Acquired Orphan Blood Diseases Therapeutics Market to grow with a CAGR of 7.80%
Increasing
healthcare infrastructure and a supportive regulatory environment are the major
drivers for the Global Acquired Orphan Blood Diseases Therapeutics Market.
According
to TechSci Research report, “Global Acquired Orphan Blood Diseases
Therapeutics Market Industry Size, Share, Trends, Competition, Opportunity and
Forecast, 2018-2028”, Global
Acquired Orphan Blood Diseases Therapeutics Market has valued at USD 7.95
billion in 2022 and is anticipated to witness an impressive growth in the
forecast period with a CAGR of 7.80% through 2028. This can be due to collaborations and
partnerships among leading companies with a diverse approach to merge the
expertise of individual companies and to strengthen their position in the
market.
Patient advocacy and support play a crucial
role in driving the demand for Global Acquired Orphan Blood Diseases
Therapeutics. Patient advocacy groups and organizations dedicated to rare
diseases, including acquired orphan blood diseases, work tirelessly to raise
awareness about these conditions. Their efforts educate the public, healthcare
providers, and policymakers about the existence and challenges faced by
individuals with these diseases. Increased awareness can lead to earlier
diagnosis of acquired orphan blood diseases. Timely diagnosis is essential for
initiating appropriate treatment and managing the condition effectively. Patient
advocacy groups often advocate for increased research funding for rare
diseases. This funding supports scientific research aimed at understanding the
underlying causes of these diseases and developing new therapeutic approaches. Patient
advocacy groups encourage their members to participate in clinical trials for
potential therapies. Their involvement in research is invaluable for advancing
the development of new treatments. Patient advocacy organizations provide
patients and their families with access to information about the latest
advancements in treatments and research. This knowledge empowers patients to
make informed decisions about their healthcare. These groups offer support
networks where patients can connect with others facing similar challenges.
Support from peers who understand their experiences can be emotionally and
practically beneficial. Patient advocacy organizations actively advocate for
improved access to treatments and therapies. They may lobby for policy changes,
raise awareness about insurance coverage issues, and push for fair pricing of
orphan drugs.
Acquired
orphan blood diseases encompass a group of rare blood disorders characterized
by the inability to produce an adequate quantity of red blood cells (RBCs) or
their insufficient presence in the bloodstream. This category includes various
conditions like myelodysplastic syndrome (MDS), idiopathic thrombocytopenic
purpura (ITP), paroxysmal nocturnal hemoglobinuria (PNH), myelofibrosis (MF),
polycythemia vera (PV), and acquired agranulocytosis. These diseases can
disrupt the normal functioning of the bone marrow and lead to a reduction in
platelet counts.
In
October 2022, Pfizer Inc. has successfully finalized its acquisition of Global
Blood Therapeutics, Inc. (GBT), a biopharmaceutical company with a strong focus
on discovering, developing, and delivering transformative treatments,
particularly for underserved patient populations, beginning with sickle cell
disease (SCD). This acquisition underscores Pfizer's dedication to addressing
SCD and further strengthens its position in the rare hematology sector,
building upon a 30-year history in this field. GBT comes with a portfolio and
pipeline that holds the potential to address the comprehensive requirements of
this marginalized community. Among GBT's achievements is the discovery and
development of Oxbryta (voxelotor), a pioneering medication that directly targets
the root cause of SCD. Furthermore, GBT boasts a promising lineup of
preclinical and clinical investigational assets focused on SCD, including
GBT021601 (GBT601) and inclaclumab, both of which have earned Orphan Drug and
Rare Paediatric Disease designations from the U.S. Food and Drug Administration
(FDA).
Complex diagnosis is a significant challenge
in the Global Acquired Orphan Blood Diseases Therapeutics Market. Many acquired
orphan blood diseases are rare, and healthcare providers may have limited
experience or exposure to these conditions. This can lead to misdiagnoses or
delayed diagnoses. Symptoms of acquired orphan blood diseases, such as anemia,
fatigue, and bleeding, can be non-specific and overlap with those of other more
common medical conditions. This makes it challenging to pinpoint the exact underlying
disease. Diagnostic criteria for rare blood diseases may vary among healthcare
providers and institutions, leading to inconsistencies in diagnosis. Accurate
and standardized diagnostic criteria are essential but may be lacking for some
conditions. Specialized laboratory tests, such as flow cytometry, genetic
testing, and bone marrow biopsies, may be required to confirm diagnoses of
acquired orphan blood diseases. Access to these tests may be limited in certain
regions. Some acquired orphan blood diseases, like myelodysplastic syndromes
(MDS), exhibit significant heterogeneity, with variations in disease
presentation and progression. This complexity can make diagnosis more
challenging.
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on "Acquired Orphan Blood Diseases Therapeutics Market.”
Global Acquired Orphan Blood Diseases Therapeutics Market
is segmented based on Therapy, Disease Indication, Distribution Channel, and by
region.
