Orphan Drugs Market

Orphan Drugs Market

Orphan Drugs Market Forecasts to 2030 - Global Analysis By Disease Type (Oncological Diseases, Metabolic Diseases, Hematologic & Immunologic Diseases, Infectious diseases and Neurological diseases), Drug Type (Non-Biologics and Biologics), Sale, Drug, Distribution Channel and By Geography

4.6 (99 reviews)
4.6 (99 reviews)
Published: 2023 ID: SMRC24351

This report covers the impact of COVID-19 on this global market

Years Covered


Estimated Year Value (2023)

US $175.8 BN

Projected Year Value (2030)

US $445.5 BN

CAGR (2023 - 2030)


Regions Covered

North America, Europe, Asia Pacific, South America, and Middle East & Africa

Countries Covered

US, Canada, Mexico, Germany, UK, Italy, France, Spain, Japan, China, India, Australia, New Zealand, South Korea, Rest of Asia Pacific, South America, Argentina, Brazil, Chile, Middle East & Africa, Saudi Arabia, UAE, Qatar, and South Africa

Largest Market

Asia Pacific

Highest Growing Market

North America

According to Stratistics MRC, the Global Orphan Drugs Market is accounted for $175.8 billion in 2023 and is expected to reach $445.5 billion by 2030 growing at a CAGR of 14.2% during the forecast period. Drugs used to identify and treat uncommon medical conditions are known as orphan drugs. Numerous oncological, metabolic, hematologic, chronic, progressive, viral, and neurological illnesses can be effectively treated with them. Orphan medications often employ a targeted approach, concentrating on the particular genetic or molecular abnormalities that underlie a rare disease. This results in more precise and effective treatment options, leading to improved patient outcomes, including increased survival rates, symptom management, and overall quality of life.

According to the Genetic and Rare Diseases (GARD) Information Center, there are more than 10,000 known rare diseases in the U.S. that affect about 1 in 10 people or 30 million people in the U.S.

Market Dynamics: 


Rise in disease awareness

Growing knowledge of diseases is a major factor driving the market for orphan medications since it makes rare diseases better recognized, which encourages advocacy, funding, and research. Moreover, an increase in patient, healthcare professional and advocacy group awareness drives up the need for efficient therapies, which in turn motivates pharmaceutical corporations to fund research and development of orphan drugs. In the end, it expands the market and enhances the lives of people with rare diseases by making specialized therapies more accessible. Therefore, it also results in increased support for funding, regulatory incentives, and partnerships.  


High initial costs

The process of creating medications for uncommon diseases can be costly due to the extensive research, clinical trials, and regulatory procedures involved. Additionally, orphan medications are generally costly since these expenses must be covered by drug prices. It can be difficult to deliver rare disease therapies that are both widely available and reasonably priced due to the high expenses, which can also pose access challenges for insurers, healthcare systems, and patients. Hence, this can leave patients with limited treatment options and hinder progress in addressing the unmet medical needs of individuals with rare diseases.


Developments in regulatory incentives

Extended market exclusivity, tax rebates, and accelerated approval procedures are all included in these incentives. They incentivize pharmaceutical companies to spend money on the development of orphan drugs by lowering financial risks and accelerating time to market. Furthermore, these kinds of incentives make these ventures more financially viable, which in turn encourage innovation and make more orphan medications available to meet the unmet medical requirements of people with rare diseases. Pharmaceutical businesses and those with rare illnesses benefit from this in a win-win scenario. Hence, government regulations that provide favourable incentives to encourage the development of therapies for rare diseases are a major factor driving the orphan drug industry.


Risks associated with limited potential  

The limited market potential can discourage companies from pursuing orphan drug development, resulting in fewer treatment options for patients with rare diseases and potential delays in accessing innovative therapies. Moreover, drugs known as orphans are created to treat uncommon conditions that only a tiny number of people have. The market for these medications is less than that of medications for more prevalent disorders because of the small number of patients. Furthermore, pharmaceutical corporations may find it financially undesirable to invest in the research and development of orphan medications as a result, which is a drawback of the market's expansion.  

