UK Mucopolysaccharidosis I (MPS-I) Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2585

  • Format: PPT, PDF

UK Mucopolysaccharidosis I Therapeutics Market: Segmented by Therapeutics, Route of Administration, Distribution Channels, and End Users – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

 

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Report Overview

The UK Mucopolysaccharidosis I 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.

Mucopolysaccharidosis I (MPS I) is a member of a group of hereditary metabolic diseases known as the mucopolysaccharidoses which, in turn, are part of a larger group of diseases known as lysosomal storage disorders (LSDs). Mutations in IDUA gene cause MPS I. There are seven forms of mucopolysaccharidosis. In individuals with MPS disorders, including MPS I, deficiency or improper functioning of lysosomal enzymes leads to an abnormal accumulation of a particular complex carbohydrate known as glycosaminoglycans (GAGs). When cells cannot break down these glycosaminoglycans they then accumulate within various tissues, such as the bones, joints, brain, spinal cord, heart, spleen, or liver, and this leads to the symptoms that MPS I individuals have.

Generally, most of the affected people appear healthy at birth and experience a period of normal development, followed by a decline in physical and/or mental function. As the condition progresses, it may affect appearance, physical abilities, organ and system functioning, and in most cases, cognitive development.

Market Growth Drivers

The growing necessity for advanced therapeutics will impel companies to launch novel therapies, which in turn, will boost the MPS I treatment market growth in the forthcoming years. Moreover, the growing unmet clinical needs of patients along with better treatment outcomes will spur demand for the MPS I treatment market during the forecast period.

In addition, the increasing R&D by pharmaceutical companies for the mass production of conventional medications or therapies to treat mucopolysaccharidosis, this factor will aid the MPS I treatment market revenue in the foreseeable future.

Market Restraints

The high cost of therapeutics coupled with the poor diagnosis is expected to hinder the growth of the MPS I treatment market during the forecast period. Another key limiting factor is the delay associated with the diagnosis of mucopolysaccharidosis.

TABLE OF CONTENTS

1. UK Mucopolysaccharidosis I Therapeutics Market Overview…………
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Robust Products Pipeline & Introduction of Novel Therapies
B. Rising Government Initiatives and Increased R&D Activities
C. Rise in Prevalence and Incidence of Mucopolysaccharidosis I (MPS I)
Market Restraints
A. High Cost of Approved Therapeutics
B. Poor Diagnosis
3. Major Clinical Forms of Mucopolysaccharidosis I……………………………………
A. Scheie Syndrome (Milder Form)
B. Hurler-Scheie Syndrome (Intermediate Form)
C. Hurler Syndrome (Severe Form)
4. Mucopolysaccharidosis I Therapeutics Market Segmentation………………..
A. By Therapeutics
I. Enzyme Replacement Therapy (ERP)
II. Stem Cell Therapies (SCT)
III. Bone Marrow Transplantation
IV. Umbilical Cord Blood Transplantation
V. Others
B. By Route of Administration
I. Intravenous
II. Intracerebroventicular (ICV)
III. Intrathecal
IV. Others
C. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
D. By End Users
I. Hospitals
II. Speciality Clinics
III. Medical Research Centres
IV. Home Infusion
V. Others
5. Mucopolysaccharidosis I 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. Genzyme Company overview, Product & Services, Strategies & Financials
B. Takeda Company overview, Product & Services, Strategies & Financials
C. Multiplex 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 Mucopolysaccharidosis I Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Therapeutics:

On the basis of therapeutics, the MPS I treatment market is segmented into

  • Enzyme Replacement Therapies (ERT)
  • Stem Cell Therapies (SCT)
  • Bone Marrow Transplantation
  • Umbilical Cord Blood Transplantation
  • Others

ERT consists of replacing the deficient or absent enzyme with a functional recombinant version through intravenous administration. ERT is the only major therapeutics approved by regulatory agencies for the treatment of MPS I, hence in 2020, it has been found that the ERT segment dominated the market share. Therefore, the robust growth of the ERT segment can be anticipated during the forecast period.

By Route of Administration:

Based on the route of administration, the MPS I treatment of the market can be segmented into

  • Intravenous (IV)
  • Intracerebroventricular (ICV)
  • Intrathecal
  • Others

The intravenous type is anticipated to account for the majority of the revenue share of the route of administration segment. Because most of the therapeutics for MPS I treatment are administered by intravenous injections only, thus holds the monopoly over the MPS I treatment market. Therefore, further projected to control its market share during the forecast period.

By Distribution Channels:

In terms of distribution channels, the MPS I treatment market has been bifurcated into

  • Hospital pharmacies
  • Retail pharmacies
  • 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 the major products are prescription products, which are available in hospital pharmacies.

By End Users:

Based on end-user, the MPS I treatment market is segmented into

  • Hospitals
  • Specialty Clinics
  • Medical Research Centres
  • Home-infusion
  • Others

The key factor aiding in the dominance of the hospital segment is the appropriate and safe administration of critical therapeutics used in MPS I treatment by trained medical professionals who can administer only in hospitals with care and proper guidelines. Thus, the potential growth of the hospital segment can be anticipated during the forecast period.

Top Major Players

The UK MPS I treatment market is highly a competitive one and the prominent players in the market have adopted various strategies to garner maximum market share. Major players operating in the MPS I treatment market include Genzyme Corporation, Takeda Pharmaceuticals Pvt Ltd (acquired Shire Plc. In 2019), and Multiplex Pharma Holdings LLC 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

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
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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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