As per our report, the size of the global orphan drugs market is estimated to worth USD 132.61 billion in 2020. This market is expected to be growing at a CAGR of 7.86% and will reach USD 193.59 billion by 2025.
Orphan drugs are designed with specific conditions to treat rare diseases. Majority of the rare diseases comes through genetic disorders. The other significant possibilities to get affected by these diseases are bacteria, viruses and allergies. Coronavirus severely damages the world health system. The pandemic situation caused by this virus has helped the pharmaceutical industries to focus more on manufacturing drugs to fight against this virus. For instance, on, March 25th, Remdesivir, an antiviral viral drug introduced by Gilead Sciences, Inc. has received the Compassionate Use Program, which is an orphan drug status by the FDA. As of now, it is the top-ranked drug in the market to battle against Coronavirus.
According to a report published by the FDA, it has approved more than 600 drugs since the Orphan Drug Act in 1983. Among those, 14% of orphan drugs are accepted with additional non-orphan indications. The ODA act has gained acceptance from many countries and boosted the manufacturing activities of orphan drugs to treat the diseases related. This ODA act had come up with financial and non-financial considerations to assure availability and access to orphan drugs. Under this act, the government agencies are offering tax credits and subsidies in favour of the patient, and pharmaceutical companies which are showing a positive impact on the market.
Increasing incidences of rare diseases is a crucial driving factor for the market to grow eventually.
Over 30 million Americans are recognized with various rare diseases where 200,000 people are effecting with chronic or acute conditions such as cancer, autoimmune disorders and digestive disorders, among others. In the United States, biopharmaceutical companies focused on introducing innovative vaccines or drugs for rare diseases as there are no particular treatment options for some unique conditions. There are approximately 7000 rare diseases where only five diseases have specific treatments. Advancements in the genomic studies will help the researchers to develop promising medicines. Studies found that 80% of the origin of the rare disease genetically. In 2020, COVID-19 disease is a unique, challenging factor for the researchers to find out effective treatment procedure.
Additionally, the rising demand to increase the production rate of the orphan drugs to make the drugs readily available in the stores is propelling the growth rate of the market. Creating awareness towards the availability of various treatment procedures and prevalence to improve healthcare services in hospitals and clinics with quality drugs is expanding the size of the orphan drugs market. The growing tendency to develop novel drugs, the ever-increasing scale of health care centres along with pharmaceutical companies and rise in disposable income in developed and developing countries, are further accelerating the market for orphan drugs.
Stringent rules and regulations by the government in approving new developments in concern towards people's safety remains a significant challenging factor for the orphan drugs market players. Furthermore, the lack of complete knowledge over the manufacturing process with the latest formula is slowly hindering the growth rate of the market. Also, fluctuations in the availability of raw materials due to the increasing prices of final products is limiting the growth rate of the market. Continuous changes in the economic strategies by government bodies in various countries may also impact negatively on the demand of the global orphan drugs market.
Market Size Available
2019 to 2025
2020 to 2025
By Type, Therapeutic and Region
Various Analyses Covered
Global, Regional & Country Level Analysis, Segment-Level Analysis, Drivers, Restraints, Opportunities, Challenges, PESTLE Analysis, Porter's Five Forces Analysis, Competitive Landscape, Analyst Overview on Investment Opportunities
North America, Europe, Asia Pacific, Latin America, Middle East & Africa
This research report on the Global Orphan Drugs Market has been segmented & sub-segmented into the following categories and analyzed market size & forecast for each segment until 2024.
Based on drug type, the orphan drugs market segmented into
The biological orphan drugs segment is leading with the highest shares of the market from recent years. Rising different kind of rare diseases which can be curable with biological drugs and growing investments in the development of drugs for rare diseases by private companies are leveraging the demand for this segment.
The non-biological orphan drugs segment is to have slight growth rate in coming years. Rising prevalence for genomic studies and the rise in the number of people suffering from chronic diseases are accelerating the growth of the segment.
Orphan Drugs Market Analysis - By Therapeutic
Oncology is ruling with dominant shares of the market with the increasing cancer patients. Escalating expenditure on healthcare is fuelling the demand for this segment. Companies like Johnson & Johnson, Abbvie and Bristol-Myers Squibb have special emphasize to manufacture orphan drugs related to oncology segment. It has put the oncology in the leading position in this segment.
Neurology segment is to have highest CGAR in coming years with the rise in the incidences of neurological disorders across the world. Also, growing support from the Food and Drug Administration is to boost up the growth rate of the orphan drugs market. Haematology category is to have significant shares. Increasing population is also one of the factors impacting positively on the growth of the market.
Regionally, North America is holding the most significant shares of the market by owing to the adoption of advanced technologies. Also, the manufacturing of innovative drugs in very less time when compared with the regular drugs is leveraging the growth rate of the orphan drugs market in this region. The constant economic growth rate is fuelling the size of the market in North America.
