Market Outlook
The global Orphan Drugs market size was valued at USD 143200 million in 2022 and is forecast to a readjusted size of USD 269190 million by 2029 with a CAGR of 9.4% during review period.
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.
Bristol-Myers Squibb is one of the most important global key players of orphan drugs, holds a share of about 10%, other key players include Roche and Novartis, etc. North America is the largest market, occupied for about 40 percent, followed by Asia-Pacific. In terms of type, oncology is the largest segment, with a share of over 60%, and in terms of application, the adult segment holds a share of about 40 percent.
This report is a detailed and comprehensive analysis for global Orphan Drugs market. Both quantitative and qualitative analyses are presented by company, by region & country, by Type and by Application. As the market is constantly changing, this report explores the competition, supply and demand trends, as well as key factors that contribute to its changing demands across many markets. Company profiles and product examples of selected competitors, along with market share estimates of some of the selected leaders for the year 2023, are provided.
Key Features:
Global Orphan Drugs market size and forecasts, in consumption value ($ Million), 2018-2029
Global Orphan Drugs market size and forecasts by region and country, in consumption value ($ Million), 2018-2029
Global Orphan Drugs market size and forecasts, by Type and by Application, in consumption value ($ Million), 2018-2029
Global Orphan Drugs market shares of main players, in revenue ($ Million), 2018-2023
The Primary Objectives in This Report Are:
To determine the size of the total market opportunity of global and key countries
To assess the growth potential for Orphan Drugs
To forecast future growth in each product and end-use market
To assess competitive factors affecting the marketplace
This report profiles key players in the global Orphan Drugs market based on the following parameters - company overview, production, value, price, gross margin, product portfolio, geographical presence, and key developments.
This report also provides key insights about market drivers, restraints, opportunities, new product launches or approvals, COVID-19 and Russia-Ukraine War Influence.
Key Market Players
Bristol-Myers Squibb
Roche
Novartis
Johnson & Johnson
Pfizer
Amgen
Sanofi
AstraZeneca
Takeda
Vertex Pharmaceuticals
AbbVie
Biogen
Eli Lilly
Segmentation By Type
Oncology
Pulmonary
Neurology
Hematology
Endocrinology
Cardio-vascular
Metabolic Disorders
Others
Segmentation By Application
Baby and Child
Teenager
Adult
Other
Segmentation By Region
North America (United States, Canada, and Mexico)
Europe (Germany, France, UK, Russia, Italy, and Rest of Europe)
Asia-Pacific (China, Japan, South Korea, India, Southeast Asia, Australia and Rest of Asia-Pacific)
South America (Brazil, Argentina and Rest of South America)
Middle East & Africa (Turkey, Saudi Arabia, UAE, Rest of Middle East & Africa)
Market SWOT Analysis
What are the strengths of the orphan drugs market in 2025?
The orphan drugs market is expected to continue benefiting from strong government incentives, such as extended market exclusivity and tax credits. Increased awareness of rare diseases and advancements in biotechnology will also foster innovation, improving treatment options for underserved populations.
What are the weaknesses in the orphan drugs market in 2025?
Despite these strengths, orphan drugs remain costly to develop, and market access can be limited by regulatory hurdles and pricing concerns. The small patient population often makes it harder to justify the investment, leading to challenges in scaling production.
What are the opportunities in the orphan drugs market in 2025?
There are significant opportunities in gene therapy, precision medicine, and biologics, which promise to revolutionize the treatment of rare diseases. The growing number of orphan drug designations and expanding healthcare access in emerging markets are expected to drive market growth.
What are the threats to the orphan drugs market in 2025?
Pricing pressures from healthcare payers, regulatory delays, and the potential for competition from biosimilars or alternative therapies pose ongoing threats. Additionally, economic downturns could lead to reduced healthcare spending, limiting the market’s growth potential.
Market PESTEL Analysis
What are the political factors affecting the orphan drugs market in 2025?
Government policies, including incentives like tax breaks, grants, and market exclusivity, will continue to support the orphan drug market. However, political instability or changing healthcare regulations could lead to uncertainty in pricing, reimbursement, and approval processes.
How do economic factors impact the orphan drugs market in 2025?
Economic conditions can significantly affect market growth. In periods of economic downturn, funding for rare disease treatments may face scrutiny, while budget cuts in healthcare could limit the ability of patients or providers to afford expensive orphan drugs.
What are the social factors influencing the orphan drugs market in 2025?
Rising awareness of rare diseases and a growing demand for personalized medicine will drive interest in orphan drugs. Additionally, patient advocacy groups and the increasing importance of patient-centric healthcare will push for better access to treatments for rare conditions.
What technological factors are affecting the orphan drugs market in 2025?
