Certified Global Research Member
Key Questions Answered
- Global Market Outlook
- In-depth analysis of global and regional trends
- Analyze and identify the major players in the market, their market share, key developments, etc.
- To understand the capability of the major players based on products offered, financials, and strategies.
- Identify disrupting products, companies, and trends.
- To identify opportunities in the market.
- Analyze the key challenges in the market.
- Analyze the regional penetration of players, products, and services in the market.
- Comparison of major players’ financial performance.
- Evaluate strategies adopted by major players.
- Recommendations
Why Choose Market Research Future?
- Vigorous research methodologies for specific market.
- Knowledge partners across the globe
- Large network of partner consultants.
- Ever-increasing/ Escalating data base with quarterly monitoring of various markets
- Trusted by fortune 500 companies/startups/ universities/organizations
- Large database of 5000+ markets reports.
- Effective and prompt pre- and post-sales support.
The population of orphan disease is characterized by different factors of the market that are unique, which makes it different from mainstream pharmaceutical markets. Orphan diseases or rare diseases affect few people in comparison to other more prevalent health conditions. Nonetheless, their rarity in number collectively affects a considerable part of the population and addressing them brings up both challenges and opportunities to pharmaceutical industry.
There is one major market factor that influences the orphan disease market which is limited patient population. The fact that there are no many patients suffering from these diseases leads to limited number of potential customers for drugs sold through orphan programs. This scarcity creates a specific market dynamics where companies have to think thoughtfully about how practical and financially desirable it would be to develop treatments for this type of illnesses. However, advances in genomic research and better awareness have helped improve diagnostics hence enabling earlier identification of patients with such diseases. As a result, this has increased the size of the prospective market thereby leading many pharmaceutical companies investing heavily on research and development activities targeted at manufacturing drugs for orphans.
Another significant factor shaping the orphan disease market is regulatory incentives. Governments worldwide acknowledge difficulties surrounding development of treatments for rare diseases and therefore, have implemented several regulations that encourage involvement of pharmaceutical companies in this field. Some examples include prolonged exclusivity period tax credits as well as reduced regulatory fees among others; objectives being lessening financial risks associated with developing drugs for small number of patients while encouraging innovations within orphan space.
Additionally, accelerated approval pathways established by regulatory agencies also influence orphan disease markets. Regulatory bodies like FDA and EMA have launched more rapid approval systems for these types of medications due to recognition for immediate need for drugs treating rare disorders. Such an approach allows faster entry into the market place thereby reducing time spent on drug development process as well as costs involved in acquiring necessary permissions. Companies can thus bring out their products sooner concerning orphaned children’s needs hence potentially gaining competitive edge.
Collaboration and partnerships play a pivotal role in the orphan disease market. Collaboration between pharmaceutical companies, research institutions and patient advocacy groups is essential for success given the unique nature of orphan diseases. Joint efforts such as those among partners on pooling their resources may assist in accelerating the development of orphan drugs by sharing expertise and data. Additionally, collaboration with patient advocacy groups helps ensure that the unique perspectives and needs of the affected communities are considered throughout the drug development process.
Market access and reimbursement strategies pose significant challenges in the orphan disease market. Pricing and reimbursement negotiations become critical due to high costs associated with orphan drug development and limited patient population. Pharmaceutical companies have to go through these hurdles to ensure that they get their products into market while still remaining viable in terms of finances. As a result, different models of pricing have been developed as well as use outcome-based agreements among other strategies that engage payers to sort out this complexity.”
Orphan Diseases Market Highlights:
Global Orphan Diseases Market Overview
The Orphan diseases market size is anticipated to reach USD 328.5 billion by 2030 at 10.70% of CAGR during the forecast timeframe 2024-2030. Orphan diseases are also popularly called rare diseases. The market for the orphan disease is growing rapidly in the forecast period. This disease affects a small percentage of the population across the World. Research says that most orphan diseases remain with the patient throughout life as it is genetic. The exact number of diseases are not found yet, but there are nearly more than 7000 various rare disorders and diseases are present across the World.