U.S accounted for the largest share in the global Orphan Drugs market in 2019, as it spends nearly 20% of its GDP on health. The global market outlook is projected to be positive as more and more governments are adopting policies to encourage this sector, especially in developing countries.
Following North America, Europe is leading the share of the market with the growing prevalence of the novel drugs. Increasing incidences of rare diseases across the region are also one of the factors levelling up the demand of the market.
Asia Pacific to hit the highest CAGR in the coming years due to the rising population is enhancing the shares of the orphan drugs market. India and China are significant countries contributing the largest shares for the market growth. Launch of reimbursement schemes in favour of the ordinary people is also magnifying the demand of the market.
Presently, the market for orphan drugs represents only about 6% of the entire pharmaceutical market. An extremely conducive environment currently prevails for the growth of the orphan drugs market, thereby encouraging the entry of more companies into the market.
Top companies in the market
Johnson and Johnson, Novartis, Celgene and AbbVie are the global market leaders for orphan drugs. Companies like GlaxoSmithKline, Roche, Alexion, Sanofi, Bristol Myers Squibb, Pfizer, Vertex and Merck are also playing a promising role in the orphan drugs market.
Significant Developments in this market in the recent past
On February 25th 2019, Roche bought Spark Therapeutics. This deal helped Roche to make their presence more robust in the advanced therapies. On the other end, Spark Therapeutics was named as the first company in the United States to receive a gene therapy approval for their Luxturna. Luxturna is a therapy to treat rare inherited eye disease.
On January 16th 2019, an agreement between Medunik Canada and HRA pharma had got legal rights for marketing and distribution of an orphan drug named Lysodren which helps to treat inoperable adrenal cortical carcinomas.
On April 9th 2018, AveXis was purchased by Novartis. AveXis is a Biotechnology Company based out of the United States and develops therapies for rare disorders related to neurological genetics. AVXS-101 is the lead candidate of AveXis, is a gene-replacement therapy to treat a rare disease called SMA (spinal muscular atrophy)
On December 12th 2017, Aprecia Pharmaceuticals had a partnership with the Cycle Pharmaceuticals to introduce 3D printed orphan drugs in foreseen years.
On November 30th 2016, AbbVie, one of a leader in manufacturing the orphan drugs, has received the Orphan Drug Designation by the FDA. It was for their risankizumab, which is an investigational treatment for a rare disease called pediatric Crohn's disease.
1.1 Market Definition
1.2 Study Deliverables
1.3 Base Currency, Base Year and Forecast Periods
1.4 General Study Assumptions
2. Research Methodology
2.2 Research Phases
2.2.1 Secondary Research
2.2.2 Primary Research
2.2.3 Econometric Modelling
2.2.4 Expert Validation
2.3 Analysis Design
2.4 Study Timeline
3.1 Executive Summary
3.2 Key Inferences
4. Drivers, Restraints, Opportunities, and Challenges Analysis (DROC)
4.1 Market Drivers
4.2 Market Restraints
4.3 Key Challenges
4.4 Current Opportunities in the Market
5. Market Segmentation
5.1.3 Non Biological
5.1.4 Y-o-Y Growth Analysis, By Type
5.1.5 Market Attractiveness Analysis, By Type
5.1.6 Market Share Analysis, By Type
5.2.5 Y-o-Y Growth Analysis, By Therapeutic
5.2.6 Market Attractiveness Analysis, By Therapeutic
5.2.7 Market Share Analysis, By Therapeutic
6. Geographical Analysis
6.1.1 Regional Trends
6.1.2 Impact Analysis
6.1.3 Y-o-Y Growth Analysis
220.127.116.11 By Geographical Area
18.104.22.168 By Type
22.214.171.124 By Therapeutic
6.1.4 Market Attractiveness Analysis
126.96.36.199 By Geographical Area
188.8.131.52 By Type
184.108.40.206 By Therapeutic
6.1.5 Market Share Analysis
220.127.116.11 By Geographical Area
18.104.22.168 By Type
22.214.171.124 By Therapeutic
6.2 North America
6.1.2 United States
6.3.6 South Korea
6.5 Latin America
6.4.5 Rest of Latin America
6.6 Middle East & Africa
7.1 PESTLE analysis
7.2 Porter’s Five analysis
7.2.1 Bargaining Power of Suppliers
7.2.2 Bargaining Power of Consumers
7.2.3 Threat of New Entrants
7.2.4 Threat of Substitute Products and Services
7.2.5 Competitive Rivalry within the Industry
8.Market Leaders' Analysis
8.1.2 Product Analysis
8.1.3 Financial analysis
8.1.4 Recent Developments
8.1.5 SWOT analysis
8.1.6 Analyst View
8.6 Bristol Myers Squibb
9.1 Market share analysis
9.2 Merger and Acquisition Analysis
9.3 Agreements, collaborations and Joint Ventures
9.4 New Product Launches
10.Market Outlook and Investment Opportunities
a) List of Tables
b) List of Figures