Global Orphan Drugs
Market Report
2025
Delivery Includes:- Market Timeline 2019 till 2031, Market Size, Revenue/Volume Share, Forecast and CAGR, Competitor Analysis, Regional Analysis, Country Analysis, Segment Analysis, Market Trends, Drivers, Opportunities, Restraints, ESG Analysis, Porters Analysis, PESTEL Analysis, Market Attractiveness, Patent Analysis, Technological Trend, SWOT Analysis, COVID-19 Analysis, Consumer Behavior Analysis, etc.
The base year for the calculation is 2023 and 2019 to 2023 will be historical period. The year 2024 will be estimated one while the forecasted data will be from year 2025 to 2031. When we deliver the report that time we updated report data till the purchase date.
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According to cognitive market research, the global orphan drugs market size was valued at USD xx billion in 2024 and is expected to reach USD xx billion at a CAGR of xx% during the forecast period.
Base Year | 2023 |
Historical Data Time Period | 2019-2023 |
Forecast Period | 2024-2031 |
Global Orphan Drugs Market Sales Revenue 2020 | $ 138460 Million |
Global Orphan Drugs Market Sales Revenue 2030 | $ 388738 Million |
Global Orphan Drugs Market Compound Annual Growth Rate (CAGR) for 2024 to 2031 | 12.15% |
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Market Split by Distribution channel |
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List of Competitors |
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Regional Analysis |
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Country Analysis |
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Report scope is customizable as we have a huge database of Orphan Drugs industry. We can deliver an exclusive report Edition/Consultation as per your data requirements. Request for your Free Sample Pages.
Orphan Drugs Market is Segmented as below. Particular segment of your interest can be provided without any additional cost. Download the Sample Pages!
Orphan pharmaceuticals are pharmaceuticals that are used to diagnose and treat rare medical illnesses. These medications are developed to address a specific public health need and typically have a limited market because they are designed for a small patient population.
Numerous oncological, metabolic, hematologic, chronic, progressive, viral, and neurological disorders can be effectively treated with them. All of these diseases lymphoma, glioma, leukemia, cystic fibrosis, ovarian cancer, immunologic, multiple myeloma, and renal cell carcinoma are degenerative, life-threatening illnesses that need specialized care tailored to each patient's unique set of symptoms.
About 300 million people are predicted to be impacted globally by recurrent, chronic, and uncommon diseases as well as a rise in the need for large-scale orphan medication manufacture. The need for pharmaceuticals that support the growth of the market is rising for rare illness medications, including Waardenburg Syndrome, von Willebrand illness, Yellow Fever, Diabetes Macular Edoema, Abetalipoproteinemia, Autism Spectrum Disorder, Von Hippel Lindau Syndrome, Yellow Nail Syndrome, and others. Furthermore, about 400 million individuals worldwide suffer from orphan diseases, even though they are uncommon. Approximately 7000 distinct forms of orphan diseases have been identified. Players in the orphan medication industry now have a tonne of opportunities.
https://genomemedicine.biomedcentral.com/articles/10.1186/s13073-022-01026-w
• For instance, according to an article published by Euronews in 2023, there are 5000 to 8000 rare diseases in Europe that affect the daily lives of almost 30.0 million people in the region and 300.0 million across the world.
(Source:https://www.euronews.com/health/2023/06/15/30-million-europeans-affected-by-rare-diseases)
To incentivize pharmaceutical companies to invest in research and development for rare diseases, the U.S. Congress passed The Orphan Drug Act in 1983. This act grants tax credits, grants, and exclusive marketing rights to pharmaceutical companies that manufacture orphan drugs. The governments provide advantageous laws for the development and approval of orphan medications, hence providing further assistance to the pharmaceutical industry. Additionally, through insurance coverage and payment regulations, governments have supported patient access to orphan medications and funded their research and development.
• For instance, in October 2023, the Indian government launched the National Policy On Research and Development and Innovation in The Pharma-Med Tech Sector in India and the Scheme for The Promotion of Research and Innovations in The Pharma-Medtech Sector (PRIP). They focus on fostering research, development, and innovation in the pharmaceutical and medical technology sectors in India. The policy’s parts focus on rare diseases, gene therapy, orphan drugs, and precision medicines.
