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The Rare Hematology Market size is forecast to increase by USD 4.59 billion, at a CAGR of 5.7% between 2023 and 2028. The market is witnessing significant growth due to the enhancement of strategic portfolios and approvals from regulatory bodies like the Food and Drug Administration (FDA) for advanced healthcare solutions. The increasing prevalence of blood diseases such as Anemia, Myeloma, Leukemia, and Sickle cell anemia necessitates the development of innovative treatments. The high treatment costs associated with these diseases create a lucrative market for market players. Hematologists are constantly seeking effective and cost-efficient detection processes to diagnose and treat these complex conditions, driving the market's growth. The market's future looks promising with ongoing research and development efforts to improve patient outcomes and expand treatment options.
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The market research report provides comprehensive data (region-wise segment analysis), with forecasts and estimates in "USD billion" for the period 2024-2028, as well as historical data from 2018 - 2022 for the following segments.
The hemophilia segment is estimated to witness significant growth during the forecast period. The market, specifically focusing on the treatment of hemophilia, is experiencing substantial progress and innovation. Hemophilia, a genetic disorder characterized by a deficiency in clotting factors, predominantly affects males and is classified into hemophilia A (factor VIII deficiency) and hemophilia B (factor IX deficiency). According to estimates, over 400,000 individuals worldwide were diagnosed with hemophilia in September 2024.
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The hemophilia segment was valued at USD 3.65 billion in 2018. A significant challenge in managing hemophilia is the emergence of inhibitors to factor VIII and factor IX, which affects approximately 25% to 30% of hemophilia A patients and 3% to 5% of hemophilia B patients. These inhibitors render traditional factor replacement therapies ineffective. To address this issue, the pharmaceutical industry is investing in the development of effective and affordable treatments, including gene editing, targeted therapies, and novel drugs. Biotechnologies are also playing a pivotal role in creating innovative solutions for rare hematological disorders.
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North America is estimated to contribute 35% 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 in the Pharmaceutical sector is witnessing significant growth due to the development of Effective Treatments and Affordable Treatments for various rare blood disorders. In North America, the market holds a prominent position, driven by advanced healthcare infrastructure, intensive research and development activities, and regulatory support. For instance, the Texas Children's Cancer and Hematology Center in the US is a leading institution specializing in diagnosing and treating rare hematological disorders, including pediatric sarcomas. This center's multidisciplinary team consists of specialists in oncology, surgery, diagnostic imaging, molecular pathology, and psychosocial care, ensuring comprehensive treatment for patients. Novel Drugs and Gene Editing technologies, along with Targeted Therapies, are some of the Biotechnologies contributing to the market's growth. These advancements aim to improve patient outcomes and reduce the financial burden on patients and healthcare systems.
Companies 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 focuses on researching medicines for blood cancer and solid malignancies.
Technavio provides the ranking index for the top 20 companies along with insights on the market positioning of:
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Market structure |
Fragmented |
YoY growth 2023-2024 |
5.1 |
The market encompasses the diagnosis, treatment, and management of various blood disorders, including hematologic diseases, plasma disorders, anemia, myeloma, leukemia, sickle cell anemia, and hereditary cancer. Diagnostic techniques play a crucial role in the market, with genetic testing technologies and innovative diagnostic capabilities enabling early intervention and personalized treatment. Effective and affordable treatments are in high demand, with therapeutic avenues including gene therapies, targeted therapies, and orphan drug designation. Pharmaceutical companies are investing heavily in clinical trial data to develop new treatments for these complex conditions. Hematologists rely on their diagnostic skills to navigate the intricacies of the medical diagnosis process and provide patient-centric approaches to treatment. The market is characterized by a focus on personalized medicine and a growing need for advanced diagnostic and treatment solutions. Clinical trials continue to uncover new therapeutic avenues, offering hope for those affected by these often debilitating conditions. Our researchers analyzed the data with 2023 as the base year, along with the key drivers, trends, and challenges. A holistic analysis of drivers will help companies refine their marketing strategies to gain a competitive advantage.
Enhancement of strategic portfolios is notably driving market growth. The market is witnessing significant growth as companies invest in expanding their product portfolios to address unmet medical needs and drive advancements in the diagnosis and treatment of various blood disorders. One notable example is Sobi, a Swedish drugmaker, which reported a 13% year-over-year increase in sales for its hematology segment during the second quarter of 2024, amounting to USD 363 million (3.86 billion Swedish krona). This growth was a significant contributor to the company's overall performance, highlighting the importance of a comprehensive hematology portfolio. Sobi's hematology offerings include diagnostic techniques such as genetic testing, treatments, and personalized medicine, as well as innovative solutions like gene therapies.
The orphan drug designation of these products enables companies to focus on developing treatments for rare diseases with limited therapeutic options, making a substantial impact on patient care. The market is further driven by the increasing demand for personalized medicine and the development of advanced diagnostic techniques and treatments. Thus, such factors are driving the growth of the market during the forecast period.
