Global Gene Therapy for Hemophilia Market

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  • Type: Common Disease Area
  • Published : March 2021
  • Region: Worldwide
  • Country: Other
  • Report ID : 491

  • Format: PPT, PDF

Global Gene Therapy for Hemophilia Market – Growth, Trends, And Forecasts (2019 – 2028)

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Report Overview

The global Gene Therapy for Hemophilia market size is projected xx billion in 2019 and expected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Gene Therapy is expected to witness fast growth owing to the rigorous Research and Development in the respective field and is expected to grow at the CAGR of 5.04% during the forecast period. Gene therapy is expected to be a significant treatment option for individuals during the forecast period. Many pharmaceutical companies are making progress for generating gene therapy for Hemophilia. The final goal of hemophilia gene therapy is the restoration of a corrected gene for the rest of the life of the patient which is a challenging and yet not accomplished. Hemophilia gene therapy treatment has a low brink for success. Moreover, hemophilia gene therapy is more likely to interact with the immune system if the doses are increased. The scientists and the biopharmaceutical industries are trying their best to cure Hemophilia A and B by gene therapy.

Market Drivers

Increase in funding for R&D activities pertaining to gene therapy and rise in awareness regarding gene therapy are the major factors that drive the global gene therapy market growth. In addition, increase in government support for research in gene therapy, ethical acceptance of gene therapy for cancer treatment, and rise in prevalence of cancer boost the growth of the gene therapy market. However, high cost associated with the treatment and unwanted immune responses are expected to hamper the growth of the gene therapy market. On the contrary, untapped potential for emerging markets are expected to provide significant profitable opportunities for the manufacturers in the gene therapy industry.

1. Global Gene Therapy for Hemophilia Market Overview………………………….
A. Market Size
2. Market Growth Drivers…………………………………………………………………..
A. Rise in Global Prevalence of Gene Therapy for Hemophilia
B. Growing R&D Investments and Reimbursement Policies
3. Gene Therapy for Hemophilia Market Segmentation………………………….
A. By Class
I. Viral Vector
II. Non-Viral Vector
B. Distribution Channel
I. Hospital Pharmacies
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
4. Gene Therapy for Hemophilia Disease Major Market Share…………………..
A. Market Analysis, Insights and Forecast – By Revenue
5. Competitive Landscape………………………………………………………………….
A. Major Players
B. Products in Pipeline
6. Key Company Profiles……………………………………………………………………
A. Pfizer overview, Product & Services, Strategies & Financials
B. Shire PLC Company overview, Product & Services, Strategies & Financials
C. Roche Company, Product & Services, Strategies & Financials
7. Healthcare Policies and Regulatory Landscape………………………………….
A. Policy changes and Reimbursement scenario
8. Factors Driving Future Growth………………………………………………………..
A. New Trends and Development of Gene Therapy for Hemophilia market
B. Future Opportunities
9. Barriers in Future Growth………………………………………………………………………….
10. Conclusion

Market Segmentation

By Class

Based on vector type, the market is categorized into viral vector and nonviral vector. Among these, the viral vector segment was the largest contributor toward the market growth, owing to easier modifications of many viruses such as Lentivirus, Adeno-Associated Virus (AAV), RetroVirus & Gamma RetroVirus to deliver gene therapy drugs, advancements on DNA, RNA and oncolytic viral vectors gene therapy. However, the nonviral vector segment is anticipated to exhibit fastest growth during the forecast period, due to advancements in physicochemical approaches such as physical methods and chemical methods of non-viral vector for gene therapies during preclinical and clinical trials for the treatment of various diseases.

By Disease Indication:

Gene Therapy for Hemophilia is projected to be the fastest growing segment at a CAGR of 5.7%. However, low availability of treatment for the segment is a major challenge for the market growth for this segment. Furthermore, type B constitutes approximately 20% of hemophilia cases globally, out of which 50% of them have factor IX levels greater than 1%.

By Distribution Channel:

In terms of distribution channel, the gene therapy market is segmented into hospitals, clinics, and others. The hospitals segment is expected to have the highest share in the market during the forecast period. The dominance is due to the high availability and accessibility of the treatment methods in such facilities.

By Region:

Geographically, global Hemophilia Gene therapy Market is split into regions viz. North America, Latin America, Middle East & Africa, Asia Pacific, Western Europe and Eastern Europe. North America’s Hemophilia Gene therapy Market is expected to grow because of evolution and progression in the technology and advancements to improve the patients’ health. However, the willingness of payers and government to arrange funding or insurance coverage for Hemophilia Gene therapy is not well established. If the manufacturers that bring Hemophilia Gene therapy to the market have conventional and older hemophilia therapies within their product portfolio their consideration to offer gene therapy for low price may lack as the new technology would disrupt their present market.

Top 5 Major Players

The Gene Therapy for Hemophilia market is moderately competitive and consists of several major players. Few of the key players are developing the novel product and technologies to compete with the existing products while others are acquiring and partnering with the other companies trending in the market. Some of the companies which are currently dominating the market are F. Hoffmann-La Roche AG, Pfizer Inc., BioMarin Pharmaceuticals, uniQure and, Shire PLC.

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:

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  • 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

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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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