Technological advancements in genomics, biotechnology, and data analytics will enhance the development of orphan drugs. Innovations like gene editing, gene therapy, and biologics will be key drivers, enabling more effective treatments and expanding market opportunities.
What environmental factors are relevant to the orphan drugs market in 2025?
Environmental factors like the sustainability of production processes and the carbon footprint of manufacturing orphan drugs may play a role in industry practices. Furthermore, increasing focus on environmental responsibility may influence regulatory standards for production and distribution.
What are the legal factors influencing the orphan drugs market in 2025?
The orphan drug market is highly regulated, with laws regarding market exclusivity, patent protections, and pricing policies. Changes in intellectual property rights, regulatory approvals, or international trade agreements could impact the market dynamics.
Market SIPOC Analysis
What are the suppliers in the orphan drugs market in 2025?
Suppliers include biotech companies, pharmaceutical manufacturers, research institutions, and contract manufacturers that provide the raw materials, active pharmaceutical ingredients (APIs), and advanced technologies needed for developing orphan drugs.
Who are the inputs in the orphan drugs market in 2025?
Inputs include patient data, rare disease research, funding for clinical trials, regulatory frameworks, and the expertise of healthcare professionals and researchers working on orphan drug development. Technological advances in biotechnology and genomics also serve as crucial inputs.
What are the processes involved in the orphan drugs market in 2025?
The process involves drug discovery, preclinical and clinical trials, regulatory approval, manufacturing, distribution, and post-market surveillance. It also includes ongoing research to optimize treatments and address the unique needs of patients with rare diseases.
What are the outputs of the orphan drugs market in 2025?
Outputs consist of approved orphan drugs, patient access to these treatments, and improvements in the quality of life for patients with rare diseases. There are also intellectual property outcomes, such as patents, and financial results, including drug sales and market penetration.
Who are the customers of the orphan drugs market in 2025?
Customers include healthcare providers, hospitals, patients with rare diseases, and healthcare payers, including insurance companies and government healthcare systems, that reimburse or support the cost of orphan drugs. Additionally, patient advocacy groups play a critical role in driving demand and awareness.
Market Porter's Five Forces
What is the threat of new entrants in the orphan drugs market in 2025?
The threat of new entrants is moderate. While the orphan drugs market offers high potential profits, the complexity of regulatory approvals, high development costs, and the need for specialized expertise act as barriers to entry, limiting the number of new competitors.
How intense is the bargaining power of suppliers in the orphan drugs market in 2025?
The bargaining power of suppliers is relatively high. The market relies on specialized raw materials, advanced technology, and unique biotech capabilities that are often provided by a limited number of suppliers, giving them more leverage in pricing and supply agreements.
What is the bargaining power of buyers in the orphan drugs market in 2025?
The bargaining power of buyers is low to moderate. While patients and healthcare providers require orphan drugs, the limited availability of effective treatments for rare diseases reduces the ability of buyers to negotiate lower prices. However, healthcare payers may exert some influence on pricing.
How strong is the threat of substitute products in the orphan drugs market in 2025?
The threat of substitute products is low. Given the highly specialized nature of orphan drugs for rare diseases, alternatives are limited, and effective substitutes are often unavailable. However, new therapies such as gene therapies could pose a potential threat over time.
How intense is the rivalry among existing competitors in the orphan drugs market in 2025?
Rivalry is moderate to high. The orphan drugs market is growing rapidly, but competition remains limited due to the niche nature of the market. Nonetheless, as more companies develop treatments for rare diseases, competition will increase, especially for high-demand drug categories.
Market Upstream Analysis
What are the key upstream factors affecting the orphan drugs market in 2025?
Upstream factors include the availability and cost of raw materials, such as active pharmaceutical ingredients (APIs), and advancements in biotechnology and genetic research. The development of new technologies for drug discovery and manufacturing processes also plays a critical role in shaping the market.
How do suppliers impact the orphan drugs market in 2025?
Suppliers, including biotech companies, contract manufacturers, and research institutions, provide the necessary resources, technologies, and expertise for the development of orphan drugs. Their ability to supply high-quality raw materials, advanced production methods, and research support significantly influences the speed and cost of drug development.
What role do regulatory bodies play in the upstream process of orphan drugs in 2025?
Regulatory bodies like the FDA and EMA play a significant role in the upstream process by setting the standards for drug approval, safety, and efficacy. Their policies on orphan drug designation, expedited approval pathways, and pricing regulations directly impact the development timelines and market access for these drugs.
How does research and development (R&D) influence the upstream dynamics in the orphan drugs market in 2025?
R&D is a key upstream driver, as innovations in biotechnology, genomics, and rare disease research create new opportunities for developing orphan drugs. The ability to identify novel drug targets and create specialized therapies for underserved patient populations is crucial to meeting market demands.