(Source:https://pib.gov.in/PressReleasePage.aspx?PRID=1960812)
The market for orphan pharmaceuticals is predicted to expand slowly due to the high costs connected with these medication innovations; nevertheless, during the projection period, the industry's growth may be hampered by high per-patient treatment costs and regulatory limitations on profitability. Orphan medications are selectively available because of the rarity of orphan illnesses. Orphan medications are expensive due to their limited availability and significant investments in research & development. Furthermore, the market's growth pace is impeded by factors like a small patient population, low public awareness of rare illnesses and orphan pharmaceuticals, and problems with payment in some nations.
• For instance, as a news article published by USA in 2023, Zolgensma, a one-time treatment approved for treating spinal muscular atrophy, is among the highest-priced drugs in the US market. The drugs cost around USD 2.25 million and have treated more than 3000 patients globally.
(Source:https://www.drugs.com/medical-answers/zolgensma-expensive-3552644/)
The COVID-19 pandemic has had a significant global impact on business operations. Furthermore, distinct sectors were affected by the COVID-19 pandemic in both good and bad ways. A few industries, notably those that provided healthcare services, were forced to close their doors. In addition, the COVID-19 pandemic negatively impacted the market for orphan pharmaceuticals because of financial shortages, operational challenges in research, and disruptions in major industry supply chains. The worldwide market for orphan pharmaceuticals declined as a result of several factors, including restricted access to specialists, temporary institution closures, and other difficulties the research institutes experienced. Due to social isolation and supply chain disruption, the COVID-19 epidemic is an exceptional public health risk that has had a substantial impact on the worldwide orphan drug market. Hospital and healthcare services have been severely constrained.
• For instance, In September 2020, following criticism that treating COVID-19 as a rare disease was "disingenuous," the United States Food and Drug Administration (FDA) withdrew orphan drug designation from a potential treatment for COVID-19 from Gilead Science.
(Source:https://pharmafeatures.com/understanding-the-regulation-of-orphan-drugs/)
The rising R&D expenditures by major companies for the creation of innovative items are one of the crucial factors that dominate the worldwide market. Strong pipeline candidates for orphan pharmaceuticals at various phases of clinical trials are being developed by several prominent clinical-stage biopharmaceutical firms and existing market players in response to the growing knowledge and comprehension of uncommon illnesses. The growing interest in treating rare illnesses is a result of the fact that, in contrast to standard pharmacological portfolios, uncommon disorders are a good candidate for significant pharmaceutical discoveries that might lead to the creation of blockbuster medications.
• For instance, in 2021, over half of the FDA’s Center for Drug Evaluation and Research (CDER), approvals had orphan designation and two CAR-T cell therapies were approved by the Centre for Biologics Evaluation and Research (CBER).
(Source:https://info.evaluate.com/rs/607-YGS-364/images/Evaluate%20Orphan%20Drug%20Report.pdf)
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Major market participants who are essential in determining consumer preferences and market dynamics have a major impact on the orphan drugs market. These major players provide consumers with easy access to a vast array of wardrobe options through their extensive retail networks and online platforms. Product adoption has increased as a result of their strong worldwide presence and a well-known brand, which has also increased consumer trust and loyalty. In addition, these industry titans never stop funding R&D, bringing cutting-edge styles, materials, and practical features to clothing wardrobes to meet changing customer demands and preferences. These big players' combined efforts have a big impact on the market's future direction and level of competition.
Top Companies Market Share in Orphan Drugs Industry: (In no particular order of Rank)
If any Company(ies) of your interest has/have not been disclosed in the above list then please let us know the same so that we will check the data availability in our database and provide you the confirmation or include it in the final deliverables.
North America is dominating in the global market. The market is growing due to several variables, such as the support of legislation that provides the quick approval of such products, the exclusivity of drug status for such drugs, and the presence of robust health systems. Regional expansion is being accelerated by the existence of a well-established healthcare network, important players, and an expanding patient base. Moreover, financing and supportive policies from the government are promoting regional prosperity. The fact that orphan pharmaceuticals in the US enjoy tax benefits, a user fee waiver, and seven years of marketing exclusivity following FDA clearance for a particular purpose is one factor contributing to the market's expansion. Furthermore, it is projected that the market will increase at a faster rate as more medications receive the status of orphan medicine.
