Global Orphan Drugs Market Size, Trends, and Growth Opportunity, By Therapy Area (Neurology and Hematology), By Drug Type (Biologics and Non-Biologics), By Distribution Channel (Hospital Pharmacy and Online pharmacy), By Regional and Forecast to 2030  

Global Orphan Drugs Market 

The Global Orphan Drugs Market was valued at USD 168.32 billion in 2022 and is expected to reach USD 433.59 billion by 2030, at a CAGR 15.49 % during the forecast period 2023- 2030.

Pharmaceuticals used to detect and treat unusual medical conditions are known as orphan drugs. These drugs often have a tiny market since they are made for a small patient population and are created to meet a particular public health need.

Many oncological, metabolic, hematologic, chronic, progressive, viral, and neurological conditions can be successfully treated with them. Lymphoma, glioma, leukemia, cystic fibrosis, ovarian cancer, immunologic, multiple myeloma, and renal cell carcinoma are all life-threatening, degenerative, and disabling illnesses that call for certain treatment methods that are efficient for their particular symptoms.

Market Drivers

The increasing prevalence of the rare disease is anticipated to drive market growth during the forecast period. Numerous emerging and developed countries enacted legislation to ensure that these drugs are freely accessible to patients and to support the development of orphan medications. Additionally, FDA approvals for several cancer-related orphan pharmaceuticals are likely to result in greater commercialization of these treatments, particularly in the oncology therapy field, anticipated to boost the market growth throughout the projection period. 

Furthermore, higher demand for immunomodulators, which normalize or augment the immune system, will drive market expansion. The rising prescription prices as well as an increase in the prevalence of infectious disorders in both developed and developing countries are expected to drive demand for immunomodulators, which will aid the orphan drugs market to grow throughout the forecast period.

Market Restraints

High Product, as well as developing costs of drugs, are anticipated to hamper the market growth. Due to the small patient populations and specialized research and development needed, developing and producing orphan medications is expensive.

Additionally, regulatory approval and reimbursement issues: Orphan medications may have difficulty receiving regulatory approval and funding, which raises their already high development costs and can restrain market expansion.

COVID-19 Impact

Due to the postponement of non-life-threatening diagnosis and treatment, the effect of the COVID-19 pandemic on particular pharmaceutical markets is anticipated to be larger. Given that the pandemic has received increased attention internationally in terms of funding and healthcare resources, this is particularly true for those who suffer from rare diseases. Patients with rare diseases are expected to be significantly impacted by this, as a lack of diagnostic and consulting services from medical professionals may delay the prompt prescription of orphan drugs. The scarcity of pharmaceuticals caused several enterprises to close their doors.

Market Segmentation

Global Orphan Drugs Market is segmented into Therapy Area, Drug Type, and Distribution Channel.

 Based Therapy Area market is segmented into Neurology and Hematology

Based on Drug Type market is divided into Biologics and Non-Biologics

Based on the Distribution Channel market is segmented into Hospital pharmacy and Online pharmacy

Regional Analysis

Global Orphan Drug Market is segmented into five regions such as North America, Latin America, Europe, Asia Pacific, and Middle East & Africa.

North America is estimated to dominate the Orphan Drug Market during the forecast period. The United States has the greatest market share in North America. The fact that a drug with orphan drug classification in the US obtains seven years of marketing exclusivity after FDA clearance for tax credits, particular indications, and user fee waivers is one of the factors driving market expansion.

Moreover, according to statistics revised in May 2022, 3.1 million Canadians and around 30 million Americans have rare diseases, by the Genetic and Rare Diseases Information Center (GARD) and the Canadian Organization for Rare Disorders (CORD) 2021 update. With research grants and subsidies, the government has assisted patients with uncommon diseases, due to the increasing occurrence of a rare diseases in this region. All the factors anticipated to propel the market growth in the coming years.

Key Development

In March 2022, CER-001, created by ABIONYX Pharma, was granted FDA Orphan Drugs Designation (ODD) for the treatment of LCAT deficiency that presents as kidney disease and/or ophthalmologic sickness.

Key Players

Various key players are discussed in the Global Orphan Drug Market Report including: Celgene Corporation, Takeda Pharmaceutical Company Limited, Novartis AG, Johnson & Johnson, and F. Hoffmann-La Roche Ltd.

