Blood develops from hematopoietic stem cells (HSC) and formation takes place in the bone marrow through a sequence of the regulated process known as hematopoiesis. Blood contains component such as plasma, RBS, WBC, and platelets. Blood disorders include bleeding disorders such as hemophilia, blood clots; blood cancers (leukemia, lymphoma, and myeloma). When the normal process of the blood development fails, abnormal blood cell type is produced, which causes blood cancer and other blood disorders. The rare hematological disease includes anemia-type red blood cell conditions, white blood cell dysfunctions, immuno-disorders and other platelet-based abnormalities. In the United States, the condition is considered as rare when it affects less than 200,000 people in the United States or prevalence of 5 per 10,000. In Europe, 5 in 10,000 citizens are considered as rare diseases. According to Eurordis (European organization of rare diseases), there are approximately 6000 to 8000 rare diseases.
Rare blood diseases include sickle cell disease, beta thalassemia, follicular lymphoma, multiple myeloma, antiphospholipid syndrome, thrombotic thrombocytopenic purpura, and others. Around 15 million people across the globe suffer from thalassemic disorders and nearly 1.5% of the world population is estimated to be a carrier of β-thalassemia. The other hemoglobinopathies are also on the rise due to increasing immigration of carriers from underdeveloped nations to developed regions of the world. The rare blood disorders are difficult to diagnose and treat. Government organization and pharmaceutical companies dealing with hematological disorders are focusing on research and development in hematology area to new, effective and advanced treatment for diseases and their complications.
The government initiatives such as by Canadian Institute of Health Research (CIHR), is conducting research and development in hematology area and is playing a leading role in international research initiatives. For example, Canadian Institute of Health Research supported the drug development of, the Canadian branch of Orphanet known as of Orphanet-Canada, an online portal for rare diseases. The rise in prevalence of hematology disorders represents additional opportunities for rare diseases treatment manufacturers. The Food and Drug Administration of Orphan Products Development is providing funds and grants to supports the clinical trial and drug development for the treatment of rare diseases and disorders. Manufacturers of orphan drugs are getting an advantage of tax benefits, research grants and fast-track approval are giving an opportunity for innovations.
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The global market for rare hematology disorders market is segmented on basis of disease indication, dosage form, and geography:
- Segmentation by Disease Indication
- Hemoglobin Disorders
- Beta-thalassemia
- Sickle Cell Anemias
- Diamond-Blackfan Anemia
- Rare Blood Cancers
- Mantle Cell Lymphoma
- Chronic Myeloid Leukemia
- Hairy Cell Leukemia
- Multiple Myeloma
- Platelet Based Disorders
- Idiopathic Thrombocytopenic Purpura
- Glanzmann’s Thrombasthenia
- Plasma Disorders
- Hemophilia
- Acquired Hemophilia
- Myeloproliferative Disorders
- Myelofibrosis
- Systemic Mastocytosis
- Eosinophilia
- Genetic Hematology Disorders
- Noonan syndrome
- Von Hippel-Lindau disease
- Hemoglobin Disorders
Segmentation by Dosage Form
- Oral Dosage Forms
- Injectable Dosage Forms
On the basis of indication, the global rare hematology disorders market has been segmented into rare hemoglobin disorders, rare blood cancers, platelet-based disorders, plasma disorders, myeloproliferative disorders, genetic hematology disorders. Rare blood cancers and rare hemoglobin disorders indication segments are expected to contribute highest market share due to more number of approved products for this indication. Based on the dosage form global market has been segmented in to oral and injectable. Injectable dosage form segment dominated the market and would continue to do so as a majority of new approvals are injectable dosage forms.
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On the basis of regional presence, global rare hematology disorders market is segmented into five key regions viz. North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America will continue to dominate the global rare hematology disorders market due to high patient volume and availability of reimbursement for rare hematology disorder treatment products. Rare hematology disorder treatment possesses a rich pipeline and majority of these clinical trials are being carried out in the U.S. Europe is expected to hold second largest market share in global rare hematology disorders market due to growing presence of key players. Moreover, EU provides faster approvals of orphan drugs, which reduces the time-to-market. Thus, Europe rare hematology disorders market is matured market.
Some of the key players identified in global rare hematology disorders treatment market include ADIENNE Pharma & Biotech, Bluebird Bio Inc., Emmaus Medical, Inc., Bristol-Myers Squibb Company, MERCK & CO., Inc., AstraZeneca Plc., Pfizer Inc., Millennium Pharmaceuticals, Inc., Novartis AG, Novo Nordisk A/S, Shire Plc. Hoffman-La Roche AG, Incyte Corporation,