.6 South Korea
3.6.7 India
3.6.8 Australia
3.6.9 Taiwan
3.6.10 Indonesia
3.6.11 Thailand
3.6.12 Malaysia
3.6.13 Philippines
3.7 Latin America Rare Disease Drug Market Facts & Figures by Country
3.7.1 Latin America Rare Disease Drug Market Size by Country: 2020 VS 2024 VS 2031
3.7.2 Latin America Rare Disease Drug Sales by Country (2020-2031)
3.7.3 Latin America Rare Disease Drug Revenue by Country (2020-2031)
3.7.4 Mexico
3.7.5 Brazil
3.7.6 Argentina
3.8 Middle East and Africa Rare Disease Drug Market Facts & Figures by Country
3.8.1 Middle East and Africa Rare Disease Drug Market Size by Country: 2020 VS 2024 VS 2031
3.8.2 Middle East and Africa Rare Disease Drug Sales by Country (2020-2031)
3.8.3 Middle East and Africa Rare Disease Drug Revenue by Country (2020-2031)
3.8.4 Turkey
3.8.5 Saudi Arabia
3.8.6 U.A.E
4 Segment by Type
4.1 Global Rare Disease Drug Sales by Type (2020-2031)
4.1.1 Global Rare Disease Drug Sales by Type (2020-2025)
4.1.2 Global Rare Disease Drug Sales by Type (2026-2031)
4.1.3 Global Rare Disease Drug Sales Market Share by Type (2020-2031)
4.2 Global Rare Disease Drug Revenue by Type (2020-2031)
4.2.1 Global Rare Disease Drug Revenue by Type (2020-2025)
4.2.2 Global Rare Disease Drug Revenue by Type (2026-2031)
4.2.3 Global Rare Disease Drug Revenue Market Share by Type (2020-2031)
4.3 Global Rare Disease Drug Price by Type (2020-2031)
5 Segment by Application
5.1 Global Rare Disease Drug Sales by Application (2020-2031)
5.1.1 Global Rare Disease Drug Sales by Application (2020-2025)
5.1.2 Global Rare Disease Drug Sales by Application (2026-2031)
5.1.3 Global Rare Disease Drug Sales Market Share by Application (2020-2031)
5.2 Global Rare Disease Drug Revenue by Application (2020-2031)
5.2.1 Global Rare Disease Drug Revenue by Application (2020-2025)
5.2.2 Global Rare Disease Drug Revenue by Application (2026-2031)
5.2.3 Global Rare Disease Drug Revenue Market Share by Application (2020-2031)
5.3 Global Rare Disease Drug Price by Application (2020-2031)
6 Key Companies Profiled
6.1 Novartis AG
6.1.1 Novartis AG Company Information
6.1.2 Novartis AG Description and Business Overview
6.1.3 Novartis AG Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.1.4 Novartis AG Rare Disease Drug Product Portfolio
6.1.5 Novartis AG Recent Developments/Updates
6.2 Bristol-Myers Squibb Company
6.2.1 Bristol-Myers Squibb Company Company Information
6.2.2 Bristol-Myers Squibb Company Description and Business Overview
6.2.3 Bristol-Myers Squibb Company Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.2.4 Bristol-Myers Squibb Company Rare Disease Drug Product Portfolio
6.2.5 Bristol-Myers Squibb Company Recent Developments/Updates
6.3 Celgene Corporation
6.3.1 Celgene Corporation Company Information
6.3.2 Celgene Corporation Description and Business Overview
6.3.3 Celgene Corporation Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.3.4 Celgene Corporation Rare Disease Drug Product Portfolio
6.3.5 Celgene Corporation Recent Developments/Updates
6.4 F. Hoffmann-La Roche Ltd.
6.4.1 F. Hoffmann-La Roche Ltd. Company Information
6.4.2 F. Hoffmann-La Roche Ltd. Description and Business Overview
6.4.3 F. Hoffmann-La Roche Ltd. Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.4.4 F. Hoffmann-La Roche Ltd. Rare Disease Drug Product Portfolio
6.4.5 F. Hoffmann-La Roche Ltd. Recent Developments/Updates
6.5 Pfizer, Inc.
6.5.1 Pfizer, Inc. Company Information
6.5.2 Pfizer, Inc. Description and Business Overview
6.5.3 Pfizer, Inc. Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.5.4 Pfizer, Inc. Rare Disease Drug Product Portfolio