Market Taxonomy

By Therapy Area 

• Neurology
• Hematology

By Drug Type 
• Biologics 
• Non-Biologics

By Distribution Channel 

• Hospital pharmacy
• Online Pharmacy

By Region

• North America
• Latin America
• Europe
• Asia Pacific
• Middle East & Africa
 

Global Orphan Drugs Market
1 Introduction 
1.1 Objective of the Study
1.2 Market definition
1.3 Market Scope
2 Research Methodology
2.1 Data Mining
2.2 Validation
2.3 Primary Interviews
2.4 List of Data Sources
3 Executive Summary
4 Global Orphan Drugs Market Outlook
4.1 Overview
4.2 Market Dynamics
4.2.1 Drivers
4.2.2 Restraints
4.2.3 Opportunities
4.2.4 Cumulative Impact due to recent Energy Crisis
4.2.5 Cumulative Impact due to nearing economic downturn 
4.2.6 Post covid-19 world Supply & Demand conditions
4.2.7 Cumulative Impact of Russia-Ukraine Conflict
4.3 Porters Five Force Model
4.4 Value Chain Analysis
5 Global Orphan Drugs Market, By Therapy Area 
5.1 Y-o-Y Growth Comparison, By Therapy Area  
5.2 Global Orphan Drugs Market Share Analysis, By Therapy Area  
5.3 Global Orphan Drugs Market Size and Forecast, By Therapy Area 
5.3.1. Neurology 
5.3.2. Hematology
6 Global Orphan Drugs Market, By Drug Type
6.1 Y-o-Y Growth Comparison, By Drug Type
6.2 Global Orphan Drugs Market Share Analysis, By Drug Type
6.3 Global Orphan Drugs Market Size and Forecast, By Drug Type
6.3.1 Biologics 
6.3.2. Non-Biologics
7 Global Orphan Drugs Market, By Distribution Channel
7.1 Y-o-Y Growth Comparison, By Distribution Channel
7.2 Global Orphan Drugs Market Share Analysis, By Distribution Channel
7.3 Global Orphan Drugs Market Size and Forecast, By Distribution Channel
7.3.1. Hospital pharmacy 
7.3.2. Online pharmacy
8 Global Orphan Drugs Market, By Region
8.1 Global Orphan Drugs Market Share Analysis, By Region
8.2 Global Orphan Drugs Market Size and Forecast, By Region
9 North America Orphan Drugs Market Analysis and Forecast (2023-2027)
9.1 Introduction
9.2 North America Orphan Drugs Market Share Analysis, By Therapy Area  
9.3 North America Orphan Drugs Market Size and Forecast, By Drug Type
9.4 North America Orphan Drugs Market Size and Forecast, By Distribution Channel
9.5 North America Orphan Drugs Market Size and Forecast, By Country
9.5.1 U.S.
9.5.2 Canada
9.5.3 Mexico
10 Europe Orphan Drugs Market Analysis and Forecast (2023-2030)
10.1 Introduction
10.2 Europe Orphan Drugs Market Share Analysis, By Therapy Area  
10.3 Europe Orphan Drugs Market Size and Forecast, By Drug Type
10.4 Europe Orphan Drugs Market Size and Forecast, By Distribution Channel
10.5 Europe Orphan Drugs Market Size and Forecast, By Country
10.5.1 Germany
10.5.2 France
10.5.3 UK
10.5.4. Rest of Europe
11 Asia Pacific Orphan Drugs Market Analysis and Forecast (2023-2030)
11.1 Introduction
11.2 Asia Pacific Orphan Drugs Market Share Analysis, By Therapy Area  
11.3 Asia Pacific Orphan Drugs Market Size and Forecast, By Drug Type
11.4 Asia Pacific Orphan Drugs Market Size and Forecast, By Distribution Channel
11.5 Asia Pacific Orphan Drugs Market Size and Forecast, By Country
11.5.1 China 
11.5.2 Japan
11.5.3 India
11.5.4. Rest of Asia Pacific
12 Latin America Orphan Drugs Market Analysis and Forecast (2023-2030)
12.1 Introduction
12.2 Latin America Orphan Drugs Market Share Analysis, By Therapy Area  
12.3 Latin America Orphan Drugs Market Size and Forecast, By Drug Type
12.4 Latin America Orphan Drugs Market Size and Forecast, By Distribution Channel
12.5 Latin America Orphan Drugs Market Size and Forecast, Country
12.5.1. Brazil
12.5.2. Rest of Latin America
13 Middle East Orphan Drugs Market Analysis and Forecast (2023-2030)
13.1 Introduction
13.2 Middle East Orphan Drugs Market Share Analysis, By Therapy Area  
13.3 Middle East Orphan Drugs Market Size and Forecast, By Drug Type
13.4 Middle East Orphan Drugs Market Size and Forecast, By Distribution Channel
13.5 Middle East Orphan Drugs Market Size and Forecast, By Country
13.6.1. Saudi Arabia
13.5.2. UAE
13.5.3. Egypt
13.5.4. Kuwait
13.5.5. South Africa
14 Competitive Analysis
14.1 Competition Dashboard
14.2 Market share Analysis of Top Vendors
14.3 Key Development Strategies
15Company Profiles
15.1 Celgene Corporation
15.1.1 Overview
15.1.2 Offerings
15.1.3 Key Financials
15.1.4 Business Segment & Geographic Overview
15.1.5 Key Market Developments
15.1.6 Key Strategies
15.2. Takeda Pharmaceutical Company Limited
15.2.1 Overview
15.2.2 Offerings
15.2.3 Key Financials
15.2.4 Business Segment & Geographic Overview
15.2.5 Key Market Developments
15.2.6 Key Strategies
15.3. Novartis AG
15.3.1 Overview
15.3.2 Offerings
15.3.3 Key Financials
15.3.4 Business Segment & Geographic Overview
15.3.5 Key Market Developments
15.3.6 Key Strategies
15.4 Johnson & Johnson
15.4.1 Overview
15.4.2 Offerings
15.4.3 Key Financials
15.4.4 Business Segment & Geographic Overview
15.4.5 Key Market Developments
15.4.6 Key Strategies
15.5 F. Hoffmann-La Roche
15.5.1 Overview
15.5.2 Offerings
15.5.3 Key Financials
15.5.4 Business Segment & Geographic Overview
15.5.5 Key Market Developments
15.5.6 Key Strategies