Covid-19 Impact

The outbreak of COVID-19 has disrupted workflows in the healthcare sector around the world. Numerous industries, including some healthcare-related sub-sectors, have been forced to temporarily close their doors due to the disease. The market for orphan drugs has also experienced negative growth, which can be linked to a number of issues, including a drop in screening services, a decrease in access to specialists, treatment interruptions, restricted operations in the majority of industries, insufficient funding for academic institutions and research, the temporary closure of significant academic institutions, supply chain disruptions, and difficulties delivering necessary or post-sales services.

The oncological diseases segment is expected to be the largest during the forecast period

The oncological diseases segment is estimated to hold the largest share. Orphan drugs play a crucial role in the treatment of oncological diseases. A medication created especially to treat uncommon diseases that only affect a small number of patients is known as an orphan drug. Certain subtypes or mutations within a cancer type can be classified as rare diseases, even though individual cancer types might not be. These orphan medications target particular genetic defects or molecular pathways that contribute to the development and spread of cancer. Due to favourable regulatory incentives, a shift towards precision medicine, and the rising prevalence of rare cancer subtypes, the market for orphan drugs for oncological diseases has experienced significant growth.

The biologics segment is expected to have the highest CAGR during the forecast period

The biologics segment is anticipated to have lucrative growth during the forecast period. Biologics can be created to target the underlying genetic abnormalities or molecular pathways that cause the disease in the case of orphan diseases, where the patient population is small. This focused approach lowers possible side effects and improves treatment efficacy. One sort of biologic that has been developed is the monoclonal antibody. It binds specifically to certain proteins on cancer cells, causing the immune system to attack the tumor. The orphan drug market has witnessed a growing number of biologic drugs being developed and approved for rare diseases, including rare forms of cancer. 

Region with largest share:

Asia Pacific commanded the largest market share during the extrapolated period owing to the growing infrastructure of industries, rising disposable incomes, and favourable government policies in the region. Furthermore, clinical trial growth in Asia-Pacific is anticipated to accelerate beyond that of the United States and Europe, contributing to the region's market expansion. Moreover, it is anticipated that an increase in government initiatives to expand the number of oncology and community health centers will broaden the availability of orphan medications in developing nations.

Region with highest CAGR:

North America is expected to witness profitable growth over the projection period, due to the presence of major market players and a large population of patients with orphan diseases. Additionally, a growing number of orphan product approvals, a sizable number of orphan drugs in the development stage, and rising orphan drug spending in the area are the other factors driving the orphan drug market in the North American region.

Key players in the market

Some of the key players in the Orphan Drugs Market include Bayer AG, Novartis AG, F. Hoffmann-La Roche Ltd, Bristol-Myers Squibb Company, Amgen Inc., Novo Nordisk A/S, Alexion Pharmaceuticals Inc., GlaxoSmithKline plc, AstraZeneca, Celgene Corporation, Mylan, Biogen, Merck KGaA, Takeda Pharmaceutical Company Limited, Eli Lilly and Company and Shire Pharmaceutical.

Key Developments:

In March 2023, F. Hoffmann-La Roche Ltd: Roche announces collaboration with Lilly to enhance early diagnosis of Alzheimer's disease.

In January 2022, Amgen and Generate Biomedicines announced a research collaboration agreement to discover and create protein therapeutics for five clinical targets across several therapeutic areas and multiple modalities.