6.5.5 Pfizer, Inc. Recent Developments/Updates
6.6 Sanofi S.A.
6.6.1 Sanofi S.A. Company Information
6.6.2 Sanofi S.A. Description and Business Overview
6.6.3 Sanofi S.A. Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.6.4 Sanofi S.A. Rare Disease Drug Product Portfolio
6.6.5 Sanofi S.A. Recent Developments/Updates
6.7 Alexion Pharmaceuticals, Inc.
6.7.1 Alexion Pharmaceuticals, Inc. Company Information
6.7.2 Alexion Pharmaceuticals, Inc. Description and Business Overview
6.7.3 Alexion Pharmaceuticals, Inc. Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.7.4 Alexion Pharmaceuticals, Inc. Rare Disease Drug Product Portfolio
6.7.5 Alexion Pharmaceuticals, Inc. Recent Developments/Updates
6.8 Eli Lilly and Company
6.8.1 Eli Lilly and Company Company Information
6.8.2 Eli Lilly and Company Description and Business Overview
6.8.3 Eli Lilly and Company Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.8.4 Eli Lilly and Company Rare Disease Drug Product Portfolio
6.8.5 Eli Lilly and Company Recent Developments/Updates
6.9 Novo Nordisk A/S
6.9.1 Novo Nordisk A/S Company Information
6.9.2 Novo Nordisk A/S Description and Business Overview
6.9.3 Novo Nordisk A/S Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.9.4 Novo Nordisk A/S Rare Disease Drug Product Portfolio
6.9.5 Novo Nordisk A/S Recent Developments/Updates
6.10 AstraZeneca
6.10.1 AstraZeneca Company Information
6.10.2 AstraZeneca Description and Business Overview
6.10.3 AstraZeneca Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.10.4 AstraZeneca Rare Disease Drug Product Portfolio
6.10.5 AstraZeneca Recent Developments/Updates
6.11 Eisai Co., Ltd.
6.11.1 Eisai Co., Ltd. Company Information
6.11.2 Eisai Co., Ltd. Description and Business Overview
6.11.3 Eisai Co., Ltd. Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.11.4 Eisai Co., Ltd. Rare Disease Drug Product Portfolio
6.11.5 Eisai Co., Ltd. Recent Developments/Updates
6.12 Daiichi Sankyo Company Limited
6.12.1 Daiichi Sankyo Company Limited Company Information
6.12.2 Daiichi Sankyo Company Limited Description and Business Overview
6.12.3 Daiichi Sankyo Company Limited Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.12.4 Daiichi Sankyo Company Limited Rare Disease Drug Product Portfolio
6.12.5 Daiichi Sankyo Company Limited Recent Developments/Updates
6.13 Bayer AG
6.13.1 Bayer AG Company Information
6.13.2 Bayer AG Description and Business Overview
6.13.3 Bayer AG Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.13.4 Bayer AG Rare Disease Drug Product Portfolio
6.13.5 Bayer AG Recent Developments/Updates
6.14 GlaxoSmithKline
6.14.1 GlaxoSmithKline Company Information
6.14.2 GlaxoSmithKline Description and Business Overview
6.14.3 GlaxoSmithKline Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.14.4 GlaxoSmithKline Rare Disease Drug Product Portfolio
6.14.5 GlaxoSmithKline Recent Developments/Updates
6.15 Merck & Co., Inc.
6.15.1 Merck & Co., Inc. Company Information
6.15.2 Merck & Co., Inc. Description and Business Overview
6.15.3 Merck & Co., Inc. Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.15.4 Merck & Co., Inc. Rare Disease Drug Product Portfolio
6.15.5 Merck & Co., Inc. Recent Developments/Updates
6.16 Johnson & Johnson
6.16.1 Johnson & Johnson Company Information
6.16.2 Johnson & Johnson Description and Business Overview
6.16.3 Johnson & Johnson Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.16.4 Johnson & Johnson Rare Disease Drug Product Portfolio
