UK Paroxysmal Noctural Hemglobinuria (PNH) Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2575

  • Format: PPT, PDF

UK Paroxysmal Nocturnal Haemoglobinuria Therapeutics Market: Segmented by Diagnosis, Treatment, and Distribution Channels – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

 

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SKU: 2575 Categories: ,

Report Overview

The UK Paroxysmal Nocturnal Haemoglobinuria 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.

The overall incidence rate was estimated at 0.35 cases per 100,000 people per year. This equates to 220 cases newly diagnosed in the UK each year. The overall prevalence rate was 3.81 per 100,000, this equates to an estimated 2,400 prevalent cases in the UK. PNH is believed to affect males and females in equal numbers. The disorder has been described in many racial groups.

Market Growth Drivers

Factors driving the PNH treatment market include a rise in the number of blood & bone marrow related disorders, increase in incidence and prevalence of patients suffering from PNH disease, rise in aging population, growing disposable income coupled with the 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 and anticipated to account for the higher market share over the forecast period.

Market Restraints

However, an increase in the cost of medical equipment, specifically surgical equipment required for stem cell transformation, lack of reimbursement policies, and occurrence of side effects in related currently available treatments may hamper the PNH treatment market over the forecast period.

TABLE OF CONTENTS

1. UK Paroxysmal Nocturnal Haemoglobinuria Therapeutics Market Overview……………………..
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Rise in number of blood and bone marrow related disorders
B. Technological advancements in stem cells transplantation
C. Emergence of biologics & Adoption of novel therapeutics
Market Restraints
A. Increase in cost of medical & surgical equipment
B. Lack of reimbursement policies
3. Major Types of Paroxysmal Nocturnal Haemoglobinuria……………………..
A. Classical Paroxysmal Nocturnal Haemoglobinuria
B. Subclinical Paroxysmal Nocturnal Haemoglobinuria
C. Other Primary Bone Marrow Disorders
4. PNH Therapeutics Market Segmentation…………………..
A. By Diagnosis
I. Complete Blood Count Test
II. Lactate Dehydrogenase Test (LDH)
III. Bilirubin Test
IV. Bone Marrow Examination
V. Flow Cytometry
VI. Others
B. By Treatment
I. Soliris (eculizumab)
II. Transfusions of red blood cells
III. Folic acid therapy
IV. Steroids (e.g., androgens, glucocorticoids)
V. Anticoagulants
VI. Potential adjunctive therapies
VII. Others
C. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. Paroxysmal Nocturnal Haemoglobinuria Major Drugs 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
B. BioCryst Company overview, Product & Services, Strategies & Financials
C. Alnylam Company overview, Product & Services, Strategies & Financials
D. Volution Company overview, Product & Services, Strategies & Financials
E. Akari 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 PNH Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Diagnosis:

On the basis of diagnosis, the PNH treatment market can be classified into

  • Complete Blood Count Test
  • Lactate Dehydrogenase Test (LDH)
  • Bilirubin Test
  • Bone Marrow Examination
  • Flow Cytometry
  • Others

By Treatment:

Based on the treatment, the PNH treatment market can be bifurcated into

  • Soliris (eculizumab)
  • Transfusions of red blood cells
  • Folic acid therapy
  • Steroids (e.g., androgens, glucocorticoids)
  • Anticoagulants
  • Potential adjunctive therapies
  • Others

The treatment of PNH is directed at the specific symptoms that are present in each individual and includes a variety of different therapeutic options. The regulatory authority approved the orphan drug Soliris (eculizumab) as a treatment for PNH disorder.

Pre-complement inhibitor treatment includes transfusions of red blood cells, iron and/or folic acid therapy, steroids (e.g., androgens, glucocorticoids), and anticoagulants.

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 can be continued during eculizumab therapy if the treating hematologist considers it appropriate. For a hemolytic crisis, narcotic pain relief and fluids may be appropriate. Blood transfusions may be required to support hemoglobin levels.

By Distribution Channels:

In terms of distribution channel, the primary hyperoxaluria treatment market can be classified into

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies

In terms of value (around more than 70%), the hospital pharmacies accounted for the leading market share in 2020. Moreover, it is anticipated to witness growth during the forecast period as most of the products are prescription products, which are available in hospital pharmacies.

Top Major Players

The PNH treatment market is highly competitive, and the prominent players in the market have adopted various strategies to garner maximum market share. These include collaboration, product launch, partnership, and acquisition. The major player operating in the market include Alexion Pharmaceuticals Ltd, RA Pharmaceuticals Ltd, Volution Immuno Pharmaceuticals Ltd, Akari Therapeutics Plc, BioCryst Pharmaceuticals Inc, and Alnylam Pharmaceuticals Inc. among others.

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”.

Our analysis methodology involves three critical stages:

Stage I: Market Data Collection

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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:

Data triangulation of various sources and results of the research are carried out by benchmarking with reliable sources such as industry statistics, statistical databases and the company level averages etc.

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.

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analysed and synthesized. A second series of interviews are done if necessary to check and validate. The future opportunities are analysed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results are then, interpreted by considering following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
  • Analyzing current needs and determining penetration to estimate market

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Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

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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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