USD 87.44 billion
Orphan Drugs Market Size, Share, Growth Analysis, By Drug Type (Biological, Non-biological), By Disease Type (Oncology, Hematology), By Distribution Channel (Pharmacy, Hospital), By Region - Industry Forecast 2025-2032
Report ID:
SQMIG30A2005 |
Region:
Global |
Published Date: November, 2024
Pages:
211
|Tables:
65
|Figures:
75
Orphan Drug Market Insights
Orphan Drug Market size was valued at USD 195.76 Billion in 2023 and is poised to grow from USD 218.9 Billion in 2024 to USD 535.05 Billion by 2032, growing at a CAGR of 11.82% during the forecast period (2025-2032).
Orphan drugs are designed to treat illnesses that are so uncommon that their sponsors are reluctant to develop them under standard marketing guidelines.
According to the World Health Organization (WHO), this condition occurs less frequently than 6.5 to 10 times per 10,000 people.
The journey from a new molecule's discovery to its commercialization often takes ten years, costs several tens of millions of euros, and is fraught with uncertainty (among ten molecules tested, only one may have a therapeutic effect). The money spent on its research cannot be recouped when a medicine is developed to treat a rare condition.
Since the introduction of the Orphan Drug Act in 2019, intended to promote development of treatments for rare diseases, at least 378 orphan drugs have been approved. Kalydeco may be the orphan drug of all orphan drugs.
The U.S. dominated development of orphan drugs, with more than 300 in clinical trials, followed by Europe. Cancer treatment was the indication in more than 30% of orphan drug trials.
There are currently approximately 7000 rare diseases listed on the National Institute of Health’s (NIH) Office of Rare Diseases website, affecting an estimated 25–30 million people within the United States3, most of these rare diseases are genetic and many are neurological. Approximately 250 new rare diseases are described each year4, and with advances in genetics, many more common disorders will be subdivided into genetically distinct “rare” diseases.
US Orphan Drug Market is poised to grow at a sustainable CAGR for the next forecast year.
Market snapshot - 2024-2031
Global Market Size
Largest Segment
Biologics
Fastest Growth
Non-biologics
Growth Rate
9.5% CAGR
Global Orphan Drug Market, 2024-2031($ Bn)
Country Share for North America Region- 2024-2031 (%)
To get more reports on the above market click here to Buy The Report
Orphan Drug Market Segmental Analysis
Global Orphan Drugs Market is segmented by Drug Type, Disease Type, Distribution Channel, and Region. Based on Drug Type, the market is segmented into Biological, and Non-biological. Based on Disease Type, the market is segmented into O Hematology, Neurology, Endocrinology, Cardiovascular, Respiratory, Immunotherapy, Infectious Diseases, and Others. Based on Distribution Channel, the market is segmented into Hospital, Pharmacy, Online sales, Others. Based on region, the market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa.
Orphan Drug Market Analysis by Disease Type
Oncological diseases, metabolic diseases, hematologic & immunologic diseases, infectious diseases, neurologic diseases, and other rare diseases, which also include transplantation diseases, gastrointestinal diseases, dermatologic diseases, perinatal & congenital abnormalities, and others, are the disease types that the orphan drugs market is divided into by category. Acute myeloid leukaemia (AML), pancreatic cancer, ovarian cancer, multiple myeloma, renal cell carcinoma, and other cancers are included in the classification of oncological illnesses. Huntington syndrome, Fabry disease, Gaucher disease, hypoparathyroidism, and other are further subtypes of metabolic illnesses. Hematologic and immunologic illnesses are further divided into haemophilia, hereditary angioedema, and other conditions. Duchenne muscular dystrophy (DMD), Alzheimer's disease, Huntington's disease, and other neurologic illnesses are further divided into subcategories.
Due to an increase in the prevalence of oncological illnesses, increased public awareness of cancer, the availability of oncological orphan medications for the treatment of cancer, and a surge in early cancer detection, the oncological disorders segment had the largest shareholder in 2021.
Orphan Drug Market Analysis by Drug Type
The Global Orphan Drug Market has been segmented on the basis of Drug Type as biologics and non-biologics. One of the main causes of the segment's domination in 2019 is the preponderance of product offerings that are categorized as biologics. The U.S. Food and Drug Administration (U.S. FDA) estimates that over 600 medicines and biologic products for rare conditions have been developed and marketed thanks to the organization's Orphan Products Development (OOPD) since 1983. The non-biologics market sector had a smaller market share overall and a lower CAGR than other segments.