What are the challenges faced in the upstream process of orphan drugs in 2025?
Challenges in the upstream process include high R&D costs, lengthy development timelines, and difficulties in obtaining patient data for rare diseases. Additionally, the complexity of manufacturing these drugs at scale, particularly for biologics and gene therapies, presents logistical and financial challenges.
Market Midstream Analysis
What are the key midstream factors affecting the orphan drugs market in 2025?
Midstream factors include the manufacturing and production processes of orphan drugs, distribution networks, and the role of contract manufacturing organizations (CMOs). Efficient scaling of production, quality control, and distribution to global markets are crucial for ensuring timely access to these treatments.
How does the manufacturing process impact the orphan drugs market in 2025?
The manufacturing process directly affects the cost, quality, and availability of orphan drugs. Given the complexity of these drugs, particularly biologics and gene therapies, manufacturing needs to be specialized and capable of handling small-scale but highly specialized production for rare diseases.
What role do distribution channels play in the orphan drugs market in 2025?
Distribution channels are vital for ensuring that orphan drugs reach the targeted patient populations, especially given the niche nature of these drugs. Specialized distribution networks and partnerships with healthcare providers and pharmacies ensure that rare disease treatments are accessible to patients worldwide.
How do logistics and supply chain issues affect the orphan drugs market in 2025?
Logistics and supply chain management are critical in the midstream phase, particularly because orphan drugs often require cold-chain storage or handling due to their sensitive nature. Any disruptions in the supply chain, whether from regulatory delays, manufacturing issues, or transportation challenges, can hinder timely access to treatments.
What challenges are faced in the midstream phase of orphan drug development in 2025?
Challenges include maintaining the stability and quality of small batches of drugs, ensuring regulatory compliance across various markets, and managing the costs associated with scaling production. Additionally, the need for specialized storage and transportation systems adds complexity to the supply chain.
Market Downstream Analysis
What are the key downstream factors affecting the orphan drugs market in 2025?
Downstream factors include the pricing, marketing, and accessibility of orphan drugs to patients. The role of healthcare providers, insurance companies, and patient advocacy groups in facilitating access, as well as the regulatory environment surrounding pricing and reimbursement, are essential for downstream success.
How does the pricing and reimbursement system affect the orphan drugs market in 2025?
Pricing and reimbursement systems directly influence market accessibility. Given the high cost of orphan drugs, reimbursement from insurance providers and government healthcare systems is critical to ensuring patient access. However, this can lead to challenges related to cost containment and affordability.
What role do healthcare providers play in the downstream phase of orphan drugs in 2025?
Healthcare providers are essential in diagnosing rare diseases, prescribing orphan drugs, and monitoring patient outcomes. Their involvement in clinical trials and post-market surveillance also contributes to the ongoing development of treatment strategies for rare diseases.
How do patient advocacy groups impact the orphan drugs market in 2025?
Patient advocacy groups play a significant role in raising awareness, advocating for regulatory support, and driving demand for orphan drugs. These groups can influence policy changes and help create a sense of urgency for new treatments, benefiting both patients and pharmaceutical companies.
What challenges are faced in the downstream phase of orphan drug development in 2025?
Challenges include navigating complex reimbursement processes, ensuring widespread access to orphan drugs in different regions, and addressing public perception of high drug prices. Additionally, limited healthcare infrastructure in some markets may hinder the availability of treatments to those who need them most.
Chapter 1, to describe Orphan Drugs product scope, market overview, market estimation caveats and base year.
Chapter 2, to profile the top players of Orphan Drugs, with revenue, gross margin and global market share of Orphan Drugs from 2018 to 2023.
Chapter 3, the Orphan Drugs competitive situation, revenue and global market share of top players are analyzed emphatically by landscape contrast.
Chapter 4 and 5, to segment the market size by Type and application, with consumption value and growth rate by Type, application, from 2018 to 2029.
Chapter 6, 7, 8, 9, and 10, to break the market size data at the country level, with revenue and market share for key countries in the world, from 2018 to 2023.and Orphan Drugs market forecast, by regions, type and application, with consumption value, from 2024 to 2029.
Chapter 11, market dynamics, drivers, restraints, trends, Porters Five Forces analysis, and Influence of COVID-19 and Russia-Ukraine War
Chapter 12, the key raw materials and key suppliers, and industry chain of Orphan Drugs.
Chapter 13, to describe Orphan Drugs research findings and conclusion.
