Rare hematology disorders market Outlook - 2027
Rare blood diseases include sickle cell disease, beta thalassemia, follicular lymphoma, multiple myeloma, antiphospholipid syndrome, thrombotic thrombocytopenic purpura, and others. 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. 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.
Covid-19 Scenario analysis:
The coronavirus COVID-19 has affected 213 countries and territories around the world. COVID-19 has created new challenges that are requiring manufacturers to innovate at speeds they never have before. Supply chain disruptions, social distancing on high-touch assembly lines, limited ability to travel, and the need for oversight add significant complexity to today’s processes. The outbreak of coronavirus disease 2019 (COVID-19) has created a global health crisis that has had a deep impact on the manufacturing market there is a shortage in supply of raw materials from Chinese manufacturers which has resulted in a severe demand-supply gap. The manufacturers are further expected to be stranded on raw material orders owing to the logistics industry being significantly impacted due to lockdown amid COVID-19. This has all affected the market adversely causing a slowdown in the rare hematology disorders market due to shortage of supply and manufacturing drugs.
Top impacting factors: Market Scenario Analysis, Trends, Drivers and Impact analysis:
Rare diseases comprise a growing public health priority, as they affect above 300 million people globally and they are difficult to diagnose and treat. There is a pressing need for better ways to detect and diagnose rare diseases, as well as to provide companion diagnostics for therapy guidance, clinical trials enrollment and therapy monitoring applications. In the U.S., 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. 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. Rare hematology disorder treatment among the geriatric population base will show boast in future. An overall occurrence of anemia ranges from 10% to 24% among the senior adults. As the age of patient increases, the need for hematology disorder care also increases. Thus, the growing population of geriatric age group will increase the chances of developing rare blood disorders among adults, thereby boosting the demand for hematology disorder treatment in the forecast period.
Development of new therapies:
Global Blood Therapeutics (GBT) is developing biologics for the treatment of sickle cell disease. The company lead candidate, voxelotor (GBT440) is in Phase 3 trials and being investigated for the treatment of sickle cell disease. The candidate is a hemoglobin S polymerization inhibitor and its mechanism of action is modulation hemoglobin affinity for oxygen. FDA has granted has breakthrough therapy, fast track, orphan drug and rare pediatric disease designations to Vozelotor. Imara is developing novel therapeutics for patients suffering from sickle cell disease and other hemoglobinopathies. Sickle cell disease is a rare, genetic blood disease that causes red blood cells to sickle and become damaged, activating immune cells and blocking blood flow in capillaries, injuring many organs and causing daily pain. Its lead candidate is IMR-687, a small molecule inhibitor of phosphodiesterase-9 (PDE9i) for the treatment of sickle cell disease. Launched in April 2016 by orphan drug accelerator Cydan Development. Cyclerion is involved in developing therapies for treating sickle cell disease (SCD) and diabetic nephropathy. They are focused on the nitric oxide-cyclic guanosine monophosphate (cGMP) signaling pathway. Their lead candidate, Olinciguat, which is a vascular guanylate cyclase agonist. The candidate is in phase II clinical trial. They have granted Orphan Drug Designation for SCD by the U.S. Food and Drug Administration. The development of such therapies and treatment for the diseases will drive the Rare hematology disorders market growth.
High cost of treatment and late detection in developing countries will prove as the restrain for market growth.
Key benefits of the report:
- This study presents the analytical depiction of the global rare hematology disorders industry along with the current trends and future estimations to determine the imminent investment pockets.
- The report presents information related to key drivers, restraints, and opportunities along with detailed analysis of the global rare hematology disorders market share.
- The current market is quantitatively analyzed to highlight the global rare hematology disorders market growth scenario.
- Porter’s five forces analysis illustrates the potency of buyers & suppliers in the market.
- The report provides a detailed global rare hematology disorders market analysis based on competitive intensity and how the competition will take shape in coming years.
Questions answered in the rare hematology disorders Market research report:
- Who are the leading market players active in the rare hematology disorders market?
- What are the current trends that will influence the market in the next few years?
- What are the driving factors, restraints, and opportunities in the market?
- What are the projections for the future that would help in taking further strategic steps?
Rare hematology disorders Market Report Highlights
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Key Market Players
Bayer Healthcare AG, Shire Plc, CSL Behring LLC, Bristol Myers Squibb, Novartis AG, Celgene Corporation, Biogen Inc, Amgen Inc, Pfizer Inc, Novo Nordisk A/S