The European orphan drugs market accounted for a substantial share of the global market in 2024 and is expected to grow at a promising CAGR during the forecast period. The European market is expanding at a faster rate than other regions of the world due to factors including the rising frequency of uncommon illnesses and the growing prevalence of innovative medications. Furthermore, the European market is growing due to the presence of many biotech and pharmaceutical businesses as well as regulatory agencies like the European Medicines Agency (EMA) that have favorable policies for the development of orphan medications. In 2024, Germany, France, and the UK accounted for the largest shares of the European market
The current report Scope analyzes Orphan Drugs Market on 5 major region Split (In case you wish to acquire a specific region edition (more granular data) or any country Edition data then please write us on info@cognitivemarketresearch.com
The above graph is for illustrative purposes only.
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Global Orphan Drugs Market Report 2024 Edition talks about crucial market insights with the help of segments and sub-segments analysis. In this section, we reveal an in-depth analysis of the key factors influencing Orphan Drugs Industry growth. Orphan Drugs market has been segmented with the help of its Type, Distribution channel , and others. Orphan Drugs market analysis helps to understand key industry segments, and their global, regional, and country-level insights. Furthermore, this analysis also provides information pertaining to segments that are going to be most lucrative in the near future and their expected growth rate and future market opportunities. The report also provides detailed insights into factors responsible for the positive or negative growth of each industry segment.
Depending on the drug type, the orphan drugs market can be further segmented into biological and non-biological orphan products. The World Health Organisation (WHO) defines biologics, also known as biological products, as a broad category of pharmaceuticals that includes human blood and plasma products, growth factors, monoclonal antibodies, vaccines, and immune modulators. The biological orphan drug category is the result of key market participants supplying a greater number of biological products. Over the course of the projection period, the biological sector is anticipated to develop at a faster rate than the non-biological segment due to the increasing number of cutting-edge biomedical research projects. Furthermore, the biological orphan medication market is dominated by the growing number of biological orphan medicines that are undergoing various stages of clinical studies.
• For instance, according to an estimate by the US Food and Drug Administration (FDA), the Office of Orphan Products Development (OOPD) of the organization has developed and marketed over 600 drugs and biological products to treat rare disorders since 1983.
(Source:https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products)
The above Chart is for representative purposes and does not depict actual sale statistics. Access/Request the quantitative data to understand the trends and dominating segment of Orphan Drugs Industry. Request a Free Sample PDF!
Based on the distribution channel, the market is segmented into hospital pharmacies and retail pharmacies. The hospital pharmacies segment is expected to share the largest market share in the orphan drugs market. Drug dosages supplied intravenously (IV) to patients with orphan illnesses must only be handled and monitored by qualified healthcare providers. This guarantees the supply of orphan medications, ideally in hospital pharmacies, further controlling this market sector for orphan medications. This concentration allows for better organization and accessibility to the relevant medications and therapies. Moreover, government assistance is one of the reasons driving the growth of the hospital pharmacy industry.
• For instance, the FIP (International Pharmaceutical Federation) Hospital Pharmacy Section (HPS) is dedicated to advancing the practice of hospital pharmacy through various initiatives and grants. The HPS aims to encourage innovative research in the field of hospital pharmacy.
(Source:https://www.fip.org/files/fip/HPS/FIP_HPS_Strategic_Plan_2022-2027.pdf)
The above Graph is for representation purposes only. This chart does not depict actual Market share.
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Senior Research Associate at Cognitive Market Research
Manjiri Kanhere is an experienced market researcher focused on the Pharma & Healthcare industry. With over 3+ years of experience, She has worked with major pharmaceutical companies and healthcare providers, helping them to understand market trends, identify new business opportunities, and develop effective sales & marketing strategies. Her expertise lies in the ability to analyze complex data sets and deliver insights that drive business decisions. At Cognitive Market Research, she handles the market research related to Pharma and healthcare industry.
Manjiri Kanhere is an experienced market researcher focused on the Pharma & Healthcare industry. With over three years of experience, She has worked with major pharmaceutical companies and healthcare providers, helping them to understand market trends, identify new business opportunities, and develop effective sales & marketing strategies.
In her current role, Manjiri handles the market research related to Pharma and healthcare industry. Her passion lies in utilizing innovative approaches to distill complex information into strategic insights that empower organizations to make informed decisions.Manjiri remains an invaluable asset in the dynamic landscape of market research.
