Orphan Drugs are pharmaceutical products that treat a rare medical condition or disease. The development of orphan drugs has been financially incentivized through U.S. law via the Orphan Drug Act of 1983.
The Global Orphan Drugs market was worth $XX Billion in 2017 and is forecasted to reach $323.4 Billion by 2025, growing at a CAGR of 11.5% during the forecast period (2018-2025).
Global Orphan Drugs Market 2016-2025, in USD Billion
The National Organization for Rare Disorders (NORD), which was instrumental in establishing the Act, currently estimates that there are as many as 7,000 rare diseases and that up to 30 Billion Americans suffer from a rare disease. The success of the original Orphan Drug Act in the USA led to it being adopted in other key markets, most notably in Japan in 1993 and the European Union in 2000.
Global Orphan Drugs Market – Market Dynamics
Incentives for developing orphan drugs
The financial incentives and overall rewards for the successful development of drugs for the treatment and diagnosis of rare diseases and conditions are significant, and as such companies are motivated to design commercially viable end-products to obtain such benefits. For instance, the FDA Office of Orphan Products Development (OOPD) in the U.S. evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare disease and to further advance scientific development of such promising medical products. The committee also offers incentives for sponsors enabling the development and marketing of drugs, biologics, and medical devices for rare diseases. The rights for market exclusivity after the designation of orphan drugs would be extended to a period of seven years in the U.S., ten years in Europe, and ten years in Japan, further propelling the growth of the market. The companies are thus focused on entering the market. For instance, in June 2018, Cardax, Inc. announced that it engaged industry veteran and orphan drug expert, Frederick D. Sancilio, Ph.D., to launch the Company's orphan drug development program.
High per Patient Treatment Cost
The high initial investments result in higher per patient treatment cost, acting as one of the significant barriers to market entry for the orphan drugs market. The mean orphan drugs treatment cost for a rare disease per patient per year is estimated to be $147,308 in the U.S. in 2017, which has witnessed a growth of 1.4% when compared to the previous year. The cost of development of drugs is also significantly higher, requiring a continued influx of cash to remain commercially competitive and relevant in the overall market scenario.
Global Orphan Drugs Market – Segment Analysis
The global orphan drugs market is segmented by type into biological and non-biological orphan drugs. The biological segment is leading by market share owing to its precise clinical targeting, which would result in better outcomes and fewer toxic side-effects compared to existing drugs. The companies are thus focused on obtaining orphan drug development for commercial products to increase company revenue. For instance, in February 2018, Yisheng Biopharma Co., Ltd. announced that the U.S. Food and Drug Administration (FDA) had granted orphan drug designation (ODD) for its lead immuno-oncology candidate, YS-ON-001, for the treatment of pancreatic cancer.
The market is further categorized by therapy into oncology, hematology diseases, central nervous system, cardiovascular, endocrine, respiratory, immunomodulators, gastro-intestinal, musculoskeletal systemic anti-infectives, dermatology, and others. The hematology diseases orphan drug segment is dominating the market scenario owing to the prevalence of blood-related disorders such as atypical hemolytic uremic syndrome (aHUS), beta-thalassemia (sickle cell beta-thalassemia), and paroxysmal nocturnal hemoglobinuria. For instance, in March 2018, ADDMEDICA has announced that the US Food and Drug Administration (FDA) has approved Siklos® (hydroxyurea tablets) in pediatric patients, two years of age and older, suffering from sickle cell anemia. Approval was obtained under a priority review and orphan drug designation.
Global Orphan Drugs Market – Geographical Analysis
Global Orphan Drugs Market Share (%), 2017
North America 45.54%
Asia Pacific 18.78%
South America 7.22%
The global orphan drugs market is segmented geographically by region into North America, South America, Europe, Asia-Pacific, and Rest of the World (RoW).
North America accounted for the largest share in the global orphan drugs market, with the U.S. contributing a significant portion to the overall regional growth.
The increase in government incentives coupled with high demand for end-products are the primary factors that are positively influencing the market growth in North America. The extended period of seven years of market exclusivity permitted after orphan drug designation have further driven the growth of the market. The North America-based companies are focused on obtaining drug designation for proprietary products to increase market share. For instance, in October 2018, AstraZeneca and Merck announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) for LYNPARZA for the treatment of pancreatic cancer.