6.16.5 Johnson & Johnson Recent Developments/Updates
6.17 Biogen, Inc.
6.17.1 Biogen, Inc. Company Information
6.17.2 Biogen, Inc. Description and Business Overview
6.17.3 Biogen, Inc. Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.17.4 Biogen, Inc. Rare Disease Drug Product Portfolio
6.17.5 Biogen, Inc. Recent Developments/Updates
6.18 Takeda
6.18.1 Takeda Company Information
6.18.2 Takeda Description and Business Overview
6.18.3 Takeda Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.18.4 Takeda Rare Disease Drug Product Portfolio
6.18.5 Takeda Recent Developments/Updates
6.19 Amgen, Inc.
6.19.1 Amgen, Inc. Company Information
6.19.2 Amgen, Inc. Description and Business Overview
6.19.3 Amgen, Inc. Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.19.4 Amgen, Inc. Rare Disease Drug Product Portfolio
6.19.5 Amgen, Inc. Recent Developments/Updates
6.20 Deciphera
6.20.1 Deciphera Company Information
6.20.2 Deciphera Description and Business Overview
6.20.3 Deciphera Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.20.4 Deciphera Rare Disease Drug Product Portfolio
6.20.5 Deciphera Recent Developments/Updates
6.21 Atara Biotherapeutics
6.21.1 Atara Biotherapeutics Company Information
6.21.2 Atara Biotherapeutics Description and Business Overview
6.21.3 Atara Biotherapeutics Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.21.4 Atara Biotherapeutics Rare Disease Drug Product Portfolio
6.21.5 Atara Biotherapeutics Recent Developments/Updates
6.22 ProQR
6.22.1 ProQR Company Information
6.22.2 ProQR Description and Business Overview
6.22.3 ProQR Rare Disease Drug Sales, Revenue and Gross Margin (2020-2025)
6.22.4 ProQR Rare Disease Drug Product Portfolio
6.22.5 ProQR Recent Developments/Updates
7 Industry Chain and Sales Channels Analysis
7.1 Rare Disease Drug Industry Chain Analysis
7.2 Rare Disease Drug Raw Material Supply Analysis
7.2.1 Key Raw Materials
7.2.2 Raw Materials Key Suppliers
7.3 Rare Disease Drug Production Mode & Process Analysis
7.4 Rare Disease Drug Sales and Marketing
7.4.1 Rare Disease Drug Sales Channels
7.4.2 Rare Disease Drug Distributors
7.5 Rare Disease Drug Customer Analysis
8 Rare Disease Drug Market Dynamics
8.1 Rare Disease Drug Industry Trends
8.2 Rare Disease Drug Market Drivers
8.3 Rare Disease Drug Market Challenges
8.4 Rare Disease Drug Market Restraints
9 Research Findings and Conclusion
10 Methodology and Data Source
10.1 Methodology/Research Approach
10.1.1 Research Programs/Design
10.1.2 Market Size Estimation
10.1.3 Market Breakdown and Data Triangulation
10.2 Data Source
10.2.1 Secondary Sources
10.2.2 Primary Sources
10.3 Author List
10.4 Disclaimer
FEATURED COMPANIES
Novartis
Bristol Myers Squibb Company
Celgene Corporation
F Hoffmann La Roche Ltd
Pfizer Inc
Sanofi
Alexion Pharmaceuticals Inc
Eli Lilly and Company
Novo Nordisk A S
Astrazeneca plc
Eisai Co Ltd
Daiichi Sankyo Company Limited
Bayer
GlaxoSmithKline PLC
Merck& Co. Inc
Johnson & Johnson
Biogen
Takeda
Amgen Inc
Deciphera
Atara Biotherapeutics
ProQR
List of Tables
 Table 1. Global Rare Disease Drug Market Value Comparison by Type (2024 VS 2031) & (US$ Million)
 Table 2. Global Rare Disease Drug Market Value by Application (2024 VS 2031) & (US$ Million)
 Table 3. Global Rare Disease Drug Market Competitive Situation by Manufacturers in 2024
 Table 4. Global Rare Disease Drug Sales (K Doses) of Key Manufacturers (2020-2025)
 Table 5. Global Rare Disease Drug Sales Market Share by Manufacturers (2020-2025)
 Table 6. Global Rare Disease Drug Revenue (US$ Million) by Manufacturers (2020-2025)
 Table 7. Global Rare Disease Drug Revenue Share by Manufacturers (2020-2025)
 Table 8. Global Market Rare Disease Drug Average Price (USD/Dose) of Key Manufacturers (2020-2025)
 Table 9. Global Key Players of Rare Disease Drug, Industry Ranking, 2022 VS 2023 VS 2024
 Table 10. Global Key Manufacturers of Rare Disease Drug, Manufacturing Sites & Headquarters
 Table 11. Global Key Manufacturers of Rare Disease Drug, Product Type & Application
 Table 12. Global Key Manufacturers of Rare Disease Drug, Date of Enter into This Industry