The increasing patient population with a rare disease contributes to the global market throughout the review period. Moreover, the research and development initiatives are increasing day by day, fuelling the worldwide market growth. Also, the increasing adoption of orphan drugs is enhancing the market growth.
According to the Orphan Diseases Market Trends report, the current market has some intended audiences who contribute to generating more scope and opportunities for the global market. These intended audiences are hospitals and clinics, medical research laboratories, orphan diseases drug manufacturers and suppliers, potential investors, government and independent regulatory authorities, market research and consulting service providers, and research and development (R&D) companies.
COVID-19 Analysis
In the health care sector, the workflow has been affected due to the arrival of the Covid-19 pandemic. At this time, many sub-domains of healthcare industries have shut their doors for a temporary period.
Also, the global Orphan Diseases Market Value has decreased with the reduced access to healthcare specialists, limited operations in most industries, the decline in screening services, and inadequate funding.
Orphan Diseases Market Trends
Key Market Drivers
The global Orphan Diseases Market growth is driven due to the increasing prevalence of rare diseases across the World. Another important factor that enhances market growth is favorable government policies.
The US food and Drug Administration (US FDA) has estimated that since 1983, the Orphan Products Development (OOPD) organization has resulted in over 600 drugs and biologic products development and marketing for rare disorders. These kinds of initiatives are growing day-by-day that is fuelling the growth of the market during the assessment period.
Market Challenges
The Orphan Diseases Market might face challenges due to the high cost of the orphan drug and the stringent rules and regulations of the government relating to the orphan drug.
Moreover, the manufacturing chain and supply chain have been affected during the pandemic period, which has brought major challenges for the global market due to the lack of transportation, workforce, etc.
Market Opportunities
The growing novel indications for known orphan drugs are expected to bring lucrative opportunities for the global market players and manufacturers.
Moreover, the market might go through significant opportunities due to the untapped emerging economies across the World.
Market Restraints
The global orphan diseases market growth might experience restraints due to the correct treatments, correct diagnosis and availability of healthcare facilities during the research period.
Moreover, the lack of awareness might affect the growth of the market in the forecast period. In addition, the high cost associated with orphan drugs might hamper the growth of the global market.
Cumulative Evaluation of The Market
While looking into the past records, the global Market has witnessed significant growth and is expected to continue to maintain the trend in the upcoming years.
As per the orphan diseases market analysis, the regional market players exist across regions like North America, Asia Pacific, and Europe, who contribute efficiently to enhance the market growth in the coming years.
Orphan Diseases Market Segment Insights
Orphan Diseases types of diseases Insights
The global market has been classified as genetic disorders, oncological diseases, cardiovascular diseases, respiratory disorders, eye disorders, autoimmune disease, blood disorders, growth disorders, neurological disorders and others.
Further, the oncological disease is sub-divided into Pancreatic cancer, multiple myeloma, acute myeloid leukemia (AML), ovarian cancer, renal cell carcinoma, and others. In 2020, the oncological disease segment was anticipated to hold the largest orphan diseases market share due to the increasing awareness about cancer among the population, rise in prevalence of oncological disease, availability of oncological orphan drugs for all to treat cancer, and early screening of cancer. Further, this disease is estimated to maintain the dominating place throughout the research period.
Orphan Diseases End-users Insights
The global market has been segregated into a research laboratory, hospital and clinics, and others depending on the end-user segment.
Among these, the hospital and clinic segments are estimated to generate the largest orphan diseases market size during the forecast period. This segment is growing due to the rising number of drugs administered by professional healthcare workers at the hospital.
Orphan Diseases drug type Insights
On the basis of drug type segment, the global market has been divided into non-biologics and biologics.
In 2019, the biologics segment was projected to dominate the global market by generating significant market share. On the contrary, the non-biologics segment is registered a low CAGR comparatively and generated a lower market share for the year.