Global Orphan Drug Market By Drug Type , 2024(%)
To get detailed analysis on other segments, Request For Free Sample Report
Orphan Drug Market Regional Insights
In terms of CAGR, North America is expected to grow quickly in the years to come. This is attributed to the increase in patients in this area who are susceptible to rare diseases. The unhealthy lifestyle and stressful lifestyle are the key factors that cause this disease to spread in this area. In order to reduce the number of patients brought on by this disease, government organisations like NGO are investing a significant amount of money in this region.
Also, the European Orphan Drug Market would grow in the coming years, due to the large patient population in the area and the widespread use of complex medicines for uncommon diseases. Due to rising healthcare costs and greater awareness of rare diseases, the market in Asia Pacific is expected to expand at the fastest rate. Latin America, the Middle East, and Africa made up the Rest of the World, which accounted for a smaller share of the worldwide market due to the under-penetration of the market.
Global Orphan Drug Market By Geography, 2024-2031
- Largest
- Fastest
To know more about the market opportunities by region and country, click here to
Buy The Complete Report
Orphan Drug Market Dynamics
Orphan Drug Market Driver
- The development of orphan medications under research has been significantly influenced by the development of new technologies. Due recent advancements in genomics and the wider application of gene sequencing, made it possible in case of rare disease. furthermore, additional technologies like gene therapy and antisense therapy have made it feasible for us to target certain disorders in ways that were not previously possible. Combined, technological advancements have made it easier to both diagnose and cure uncommon genetic disorders, which has made them more manageable for pharmaceutical companies.
- Even though there is a small patient pool, factors including incentives, accelerated development schedules, and high regulatory approval rates make developing drugs for rare diseases cost effective.
Orphan Drug Market Restraint
- Orphan medications frequently fall short of established health technology assessment criteria, particularly cost-effectiveness, which may affect access, due to high costs and a lack of data.
- One of the major challenges in these markets is finding qualified patients for clinical trials of orphan drugs. Additionally, a thorough understanding of the biology of the disease is necessary for efficient recruiting in uncommon disorders.
Request Free Customization of this report to help us to meet your business objectives.
Orphan Drug Market Competitive Landscape
The Global Orphan Drug Market is relatively fragmented, with a high level of competition. The prominent players operating in the orphan drug market are constantly
Orphan Drug Market Top Player's Company Profiles
- Novartis AG
- Pfizer Inc.
- Sanofi
- Bristol-Myers Squibb Company
- GSK plc
- AbbVie Inc.
- Merck & Co., Inc.
- Johnson & Johnson Private Limited
- F. Hoffmann-La Roche Ltd
- Vertex Pharmaceuticals
- BioMarin Pharmaceutical
- Takeda Pharmaceutical Company Limited
- Roche
- Amgen
- Ultragenyx Pharmaceutical
- CSL Behring
- Grifols
- Sobi (Swedish Orphan Biovitrum)
- Ipsen
- Jazz Pharmaceuticals
- AstraZeneca
Orphan Drug Market Recent Developments
- In April 2022, according to news, the product's creator Shanghai Henlius Biotech, the FDA has qualified serplulimab, also known as Hansizhuang, as an orphan medication for the treatment of small-cell lung cancer.
- In March 2022, ABIONYX Pharma reported that the USFDA had granted Bio-HDL CER-001 orphan-drug designation (ODD) for the treatment of lecithin-cholesterol acyltransferase deficiency, also known as LCAT deficiency.
Orphan Drug Key Market Trends
- Since companies employ far lower criteria for showing treatment effectiveness, the expansion of the use of auxiliary or surrogate endpoints within clinical trials for orphan pharmaceuticals has significantly reduced the time and cost needed to undertake R&D. This can accelerate the growth of the Orphan Drug Market.
- Patients throughout the world looking for effective and efficient therapy for rare diseases. Newer and more effective product offerings are being introduced as a result of growing R&D initiatives and growing patient advocacy for effective therapy. These developments have resulted in the creation and distribution of multiple blockbuster medications for the treatment of various malignancies, immune disorders, and other rare diseases.