Disease Types Covered:
• Oncological Diseases
• Metabolic Diseases
• Hematologic & Immunologic Diseases
• Infectious diseases
• Neurological diseases

Drug Types Covered:
• Non-Biologics
• Biologics

Sales Covered:
• Prescribed
• Generics

Drugs Covered:
• Rituxan 
• Revlimid 
• Imbruvica 
• Opdivo 
• Darzalex 
• Pomalyst 
• Other Drugs 

Distribution Channels Covered:
• Online Pharmacies  
• Hospital Pharmacies  
• Retail Pharmacies  
• Other Distribution Channels  

Regions Covered:
• North America
o US
o Canada
o Mexico
• Europe
o Germany
o UK
o Italy
o France
o Spain
o Rest of Europe
• Asia Pacific
o Japan        
o China        
o India        
o Australia  
o New Zealand
o South Korea
o Rest of Asia Pacific    
• South America
o Argentina
o Brazil
o Chile
o Rest of South America
• Middle East & Africa 
o Saudi Arabia
o Qatar
o South Africa
o Rest of Middle East & Africa

What our report offers:
- Market share assessments for the regional and country-level segments
- Strategic recommendations for the new entrants
- Covers Market data for the years 2021, 2022, 2023, 2026, and 2030
- Market Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, and recommendations)
- Strategic recommendations in key business segments based on the market estimations
- Competitive landscaping mapping the key common trends
- Company profiling with detailed strategies, financials, and recent developments
- Supply chain trends mapping the latest technological advancements

Free Customization Offerings: 
All the customers of this report will be entitled to receive one of the following free customization options:
• Company Profiling
o Comprehensive profiling of additional market players (up to 3)
o SWOT Analysis of key players (up to 3)
• Regional Segmentation
o Market estimations, Forecasts and CAGR of any prominent country as per the client's interest (Note: Depends on feasibility check)
• Competitive Benchmarking
o Benchmarking of key players based on product portfolio, geographical presence, and strategic alliances

