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Increasing Prevalence Of Rare Blood Disorders Fueling The Growth Of The Market Due To Genetic Mutations And Inherited Conditions Emerges as a Core Driver of the Rare Hematology Market in 2025

10-09-2025 09:00 AM CET | Health & Medicine

Press release from: The Business Research Company

Rare Hematology Market

Rare Hematology Market

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What Is the Expected CAGR for the Rare Hematology Market Through 2025?
The market size of rare hematology has observed significant growth in the past few years. It is projected to expand from $14.46 billion in 2024 to $15.47 billion in 2025, yielding a compound annual growth rate (CAGR) of 7.0%. The prior growth can be credited to factors such as an increase in specialized treatment facilities, enhanced funding for academic research in hematology, growing awareness amongst physicians regarding rare blood disorders, the surge in the use of biobanking and registries, and an increase in reported incidences thanks to improved surveillance systems.

What's the Projected Size of the Global Rare Hematology Market by 2029?
Anticipations are high for considerable expansion in the rare hematology market size over the impending years. The projections suggest a surge to $19.98 billion in 2029, with a compound annual growth rate (CAGR) of 6.6%. This forecasted growth is attributed to numerous factors such as an expanding pipeline of targeted biologics, the rising uptake of precision diagnostics, an increase in international collaborations for rare disease research, the growth of digital platforms to provide patient assistance, and an intensified focus on preventive genetic counseling. The forecast period is likely to witness trends like progress in immunotherapy approaches, advances in methods of stem cell transplantation, the inclusion of digital health platforms in hematology, the application of technology in drug discovery models, and advancements in biomarker-based diagnostics.

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Top Growth Drivers in the Rare Hematology Industry: What's Accelerating the Market?
The growth of the rare hematology market is predicted to be driven by the growing incidence of uncommon blood disorders. Such disorders, which impact the production, function, or lifespan of blood cells or clotting factors leading to anemia, bleeding, or hemolysis, are rising due to genetic changes and inherited traits because faulty genes are transferred from parents to their offspring. Rare hematology contributes to fighting these unusual blood disorders by providing precise diagnostics and bespoke therapies, making it perfectly suited for handling rare and complicated conditions. It lessens the impact of the ailment by ensuring exact treatment and sustained patient care, enhancing health conditions and life quality. For example, in April 2024, the Perth Blood Institute, a non-profit entity based in Australia, reported that the count of individuals diagnosed with hemophilia (a hereditary blood ailment in which the blood lacks sufficient clotting factors resulting in extended bleeding) raised to 257,146 in 2022 from 233,577 in 2021. As a result, the increasing incidence of rare blood disorders is stimulating the expansion of the rare hematology market.

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What Trends Will Shape the Rare Hematology Market Through 2029 and Beyond?
Leading firms in the rare hematology sector are concentrating their efforts on developing cutting-edge therapies and strategic programs such as multi-stakeholder advocacy councils in a bid to meet outstanding medical requirements, improve patient support, and foster solutions that can be applied across various indicators. These multi-stakeholder advocacy councils consist of patients, caregivers, and specialists working towards the enhancement of care, awareness, and treatment options for specific health issues. For example, in February 2023, Agios Pharmaceuticals Inc., a biopharmaceutical firm based in the US, instituted the Red Cell Revolution advocacy advisory council. This endeavor brings together patients, caregivers, and healthcare experts afflicted with rare hemolytic anemias such as pyruvate kinase deficiency, thalassemia, and sickle cell disease. This council aims to fortify community bonds, produce documented evidence, establish practical solutions and amplify awareness of collective challenges. Their strategies include initiatives like sponsored podcasts, video content, and collaborations with entities such as the National Organization for Rare Disorders to promote healthcare equality and education.

What Are the Main Segments in the Rare Hematology Market?
The rare hematology market covered in this report is segmented as

1) By Disease Type: Hemophilia, Sickle Cell Disease, Thalassemia, Myelodysplastic Syndromes, Other Disease Types
2) By Treatment Type: Gene Therapy, Stem Cell Transplantation, Blood Transfusion, Pharmacotherapy
3) By Diagnostic Technique: Laboratory Tests, Blood Tests, Bone Marrow Biopsies, Imaging Techniques, Magnetic Resonance Imaging (MRI) Scans, Ultrasounds, Genetic Testing, Carrier Screening, Whole Genome Sequencing
4) By Application: Pediatric, Adult
5) By End-User: Hospitals, Specialty Clinics, Research Institutes

Subsegments:
1) By Hemophilia: Hemophilia A, Hemophilia B, Hemophilia C, Acquired Hemophilia
2) By Sickle Cell Disease: Sickle Cell Anemia, Sickle Hemoglobin C Disease, Sickle Beta Thalassemia
3) By Thalassemia: Alpha Thalassemia, Beta Thalassemia
4) By Myelodysplastic Syndromes: Refractory Anemia (RA), Refractory Anemia With Ring Sideroblasts (RARS), Refractory Cytopenia With Multilineage Dysplasia (RCMD), Refractory Anemia With Excess Blasts (RAEB), Myelodysplastic Syndrome With Isolated Del(5Q), Unclassified Myelodysplastic Syndromes
5) By Other Disease Types: Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Rare Coagulation Disorders

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Which Top Companies are Driving Growth in the Rare Hematology Market?
Major companies operating 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.

Which Regions Will Dominate the Rare Hematology Market Through 2029?
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.

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