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The orphan drugs market size is estimated to grow at a CAGR of 14.02% between 2022 and 2027. The orphan drugs market size is forecast to increase by USD 170.8 billion. The growth of the market depends on several factors, including robust pipeline and recent drug approvals, the rising incidence of rare diseases, and the availability of incentives for orphan drug development.
This orphan drugs market report extensively covers market segmentation by distribution channel (hospital pharmacy, specialty pharmacy, retail pharmacy, and others), product (biologics and non-biologics), and geography (North America, Europe, Asia, and Rest of World (ROW). It also includes an in-depth analysis of drivers, trends, and challenges. Furthermore, the report includes historic market data from 2017 to 2021.
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The rising incidence of rare diseases is notably driving the market growth, although factors such as delays in diagnosis may impede the market growth. Our researchers analyzed the data with 2022 as the base year and the key drivers, trends, and challenges. A holistic analysis of drivers will help companies refine their marketing strategies to gain a competitive advantage.
The rising incidence of rare diseases is notably driving market growth. Rare diseases and a subset of non-orphan diseases are specifically treated with orphan drugs. Inherited genetic disorders, uncommon cancers, tropical infectious diseases, and neurodegenerative illnesses are examples of rare diseases. In spite of their low incidence, rare diseases paradoxically affect a sizable portion of the population worldwide. According to the National Institutes of Health (NIH), 400 million people worldwide suffer from rare diseases. One out of every two patients in the US with a rare disease is a child, according to the NIH. Monogenic or single-gene disorders are very common and frequently passed down through the germline.
However, many inherited rare diseases have no diagnostic tools available. There are about 350 rare diseases that affect 80% of all patients with rare diseases. The 350 rare diseases are therefore the subject of extensive research by numerous diagnostic and pharmaceutical companies. These studies have increased public awareness on the use of orphan products, which will fuel the market's expansion. Therefore, the high incidence of rare diseases will increase the requirement for orphan drugs, which, in turn, will drive market growth during the forecast period.
Strategic focus on alliances, partnerships, and M&A is an emerging trend in market growth. Strategic alliances and partnerships allow vendors to co-develop and market products, increase their product portfolios, and reach a wider geographic audience. As a result, it is anticipated that the market's vendors will keep looking for strategic opportunities to seize, which will fuel the market's expansion during the forecast period.
In order to deal with impending patent expiration and the requirement to restock the strong pipeline, the market is currently embracing the M&A trend. A concentration on various therapeutics fueled by rising M&A activities and the adoption of cutting-edge, economical manufacturing technologies will pave the way for the development of novel orphan drugs. Furthermore, the market players are using a variety of strategies for productive strategic alliances to overcome the difficulties posed by manufacturing procedures and translational biology. There have been several cross-disciplinary gene therapy collaborations that have been partnership-focused, including those with patient and advocacy groups, small and large biopharmaceutical companies, and data-driven technology companies. Hence, such collaborations will boost the growth of the market during the forecast period.
Delays in diagnosis are major challenges impeding market growth. A precise and timely diagnosis is the initial step to improving care for people with rare diseases. Due to inaccurate, expensive testing, general ignorance, and the asymptomatic nature of many rare diseases, the diagnosis of these conditions has historically been hampered. A delayed diagnosis can postpone the start of an effective course of treatment, which could cause serious, irreversible, and crippling disease to progress. The delayed diagnosis often results in premature deaths and delays in patient care.
Numerous rare diseases can be misdiagnosed and take longer to diagnose because of the asymptomatic nature of many relatively common symptoms. The longer it takes to diagnose a rare disease, the more physician-patient consultations take place, which may make patients and caregivers feel frustrated and scared when the diagnosis is delayed. The average wait time for patients in developing and underdeveloped nations is 5–30 years between the onset of the first symptoms and the official diagnosis of their illness. Only 30% of cases around the world have access to genetic counseling because rare diseases have genetic roots. Furthermore, the vast majority of patients around the world are initially given a false diagnosis. This results in the use of therapeutic treatments being restricted and causes medical interventions to be based on incorrect diagnoses. Hence, these limitations will negatively impact the global market during the forecast period.
The report includes the adoption lifecycle of the market, covering from the innovator’s stage to the laggard’s stage. It focuses on adoption rates in different regions based on penetration. Furthermore, the report also includes key purchase criteria and drivers of price sensitivity to help companies evaluate and develop their growth strategies.
Global Orphan Drugs Market Customer Landscape
Vendors are implementing various strategies, such as strategic alliances, partnerships, mergers and acquisitions, geographical expansion, and product/service launches, to enhance their presence in the market.
AbbVie Inc. - The company conducts research and development, manufacturing, commercialization, and sale of innovative medicines and therapies to allocate resources and assess business performance on a global basis in order to achieve established long-term strategic goals. The key offerings of the company include orphan drugs such as Humira and Venetoclax.
The report also includes detailed analyses of the competitive landscape of the market and information about 15 market vendors, including:
Qualitative and quantitative analysis of vendors has been conducted to help clients understand the wider business environment as well as the strengths and weaknesses of key market players. Data is qualitatively analyzed to categorize vendors as pure play, category-focused, industry-focused, and diversified; it is quantitatively analyzed to categorize vendors as dominant, leading, strong, tentative, and weak.