Orphan Drug Market SkyQuest Analysis
SkyQuest’s ABIRAW (Advanced Business Intelligence, Research & Analysis Wing) is our Business Information Services team that Collects, Collates, Co-relates, and Analyses the Data collected utilizing Primary Exploratory Research backed by the robust Secondary Desk research.
According to our Global Orphan Drug Market Analysis, in terms of drug type, the availability of oncological orphan medications for the treatment of cancer, and a surge in early cancer detection, the oncological disorders segment had the largest shareholder in 2021. Combined, technological advancements have made it easier to both diagnose and cure uncommon genetic disorders, which has made them more manageable for pharmaceutical companies.
| Report Metric | Details |
|---|---|
| Market size value in 2022 | USD 175.06 billion |
| Market size value in 2031 | USD 478.49 billion |
| Growth Rate | 11.82% |
| Base year | 2023 |
| Forecast period | 2024-2031 |
| Forecast Unit (Value) | USD Billion |
| Segments covered |
|
| Regions covered | North America (US, Canada), Europe (Germany, France, United Kingdom, Italy, Spain, Rest of Europe), Asia Pacific (China, India, Japan, Rest of Asia-Pacific), Latin America (Brazil, Rest of Latin America), Middle East & Africa (South Africa, GCC Countries, Rest of MEA) |
| Companies covered |
|
| Customization scope | Free report customization with purchase. Customization includes:-
|
To get a free trial access to our platform which is a one stop solution for all your data requirements for quicker decision making. This platform allows you to compare markets, competitors who are prominent in the market, and mega trends that are influencing the dynamics in the market. Also, get access to detailed SkyQuest exclusive matrix.
Buy The Complete Report to read the analyzed strategies adopted by the top vendors either to retain or gain market share
Table Of Content
Executive Summary
Market overview
- Exhibit: Executive Summary – Chart on Market Overview
- Exhibit: Executive Summary – Data Table on Market Overview
- Exhibit: Executive Summary – Chart on Orphan Drug Market Characteristics
- Exhibit: Executive Summary – Chart on Market by Geography
- Exhibit: Executive Summary – Chart on Market Segmentation
- Exhibit: Executive Summary – Chart on Incremental Growth
- Exhibit: Executive Summary – Data Table on Incremental Growth
- Exhibit: Executive Summary – Chart on Vendor Market Positioning
Parent Market Analysis
Market overview
Market size
- Market Dynamics
- Exhibit: Impact analysis of DROC, 2021
- Drivers
- Opportunities
- Restraints
- Challenges
- Exhibit: Impact analysis of DROC, 2021
- SWOT Analysis
KEY MARKET INSIGHTS
- Technology Analysis
- (Exhibit: Data Table: Name of technology and details)
- Pricing Analysis
- (Exhibit: Data Table: Name of technology and pricing details)
- Supply Chain Analysis
- (Exhibit: Detailed Supply Chain Presentation)
- Value Chain Analysis
- (Exhibit: Detailed Value Chain Presentation)
- Ecosystem Of the Market
- Exhibit: Parent Market Ecosystem Market Analysis
- Exhibit: Market Characteristics of Parent Market
- IP Analysis
- (Exhibit: Data Table: Name of product/technology, patents filed, inventor/company name, acquiring firm)
- Trade Analysis
- (Exhibit: Data Table: Import and Export data details)
- Startup Analysis
- (Exhibit: Data Table: Emerging startups details)
- Raw Material Analysis
- (Exhibit: Data Table: Mapping of key raw materials)
- Innovation Matrix
- (Exhibit: Positioning Matrix: Mapping of new and existing technologies)
- Pipeline product Analysis
- (Exhibit: Data Table: Name of companies and pipeline products, regional mapping)
- Macroeconomic Indicators
COVID IMPACT
- Introduction
- Impact On Economy—scenario Assessment
- Exhibit: Data on GDP - Year-over-year growth 2016-2022 (%)
- Revised Market Size
- Exhibit: Data Table on Orphan Drug Market size and forecast 2021-2027 ($ million)
- Impact Of COVID On Key Segments
- Exhibit: Data Table on Segment Market size and forecast 2021-2027 ($ million)
- COVID Strategies By Company
- Exhibit: Analysis on key strategies adopted by companies
MARKET DYNAMICS & OUTLOOK
- Market Dynamics
- Exhibit: Impact analysis of DROC, 2021
- Drivers
- Opportunities
- Restraints
- Challenges
- Exhibit: Impact analysis of DROC, 2021
- Regulatory Landscape
- Exhibit: Data Table on regulation from different region
- SWOT Analysis
- Porters Analysis
- Competitive rivalry
- Exhibit: Competitive rivalry Impact of key factors, 2021
- Threat of substitute products
- Exhibit: Threat of Substitute Products Impact of key factors, 2021
- Bargaining power of buyers
- Exhibit: buyers bargaining power Impact of key factors, 2021
- Threat of new entrants
- Exhibit: Threat of new entrants Impact of key factors, 2021
- Bargaining power of suppliers
- Exhibit: Threat of suppliers bargaining power Impact of key factors, 2021
- Competitive rivalry
- Skyquest special insights on future disruptions
- Political Impact
- Economic impact
- Social Impact
- Technical Impact
