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Global Orphan Indication Drugs Market Research Report 2025
Synopsis
An orphan drug is a drug used to treat a disease that cannot be produced profitably without government assistance because of the small number of patients.The classification of certain diseases, and the medicines that treat them, as "orphan" is a matter of public policy in many countries . Given the nature of the enormous economic resources required for medical research , these medicines would have no chance of producing breakthroughs that otherwise might not have been possible without government assistance.In the United States and the European Union , orphan drugs are easier to obtain marketing authorization. There may also be additional financial incentives, such as an extension of the market exclusivity period, which would allow the pharmaceutical company to have a longer exclusivity to sell the drug. All of these measures are designed to encourage drug development, and without this kind of profit motive , few pharmaceutical companies will be willing to allocate their budgets and personnel to this type of research.
Global Orphan Indication Drugs market is projected to reach US$ 734.7 million in 2029, increasing from US$ 633 million in 2022, with the CAGR of 2.3% during the period of 2023 to 2029. Influencing issues, such as economy environments, COVID-19 and Russia-Ukraine War, have led to great market fluctuations in the past few years and are considered comprehensively in the whole Orphan Indication Drugs market research.
The global pharmaceutical market is 1475 billion USD in 2022, growing at a CAGR of 5% during the next six years. 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, based on historical analysis (2018-2022) and forecast calculation (2023-2029), aims to help readers to get a comprehensive understanding of global Orphan Indication Drugs market with multiple angles, which provides sufficient supports to readers’ strategy and decision making.
By Company
Bristol-Myers Squibb
Roche
Novartis
Johnson & Johnson
Pfizer
Amgen
Sanofi
AstraZeneca
Takeda
Vertex Pharmaceuticals
AbbVie
Biogen
Eli Lilly
Segment by Type
Biologics
Non-Biologics
Segment by Application
Hospital Pharmacy
Retail Pharmacy
Online Sales
Consumption by Region
North America
United States
Canada
Europe
Germany
France
U.K.
Italy
Russia
Asia-Pacific
China
Japan
South Korea
India
Australia
China Taiwan
Indonesia
Thailand
Malaysia
Latin America
Mexico
Brazil
Argentina
Middle East & Africa
Turkey
Saudi Arabia
UAE
The Orphan Indication Drugs report covers below items:
Chapter 1: Product Basic Information (Definition, Type and Application)
Chapter 2: Manufacturers’ Competition Patterns
Chapter 3: Country Level Sales Analysis
Chapter 4: Product Type Analysis
Chapter 5: Product Application Analysis
Chapter 6: Manufacturers’ Outline
Chapter 7: Industry Chain, Market Channel and Customer Analysis
Chapter 8: Market Opportunities and Challenges
Chapter 9: Market Conclusions
Chapter 10: Research Methodology and Data Source
An orphan drug is a drug used to treat a disease that cannot be produced profitably without government assistance because of the small number of patients.The classification of certain diseases, and the medicines that treat them, as "orphan" is a matter of public policy in many countries . Given the nature of the enormous economic resources required for medical research , these medicines would have no chance of producing breakthroughs that otherwise might not have been possible without government assistance.In the United States and the European Union , orphan drugs are easier to obtain marketing authorization. There may also be additional financial incentives, such as an extension of the market exclusivity period, which would allow the pharmaceutical company to have a longer exclusivity to sell the drug. All of these measures are designed to encourage drug development, and without this kind of profit motive , few pharmaceutical companies will be willing to allocate their budgets and personnel to this type of research.
Global Orphan Indication Drugs market is projected to reach US$ 734.7 million in 2029, increasing from US$ 633 million in 2022, with the CAGR of 2.3% during the period of 2023 to 2029. Influencing issues, such as economy environments, COVID-19 and Russia-Ukraine War, have led to great market fluctuations in the past few years and are considered comprehensively in the whole Orphan Indication Drugs market research.
The global pharmaceutical market is 1475 billion USD in 2022, growing at a CAGR of 5% during the next six years. 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, based on historical analysis (2018-2022) and forecast calculation (2023-2029), aims to help readers to get a comprehensive understanding of global Orphan Indication Drugs market with multiple angles, which provides sufficient supports to readers’ strategy and decision making.