 Table 13. Global Manufacturers Market Concentration Ratio (CR5 and HHI)
 Table 14. Global Rare Disease Drug by Company Type (Tier 1, Tier 2, and Tier 3) & (based on the Revenue in Rare Disease Drug as of 2024)
 Table 15. Manufacturers Mergers & Acquisitions, Expansion Plans
 Table 16. Global Rare Disease Drug Market Size by Region (US$ Million): 2020 VS 2024 VS 2031
 Table 17. Global Rare Disease Drug Sales by Region (2020-2025) & (K Doses)
 Table 18. Global Rare Disease Drug Sales Market Share by Region (2020-2025)
 Table 19. Global Rare Disease Drug Sales by Region (2026-2031) & (K Doses)
 Table 20. Global Rare Disease Drug Sales Market Share by Region (2026-2031)
 Table 21. Global Rare Disease Drug Revenue by Region (2020-2025) & (US$ Million)
 Table 22. Global Rare Disease Drug Revenue Market Share by Region (2020-2025)
 Table 23. Global Rare Disease Drug Revenue by Region (2026-2031) & (US$ Million)
 Table 24. Global Rare Disease Drug Revenue Market Share by Region (2026-2031)
 Table 25. North America Rare Disease Drug Revenue by Country: 2020 VS 2024 VS 2031 (US$ Million)
 Table 26. North America Rare Disease Drug Sales by Country (2020-2025) & (K Doses)
 Table 27. North America Rare Disease Drug Sales by Country (2026-2031) & (K Doses)
 Table 28. North America Rare Disease Drug Revenue by Country (2020-2025) & (US$ Million)
 Table 29. North America Rare Disease Drug Revenue by Country (2026-2031) & (US$ Million)
 Table 30. Europe Rare Disease Drug Revenue by Country: 2020 VS 2024 VS 2031 (US$ Million)
 Table 31. Europe Rare Disease Drug Sales by Country (2020-2025) & (K Doses)
 Table 32. Europe Rare Disease Drug Sales by Country (2026-2031) & (K Doses)
 Table 33. Europe Rare Disease Drug Revenue by Country (2020-2025) & (US$ Million)
 Table 34. Europe Rare Disease Drug Revenue by Country (2026-2031) & (US$ Million)
 Table 35. Asia Pacific Rare Disease Drug Revenue by Region: 2020 VS 2024 VS 2031 (US$ Million)
 Table 36. Asia Pacific Rare Disease Drug Sales by Region (2020-2025) & (K Doses)
 Table 37. Asia Pacific Rare Disease Drug Sales by Region (2026-2031) & (K Doses)
 Table 38. Asia Pacific Rare Disease Drug Revenue by Region (2020-2025) & (US$ Million)
 Table 39. Asia Pacific Rare Disease Drug Revenue by Region (2026-2031) & (US$ Million)
 Table 40. Latin America Rare Disease Drug Revenue by Country: 2020 VS 2024 VS 2031 (US$ Million)
 Table 41. Latin America Rare Disease Drug Sales by Country (2020-2025) & (K Doses)
 Table 42. Latin America Rare Disease Drug Sales by Country (2026-2031) & (K Doses)
 Table 43. Latin America Rare Disease Drug Revenue by Country (2020-2025) & (US$ Million)
 Table 44. Latin America Rare Disease Drug Revenue by Country (2026-2031) & (US$ Million)
 Table 45. Middle East and Africa Rare Disease Drug Revenue by Country: 2020 VS 2024 VS 2031 (US$ Million)
 Table 46. Middle East and Africa Rare Disease Drug Sales by Country (2020-2025) & (K Doses)
 Table 47. Middle East and Africa Rare Disease Drug Sales by Country (2026-2031) & (K Doses)
 Table 48. Middle East and Africa Rare Disease Drug Revenue by Country (2020-2025) & (US$ Million)
 Table 49. Middle East and Africa Rare Disease Drug Revenue by Country (2026-2031) & (US$ Million)
 Table 50. Global Rare Disease Drug Sales (K Doses) by Type (2020-2025)
 Table 51. Global Rare Disease Drug Sales (K Doses) by Type (2026-2031)
 Table 52. Global Rare Disease Drug Sales Market Share by Type (2020-2025)
 Table 53. Global Rare Disease Drug Sales Market Share by Type (2026-2031)
 Table 54. Global Rare Disease Drug Revenue (US$ Million) by Type (2020-2025)
 Table 55. Global Rare Disease Drug Revenue (US$ Million) by Type (2026-2031)
 Table 56. Global Rare Disease Drug Revenue Market Share by Type (2020-2025)
 Table 57. Global Rare Disease Drug Revenue Market Share by Type (2026-2031)
 Table 58. Global Rare Disease Drug Price (USD/Dose) by Type (2020-2025)
 Table 59. Global Rare Disease Drug Price (USD/Dose) by Type (2026-2031)
 Table 60. Global Rare Disease Drug Sales (K Doses) by Application (2020-2025)
 Table 61. Global Rare Disease Drug Sales (K Doses) by Application (2026-2031)