- Environmental Impact
- Legal Impact
Market Size by Region
- Chart on Market share by geography 2021-2027 (%)
- Data Table on Market share by geography 2021-2027(%)
- North America
- Chart on Market share by country 2021-2027 (%)
- Data Table on Market share by country 2021-2027(%)
- USA
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Canada
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Europe
- Chart on Market share by country 2021-2027 (%)
- Data Table on Market share by country 2021-2027(%)
- Germany
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Spain
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- France
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- UK
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Rest of Europe
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Asia Pacific
- Chart on Market share by country 2021-2027 (%)
- Data Table on Market share by country 2021-2027(%)
- China
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- India
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Japan
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- South Korea
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Rest of Asia Pacific
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Latin America
- Chart on Market share by country 2021-2027 (%)
- Data Table on Market share by country 2021-2027(%)
- Brazil
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Rest of South America
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Middle East & Africa (MEA)
- Chart on Market share by country 2021-2027 (%)
- Data Table on Market share by country 2021-2027(%)
- GCC Countries
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- South Africa
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Rest of MEA
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
KEY COMPANY PROFILES
- Competitive Landscape
- Total number of companies covered
- Exhibit: companies covered in the report, 2021
- Top companies market positioning
- Exhibit: company positioning matrix, 2021
- Top companies market Share
- Exhibit: Pie chart analysis on company market share, 2021(%)
- Total number of companies covered
Methodology
For the Orphan Drug Market, our research methodology involved a mixture of primary and secondary data sources. Key steps involved in the research process are listed below:
1. Information Procurement: This stage involved the procurement of Market data or related information via primary and secondary sources. The various secondary sources used included various company websites, annual reports, trade databases, and paid databases such as Hoover's, Bloomberg Business, Factiva, and Avention. Our team did 45 primary interactions Globally which included several stakeholders such as manufacturers, customers, key opinion leaders, etc. Overall, information procurement was one of the most extensive stages in our research process.
2. Information Analysis: This step involved triangulation of data through bottom-up and top-down approaches to estimate and validate the total size and future estimate of the Orphan Drug Market.
3. Report Formulation: The final step entailed the placement of data points in appropriate Market spaces in an attempt to deduce viable conclusions.
4. Validation & Publishing: Validation is the most important step in the process. Validation & re-validation via an intricately designed process helped us finalize data points to be used for final calculations. The final Market estimates and forecasts were then aligned and sent to our panel of industry experts for validation of data. Once the validation was done the report was sent to our Quality Assurance team to ensure adherence to style guides, consistency & design.
Analyst Support
Customization Options
With the given market data, our dedicated team of analysts can offer you the following customization options are available for the Orphan Drug Market:
Product Analysis: Product matrix, which offers a detailed comparison of the product portfolio of companies.
Regional Analysis: Further analysis of the Orphan Drug Market for additional countries.
Competitive Analysis: Detailed analysis and profiling of additional Market players & comparative analysis of competitive products.
Go to Market Strategy: Find the high-growth channels to invest your marketing efforts and increase your customer base.
Innovation Mapping: Identify racial solutions and innovation, connected to deep ecosystems of innovators, start-ups, academics, and strategic partners.
Category Intelligence: Customized intelligence that is relevant to their supply Markets will enable them to make smarter sourcing decisions and improve their category management.
Public Company Transcript Analysis: To improve the investment performance by generating new alpha and making better-informed decisions.
Social Media Listening: To analyze the conversations and trends happening not just around your brand, but around your industry as a whole, and use those insights to make better Marketing decisions.
USA (+1) 351-333-4748