Orphan Diseases treatment type Insights
The global market has been segregated into drug therapy, cell therapy, gene therapy, and others in terms of treatment type segment.
Orphan Diseases treatment Regional Insights
Based on the regional segmentation, the global orphan diseases market has been spread across different geographical locations such as North America, Asia Pacific, South America, Europe, and the rest of the World.
In the present global scenario, the North American region is estimated to dominate the global market by generating the largest market share throughout the research period. This region is expanding due to the presence of key players leading to the development of innovative and sophisticated products. Moreover, the strong patient population and the significant expenditure on orphan drugs are also contributing to the North American regional market growth.
Apart from this region, the European market for Orphan Diseases is considered to be the second-largest growing market in the forecast period. This region has strong adoption of sophisticated rare disease therapeutics, which is fuelling the European market for Orphan Diseases. Moreover, the Asia Pacific market for Orphan Diseases is anticipated to expand at a significant rate throughout the research period due to the growing awareness of the rare disease and rising healthcare expenditure. Furthermore, the rest of the World, including the Middle East & Africa, and Latin America, is estimated to show steady growth during the forecast period because of the under penetration of the market for Orphan Diseases.
Competitive Intensity Within the Industry
As per the Orphan Diseases Market Outlook report, the market players are the main contributors to the growth of the market for Orphan Diseases. These major market players include:
- Bristol-Myers Squibb Company (US),
- CELGENE CORPORATION (US)
- Novartis AG (Switzerland)
- Pfizer Inc. (US)
- AbbVie Inc. (US)
- F. Hoffmann-La Roche Ltd (Switzerland)
- Alexion (US)
- Vertex Pharmaceuticals Incorporated (US)
- Merck & Co., Inc (US)
- Sanofi (French)
- GlaxoSmithKline plc. (UK).
These market players are spread across all parts of the globe and focus on the research and developmental activities relating to Orphan Diseases. Moreover, they are also doing partnerships, collaborations, mergers and acquisitions to enhance the orphan diseases market growth. Likely, they are aiming to show their presence to the World in the upcoming years.
Recent Market Developments
- In June 2020, the prominent market player Agios Pharmaceuticals, Inc. declared that the company had received the Orphan Drug Designation of FDA for their Mitapivat pipeline candidate to treat Thalassemia.
- In June 2020, the major key player Chiasma, Inc. declared that it had received approval from FDA for its Mycapssa product to treat a rare disorder, i.e., acromegaly.
Report Overview
The global market report highlights the market overview, segmentation, regional classification, competitive analysis, recent developments and market dynamics. This report is a qualitative and quantitative analysis of the market.
According to the global orphan diseases market forecast report, the current market has been segmented based on the types of disease, end-users, and treatment type.
Orphan Diseases treatment Market Segmentation
Orphan Diseases treatment types of diseases Outlook
- genetic disorders,
- cancer,
- cardiovascular diseases,
- respiratory disorders,
- eye disorders,
- autoimmune disorders,
- blood disorders,
- growth disorder,
- neurological disorders,
- digestive disorders,
- others.
Orphan Diseases drug types Outlook
- non-biologics
- biologics
Orphan Diseases End-users Outlook
- research laboratory,
- hospital and clinics,
- others.
Orphan Diseases treatment type Outlook
- drug therapy,
- cell therapy,
- gene therapy,
- others.
Leading companies partner with us for data-driven Insights
Kindly complete the form below to receive a free sample of this Report
Tailored for You
- Dedicated Research on any specifics segment or region.
- Focused Research on specific players in the market.
- Custom Report based only on your requirements.
- Flexibility to add or subtract any chapter in the study.
- Historic data from 2014 and forecasts outlook till 2040.
- Flexibility of providing data/insights in formats (PDF, PPT, Excel).
- Provide cross segmentation in applicable scenario/markets.