The market share growth by the hospital pharmacy segment will be significant during the forecast period. Inpatient medications are prepared, made, stored, and distributed by the hospital pharmacy department. Orphan diseases are rare diseases that require the quickest and easiest care and treatment. In general, the easiest place to meet these needs is a hospital pharmacy.
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The hospital pharmacy segment was valued at USD 53.66 billion in 2017 and continued to grow until 2021. This segment has several benefits, including the ability to tailor each patient's dosing schedule. assisting medical professionals in making drug-related decisions and assisting patients in understanding and taking their medications. These uses are helping this market segment grow. Additionally, studies reveal that orphan drugs that were approved in the US, Europe, and Japan had respective market availability rates of 37.8%, 24.6%, and 52.4%. At the 24 tertiary public hospitals, the median availability of 31 orphan drugs was 20.8%. Thus, the hospital pharmacy segment in the market is expected to witness growth during the forecast period.
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North America is estimated to contribute 40% to the growth of the global market during the forecast period. Technavio’s analysts have elaborately explained the regional trends and drivers that shape the market during the forecast period.
The market growth in North America is mainly attributed to six driving factors, namely, new drug approvals, growing diagnosis of rare diseases, availability of reimbursement schemes, the high premium price of orphan drugs, the strong presence of key vendors of orphan drugs, and incentives or encouragement from government regulatory agencies and non-profit organizations. Novel drugs have been flooding the US market in North America over the past ten years. To aid in the evaluation and development of products that show promise for the diagnosis and/or treatment of rare diseases, the US FDA established the Office of Orphan Products Development (OOPD). A record number of orphan drugs were approved and introduced in the US in 2022, providing patients with new treatment options. The market in North America is anticipated to experience growth during the forecast period as a result of such a favorable environment for the approval of new drugs.
Another key factor influencing the growth of the market in North America is the increasing prevalence of rare diseases. Due to the development of sophisticated and affordable genetic profiling, the diagnosis rate for rare diseases has been rising in the US and Canada over time. Approximately 7,000 rare diseases affect 25–30 million people in the US today, according to the National Institutes of Health (NIH) and the US Food and Drug Administration, with 250–280 new rare diseases being described each year. Additionally, about 350 well-known rare diseases affect 80% of all patients who receive a diagnosis of a rare disease. Thus, the high prevalence of rare diseases will help propel the market in North America during the forecast period.
The COVID-19 pandemic affected the market in 2020 owing to the demand-supply gap for orphan drugs in North America or the treatment of rare diseases. However, clinical trials conducted by multiple key players for using orphan drugs for the treatment of COVID-19 provided ample opportunities. Moreover, in 2021, with the large-scale vaccination drive, the number of COVID cases was reduced which led to the lifting of the lockdown restrictions and helped the market resume its business activities. This increased the demand for such drugs in the region. Hence, such factors will boost the market in the region during the forecast period.
The orphan drugs market report forecasts market growth by revenue at global, regional & country levels and provides an analysis of the latest trends and growth opportunities from 2017 to 2027.
Technavio categorizes the global orphan drugs market within the overall healthcare industry. The parent, the global pharmaceuticals, market covers products and companies engaged in R&D or the production of generic drugs, non-generic drugs, and veterinary drugs. Our market research report has extensively covered external factors influencing the parent market growth during the forecast period.
Orphan Drugs Market Scope |
|
Report Coverage |
Details |
Page number |
171 |
Base year |
2022 |
Historic period |
2017-2021 |
Forecast period |
2023-2027 |
Growth momentum & CAGR |
Accelerate at a CAGR of 14.02% |
Market growth 2023-2027 |
USD 170.8 billion |
Market structure |
Fragmented |
YoY growth 2022-2023(%) |
13.24 |
Regional analysis |
North America, Europe, Asia, and Rest of World (ROW) |
Performing market contribution |
North America at 40% |
Key countries |
US, Canada, UK, Germany, and China |
Competitive landscape |
Leading Vendors, Market Positioning of Vendors, Competitive Strategies, and Industry Risks |
Key companies profiled |
AbbVie Inc., Alnylam Pharmaceuticals Inc., Astellas Pharma Inc., AstraZeneca Plc, bluebird bio Inc., Bristol Myers Squibb Co., CSL Ltd., F. Hoffmann La Roche Ltd., Gilead Sciences Inc., GlaxoSmithKline Plc, Ipsen Pharma, Johnson and Johnson Services Inc., Merck and Co. Inc., Novartis AG, Novo Nordisk AS, Pfizer Inc., Ractigen Therapeutics, Sanofi SA, Takeda Pharmaceutical Co. Ltd., and Vertex Pharmaceuticals Inc. |
Market dynamics |
Parent market analysis, Market growth inducers and obstacles, Fast-growing and slow-growing segment analysis, COVID-19 impact and recovery analysis and future consumer dynamics, and Market condition analysis for the forecast period. |
Customization purview |
If our report has not included the data that you are looking for, you can reach out to our analysts and get segments customized. |
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1 Executive Summary
2 Market Landscape
3 Market Sizing
4 Historic Market Size
5 Five Forces Analysis
6 Market Segmentation by Distribution Channel
7 Market Segmentation by Product
8 Customer Landscape
9 Geographic Landscape
10 Drivers, Challenges, and Trends
11 Vendor Landscape
12 Vendor Analysis
13 Appendix
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