1 Market Overview
1.1 Product Overview and Scope of Orphan Drugs
1.2 Market Estimation Caveats and Base Year
1.3 Classification of Orphan Drugs by Type
1.3.1 Overview: Global Orphan Drugs Market Size by Type: 2018 Versus 2022 Versus 2029
1.3.2 Global Orphan Drugs Consumption Value Market Share by Type in 2022
1.3.3 Oncology
1.3.4 Pulmonary
1.3.5 Neurology
1.3.6 Hematology
1.3.7 Endocrinology
1.3.8 Cardio-vascular
1.3.9 Metabolic Disorders
1.3.10 Others
1.4 Global Orphan Drugs Market by Application
1.4.1 Overview: Global Orphan Drugs Market Size by Application: 2018 Versus 2022 Versus 2029
1.4.2 Baby and Child
1.4.3 Teenager
1.4.4 Adult
1.4.5 Other
1.5 Global Orphan Drugs Market Size & Forecast
1.6 Global Orphan Drugs Market Size and Forecast by Region
1.6.1 Global Orphan Drugs Market Size by Region: 2018 VS 2022 VS 2029
1.6.2 Global Orphan Drugs Market Size by Region, (2018-2029)
1.6.3 North America Orphan Drugs Market Size and Prospect (2018-2029)
1.6.4 Europe Orphan Drugs Market Size and Prospect (2018-2029)
1.6.5 Asia-Pacific Orphan Drugs Market Size and Prospect (2018-2029)
1.6.6 South America Orphan Drugs Market Size and Prospect (2018-2029)
1.6.7 Middle East and Africa Orphan Drugs Market Size and Prospect (2018-2029)
2 Company Profiles
2.1 Bristol-Myers Squibb
2.1.1 Bristol-Myers Squibb Details
2.1.2 Bristol-Myers Squibb Major Business
2.1.3 Bristol-Myers Squibb Orphan Drugs Product and Solutions
2.1.4 Bristol-Myers Squibb Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.1.5 Bristol-Myers Squibb Recent Developments and Future Plans
2.2 Roche
2.2.1 Roche Details
2.2.2 Roche Major Business
2.2.3 Roche Orphan Drugs Product and Solutions
2.2.4 Roche Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.2.5 Roche Recent Developments and Future Plans
2.3 Novartis
2.3.1 Novartis Details
2.3.2 Novartis Major Business
2.3.3 Novartis Orphan Drugs Product and Solutions
2.3.4 Novartis Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.3.5 Novartis Recent Developments and Future Plans
2.4 Johnson & Johnson
2.4.1 Johnson & Johnson Details
2.4.2 Johnson & Johnson Major Business
2.4.3 Johnson & Johnson Orphan Drugs Product and Solutions
2.4.4 Johnson & Johnson Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.4.5 Johnson & Johnson Recent Developments and Future Plans
2.5 Pfizer
2.5.1 Pfizer Details
2.5.2 Pfizer Major Business
2.5.3 Pfizer Orphan Drugs Product and Solutions
2.5.4 Pfizer Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.5.5 Pfizer Recent Developments and Future Plans
2.6 Amgen
2.6.1 Amgen Details
2.6.2 Amgen Major Business
2.6.3 Amgen Orphan Drugs Product and Solutions
2.6.4 Amgen Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.6.5 Amgen Recent Developments and Future Plans
2.7 Sanofi
2.7.1 Sanofi Details
2.7.2 Sanofi Major Business
2.7.3 Sanofi Orphan Drugs Product and Solutions
2.7.4 Sanofi Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.7.5 Sanofi Recent Developments and Future Plans
2.8 AstraZeneca
2.8.1 AstraZeneca Details
2.8.2 AstraZeneca Major Business
2.8.3 AstraZeneca Orphan Drugs Product and Solutions
2.8.4 AstraZeneca Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.8.5 AstraZeneca Recent Developments and Future Plans
2.9 Takeda
2.9.1 Takeda Details
2.9.2 Takeda Major Business
2.9.3 Takeda Orphan Drugs Product and Solutions
2.9.4 Takeda Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.9.5 Takeda Recent Developments and Future Plans
2.10 Vertex Pharmaceuticals
2.10.1 Vertex Pharmaceuticals Details
2.10.2 Vertex Pharmaceuticals Major Business
2.10.3 Vertex Pharmaceuticals Orphan Drugs Product and Solutions
2.10.4 Vertex Pharmaceuticals Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.10.5 Vertex Pharmaceuticals Recent Developments and Future Plans
2.11 AbbVie
2.11.1 AbbVie Details
2.11.2 AbbVie Major Business
2.11.3 AbbVie Orphan Drugs Product and Solutions
2.11.4 AbbVie Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.11.5 AbbVie Recent Developments and Future Plans
2.12 Biogen
2.12.1 Biogen Details
2.12.2 Biogen Major Business
2.12.3 Biogen Orphan Drugs Product and Solutions
2.12.4 Biogen Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.12.5 Biogen Recent Developments and Future Plans
2.13 Eli Lilly
2.13.1 Eli Lilly Details
2.13.2 Eli Lilly Major Business
2.13.3 Eli Lilly Orphan Drugs Product and Solutions
2.13.4 Eli Lilly Orphan Drugs Revenue, Gross Margin and Market Share (2018-2023)
