Australia Paroxysmal Noctural Hemoglobinuria (PNH) Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: APAC
  • Country: Australia
  • Report ID : 4075

  • Format: PPT, PDF

Australian Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market: Segmented by Therapeutics, by Diagnosis and by Distribution Channel – Growth, Trends, and Forecasts (2020 – 2028F)

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Report Overview

The Australian Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market size stood at around USD XX billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Paroxysmal Nocturnal Haemoglobinuria (PNH) is a very rare and debilitating disease of the bone marrow that affects the blood and major organs. It is a chronic, life-threatening illness and can have devastating effects upon a patient physically, mentally and emotionally.

PNH is an acquired illness caused by an injury to a bone marrow stem cell and is not hereditary. The bone marrow produces all blood cells for the body, being white cells, red cells and platelets. PNH occurs when a gene required for producing normal blood cells becomes mutated resulting in a PNH clone of bone marrow cells that produce defective blood cells. The white blood cells and platelets do not function as effectively as normal cells, leaving patients prone to infections and blood clots. While all three types of blood cells are affected by PNH, most of the symptoms experienced by patients are due to the destruction of red blood cells.

PNH is believed to affect males and females in equal numbers, although some studies show a slight female preponderance. The prevalence is estimated to be between 0.5-1.5 per million people in the general population. The disorder has been described in many racial groups.

Market Drivers

Factors driving the Paroxysmal Nocturnal Hemoglobinuria Therapeutics market include rise in number of blood & bone marrow related disorders, increase in aging population, growing disposable income coupled with unmet need of treatment of PNH and technological advancements in stem cells transplantation. In addition to this, the emergence of biologics and adoption of novel therapeutics are further pushing the market growth. However, increase in cost of medical equipment, specifically surgical equipment required for stem cell transformation; lack of reimbursement policies; and occurrence of side effects in related current available treatments may hamper the Paroxysmal Nocturnal Hemoglobinuria Therapeutics market.

 

 

1. Australian Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market Overview…………………………………………………………………………………………………
A. Market Size
2. Market Growth Drivers……………………………………………………………………
A. Increase in Awareness Levels of Rare Diseases
B. Rising Number of Blood and Bone Marrow related Disorders
C. Development in Stem Cell Research
3. Major PNH Types…………………………………………………………………………….
A. Classical PNH
B. Other Primary Bone Marrow Disorders
C. Subclinical PNH
4. Australian PNH Therapeutics Market Segmentation…………………………………..
A. By Therapeutics
I. Pre-complement inhibitor treatment
II. Complement inhibitor treatment
III. Eculizumab (Soliris®) / ravulizumab (Ultomiris)
IV. Potential adjunctive therapies
V. Curative therapy – Bone marrow Transplantation
B. By Distribution Channel
I. Hospital pharmacies
II. Retail pharmacies
III. Online pharmacies
C. By Diagnosis
I. Complete Blood Count Test
II. Lactate dehydrogenase test (LDH)
III. Bilirubin test
IV. Bone marrow examination
V. Flow Cytometry
VI. Others
5. Australian PNH Major Products Market Share………………………………………
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape…………………………………………………………………………….
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………………………..
A. Alexion Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape…………………………………….
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………………………
A. New Trends and Development of Paroxysmal Nocturnal Hemoglobinuria Therapeutics market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Therapeutics:

On the basis of therapeutics, the market is segmented into Pre-complement inhibitor treatment, Complement inhibitor treatment, Eculizumab (Soliris®) / Ravulizumab (Ultomiris), potential adjunctive therapies & Curative therapy – Bone marrow Transplantation. The therapeutics of PNH is directed at the specific symptoms that are present in each individual and includes a variety of different therapeutic options. Australian Therapeutic Goods Administration (TGA) approved the orphan drug Soliris (eculizumab) as a treatment for PNH. This is the first drug to be approved for this disorder.

Pre-complement inhibitor treatment

Treatment of PNH before monoclonal antibody focused on transfusions of red blood cells, iron and/or folic acid therapy, steroids (e.g. androgens, glucocorticoids) and anticoagulants (to treat thrombotic complications such as Budd-Chiari Syndrome.

Complement inhibitor treatment

Treatment of PNH by complement inhibitor represents a major advance in treatment. The currently available complement inhibitor (eculizumab) is a monoclonal antibody that is administered through intravenous infusion.

Potential adjunctive therapies

Supportive treatments can be continued during eculizumab therapy if the treating haematologist considers it appropriate. For a haemolytic crisis, narcotic pain relief and fluids may be appropriate. Blood transfusions may be required to support haemoglobin levels.

Complement inhibitor treatment has gained major market share in 2020.

By Distribution Channel:

In terms of distribution Channel, the Paroxysmal Nocturnal Hemoglobinuria Therapeutics market can be divided into hospital pharmacies, retail pharmacies, and online pharmacies. The hospital pharmacies segment generated the highest revenue in 2020 and is anticipated to witness growth during the forecast period due to most of major products are prescription products, which is available in hospital pharmacies.

By Diagnosis:

On the basis of diagnosis, the Paroxysmal Nocturnal Hemoglobinuria Therapeutics market is segmented into complete blood count test, lactate dehydrogenase test (LDH), bilirubin test, bone marrow examination, flow cytometry & others. Flow cytometry is the required method of diagnosis for patients with Paroxysmal Nocturnal Hemoglobinuria disease.

Top Player

The Paroxysmal Nocturnal Hemoglobinuria Market is limited. Major player operating in the market include Alexion Pharmaceuticals Australasia Pty Ltd.

 

 

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision making for business stakeholders. Our focused approach to develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

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Market Sizing and Analysis: Our expert data analytics team has created various market forecast models by employing the top-down approach i.e. start with large overall market and segment different areas and bottom-up approach i.e. start with population and epidemiology and roll up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape and policy and regulatory scenario and the quantitative analysis covers different market estimates and forecasts.

Data Triangulation & Validation:

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We make sure to finalize the numbers in alignment to the market research. Firstly, our internal experts ensure thorough validation and checking to ensure the accurate and precise analysis and then validation is also done using multiple data analysis model.  Two level validation is done and entails finalization of report scope and to way of representation pattern.

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The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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