 Table 62. Global Rare Disease Drug Sales Market Share by Application (2020-2025)
 Table 63. Global Rare Disease Drug Sales Market Share by Application (2026-2031)
 Table 64. Global Rare Disease Drug Revenue (US$ Million) by Application (2020-2025)
 Table 65. Global Rare Disease Drug Revenue (US$ Million) by Application (2026-2031)
 Table 66. Global Rare Disease Drug Revenue Market Share by Application (2020-2025)
 Table 67. Global Rare Disease Drug Revenue Market Share by Application (2026-2031)
 Table 68. Global Rare Disease Drug Price (USD/Dose) by Application (2020-2025)
 Table 69. Global Rare Disease Drug Price (USD/Dose) by Application (2026-2031)
 Table 70. Novartis AG Company Information
 Table 71. Novartis AG Description and Business Overview
 Table 72. Novartis AG Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 73. Novartis AG Rare Disease Drug Product
 Table 74. Novartis AG Recent Developments/Updates
 Table 75. Bristol-Myers Squibb Company Company Information
 Table 76. Bristol-Myers Squibb Company Description and Business Overview
 Table 77. Bristol-Myers Squibb Company Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 78. Bristol-Myers Squibb Company Rare Disease Drug Product
 Table 79. Bristol-Myers Squibb Company Recent Developments/Updates
 Table 80. Celgene Corporation Company Information
 Table 81. Celgene Corporation Description and Business Overview
 Table 82. Celgene Corporation Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 83. Celgene Corporation Rare Disease Drug Product
 Table 84. Celgene Corporation Recent Developments/Updates
 Table 85. F. Hoffmann-La Roche Ltd. Company Information
 Table 86. F. Hoffmann-La Roche Ltd. Description and Business Overview
 Table 87. F. Hoffmann-La Roche Ltd. Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 88. F. Hoffmann-La Roche Ltd. Rare Disease Drug Product
 Table 89. F. Hoffmann-La Roche Ltd. Recent Developments/Updates
 Table 90. Pfizer, Inc. Company Information
 Table 91. Pfizer, Inc. Description and Business Overview
 Table 92. Pfizer, Inc. Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 93. Pfizer, Inc. Rare Disease Drug Product
 Table 94. Pfizer, Inc. Recent Developments/Updates
 Table 95. Sanofi S.A. Company Information
 Table 96. Sanofi S.A. Description and Business Overview
 Table 97. Sanofi S.A. Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 98. Sanofi S.A. Rare Disease Drug Product
 Table 99. Sanofi S.A. Recent Developments/Updates
 Table 100. Alexion Pharmaceuticals, Inc. Company Information
 Table 101. Alexion Pharmaceuticals, Inc. Description and Business Overview
 Table 102. Alexion Pharmaceuticals, Inc. Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 103. Alexion Pharmaceuticals, Inc. Rare Disease Drug Product
 Table 104. Alexion Pharmaceuticals, Inc. Recent Developments/Updates
 Table 105. Eli Lilly and Company Company Information
 Table 106. Eli Lilly and Company Description and Business Overview
 Table 107. Eli Lilly and Company Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 108. Eli Lilly and Company Rare Disease Drug Product
 Table 109. Eli Lilly and Company Recent Developments/Updates
 Table 110. Novo Nordisk A/S Company Information
 Table 111. Novo Nordisk A/S Description and Business Overview
 Table 112. Novo Nordisk A/S Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 113. Novo Nordisk A/S Rare Disease Drug Product
 Table 114. Novo Nordisk A/S Recent Developments/Updates
 Table 115. AstraZeneca Company Information
 Table 116. AstraZeneca Description and Business Overview
 Table 117. AstraZeneca Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 118. AstraZeneca Rare Disease Drug Product
 Table 119. AstraZeneca Recent Developments/Updates
 Table 120. Eisai Co., Ltd. Company Information
 Table 121. Eisai Co., Ltd. Description and Business Overview
 Table 122. Eisai Co., Ltd. Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 123. Eisai Co., Ltd. Rare Disease Drug Product
 Table 124. Eisai Co., Ltd. Recent Developments/Updates
 Table 125. Daiichi Sankyo Company Limited Company Information
 Table 126. Daiichi Sankyo Company Limited Description and Business Overview