Table of Contents

1 Executive Summary           
2 Preface           
 2.1 Abstract          
 2.2 Stake Holders          
 2.3 Research Scope          
 2.4 Research Methodology          
  2.4.1 Data Mining         
  2.4.2 Data Analysis         
  2.4.3 Data Validation         
  2.4.4 Research Approach         
 2.5 Research Sources          
  2.5.1 Primary Research Sources         
  2.5.2 Secondary Research Sources         
  2.5.3 Assumptions         
3 Market Trend Analysis           
 3.1 Introduction          
 3.2 Drivers          
 3.3 Restraints          
 3.4 Opportunities          
 3.5 Threats          
 3.6 Emerging Markets          
 3.7 Impact of Covid-19          
4 Porters Five Force Analysis           
 4.1 Bargaining power of suppliers          
 4.2 Bargaining power of buyers          
 4.3 Threat of substitutes          
 4.4 Threat of new entrants          
 4.5 Competitive rivalry          
5 Global Orphan Drugs Market, By Disease Type           
 5.1 Introduction          
 5.2 Oncological Diseases          
  5.2.1 Pancreatic Cancer         
  5.2.2 Acute Myeloid Leukemia         
  5.2.3 Multiple Myeloma         
  5.2.4 Ovarian Cancer         
  5.2.5 Renal Cell Carcinoma         
 5.3 Metabolic Diseases          
  5.3.1 Hunter Syndrome         
  5.3.2 Gaucher Disease         
  5.3.3 Fabry Disease         
  5.3.4 Hypoparathyroidism         
 5.4 Hematologic & Immunologic Diseases          
  5.4.1 Hemophilia         
  5.4.2 Hereditary Angioedema         
 5.5 Infectious diseases          
 5.6 Neurological diseases          
  5.6.1 Huntington's Disease         
  5.6.2 Alzheimer's Disease         
6 Global Orphan Drugs Market, By Drug Type           
 6.1 Introduction          
 6.2 Non-Biologics          
 6.3 Biologics          
7 Global Orphan Drugs Market, By Sale           
 7.1 Introduction          
 7.2 Prescribed          
 7.3 Generics          
8 Global Orphan Drugs Market, By Drug           
 8.1 Introduction          
 8.2 Rituxan          
 8.3 Revlimid          
 8.4 Imbruvica          
 8.5 Opdivo          
 8.6 Darzalex          
 8.7 Pomalyst          
 8.8 Other Drugs          
9 Global Orphan Drugs Market, By Distribution Channel           
 9.1 Introduction          
 9.2 Online Pharmacies          
 9.3 Hospital Pharmacies          
 9.4 Retail Pharmacies          
 9.5 Other Distribution Channels          
10 Global Orphan Drugs Market, By Geography           
 10.1 Introduction          
 10.2 North America          
  10.2.1 US         
  10.2.2 Canada         
  10.2.3 Mexico         
 10.3 Europe          
  10.3.1 Germany         
  10.3.2 UK         
  10.3.3 Italy         
  10.3.4 France         
  10.3.5 Spain         
  10.3.6 Rest of Europe         
 10.4 Asia Pacific          
  10.4.1 Japan         
  10.4.2 China         
  10.4.3 India         
  10.4.4 Australia         
  10.4.5 New Zealand         
  10.4.6 South Korea         
  10.4.7 Rest of Asia Pacific         
 10.5 South America          
  10.5.1 Argentina         
  10.5.2 Brazil         
  10.5.3 Chile         
  10.5.4 Rest of South America         
 10.6 Middle East & Africa          
  10.6.1 Saudi Arabia         
  10.6.2 UAE         
  10.6.3 Qatar         
  10.6.4 South Africa         
  10.6.5 Rest of Middle East & Africa         
11 Key Developments           
 11.1 Agreements, Partnerships, Collaborations and Joint Ventures          
 11.2 Acquisitions & Mergers          
 11.3 New Product Launch          
 11.4 Expansions          
 11.5 Other Key Strategies          
12 Company Profiling           
 12.1 Bayer AG          
 12.2 Novartis AG          
 12.3 F. Hoffmann-La Roche Ltd          
 12.4 Bristol-Myers Squibb Company          
 12.5 Amgen Inc.          
 12.6 Novo Nordisk A/S          
 12.7 Alexion Pharmaceuticals Inc.          