By Company
Bristol-Myers Squibb
Roche
Novartis
Johnson & Johnson
Pfizer
Amgen
Sanofi
AstraZeneca
Takeda
Vertex Pharmaceuticals
AbbVie
Biogen
Eli Lilly
Segment by Type
Biologics
Non-Biologics
Segment by Application
Hospital Pharmacy
Retail Pharmacy
Online Sales
Consumption by Region
North America
United States
Canada
Europe
Germany
France
U.K.
Italy
Russia
Asia-Pacific
China
Japan
South Korea
India
Australia
China Taiwan
Indonesia
Thailand
Malaysia
Latin America
Mexico
Brazil
Argentina
Middle East & Africa
Turkey
Saudi Arabia
UAE
The Orphan Indication Drugs report covers below items:
Chapter 1: Product Basic Information (Definition, Type and Application)
Chapter 2: Manufacturers’ Competition Patterns
Chapter 3: Country Level Sales Analysis
Chapter 4: Product Type Analysis
Chapter 5: Product Application Analysis
Chapter 6: Manufacturers’ Outline
Chapter 7: Industry Chain, Market Channel and Customer Analysis
Chapter 8: Market Opportunities and Challenges
Chapter 9: Market Conclusions
Chapter 10: Research Methodology and Data Source
Index1 Orphan Indication Drugs Market Overview
1.1 Product Overview and Scope of Orphan Indication Drugs
1.2 Orphan Indication Drugs Segment by Type
1.2.1 Global Orphan Indication Drugs Market Value Comparison by Type (2023-2029)
1.2.2 Biologics
1.2.3 Non-Biologics
1.3 Orphan Indication Drugs Segment by Application
1.3.1 Global Orphan Indication Drugs Market Value by Application: (2023-2029)
1.3.2 Hospital Pharmacy
1.3.3 Retail Pharmacy
1.3.4 Online Sales
1.4 Global Orphan Indication Drugs Market Size Estimates and Forecasts
1.4.1 Global Orphan Indication Drugs Revenue 2018-2029
1.4.2 Global Orphan Indication Drugs Sales 2018-2029
1.4.3 Global Orphan Indication Drugs Market Average Price (2018-2029)
1.5 Assumptions and Limitations
2 Orphan Indication Drugs Market Competition by Manufacturers
2.1 Global Orphan Indication Drugs Sales Market Share by Manufacturers (2018-2023)
2.2 Global Orphan Indication Drugs Revenue Market Share by Manufacturers (2018-2023)
2.3 Global Orphan Indication Drugs Average Price by Manufacturers (2018-2023)
2.4 Global Orphan Indication Drugs Industry Ranking 2021 VS 2022 VS 2023
2.5 Global Key Manufacturers of Orphan Indication Drugs, Manufacturing Sites & Headquarters
2.6 Global Key Manufacturers of Orphan Indication Drugs, Product Type & Application
2.7 Orphan Indication Drugs Market Competitive Situation and Trends
2.7.1 Orphan Indication Drugs Market Concentration Rate
2.7.2 The Global Top 5 and Top 10 Largest Orphan Indication Drugs Players Market Share by Revenue
2.7.3 Global Orphan Indication Drugs Market Share by Company Type (Tier 1, Tier 2 and Tier 3)
2.8 Manufacturers Mergers & Acquisitions, Expansion Plans
3 Orphan Indication Drugs Retrospective Market Scenario by Region
3.1 Global Orphan Indication Drugs Market Size by Region: 2018 Versus 2022 Versus 2029
3.2 Global Orphan Indication Drugs Global Orphan Indication Drugs Sales by Region: 2018-2029
3.2.1 Global Orphan Indication Drugs Sales by Region: 2018-2023
3.2.2 Global Orphan Indication Drugs Sales by Region: 2024-2029
3.3 Global Orphan Indication Drugs Global Orphan Indication Drugs Revenue by Region: 2018-2029
3.3.1 Global Orphan Indication Drugs Revenue by Region: 2018-2023
3.3.2 Global Orphan Indication Drugs Revenue by Region: 2024-2029
3.4 North America Orphan Indication Drugs Market Facts & Figures by Country
3.4.1 North America Orphan Indication Drugs Market Size by Country: 2018 VS 2022 VS 2029
3.4.2 North America Orphan Indication Drugs Sales by Country (2018-2029)
3.4.3 North America Orphan Indication Drugs Revenue by Country (2018-2029)
3.4.4 United States
3.4.5 Canada