 Table 127. Daiichi Sankyo Company Limited Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 128. Daiichi Sankyo Company Limited Rare Disease Drug Product
 Table 129. Daiichi Sankyo Company Limited Recent Developments/Updates
 Table 130. Bayer AG Company Information
 Table 131. Bayer AG Description and Business Overview
 Table 132. Bayer AG Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 133. Bayer AG Rare Disease Drug Product
 Table 134. Bayer AG Recent Developments/Updates
 Table 135. GlaxoSmithKline Company Information
 Table 136. GlaxoSmithKline Description and Business Overview
 Table 137. GlaxoSmithKline Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 138. GlaxoSmithKline Rare Disease Drug Product
 Table 139. GlaxoSmithKline Recent Developments/Updates
 Table 140. Merck & Co., Inc. Company Information
 Table 141. Merck & Co., Inc. Description and Business Overview
 Table 142. Merck & Co., Inc. Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 143. Merck & Co., Inc. Rare Disease Drug Product
 Table 144. Merck & Co., Inc. Recent Developments/Updates
 Table 145. Johnson & Johnson Company Information
 Table 146. Johnson & Johnson Description and Business Overview
 Table 147. Johnson & Johnson Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 148. Johnson & Johnson Rare Disease Drug Product
 Table 149. Johnson & Johnson Recent Developments/Updates
 Table 150. Biogen, Inc. Company Information
 Table 151. Biogen, Inc. Description and Business Overview
 Table 152. Biogen, Inc. Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 153. Biogen, Inc. Rare Disease Drug Product
 Table 154. Biogen, Inc. Recent Developments/Updates
 Table 155. Takeda Company Information
 Table 156. Takeda Description and Business Overview
 Table 157. Takeda Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 158. Takeda Rare Disease Drug Product
 Table 159. Takeda Recent Developments/Updates
 Table 160. Amgen, Inc. Company Information
 Table 161. Amgen, Inc. Description and Business Overview
 Table 162. Amgen, Inc. Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 163. Amgen, Inc. Rare Disease Drug Product
 Table 164. Amgen, Inc. Recent Developments/Updates
 Table 165. Deciphera Company Information
 Table 166. Deciphera Description and Business Overview
 Table 167. Deciphera Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 168. Deciphera Rare Disease Drug Product
 Table 169. Deciphera Recent Developments/Updates
 Table 170. Atara Biotherapeutics Company Information
 Table 171. Atara Biotherapeutics Description and Business Overview
 Table 172. Atara Biotherapeutics Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 173. Atara Biotherapeutics Rare Disease Drug Product
 Table 174. Atara Biotherapeutics Recent Developments/Updates
 Table 175. ProQR Company Information
 Table 176. ProQR Description and Business Overview
 Table 177. ProQR Rare Disease Drug Sales (K Doses), Revenue (US$ Million), Price (USD/Dose) and Gross Margin (2020-2025)
 Table 178. ProQR Rare Disease Drug Product
 Table 179. ProQR Recent Developments/Updates
 Table 180. Key Raw Materials Lists
 Table 181. Raw Materials Key Suppliers Lists
 Table 182. Rare Disease Drug Distributors List
 Table 183. Rare Disease Drug Customers List
 Table 184. Rare Disease Drug Market Trends
 Table 185. Rare Disease Drug Market Drivers
 Table 186. Rare Disease Drug Market Challenges
 Table 187. Rare Disease Drug Market Restraints
 Table 188. Research Programs/Design for This Report
 Table 189. Key Data Information from Secondary Sources
 Table 190. Key Data Information from Primary Sources
 Table 191. Authors List of This Report


List of Figures
 Figure 1. Product Picture of Rare Disease Drug
 Figure 2. Global Rare Disease Drug Market Value Comparison by Type (2020-2031) & (US$ Million)
 Figure 3. Global Rare Disease Drug Market Share by Type: 2024 & 2031
 Figure 4. Above 1‰ Product Picture
 Figure 5. 0.65‰～1‰ Product Picture
 Figure 6. Below 0.1‰ Product Picture
 Figure 7. Global Rare Disease Drug Market Value by Application (2020-2031) & (US$ Million)
