Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare blood disease of bone marrow stem cells, which are genetically characterized by the somatic mutation in the phosphatidylinositol glycan protein A (PIG-A) gene. PNH generally occurs in the early 30s. Around 10% patients develop PNH symptoms at 21 years of age or earlier. Around 1 to 5 individuals per million people in the U.S. are estimated to suffer from PNH. This is much lower than the incidence rate of bone marrow aplasia. PNH often goes unrecognized; delay in diagnosis may range from one year to more than 10 years.

The global PNH treatment market is anticipated to expand at a rapid pace during the forecast period. It is a niche market, with many pharmaceutical and biotech companies investing in research of bone marrow stem cells. According to current studies, the ideal treatment available is to replace all the hematopoietic stem cells with normal stem cells via stem cells transplantation. However, this treatment is not ideal in some cases as stem cell transplantation requires a stable histocompatible donor. Complete stem cells transplantation is usually considered in severe cases of PNH, for instance aplastic anemia and transformation to leukemia, as these can be life threatening complications. Factors driving the PNH treatment market include rise in number of blood & bone marrow related disorders, increase in aging population, and technological advancements in stem cells transplantation. However, increase in cost of medical equipment, specifically surgical equipment required for stem cell transformation; lack of reimbursement policies in developing regions; and occurrence of side effects in related current available treatments may hamper the PNH treatment market.

The global PNH treatment market can be segmented based on diagnosis test, type of treatment, drugs, and end-user. In terms of diagnosis test, the market can be divided into complete blood count test (CBC), lactate dehydrogenase test (LDH), bilirubin test, bone marrow examination, urine test for hemosiderin, flow cytometry, and others.  Based on the type of treatment, the PNH treatment market can be segregated into treatment of PNH patients associated with hemolysis, treatment of PNH patients associated with thrombosis, treatment of PNH patients associated with non-hemolytic anemia, allogeneic stem cell transplant (SCT)/bone marrow transplant (BMT), treatment of pregnant PNH patients, treatment of pediatric PNH patients, and others. In terms of drugs, the market can be classified into eculizumab (Soliris), ALXN1210, and others.  Based on end-user, the PNH treatment market can be split into hospitals, pharmaceutical & biotech companies, clinics, academic & research institutes, and others.

Geographically, the market for PNH treatment can be divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa (MEA). North America dominates the global PNH treatment market due to the rise in the number of blood & bone marrow related diseases, availability of satisfactory reimbursement policies, and increase in awareness about the early diagnosis of the disease in the region. The market in Europe is also expected to expand rapidly, as key players are collaborating with research institutions and labs to develop new innovative products. The PNH treatment market in Asia Pacific is anticipated to expand at a fast pace owing to the unmet needs regarding PNH treatment of the growing population. Additionally, factors such as development of the health care network, rise in disposable income, increase in health care awareness, and availability of reimbursement facilities are boosting the PNH treatment market in Asia Pacific.

Key players operating in the PNH treatment market include Alexion Pharmaceuticals, Inc., Thermo Fisher Scientific Inc., GE Healthcare, and Johnson & Johnson.

This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

  • Customer Experience Maps
  • Insights and Tools based on data-driven research
  • Actionable Results to meet all the business priorities
  • Strategic Frameworks to boost the growth journey

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

The following regional segments are covered comprehensively:

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • The Middle East and Africa

The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.

Below is a snapshot of these quadrants.

1. Customer Experience Map

The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

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The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.

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The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

9. What are some of the value-grab opportunities in various segments?

10. What will be the barrier to entry for new players in the market?

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Paroxysmal Nocturnal Hemoglobinuria Treatment Market

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