3.5 Europe Orphan Indication Drugs Market Facts & Figures by Country
3.5.1 Europe Orphan Indication Drugs Market Size by Country: 2018 VS 2022 VS 2029
3.5.2 Europe Orphan Indication Drugs Sales by Country (2018-2029)
3.5.3 Europe Orphan Indication Drugs Revenue by Country (2018-2029)
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 Orphan Indication Drugs Market Facts & Figures by Country
3.6.1 Asia Pacific Orphan Indication Drugs Market Size by Country: 2018 VS 2022 VS 2029
3.6.2 Asia Pacific Orphan Indication Drugs Sales by Country (2018-2029)
3.6.3 Asia Pacific Orphan Indication Drugs Revenue by Country (2018-2029)
3.6.4 China
3.6.5 Japan
3.6.6 South Korea
3.6.7 India
3.6.8 Australia
3.6.9 China Taiwan
3.6.10 Indonesia
3.6.11 Thailand
3.6.12 Malaysia
3.7 Latin America Orphan Indication Drugs Market Facts & Figures by Country
3.7.1 Latin America Orphan Indication Drugs Market Size by Country: 2018 VS 2022 VS 2029
3.7.2 Latin America Orphan Indication Drugs Sales by Country (2018-2029)
3.7.3 Latin America Orphan Indication Drugs Revenue by Country (2018-2029)
3.7.4 Mexico
3.7.5 Brazil
3.7.6 Argentina
3.8 Middle East and Africa Orphan Indication Drugs Market Facts & Figures by Country
3.8.1 Middle East and Africa Orphan Indication Drugs Market Size by Country: 2018 VS 2022 VS 2029
3.8.2 Middle East and Africa Orphan Indication Drugs Sales by Country (2018-2029)
3.8.3 Middle East and Africa Orphan Indication Drugs Revenue by Country (2018-2029)
3.8.4 Turkey
3.8.5 Saudi Arabia
3.8.6 UAE
4 Segment by Type
4.1 Global Orphan Indication Drugs Sales by Type (2018-2029)
4.1.1 Global Orphan Indication Drugs Sales by Type (2018-2023)
4.1.2 Global Orphan Indication Drugs Sales by Type (2024-2029)
4.1.3 Global Orphan Indication Drugs Sales Market Share by Type (2018-2029)
4.2 Global Orphan Indication Drugs Revenue by Type (2018-2029)
4.2.1 Global Orphan Indication Drugs Revenue by Type (2018-2023)
4.2.2 Global Orphan Indication Drugs Revenue by Type (2024-2029)
4.2.3 Global Orphan Indication Drugs Revenue Market Share by Type (2018-2029)
4.3 Global Orphan Indication Drugs Price by Type (2018-2029)
5 Segment by Application
5.1 Global Orphan Indication Drugs Sales by Application (2018-2029)
5.1.1 Global Orphan Indication Drugs Sales by Application (2018-2023)
5.1.2 Global Orphan Indication Drugs Sales by Application (2024-2029)
5.1.3 Global Orphan Indication Drugs Sales Market Share by Application (2018-2029)
5.2 Global Orphan Indication Drugs Revenue by Application (2018-2029)
5.2.1 Global Orphan Indication Drugs Revenue by Application (2018-2023)
5.2.2 Global Orphan Indication Drugs Revenue by Application (2024-2029)
5.2.3 Global Orphan Indication Drugs Revenue Market Share by Application (2018-2029)
5.3 Global Orphan Indication Drugs Price by Application (2018-2029)
6 Key Companies Profiled
6.1 Bristol-Myers Squibb
6.1.1 Bristol-Myers Squibb Corporation Information
6.1.2 Bristol-Myers Squibb Description and Business Overview
6.1.3 Bristol-Myers Squibb Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.1.4 Bristol-Myers Squibb Orphan Indication Drugs Product Portfolio
6.1.5 Bristol-Myers Squibb Recent Developments/Updates
6.2 Roche
6.2.1 Roche Corporation Information
6.2.2 Roche Description and Business Overview
6.2.3 Roche Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.2.4 Roche Orphan Indication Drugs Product Portfolio
6.2.5 Roche Recent Developments/Updates
6.3 Novartis
6.3.1 Novartis Corporation Information
6.3.2 Novartis Description and Business Overview
6.3.3 Novartis Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.3.4 Novartis Orphan Indication Drugs Product Portfolio