 Figure 8. Global Rare Disease Drug Market Share by Application: 2024 & 2031
 Figure 9. Hospital Pharmacies
 Figure 10. Speciality Pharmacies
 Figure 11. Retail pharmacies
 Figure 12. Others
 Figure 13. Global Rare Disease Drug Revenue, (US$ Million), 2020 VS 2024 VS 2031
 Figure 14. Global Rare Disease Drug Market Size (2020-2031) & (US$ Million)
 Figure 15. Global Rare Disease Drug Sales (2020-2031) & (K Doses)
 Figure 16. Global Rare Disease Drug Average Price (USD/Dose) & (2020-2031)
 Figure 17. Rare Disease Drug Report Years Considered
 Figure 18. Rare Disease Drug Sales Share by Manufacturers in 2024
 Figure 19. Global Rare Disease Drug Revenue Share by Manufacturers in 2024
 Figure 20. Global 5 and 10 Largest Rare Disease Drug Players: Market Share by Revenue in Rare Disease Drug in 2024
 Figure 21. Rare Disease Drug Market Share by Company Type (Tier 1, Tier 2, and Tier 3): 2020 VS 2024
 Figure 22. Global Rare Disease Drug Market Size by Region (US$ Million): 2020 VS 2024 VS 2031
 Figure 23. North America Rare Disease Drug Sales Market Share by Country (2020-2031)
 Figure 24. North America Rare Disease Drug Revenue Market Share by Country (2020-2031)
 Figure 25. U.S. Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 26. Canada Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 27. Europe Rare Disease Drug Sales Market Share by Country (2020-2031)
 Figure 28. Europe Rare Disease Drug Revenue Market Share by Country (2020-2031)
 Figure 29. Germany Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 30. France Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 31. U.K. Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 32. Italy Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 33. Russia Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 34. Asia Pacific Rare Disease Drug Sales Market Share by Region (2020-2031)
 Figure 35. Asia Pacific Rare Disease Drug Revenue Market Share by Region (2020-2031)
 Figure 36. China Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 37. Japan Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 38. South Korea Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 39. India Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 40. Australia Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 41. Taiwan Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 42. Indonesia Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 43. Thailand Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 44. Malaysia Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 45. Philippines Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 46. Latin America Rare Disease Drug Sales Market Share by Country (2020-2031)
 Figure 47. Latin America Rare Disease Drug Revenue Market Share by Country (2020-2031)
 Figure 48. Mexico Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 49. Brazil Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 50. Argentina Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 51. Middle East and Africa Rare Disease Drug Sales Market Share by Country (2020-2031)
 Figure 52. Middle East and Africa Rare Disease Drug Revenue Market Share by Country (2020-2031)
 Figure 53. Turkey Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 54. Saudi Arabia Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 55. U.A.E Rare Disease Drug Revenue Growth Rate (2020-2031) & (US$ Million)
 Figure 56. Global Sales Market Share of Rare Disease Drug by Type (2020-2031)
 Figure 57. Global Revenue Market Share of Rare Disease Drug by Type (2020-2031)
 Figure 58. Global Rare Disease Drug Price (USD/Dose) by Type (2020-2031)
 Figure 59. Global Sales Market Share of Rare Disease Drug by Application (2020-2031)
 Figure 60. Global Revenue Market Share of Rare Disease Drug by Application (2020-2031)
 Figure 61. Global Rare Disease Drug Price (USD/Dose) by Application (2020-2031)
 Figure 62. Rare Disease Drug Value Chain
 Figure 63. Channels of Distribution (Direct Vs Distribution)
 Figure 64. Bottom-up and Top-down Approaches for This Report
 Figure 65. Data Triangulation
 Figure 66. Key Executives Interviewed
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