Global Paroxysmal Noctural Hemglobinuria (PNH) Market Analysis

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  • Type: Rare Disease Area
  • Published : March 2021
  • Region: Worldwide
  • Country: Other
  • Report ID : 195

  • Format: PPT, PDF

Paroxysmal Nocturnal Hemoglobinuria (PNH) Market – Growth, Trends and Forecast 2019-2028

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Report Overview

The global Paroxysmal Noctural Hemoglobinuria (PNH) drugs market size is USD xx billion in 2019 and projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

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 anaemia 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

Market Drivers

Increasing prevalence of blood disorders is driving the market growth. Emergence of biologics that target causal haemolytic defect or abnormal hematopoietic stem cell is boosting the market growth in the forecast period. High unmet demand for drugs in developing countries is also driving the market growth. Rise in investment from government, public & private organizations for the development of novel drugs is acting as catalyst to market growth

Major Paroxysmal Noctural Hemoglobinuria (PNH) Diseases

Based on Disease Indication, the condition has two main subtypes: classical PNH, which includes patients who have evidence of PNH in the absence of another bone marrow failure disorder; PNH in the context of other primary bone marrow disorders, such as aplastic anemia or myelodysplastic syndrome; and subclinical PNH, in which patients have small PNH clones but no clinical or laboratory evidence of hemolysis (red blood cell destruction) or thrombosis (blood clots)

1. Global Paroxysmal Noctural Hemoglobinuria (PNH) Market Overview…………
A. Market Size
2. Market Growth Drivers………………………………………………………………….
A. Rise in Global Prevalence of Paroxysmal Noctural Hemoglobinuria (PNH)
B. Growing R&D Investments and Reimbursement Policies
3. Major Paroxysmal Noctural Hemoglobinuria (PNH) Diseases………………………………
A. Classical PNH
B. Other Primary Bone Marrow Disorders
C. Subclinical PNH
4. Paroxysmal Noctural Hemoglobinuria (PNH) Market Segmentation……………
A. Drug Class
I. Eculizumab
II. ALXN1210
III. Others
B. Distribution Channel
I. Hospital
II. Clinics
III. Others
C. Geography
I. North America Market Analysis
II. Europe Market Analysis
III. Asia-Pacific Market Analysis
IV. Middle-East and Africa Market Analysis
5. Paroxysmal Noctural Hemoglobinuria (PNH) Major Drugs Market Share…………
A. Market Analysis, Insights and Forecast – By Revenue
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles…………………………………………………………………..
A. Novartis Overview, Product & Services, Strategies & Financials
B. Alexion Overview, Product & Services, Strategies & Financials
C. Roche 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 Noctural Hemoglobinuria (PNH) Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Drug Class:

In terms of drugs, the market can be classified into eculizumab (Soliris), ALXN1210, and others

By Distribution Channel:

In terms of distribution channel, the global Paroxysmal Noctural Hemoglobinuria (PNH) treatment market can be divided into hospital pharmacies, retail pharmacies, and online sales. The hospital pharmacies segment generated the highest revenue in 2019 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 Region:

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

Top 5 Major Players

The global Paroxysmal Noctural Hemoglobinuria (PNH) 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. Major players operating in the market include Novartis AG, AM‐Pharma B.V., Alexion, Bayer AG, and Pfizer

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

Primary Interviews: We have developed a network of experts, freelancers and researchers across countries through which we engage with local experts to gather key data points and assumptions around each market. We also engage regularly with some of the best market research agencies such as Atheneum, GuidePoint, GLG, etc. to conduct surveys and interviews, and build intelligence. We have language translators as a part of our team, who between them can cover 30+ languages allowing us to extract better local insights.

Secondary Data Collection: We have developed strong expertise and experience in secondary data collection methods for developing unique data sets and research material. We gather data from multiple reliable sources to maintain the high level of accuracy and consistency. The market data is analysed and forecasted using appropriate statistical and coherent models. The report offers an overall analysis of the market size, growth, market share as well as segment-level analysis of the specific market. Our report includes precise, to the point information related to the overall market, competition, growth drivers, challenges, regulatory updates, and competition.

Data Sources: We have access to multiple highly reliable free and subscription data sources. We have many years of experience to understand which sources are more dependable for what and which to prefer for the reliable and latest information. The key sources of information include the following, but are not limited to:

  • Statistical Databases
  • Company Websites/Annual Reports
  • Trade Publications
  • Online Databases
  • Published Research Reports
  • Whitepapers
  • Press Releases of Key Market Players

Stage II: Market Data Analysis and Statistical Model

Market Trends: We generally look at macro parameter and micro indicators. The macro parameters includes changes in government policies, demand and supply of the market, government intervention programs and major market share. The micro indicators GDP growth, market size, market volume etc. We also understand nuances specific to each country like US, Canada, India, Germany, etc. and have worked across 60+ countries and hence no only understand global trends, but how these differ by country, how payment models, market structure, cultural parameters, etc. differ in each country.

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

Insights: Our reports deliver actionable insights backed with supporting facts and figures to assist you in achieving exemplary growth. Our in-depth analyses are interspersed with relevant insights and statistics to offer an executive-level view of a given market. The description helps in correlating many minor factors affecting the market and its impact to the different segments within the market.
Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

Presentation & Reporting: The market research report is presented in different forms such as charts by using a scientific approach for easy understanding. Historic, current and future analysis is provided for each market in terms of both value and volume. The size of the market is interpreted in the US Dollar value and the respective unit, based on the product, for volume consumptions.
The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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