6.3.5 Novartis Recent Developments/Updates
6.4 Johnson & Johnson
6.4.1 Johnson & Johnson Corporation Information
6.4.2 Johnson & Johnson Description and Business Overview
6.4.3 Johnson & Johnson Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.4.4 Johnson & Johnson Orphan Indication Drugs Product Portfolio
6.4.5 Johnson & Johnson Recent Developments/Updates
6.5 Pfizer
6.5.1 Pfizer Corporation Information
6.5.2 Pfizer Description and Business Overview
6.5.3 Pfizer Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.5.4 Pfizer Orphan Indication Drugs Product Portfolio
6.5.5 Pfizer Recent Developments/Updates
6.6 Amgen
6.6.1 Amgen Corporation Information
6.6.2 Amgen Description and Business Overview
6.6.3 Amgen Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.6.4 Amgen Orphan Indication Drugs Product Portfolio
6.6.5 Amgen Recent Developments/Updates
6.7 Sanofi
6.6.1 Sanofi Corporation Information
6.6.2 Sanofi Description and Business Overview
6.6.3 Sanofi Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.4.4 Sanofi Orphan Indication Drugs Product Portfolio
6.7.5 Sanofi Recent Developments/Updates
6.8 AstraZeneca
6.8.1 AstraZeneca Corporation Information
6.8.2 AstraZeneca Description and Business Overview
6.8.3 AstraZeneca Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.8.4 AstraZeneca Orphan Indication Drugs Product Portfolio
6.8.5 AstraZeneca Recent Developments/Updates
6.9 Takeda
6.9.1 Takeda Corporation Information
6.9.2 Takeda Description and Business Overview
6.9.3 Takeda Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.9.4 Takeda Orphan Indication Drugs Product Portfolio
6.9.5 Takeda Recent Developments/Updates
6.10 Vertex Pharmaceuticals
6.10.1 Vertex Pharmaceuticals Corporation Information
6.10.2 Vertex Pharmaceuticals Description and Business Overview
6.10.3 Vertex Pharmaceuticals Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.10.4 Vertex Pharmaceuticals Orphan Indication Drugs Product Portfolio
6.10.5 Vertex Pharmaceuticals Recent Developments/Updates
6.11 AbbVie
6.11.1 AbbVie Corporation Information
6.11.2 AbbVie Orphan Indication Drugs Description and Business Overview
6.11.3 AbbVie Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.11.4 AbbVie Orphan Indication Drugs Product Portfolio
6.11.5 AbbVie Recent Developments/Updates
6.12 Biogen
6.12.1 Biogen Corporation Information
6.12.2 Biogen Orphan Indication Drugs Description and Business Overview
6.12.3 Biogen Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.12.4 Biogen Orphan Indication Drugs Product Portfolio
6.12.5 Biogen Recent Developments/Updates
6.13 Eli Lilly
6.13.1 Eli Lilly Corporation Information
6.13.2 Eli Lilly Orphan Indication Drugs Description and Business Overview
6.13.3 Eli Lilly Orphan Indication Drugs Sales, Revenue and Gross Margin (2018-2023)
6.13.4 Eli Lilly Orphan Indication Drugs Product Portfolio
6.13.5 Eli Lilly Recent Developments/Updates
7 Industry Chain and Sales Channels Analysis
7.1 Orphan Indication Drugs Industry Chain Analysis
7.2 Orphan Indication Drugs Key Raw Materials
7.2.1 Key Raw Materials
7.2.2 Raw Materials Key Suppliers
7.3 Orphan Indication Drugs Production Mode & Process
7.4 Orphan Indication Drugs Sales and Marketing
7.4.1 Orphan Indication Drugs Sales Channels
7.4.2 Orphan Indication Drugs Distributors
7.5 Orphan Indication Drugs Customers
8 Orphan Indication Drugs Market Dynamics
8.1 Orphan Indication Drugs Industry Trends
8.2 Orphan Indication Drugs Market Drivers
8.3 Orphan Indication Drugs Market Challenges
8.4 Orphan Indication Drugs Market Restraints
9 Research Finding 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
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