 12.8 GlaxoSmithKline plc          
 12.9 AstraZeneca          
 12.10 Celgene Corporation          
 12.11 Mylan          
 12.12 Biogen          
 12.13 Merck KGaA          
 12.14 Takeda Pharmaceutical Company Limited          
 12.15 Eli Lilly and Company          
 12.16 Shire Pharmaceutical          
List of Tables            
1 Global Orphan Drugs Market Outlook, By Region (2021-2030) ($MN)           
2 Global Orphan Drugs Market Outlook, By Disease Type (2021-2030) ($MN)           
3 Global Orphan Drugs Market Outlook, By Oncological Diseases (2021-2030) ($MN)           
4 Global Orphan Drugs Market Outlook, By Pancreatic Cancer (2021-2030) ($MN)           
5 Global Orphan Drugs Market Outlook, By Acute Myeloid Leukemia (2021-2030) ($MN)           
6 Global Orphan Drugs Market Outlook, By Multiple Myeloma (2021-2030) ($MN)           
7 Global Orphan Drugs Market Outlook, By Ovarian Cancer (2021-2030) ($MN)           
8 Global Orphan Drugs Market Outlook, By Renal Cell Carcinoma (2021-2030) ($MN)           
9 Global Orphan Drugs Market Outlook, By Metabolic Diseases (2021-2030) ($MN)           
10 Global Orphan Drugs Market Outlook, By Hunter Syndrome (2021-2030) ($MN)           
11 Global Orphan Drugs Market Outlook, By Gaucher Disease (2021-2030) ($MN)           
12 Global Orphan Drugs Market Outlook, By Fabry Disease (2021-2030) ($MN)           
13 Global Orphan Drugs Market Outlook, By Hypoparathyroidism (2021-2030) ($MN)           
14 Global Orphan Drugs Market Outlook, By Hematologic & Immunologic Diseases (2021-2030) ($MN)           
15 Global Orphan Drugs Market Outlook, By Hemophilia (2021-2030) ($MN)           
16 Global Orphan Drugs Market Outlook, By Hereditary Angioedema (2021-2030) ($MN)           
17 Global Orphan Drugs Market Outlook, By Infectious diseases (2021-2030) ($MN)           
18 Global Orphan Drugs Market Outlook, By Neurological diseases (2021-2030) ($MN)           
19 Global Orphan Drugs Market Outlook, By Huntington's Disease (2021-2030) ($MN)           
20 Global Orphan Drugs Market Outlook, By Alzheimer's Disease (2021-2030) ($MN)           
21 Global Orphan Drugs Market Outlook, By Drug Type (2021-2030) ($MN)           
22 Global Orphan Drugs Market Outlook, By Non-Biologics (2021-2030) ($MN)           
23 Global Orphan Drugs Market Outlook, By Biologics (2021-2030) ($MN)           
24 Global Orphan Drugs Market Outlook, By Sale (2021-2030) ($MN)           
25 Global Orphan Drugs Market Outlook, By Prescribed (2021-2030) ($MN)           
26 Global Orphan Drugs Market Outlook, By Generics (2021-2030) ($MN)           
27 Global Orphan Drugs Market Outlook, By Drug (2021-2030) ($MN)           
28 Global Orphan Drugs Market Outlook, By Rituxan (2021-2030) ($MN)           
29 Global Orphan Drugs Market Outlook, By Revlimid (2021-2030) ($MN)           
30 Global Orphan Drugs Market Outlook, By Imbruvica (2021-2030) ($MN)           
31 Global Orphan Drugs Market Outlook, By Opdivo (2021-2030) ($MN)           
32 Global Orphan Drugs Market Outlook, By Darzalex (2021-2030) ($MN)           
33 Global Orphan Drugs Market Outlook, By Pomalyst (2021-2030) ($MN)           
34 Global Orphan Drugs Market Outlook, By Other Drugs (2021-2030) ($MN)           
35 Global Orphan Drugs Market Outlook, By Distribution Channel (2021-2030) ($MN)           
36 Global Orphan Drugs Market Outlook, By Online Pharmacies (2021-2030) ($MN)           
37 Global Orphan Drugs Market Outlook, By Hospital Pharmacies (2021-2030) ($MN)           
38 Global Orphan Drugs Market Outlook, By Retail Pharmacies (2021-2030) ($MN)           
39 Global Orphan Drugs Market Outlook, By Other Distribution Channels (2021-2030) ($MN)           
Note: Tables for North America, Europe, APAC, South America, and Middle East & Africa Regions are also represented in the same manner as above.            