Approvals from Food and Drug Administration (FDA) is the key trend in the market. The market experiences continuous growth due to the approval of innovative therapies for managing various blood disorders by regulatory bodies, such as the Food and Drug Administration (FDA). These approvals significantly impact the treatment landscape and drive market expansion. On February 23, 2023, Sanofi and Sobi received early FDA approval for Altuviiio, an investigational factor VIII replacement therapy for hemophilia A. This therapy is designed for routine prophylaxis, on-demand treatment, and surgical management in both adults and children. The expedited approval underscores the urgency to bring advanced treatments to patients with rare blood disorders. Diagnostic techniques, including genetic testing, play a vital role in identifying these disorders and guiding personalized medicine approaches. Furthermore, the development of gene therapies and orphan drug designations continues to fuel market growth in the rare hematology sector. Thus, such trends will shape the growth of the market during the forecast period.
High treatment costs are the major challenge that affects the growth of the market. The market is witnessing significant growth due to the development of innovative diagnostic techniques, genetic testing, and advanced treatments for various blood disorders. These advancements, including personalized medicine, gene therapies, and orphan drug designations, are revolutionizing the way rare hematological conditions are diagnosed and managed. However, the high cost of these treatments remains a significant challenge.
For instance, recently approved gene therapies, such as Libmeldy, Hemgenix, Zynteglo, and Skysona, carry extraordinary price tags, with Libmeldy having a wholesale acquisition cost of USD 4.25 million, making it one of the most expensive drugs globally. Other high-cost therapies range from USD 2.8 million to USD 3.5 million per dose. The lifetime cost of Vyjuvek, a topical gene therapy for dystrophic epidermolysis bullosa, could range between USD 15 million and USD 22 million per patient. These exorbitant costs are a major concern for healthcare systems and patients alike, necessitating continued research for cost-effective solutions in the market. Hence, the above factors will impede the growth of the market during the forecast period
The market forecasting report includes the adoption lifecycle of the market research and growth, 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 market growth analysis strategies.
Customer Landscape
Rare hematology refers to the branch of medicine that deals with the diagnosis, treatment, and management of various rare blood disorders and plasma diseases. These conditions, including sickle cell disease, hemophilia, myeloma, leukemia, anemia, and other hereditary and acquired hematologic diseases, can significantly impact patients' quality of life and require specialized knowledge and care. Diagnostic techniques in rare hematology have advanced with the advent of genetic testing and molecular biology. These technologies enable early intervention and personalized treatment, leading to effective and affordable treatments for patients. Gene therapies, gene editing, and targeted therapies are novel therapeutic avenues for rare hematologic diseases, with curative potential for some conditions. Orphan drug designation and innovative therapies have expanded the pharmaceutical landscape for rare hematology, providing new treatment options. Healthcare infrastructure and solutions play a crucial role in the management of rare hematology patients.
Hematologists require advanced diagnostic capabilities and treatment techniques to accurately diagnose and manage these complex conditions. The detection and diagnosis process for rare hematologic diseases can be challenging, requiring specialized skills and expertise. Effective treatments for these conditions can significantly improve patients' lives, and ongoing clinical trials explore new therapeutic avenues for various blood diseases. In summary, rare hematology is a critical area of medicine, focusing on the diagnosis, treatment, and management of various rare blood disorders and plasma diseases. Genetic testing, gene therapies, and targeted therapies are driving innovation in this field, providing new treatment options and hope for patients. The importance of early intervention, personalized treatment, and patient-centric approaches cannot be overstated in the effective management of these complex conditions.
Market Scope |
|
Report Coverage |
Details |
Page number |
210 |
Base year |
2023 |
Historic period |
2018 - 2022 |
Forecast period |
2024-2028 |
Growth momentum & CAGR |
Accelerate at a CAGR of 5.7% |
Market Growth 2024-2028 |
USD 4.56 billion |
Regional analysis |
North America, Europe, Asia, and Rest of World (ROW) |
Performing market contribution |
North America at 35% |
Key countries |
US, China, UK, Germany, Japan, India, France, Italy, South Korea, and Singapore |
Competitive landscape |
Leading Companies, Market Positioning of Companies, Competitive Strategies, and Industry Risks |
Key companies profiled |
AbbVie Inc., AstraZeneca Plc, Bayer AG, Biogen Inc., bluebird bio Inc., Bristol Myers Squibb Co., CSL Ltd., Emmaus Medical Inc., F. Hoffmann La Roche Ltd., GlaxoSmithKline Plc, Grifols SA, Jazz Pharmaceuticals Plc, Johnson and Johnson Services Inc., Merck and Co. Inc., Novartis AG, Novo Nordisk AS, Octapharma AG, Pfizer Inc., Sanofi SA, and Takeda Pharmaceutical Co. Ltd. |
Market dynamics |
Parent market analysis, market growth inducers and obstacles, fast-growing and slow-growing segment analysis, AI impact on market trends, COVID-19 impact and recovery analysis and future consumer dynamics, market condition analysis for the market forecast period |
Customization purview |
If our market report has not included the data that you are looking for, you can reach out to our analysts and get segments customized. |
1 Executive Summary
2 Technavio Analysis
3 Market Landscape
4 Market Sizing
5 Historic Market Size
6 Qualitative Analysis
7 Five Forces Analysis
8 Market Segmentation by Disease Type
9 Market Segmentation by End-user
10 Customer Landscape
11 Geographic Landscape
12 Drivers, Challenges, and Opportunity/Restraints
13 Competitive Landscape
14 Competitive Analysis
15 Appendix
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