Orphan pharmaceuticals are pharmaceuticals that are used to diagnose and treat rare medical illnesses. These medications are developed to address a specific public health need and typically have a limited market because they are designed for a small patient population. Orphan medications are selectively available because of the rarity of orphan illnesses. Orphan medications are expensive due to their limited availability and significant investments in research & development. Major market participants who are essential in determining consumer preferences and market dynamics have a major impact on the orphan drugs market
Please note, we have not disclose, all the sources consulted/referred during a market study due to confidentiality and paid service concern. However, rest assured that upon purchasing the service or paid report version, we will release the comprehensive list of sources along with the complete report and we also provide the data support where you can intract with the team of analysts who worked on the report.
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Type | Biological, Non-biological |
Distribution channel | Hospital Pharmacies, Speciality Pharmacies, Retail pharmacies |
List of Competitors | 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., Shire, Amgen Inc. |
This chapter will help you gain GLOBAL Market Analysis of Orphan Drugs. Further deep in this chapter, you will be able to review Global Orphan Drugs Market Split by various segments and Geographical Split.
Chapter 1 Global Market Analysis
Global Market has been segmented on the basis 5 major regions such as North America, Europe, Asia-Pacific, Middle East & Africa, and Latin America.
You can purchase only the Executive Summary of Global Market (2019 vs 2024 vs 2031)
Global Market Dynamics, Trends, Drivers, Restraints, Opportunities, Only Pointers will be deliverable
This chapter will help you gain North America Market Analysis of Orphan Drugs. Further deep in this chapter, you will be able to review North America Orphan Drugs Market Split by various segments and Country Split.
Chapter 2 North America Market Analysis
This chapter will help you gain Europe Market Analysis of Orphan Drugs. Further deep in this chapter, you will be able to review Europe Orphan Drugs Market Split by various segments and Country Split.
Chapter 3 Europe Market Analysis
This chapter will help you gain Asia Pacific Market Analysis of Orphan Drugs. Further deep in this chapter, you will be able to review Asia Pacific Orphan Drugs Market Split by various segments and Country Split.
Chapter 4 Asia Pacific Market Analysis
This chapter will help you gain South America Market Analysis of Orphan Drugs. Further deep in this chapter, you will be able to review South America Orphan Drugs Market Split by various segments and Country Split.
Chapter 5 South America Market Analysis
This chapter will help you gain Middle East and Africa Market Analysis of Orphan Drugs. Further deep in this chapter, you will be able to review Middle East and Africa Orphan Drugs Market Split by various segments and Country Split.
Chapter 6 Middle East and Africa Market Analysis
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Chapter 7 Top 10 Countries Analysis (Only Available with Corporate User License)
This chapter provides an in-depth analysis of the market share among key competitors of Orphan Drugs. The analysis highlights each competitor's position in the market, growth trends, and financial performance, offering insights into competitive dynamics, and emerging players.
Chapter 8 Competitor Analysis (Subject to Data Availability (Private Players))
(Subject to Data Availability (Private Players))
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
Data Subject to Availability as we consider Top competitors and their market share will be delivered.
This chapter would comprehensively cover market drivers, trends, restraints, opportunities, and various in-depth analyses like industrial chain, PESTEL, Porter’s Five Forces, and ESG, among others. It would also include product life cycle, technological advancements, and patent insights.
Chapter 9 Qualitative Analysis (Subject to Data Availability)
Segmentation Type Analysis 2019 -2031, will provide market size split by Type. This Information is provided at Global Level, Regional Level and Top Countries Level The report with the segmentation perspective mentioned under this chapters will be delivered to you On Demand. So please let us know if you would like to receive this additional data as well. No additional cost will be applicable for the same.
Chapter 10 Market Split by Type Analysis 2019 -2031
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Chapter 11 Market Split by Distribution channel Analysis 2019 -2031
This chapter helps you understand the Key Takeaways and Analyst Point of View of the global Orphan Drugs market
Chapter 12 Research Findings
Here the analyst will summarize the content of entire report and will share his view point on the current industry scenario and how the market is expected to perform in the near future. The points shared by the analyst are based on his/her detailed in-depth understanding of the market during the course of this report study. You will be provided exclusive rights to interact with the concerned analyst for unlimited time pre purchase as well as post purchase of the report.
Why Biological have a significant impact on Orphan Drugs market? |
What are the key factors affecting the Biological and Non-biological of Orphan Drugs Market? |
What is the CAGR/Growth Rate of Hospital Pharmacies during the forecast period? |
By type, which segment accounted for largest share of the global Orphan Drugs Market? |
Which region is expected to dominate the global Orphan Drugs Market within the forecast period? |
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