2.13.5 Eli Lilly Recent Developments and Future Plans
3 Market Competition, by Players
3.1 Global Orphan Drugs Revenue and Share by Players (2018-2023)
3.2 Market Share Analysis (2022)
3.2.1 Market Share of Orphan Drugs by Company Revenue
3.2.2 Top 3 Orphan Drugs Players Market Share in 2022
3.2.3 Top 6 Orphan Drugs Players Market Share in 2022
3.3 Orphan Drugs Market: Overall Company Footprint Analysis
3.3.1 Orphan Drugs Market: Region Footprint
3.3.2 Orphan Drugs Market: Company Product Type Footprint
3.3.3 Orphan Drugs Market: Company Product Application Footprint
3.4 New Market Entrants and Barriers to Market Entry
3.5 Mergers, Acquisition, Agreements, and Collaborations
4 Market Size Segment by Type
4.1 Global Orphan Drugs Consumption Value and Market Share by Type (2018-2023)
4.2 Global Orphan Drugs Market Forecast by Type (2024-2029)
5 Market Size Segment by Application
5.1 Global Orphan Drugs Consumption Value Market Share by Application (2018-2023)
5.2 Global Orphan Drugs Market Forecast by Application (2024-2029)
6 North America
6.1 North America Orphan Drugs Consumption Value by Type (2018-2029)
6.2 North America Orphan Drugs Consumption Value by Application (2018-2029)
6.3 North America Orphan Drugs Market Size by Country
6.3.1 North America Orphan Drugs Consumption Value by Country (2018-2029)
6.3.2 United States Orphan Drugs Market Size and Forecast (2018-2029)
6.3.3 Canada Orphan Drugs Market Size and Forecast (2018-2029)
6.3.4 Mexico Orphan Drugs Market Size and Forecast (2018-2029)
7 Europe
7.1 Europe Orphan Drugs Consumption Value by Type (2018-2029)
7.2 Europe Orphan Drugs Consumption Value by Application (2018-2029)
7.3 Europe Orphan Drugs Market Size by Country
7.3.1 Europe Orphan Drugs Consumption Value by Country (2018-2029)
7.3.2 Germany Orphan Drugs Market Size and Forecast (2018-2029)
7.3.3 France Orphan Drugs Market Size and Forecast (2018-2029)
7.3.4 United Kingdom Orphan Drugs Market Size and Forecast (2018-2029)
7.3.5 Russia Orphan Drugs Market Size and Forecast (2018-2029)
7.3.6 Italy Orphan Drugs Market Size and Forecast (2018-2029)
8 Asia-Pacific
8.1 Asia-Pacific Orphan Drugs Consumption Value by Type (2018-2029)
8.2 Asia-Pacific Orphan Drugs Consumption Value by Application (2018-2029)
8.3 Asia-Pacific Orphan Drugs Market Size by Region
8.3.1 Asia-Pacific Orphan Drugs Consumption Value by Region (2018-2029)
8.3.2 China Orphan Drugs Market Size and Forecast (2018-2029)
8.3.3 Japan Orphan Drugs Market Size and Forecast (2018-2029)
8.3.4 South Korea Orphan Drugs Market Size and Forecast (2018-2029)
8.3.5 India Orphan Drugs Market Size and Forecast (2018-2029)
8.3.6 Southeast Asia Orphan Drugs Market Size and Forecast (2018-2029)
8.3.7 Australia Orphan Drugs Market Size and Forecast (2018-2029)
9 South America
9.1 South America Orphan Drugs Consumption Value by Type (2018-2029)
9.2 South America Orphan Drugs Consumption Value by Application (2018-2029)
9.3 South America Orphan Drugs Market Size by Country
9.3.1 South America Orphan Drugs Consumption Value by Country (2018-2029)
9.3.2 Brazil Orphan Drugs Market Size and Forecast (2018-2029)
9.3.3 Argentina Orphan Drugs Market Size and Forecast (2018-2029)
10 Middle East & Africa
10.1 Middle East & Africa Orphan Drugs Consumption Value by Type (2018-2029)
10.2 Middle East & Africa Orphan Drugs Consumption Value by Application (2018-2029)
10.3 Middle East & Africa Orphan Drugs Market Size by Country
10.3.1 Middle East & Africa Orphan Drugs Consumption Value by Country (2018-2029)
10.3.2 Turkey Orphan Drugs Market Size and Forecast (2018-2029)
10.3.3 Saudi Arabia Orphan Drugs Market Size and Forecast (2018-2029)
10.3.4 UAE Orphan Drugs Market Size and Forecast (2018-2029)
11 Market Dynamics
11.1 Orphan Drugs Market Drivers
11.2 Orphan Drugs Market Restraints
11.3 Orphan Drugs Trends Analysis
11.4 Porters Five Forces Analysis
11.4.1 Threat of New Entrants
11.4.2 Bargaining Power of Suppliers
11.4.3 Bargaining Power of Buyers
11.4.4 Threat of Substitutes
11.4.5 Competitive Rivalry
11.5 Influence of COVID-19 and Russia-Ukraine War
11.5.1 Influence of COVID-19
11.5.2 Influence of Russia-Ukraine War
12 Industry Chain Analysis
12.1 Orphan Drugs Industry Chain
12.2 Orphan Drugs Upstream Analysis
12.3 Orphan Drugs Midstream Analysis
12.4 Orphan Drugs Downstream Analysis
13 Research Findings and Conclusion
14 Appendix
14.1 Methodology
14.2 Research Process and Data Source
14.3 Disclaimer
List of Tables