Based on Disease
Indication, Global Acquired Orphan Blood Diseases Therapeutics Market is
segmented into Acquired Agranulocytosis, Acquired Hemophilia, Acquired Von
Willebrand Syndrome, Paroxysmal Nocturnal Hemoglobinuria (PNH), Myelodysplastic
Syndrome, Others. Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare
and serious blood disorder characterized by the abnormal breakdown of red blood
cells, which leads to various symptoms and complications. PNH is caused by a
genetic mutation in bone marrow stem cells, resulting in the deficiency of
certain protective proteins on the surface of red blood cells. These protective
proteins are responsible for safeguarding red blood cells from the body's
immune system. The most common symptoms of PNH include fatigue, weakness,
paleness (pallor), and shortness of breath due to anemia. Patients may also
experience dark urine, which results from the presence of hemoglobin breakdown
products in the urine. In addition to hemolysis and thrombosis, PNH can lead to
bone marrow dysfunction. This may result in reduced production of other blood
cell types, including white blood cells and platelets, increasing the risk of
infections and bleeding disorders.
Based on Region, North America dominated the Global Acquired Orphan Blood Diseases Therapeutics Market.
North America has a strong network of patient advocacy groups and organizations
dedicated to rare diseases. These groups raise awareness, provide support to
patients and their families, and actively engage in research and treatment
development efforts. Many major pharmaceutical companies with global reach are
headquartered or have a significant presence in North America. This enables
these companies to invest in research, development, and commercialization of
orphan blood disease therapies. North America attracts a significant portion of
clinical trial activity for orphan disease therapeutics. The availability of
diverse patient populations and clinical trial infrastructure facilitates the
testing of new treatments. Governments in North America offer incentives to
promote the development of orphan drugs, including tax credits, research
grants, and market exclusivity periods. These incentives encourage
pharmaceutical companies to invest in rare disease therapeutics.
Asia-pacific region to witness fastest growth in the
Global Acquired Orphan Blood Diseases
Therapeutics Market. Awareness about rare diseases, including acquired
orphan blood diseases, has been on the rise in the Asia-Pacific region.
Improved disease recognition and diagnosis contribute to the growing patient
population seeking treatment. The Asia-Pacific region is the most populous in
the world, with a diverse and large population. While the prevalence of each
specific rare disease may be low, the sheer number of individuals in this
region means that a significant number of patients are affected, creating a
substantial market. Many countries in the Asia-Pacific region have experienced
rapid economic growth, resulting in increased healthcare spending and improved
access to healthcare services. This economic development can lead to greater
demand for advanced therapies. Investments in healthcare infrastructure,
including specialized treatment centers, research institutions, and advanced
medical technologies, have been made in the Asia-Pacific region. This
infrastructure supports the diagnosis and treatment of rare diseases.
Some
of the major companies operating in the Global
Acquired Orphan Blood Diseases Therapeutics Market include:
- Alexion
Pharmaceuticals, Inc.
- Amgen,
Inc.
- Celgene
Corporation
- Eli
Lilly and Company
- Sanofi
S.A.
- GlaxoSmithKline
plc,
- Cyclacel
Pharmaceuticals, Inc.
- Onconova
Therapeutics, Inc.
- Incyte
Corporation,
- CTI
BioPharma Corp
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“Certain areas, particularly in North
America, are projected to exert significant demand for Acquired Orphan Blood
Diseases Therapeutics. The growth in the competitive landscape and the presence
of well-established companies in the market, committed to enhance the overall
wellbeing of people each year, are expected to contribute to a remarkable
growth of the Global Acquired Orphan Blood Diseases Therapeutics Market in the
forecast period," said Mr. Karan Chechi, Research Director with TechSci
Research, a research-based Global management consulting firm.
Acquired Orphan Blood Diseases Therapeutics Market – Global Industry
Size, Share, Trends, Opportunity, and Forecast, 2018-2028 Segmented By Therapy (Recombinant Factor, Immunoglobulin
Infusion Therapy, Activated Prothrombin Complex Concentrate, Thrombopoietin
Receptor Agonists, Others), By Disease Indication (Acquired Agranulocytosis, Acquired
Hemophilia, Acquired Von Willebrand Syndrome, Paroxysmal Nocturnal
Hemoglobinuria (PNH), Myelodysplastic Syndrome, Others), By Distribution
Channel (Hospital Pharmacy, Retail Pharmacy, Others), by region, and Competition evaluated
the future growth potential of Global Acquired Orphan Blood Diseases
Therapeutics Market and provides statistics & information on market size, structure,
and future market growth. The report intends to provide innovative market
intelligence and help decision makers take sound investment decisions. Besides,
the report also identifies and analyzes the emerging trends along with
essential drivers, challenges, and opportunities in Global Acquired Orphan
Blood Diseases Therapeutics Market.
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