List of Figures


Research Methodology

We at Stratistics opt for an extensive research approach which involves data mining, data validation, and data analysis. The various research sources include in-house repository, secondary research, competitor’s sources, social media research, client internal data, and primary research.

Our team of analysts prefers the most reliable and authenticated data sources in order to perform the comprehensive literature search. With access to most of the authenticated data bases our team highly considers the best mix of information through various sources to obtain extensive and accurate analysis.

Each report takes an average time of a month and a team of 4 industry analysts. The time may vary depending on the scope and data availability of the desired market report. The various parameters used in the market assessment are standardized in order to enhance the data accuracy.

Data Mining

The data is collected from several authenticated, reliable, paid and unpaid sources and is filtered depending on the scope & objective of the research. Our reports repository acts as an added advantage in this procedure. Data gathering from the raw material suppliers, distributors and the manufacturers is performed on a regular basis, this helps in the comprehensive understanding of the products value chain. Apart from the above mentioned sources the data is also collected from the industry consultants to ensure the objective of the study is in the right direction.

Market trends such as technological advancements, regulatory affairs, market dynamics (Drivers, Restraints, Opportunities and Challenges) are obtained from scientific journals, market related national & international associations and organizations.

Data Analysis

From the data that is collected depending on the scope & objective of the research the data is subjected for the analysis. The critical steps that we follow for the data analysis include:

  • Product Lifecycle Analysis
  • Competitor analysis
  • Risk analysis
  • Porters Analysis
  • PESTEL Analysis
  • SWOT Analysis

The data engineering is performed by the core industry experts considering both the Marketing Mix Modeling and the Demand Forecasting. The marketing mix modeling makes use of multiple-regression techniques to predict the optimal mix of marketing variables. Regression factor is based on a number of variables and how they relate to an outcome such as sales or profits.

Data Validation

The data validation is performed by the exhaustive primary research from the expert interviews. This includes telephonic interviews, focus groups, face to face interviews, and questionnaires to validate our research from all aspects. The industry experts we approach come from the leading firms, involved in the supply chain ranging from the suppliers, distributors to the manufacturers and consumers so as to ensure an unbiased analysis.

We are in touch with more than 15,000 industry experts with the right mix of consultants, CEO's, presidents, vice presidents, managers, experts from both supply side and demand side, executives and so on.

The data validation involves the primary research from the industry experts belonging to:

  • Leading Companies
  • Suppliers & Distributors
  • Manufacturers
  • Consumers
  • Industry/Strategic Consultants

Apart from the data validation the primary research also helps in performing the fill gap research, i.e. providing solutions for the unmet needs of the research which helps in enhancing the reports quality.

For more details about research methodology, kindly write to us at info@strategymrc.com

Frequently Asked Questions

In case of any queries regarding this report, you can contact the customer service by filing the “Inquiry Before Buy” form available on the right hand side. You may also contact us through email: info@strategymrc.com or phone: +1-301-202-5929

Yes, the samples are available for all the published reports. You can request them by filling the “Request Sample” option available in this page.

Yes, you can request a sample with your specific requirements. All the customized samples will be provided as per the requirement with the real data masked.

All our reports are available in Digital PDF format. In case if you require them in any other formats, such as PPT, Excel etc you can submit a request through “Inquiry Before Buy” form available on the right hand side. You may also contact us through email: info@strategymrc.com or phone: +1-301-202-5929

We offer a free 15% customization with every purchase. This requirement can be fulfilled for both pre and post sale. You may send your customization requirements through email at info@strategymrc.com or call us on +1-301-202-5929.

We have 3 different licensing options available in electronic format.

  • Single User Licence: Allows one person, typically the buyer, to have access to the ordered product. The ordered product cannot be distributed to anyone else.
  • 2-5 User Licence: Allows the ordered product to be shared among a maximum of 5 people within your organisation.
  • Corporate License: Allows the product to be shared among all employees of your organisation regardless of their geographical location.

All our reports are typically be emailed to you as an attachment.

To order any available report you need to register on our website. The payment can be made either through CCAvenue or PayPal payments gateways which accept all international cards.

We extend our support to 6 months post sale. A post sale customization is also provided to cover your unmet needs in the report.

Request Customization

We provide a free 15% customization on every purchase. This requirement can be fulfilled for both pre and post sale. You may send your customization requirements through email at info@strategymrc.com or call us on +1-301-202-5929.

Note: This customization is absolutely free until it falls under the 15% bracket. If your requirement exceeds this a feasibility check will be performed. Post that, a quote will be provided along with the timelines.


Assured Quality

Assured Quality

Best in class reports with high standard of research integrity

24X7 Research Support

24X7 Research Support

Continuous support to ensure the best customer experience.

Free Customization

Free Customization

Adding more values to your product of interest.

Safe and Secure Access

Safe & Secure Access

Providing a secured environment for all online transactions.

Trusted by 600+ Brands

Trusted by 600+ Brands

Serving the most reputed brands across the world.