Table 1. Global Orphan Drugs Consumption Value by Type, (USD Million), 2018 & 2022 & 2029
Table 2. Global Orphan Drugs Consumption Value by Application, (USD Million), 2018 & 2022 & 2029
Table 3. Global Orphan Drugs Consumption Value by Region (2018-2023) & (USD Million)
Table 4. Global Orphan Drugs Consumption Value by Region (2024-2029) & (USD Million)
Table 5. Bristol-Myers Squibb Company Information, Head Office, and Major Competitors
Table 6. Bristol-Myers Squibb Major Business
Table 7. Bristol-Myers Squibb Orphan Drugs Product and Solutions
Table 8. Bristol-Myers Squibb Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 9. Bristol-Myers Squibb Recent Developments and Future Plans
Table 10. Roche Company Information, Head Office, and Major Competitors
Table 11. Roche Major Business
Table 12. Roche Orphan Drugs Product and Solutions
Table 13. Roche Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 14. Roche Recent Developments and Future Plans
Table 15. Novartis Company Information, Head Office, and Major Competitors
Table 16. Novartis Major Business
Table 17. Novartis Orphan Drugs Product and Solutions
Table 18. Novartis Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 19. Novartis Recent Developments and Future Plans
Table 20. Johnson & Johnson Company Information, Head Office, and Major Competitors
Table 21. Johnson & Johnson Major Business
Table 22. Johnson & Johnson Orphan Drugs Product and Solutions
Table 23. Johnson & Johnson Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 24. Johnson & Johnson Recent Developments and Future Plans
Table 25. Pfizer Company Information, Head Office, and Major Competitors
Table 26. Pfizer Major Business
Table 27. Pfizer Orphan Drugs Product and Solutions
Table 28. Pfizer Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 29. Pfizer Recent Developments and Future Plans
Table 30. Amgen Company Information, Head Office, and Major Competitors
Table 31. Amgen Major Business
Table 32. Amgen Orphan Drugs Product and Solutions
Table 33. Amgen Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 34. Amgen Recent Developments and Future Plans
Table 35. Sanofi Company Information, Head Office, and Major Competitors
Table 36. Sanofi Major Business
Table 37. Sanofi Orphan Drugs Product and Solutions
Table 38. Sanofi Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 39. Sanofi Recent Developments and Future Plans
Table 40. AstraZeneca Company Information, Head Office, and Major Competitors
Table 41. AstraZeneca Major Business
Table 42. AstraZeneca Orphan Drugs Product and Solutions
Table 43. AstraZeneca Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 44. AstraZeneca Recent Developments and Future Plans
Table 45. Takeda Company Information, Head Office, and Major Competitors
Table 46. Takeda Major Business
Table 47. Takeda Orphan Drugs Product and Solutions
Table 48. Takeda Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 49. Takeda Recent Developments and Future Plans
Table 50. Vertex Pharmaceuticals Company Information, Head Office, and Major Competitors
Table 51. Vertex Pharmaceuticals Major Business
Table 52. Vertex Pharmaceuticals Orphan Drugs Product and Solutions
Table 53. Vertex Pharmaceuticals Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 54. Vertex Pharmaceuticals Recent Developments and Future Plans
Table 55. AbbVie Company Information, Head Office, and Major Competitors
Table 56. AbbVie Major Business
Table 57. AbbVie Orphan Drugs Product and Solutions
Table 58. AbbVie Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 59. AbbVie Recent Developments and Future Plans
Table 60. Biogen Company Information, Head Office, and Major Competitors
Table 61. Biogen Major Business
Table 62. Biogen Orphan Drugs Product and Solutions
Table 63. Biogen Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 64. Biogen Recent Developments and Future Plans
Table 65. Eli Lilly Company Information, Head Office, and Major Competitors
Table 66. Eli Lilly Major Business
Table 67. Eli Lilly Orphan Drugs Product and Solutions
Table 68. Eli Lilly Orphan Drugs Revenue (USD Million), Gross Margin and Market Share (2018-2023)
Table 69. Eli Lilly Recent Developments and Future Plans
Table 70. Global Orphan Drugs Revenue (USD Million) by Players (2018-2023)
Table 71. Global Orphan Drugs Revenue Share by Players (2018-2023)
Table 72. Breakdown of Orphan Drugs by Company Type (Tier 1, Tier 2, and Tier 3)