Global Orphan Drugs Market –Competitive Analysis
The market for orphan drugs is moderately competitive, with the development and commercial launch of end-products still at a nascent stage. The key players of the market include Celgene Corporation, Johnson & Johnson, Novartis AG, Roche Holding AG, and Merck & Co., Inc. The companies focus on obtaining drug designation for proprietary products to increase market share. For instance, in October 2018, AstraZeneca and Merck announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) for LYNPARZA for the treatment of pancreatic cancer.
• The biological segment is leading by market share owing to its precise clinical targeting, which would result in better outcomes and fewer toxic side-effects compared to existing drugs.
• North America accounted for the largest share in the global orphan drugs market, with the U.S. contributing a significant portion to the overall regional growth. The increase in government incentives coupled with high demand for end-products are the primary factors that are positively influencing the market growth in North America. The extended period of seven years of market exclusivity permitted after orphan drug designation have further driven the growth of the market.
• The companies are focused on the launch of new products to strengthen market position and increase company revenue. For instance, in January 2018, Astellas Pharma Inc. announced that the European Commission (EC) had issued Orphan Designation to gilteritinib for the treatment of patients with acute myeloid leukemia (AML).
Scope of the Report
The report covers the factors impacting the market, Porter 5 Forces, Competitive Scenario, Product Benchmarking, and company profiles. Orphan Drugs Market is segmented by product type biological and non-biological orphan drugs. By therapy area, the global orphan drugs market is segmented into oncology, hematology diseases, central nervous system, cardiovascular, endocrine, respiratory, immunomodulators, gastro-intestinal, musculoskeletal, systemic, anti-infectives, dermatology, and others. Geographically, the market is segmented into North America, South America, Europe, Asia Pacific and Rest of the World.
Why Purchase the Report?
• Visualize the composition of the Orphan Drugs market across each indication, regarding type and treatment options, highlighting the vital commercial assets and players.
• Identify commercial opportunities in Orphan Drugs market by analyzing trends and co-development deals.
• Excel data sheet with thousands of data points of the Orphan Drugs market - level 4/5 segmentation.
• PDF report with the most relevant analysis cogently put together after exhaustive qualitative interviews and in-depth market study.
• Product mapping in excel for the principal products of all major market players
• Equipment Suppliers/ Buyers
• Service Providers/ Buyers
• Industry Investors/Investment Bankers
• Education & Research Institutes
• Research Professionals
• Emerging Companies
Chapter 1 Methodology and Scope
1.1 Research methodology
1.2 Scope of the Report
Chapter 2 Global Orphan Drugs Market– Trends
2.1 Key Trends & Developments
Chapter 3 Global Orphan Drugs Market – Industry Analysis
3.1. Industry Impact Factors
3.1.1. Market Drivers
3.1.2. Market Restraints
3.1.3. Market Opportunities
3.1.4. Impact Analysis
3.2. Pipeline Products
3.3. Pricing Analysis
3.4. Regulatory Analysis
3.5. Technology Development
3.6. Porter 5 Forces
3.7. Major Marketed Products
Chapter 4 Global Orphan Drugs Market- Product Analysis
4.1. By Product Type
4.2. By Therapy Area
4.2.2. Hematology Diseases
4.2.3. Central Nervous System
4.2.10. Systemic Anti-Infectives
Chapter 5 Global Orphan Drugs Market- Geographical Analysis
5.1. North America
5.2. South America
5.2.3. Rest of South America
5.3.2. United Kingdom
5.3.4. Rest of Europe
5.4. Asia Pacific
5.4.5. Rest of Asia-Pacific
Chapter 6 Global Orphan Drugs Market- Competitive Landscape
6.1. Competitive scenario
6.2. Key Strategies adopted by Manufacturers
6.3. Product Benchmarking
Chapter 7 Global Orphan Drugs Market- Company Profiles
7.1. AbbVie Inc.
7.2. Alexion Pharmaceuticals, Inc.
7.3. Bristol-Myers Squibb Company
7.4. Celgene Corporation
7.5. Johnson & Johnson
7.6. Merck & Co., Inc.
7.7. Novartis AG
7.8. Roche Holding AG
7.9. Sanofi SA
7.10. Shire plc
Chapter 8 Global Orphan Drugs Market- Appendix