Rare Hematology Market Report 2025

The rare hematology market size has grown strongly in recent years. It will grow from $14.46 billion in 2024 to $15.47 billion in 2025 at a compound annual growth rate (CAGR) of 7%. The growth during the historic period was driven by increasing availability of specialized treatment centers, rising academic research funding in hematology, growing physician awareness of rare blood conditions, expanding use of biobanking and registries, and improved incidence reporting through enhanced surveillance systems.

The rare hematology market size is expected to see strong growth in the next few years. It will grow to $19.98 billion in 2029 at a compound annual growth rate (CAGR) of 6.6%. The growth during the forecast period is fueled by a growing pipeline of targeted biologics, rising adoption of precision diagnostics, expanding cross-border collaborations for rare disease research, development of digital platforms for patient support, and increasing emphasis on preventive genetic counseling. The primary trends in the forecast period include advancements in immunotherapy approaches, improvements in stem cell transplantation methods, integration of digital health platforms in hematology, technology-driven drug discovery models, and developments in biomarker-based diagnostics.

The increasing prevalence of rare blood disorders is expected to drive the growth of the rare hematology market in the coming years. Rare blood disorders are uncommon conditions that affect the production, function, or lifespan of blood cells or clotting factors, leading to anemia, bleeding, or hemolysis. The rising prevalence of these disorders is primarily due to genetic mutations and inherited conditions, where abnormal genes are passed down from parents to children. The rare hematology market plays a crucial role in addressing these conditions by providing specialized diagnostics and targeted therapies, ideal for managing complex and uncommon blood disorders. These services reduce complications by enabling precise treatment and continuous patient care, ultimately improving health outcomes and quality of life. For example, in April 2024, the Perth Blood Institute, an Australia-based nonprofit, reported that the number of people diagnosed with hemophilia, a genetic blood disorder that impairs blood clotting, increased to 257,146 in 2022 from 233,577 in 2021. As a result, the growing prevalence of rare blood disorders is fueling the expansion of the rare hematology market.

Companies in the rare hematology market are increasingly focusing on developing innovative therapies and establishing strategic initiatives, such as multi-stakeholder advocacy councils, to address unmet medical needs, enhance patient support, and drive solutions across various conditions. Multi-stakeholder advocacy councils bring together patients, caregivers, and experts to collaborate on improving care, awareness, and solutions for specific health conditions. For instance, in February 2023, Agios Pharmaceuticals Inc., a US-based biopharmaceutical company, launched the Red Cell Revolution advocacy advisory council. This initiative focuses on rare hemolytic anemias, including pyruvate kinase deficiency, thalassemia, and sickle cell disease. The council aims to strengthen community connections, generate published evidence, create practical solutions, and raise awareness of common challenges. It includes initiatives such as sponsored podcasts, video content, and partnerships with organizations such as the National Organization for Rare Disorders to promote health equity and education.

In October 2022, Pfizer Inc., a US-based biopharmaceutical company, acquired Global Blood Therapeutics Inc. for an undisclosed amount. This acquisition allows Pfizer to expand its rare hematology portfolio, particularly in sickle cell disease, enhancing its pipeline of therapies for underserved patient populations. Global Blood Therapeutics Inc. is a US-based biopharmaceutical company focused on developing therapies for rare blood disorders, especially sickle cell disease.

Major players in the rare hematology market are F. Hoffmann-La Roche Ltd, Pfizer Inc., Abbvie Inc., Bayer AG, Sanofi S.A., Novartis AG, Astrazeneca Plc, Bristol-Myers Squibb Company, Takeda Pharmaceutical Company Limited, Amgen Inc., CSL Behring LLC, Biogen Inc., Jazz Pharmaceuticals Plc, Octapharma AG, PharmaEssentia Corporation, Disc Medicine Inc., BioInvent International AB, Mustang Bio Inc., BlueSphere Bio Inc., and Rallybio LLC.

North America was the largest region in the rare hematology market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in rare hematology report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the rare hematology market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report’s Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.

The sudden escalation of U.S. tariffs and the consequent trade frictions in spring 2025 are severely impacting the healthcare sector, particularly in the supply of critical medical devices, diagnostic equipment, and pharmaceuticals. Hospitals and healthcare providers are facing higher costs for imported surgical instruments, imaging equipment, and consumables such as syringes and catheters, many of which have limited domestic alternatives. These increased costs are straining healthcare budgets, leading some providers to delay equipment upgrades or pass on expenses to patients. Additionally, tariffs on raw materials and components are disrupting the production of essential drugs and devices, causing supply chain bottlenecks. In response, the industry is diversifying sourcing strategies, boosting local manufacturing where possible, and advocating for tariff exemptions on life-saving medical products.

Rare hematology refers to the study, diagnosis, and treatment of uncommon blood disorders, including rare anemias, clotting disorders, bone marrow failure syndromes, and rare blood cancers. These conditions often affect small patient populations and have limited treatment options. Rare hematology advances understanding, improves diagnosis, and supports the development of targeted therapies to enhance patient outcomes and quality of life.

The primary disease types in rare hematology include hemophilia, sickle cell disease, thalassemia, myelodysplastic syndromes, and others. Hemophilia is a genetic bleeding disorder characterized by insufficient clotting factors, leading to excessive bleeding and difficulty controlling hemorrhages. Treatments include gene therapy, stem cell transplantation, blood transfusion, and pharmacotherapy, supported by diagnostic techniques such as laboratory tests, blood tests, bone marrow biopsies, imaging methods, MRI scans, ultrasounds, genetic testing, carrier screening, and whole genome sequencing. Applications span both pediatric and adult populations, serving end users including hospitals, specialty clinics, and research institutes.

The rare hematology market research report is one of a series of new reports that provides rare hematology market statistics, including rare hematology industry global market size, regional shares, competitors with a rare hematology market share, detailed rare hematology market segments, market trends and opportunities, and any further data you may need to thrive in the rare hematology industry. This rare hematology market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The rare hematology market includes revenues earned by entities through genetic testing, disease monitoring, blood transfusion support, patient care management, and clinical trial services. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values and are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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