Table 73. Market Position of Players in Orphan Drugs, (Tier 1, Tier 2, and Tier 3), Based on Revenue in 2022
Table 74. Head Office of Key Orphan Drugs Players
Table 75. Orphan Drugs Market: Company Product Type Footprint
Table 76. Orphan Drugs Market: Company Product Application Footprint
Table 77. Orphan Drugs New Market Entrants and Barriers to Market Entry
Table 78. Orphan Drugs Mergers, Acquisition, Agreements, and Collaborations
Table 79. Global Orphan Drugs Consumption Value (USD Million) by Type (2018-2023)
Table 80. Global Orphan Drugs Consumption Value Share by Type (2018-2023)
Table 81. Global Orphan Drugs Consumption Value Forecast by Type (2024-2029)
Table 82. Global Orphan Drugs Consumption Value by Application (2018-2023)
Table 83. Global Orphan Drugs Consumption Value Forecast by Application (2024-2029)
Table 84. North America Orphan Drugs Consumption Value by Type (2018-2023) & (USD Million)
Table 85. North America Orphan Drugs Consumption Value by Type (2024-2029) & (USD Million)
Table 86. North America Orphan Drugs Consumption Value by Application (2018-2023) & (USD Million)
Table 87. North America Orphan Drugs Consumption Value by Application (2024-2029) & (USD Million)
Table 88. North America Orphan Drugs Consumption Value by Country (2018-2023) & (USD Million)
Table 89. North America Orphan Drugs Consumption Value by Country (2024-2029) & (USD Million)
Table 90. Europe Orphan Drugs Consumption Value by Type (2018-2023) & (USD Million)
Table 91. Europe Orphan Drugs Consumption Value by Type (2024-2029) & (USD Million)
Table 92. Europe Orphan Drugs Consumption Value by Application (2018-2023) & (USD Million)
Table 93. Europe Orphan Drugs Consumption Value by Application (2024-2029) & (USD Million)
Table 94. Europe Orphan Drugs Consumption Value by Country (2018-2023) & (USD Million)
Table 95. Europe Orphan Drugs Consumption Value by Country (2024-2029) & (USD Million)
Table 96. Asia-Pacific Orphan Drugs Consumption Value by Type (2018-2023) & (USD Million)
Table 97. Asia-Pacific Orphan Drugs Consumption Value by Type (2024-2029) & (USD Million)
Table 98. Asia-Pacific Orphan Drugs Consumption Value by Application (2018-2023) & (USD Million)
Table 99. Asia-Pacific Orphan Drugs Consumption Value by Application (2024-2029) & (USD Million)
Table 100. Asia-Pacific Orphan Drugs Consumption Value by Region (2018-2023) & (USD Million)
Table 101. Asia-Pacific Orphan Drugs Consumption Value by Region (2024-2029) & (USD Million)
Table 102. South America Orphan Drugs Consumption Value by Type (2018-2023) & (USD Million)
Table 103. South America Orphan Drugs Consumption Value by Type (2024-2029) & (USD Million)
Table 104. South America Orphan Drugs Consumption Value by Application (2018-2023) & (USD Million)
Table 105. South America Orphan Drugs Consumption Value by Application (2024-2029) & (USD Million)
Table 106. South America Orphan Drugs Consumption Value by Country (2018-2023) & (USD Million)
Table 107. South America Orphan Drugs Consumption Value by Country (2024-2029) & (USD Million)
Table 108. Middle East & Africa Orphan Drugs Consumption Value by Type (2018-2023) & (USD Million)
Table 109. Middle East & Africa Orphan Drugs Consumption Value by Type (2024-2029) & (USD Million)
Table 110. Middle East & Africa Orphan Drugs Consumption Value by Application (2018-2023) & (USD Million)
Table 111. Middle East & Africa Orphan Drugs Consumption Value by Application (2024-2029) & (USD Million)
Table 112. Middle East & Africa Orphan Drugs Consumption Value by Country (2018-2023) & (USD Million)
Table 113. Middle East & Africa Orphan Drugs Consumption Value by Country (2024-2029) & (USD Million)
Table 114. Orphan Drugs Raw Material
Table 115. Key Suppliers of Orphan Drugs Raw Materials
List of Figures
Figure 1. Orphan Drugs Picture
Figure 2. Global Orphan Drugs Consumption Value by Type, (USD Million), 2018 & 2022 & 2029
Figure 3. Global Orphan Drugs Consumption Value Market Share by Type in 2022
Figure 4. Oncology
Figure 5. Pulmonary
Figure 6. Neurology
Figure 7. Hematology
Figure 8. Endocrinology
Figure 9. Cardio-vascular
Figure 10. Metabolic Disorders
Figure 11. Others
Figure 12. Global Orphan Drugs Consumption Value by Type, (USD Million), 2018 & 2022 & 2029
Figure 13. Orphan Drugs Consumption Value Market Share by Application in 2022
Figure 14. Baby and Child Picture
Figure 15. Teenager Picture
Figure 16. Adult Picture
Figure 17. Other Picture
Figure 18. Global Orphan Drugs Consumption Value, (USD Million): 2018 & 2022 & 2029
Figure 19. Global Orphan Drugs Consumption Value and Forecast (2018-2029) & (USD Million)
Figure 20. Global Market Orphan Drugs Consumption Value (USD Million) Comparison by Region (2018 & 2022 & 2029)
Figure 21. Global Orphan Drugs Consumption Value Market Share by Region (2018-2029)
Figure 22. Global Orphan Drugs Consumption Value Market Share by Region in 2022
Figure 23. North America Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 24. Europe Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 25. Asia-Pacific Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 26. South America Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 27. Middle East and Africa Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 28. Global Orphan Drugs Revenue Share by Players in 2022
Figure 29. Orphan Drugs Market Share by Company Type (Tier 1, Tier 2 and Tier 3) in 2022
Figure 30. Global Top 3 Players Orphan Drugs Market Share in 2022
Figure 31. Global Top 6 Players Orphan Drugs Market Share in 2022
Figure 32. Global Orphan Drugs Consumption Value Share by Type (2018-2023)
Figure 33. Global Orphan Drugs Market Share Forecast by Type (2024-2029)
Figure 34. Global Orphan Drugs Consumption Value Share by Application (2018-2023)
Figure 35. Global Orphan Drugs Market Share Forecast by Application (2024-2029)
Figure 36. North America Orphan Drugs Consumption Value Market Share by Type (2018-2029)
Figure 37. North America Orphan Drugs Consumption Value Market Share by Application (2018-2029)
Figure 38. North America Orphan Drugs Consumption Value Market Share by Country (2018-2029)
Figure 39. United States Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 40. Canada Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 41. Mexico Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 42. Europe Orphan Drugs Consumption Value Market Share by Type (2018-2029)
Figure 43. Europe Orphan Drugs Consumption Value Market Share by Application (2018-2029)
Figure 44. Europe Orphan Drugs Consumption Value Market Share by Country (2018-2029)
Figure 45. Germany Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 46. France Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 47. United Kingdom Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 48. Russia Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 49. Italy Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 50. Asia-Pacific Orphan Drugs Consumption Value Market Share by Type (2018-2029)
Figure 51. Asia-Pacific Orphan Drugs Consumption Value Market Share by Application (2018-2029)
Figure 52. Asia-Pacific Orphan Drugs Consumption Value Market Share by Region (2018-2029)
Figure 53. China Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 54. Japan Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 55. South Korea Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 56. India Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 57. Southeast Asia Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 58. Australia Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 59. South America Orphan Drugs Consumption Value Market Share by Type (2018-2029)
Figure 60. South America Orphan Drugs Consumption Value Market Share by Application (2018-2029)
Figure 61. South America Orphan Drugs Consumption Value Market Share by Country (2018-2029)
Figure 62. Brazil Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 63. Argentina Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 64. Middle East and Africa Orphan Drugs Consumption Value Market Share by Type (2018-2029)
Figure 65. Middle East and Africa Orphan Drugs Consumption Value Market Share by Application (2018-2029)
Figure 66. Middle East and Africa Orphan Drugs Consumption Value Market Share by Country (2018-2029)
Figure 67. Turkey Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 68. Saudi Arabia Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 69. UAE Orphan Drugs Consumption Value (2018-2029) & (USD Million)
Figure 70. Orphan Drugs Market Drivers
Figure 71. Orphan Drugs Market Restraints
Figure 72. Orphan Drugs Market Trends
Figure 73. Porters Five Forces Analysis
Figure 74. Manufacturing Cost Structure Analysis of Orphan Drugs in 2022
Figure 75. Manufacturing Process Analysis of Orphan Drugs
Figure 76. Orphan Drugs Industrial Chain
Figure 77. Methodology
Figure 78. Research Process and Data Source