UK Freidreich’s Ataxia Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2596

  • Format: PPT, PDF

UK Friedreich Ataxia 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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Report Overview

The UK Friedreich Ataxia (FRDA) 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.

Friedreich’s ataxia (FRDA) is the most common inherited ataxia in the UK. FRDA is a genetic, progressive, neurodegenerative movement disorder, that primarily affects the nervous system and the heart with a typical age of onset between 10 and 15 years. The disease begins in childhood resulting in the impairment of muscle coordination that might get worse over time. FRDA is caused by a mutation in a gene labeled FXN and people with FRDA inherit a mutation from each parent, so they have mutations in both copies of their FXN gene. This gene carries the genetic code for the protein ‘frataxin’.

Initial symptoms may include unsteady posture, frequent falling, and progressive difficulty in walking due to impaired ability to coordinate voluntary movements (ataxia). Affected individuals often develop slurred speech (dysarthria), characteristic foot deformities, and an irregular curvature of the spine (scoliosis), and cardiomyopathy. About a third of the people with FRDA develop diabetes mellitus. Estimates from a study suggest that around one person in 30,000 has FRDA. This would mean there are about 2000 people with FRDA in the UK and correspondingly prevalence is about 1.8 per 100,000 in the UK.

Market Growth Drivers

The UK Friedreich ataxia management market is primarily driven by promising pipeline drugs targeting treatment for Friedreich ataxia, an increase in the geriatric population, and the growing application of precision medicine in neurodegenerative diseases. Increasing incidence and prevalence of Friedreich ataxia expected to boost the market growth during the forecast period.

Higher patient population with genetic strains of ataxia, and government initiatives to curb these disorders are anticipated to propel the market during the forecast period and various

support organizations for patient diagnosis & treatment. An increase in the number of drugs in clinical trials and a rise in research & development expenditure for medication treating neurological conditions is projected to drive the market.

Market Restraints

However, an increase in the cost of medical treatment, lack of awareness of rare diseases, and occurrence of side effects in related currently available treatments may hamper the Friedreich ataxia treatment market growth.

TABLE OF CONTENTS

1. UK Friedreich Ataxia Therapeutics Market Overview………………….……
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Increasing Incidence and Prevalence of Friedreich Ataxia
B. Government Initiatives and Support Organisations Propels Market Growth
C. Numerous Drugs in Clinical Trials and Rise in R&D Expenditure
Market Restraints
A. Huge Treatment Costs
B. Lack of Awareness
3. Major Clinical Forms of Friedreich Ataxia
A. Late-onset Friedreich Ataxia (LOFA)/ Very Late-onset Friedreich Ataxia (VLOFA)
B. Friedreich Ataxia with Retained Reflexes (FARR)
4. Friedreich Ataxia Therapeutics Market Segmentation………………………….
A. By Diagnosis
I. Nerve Conduction Studies
II. Blood Tests for FBC, U&E, Glucose
III. Genetic Counselling
IV. Echocardiography (ECH)
V. Magnetic Resonance Imaging (MRI)
VI. Vitamin E Deficiency
VII. Others
B. By Treatment
I. Physical Therapy
II. Speech Therapy
III. Occupational Therapy
IV. Gene Therapy
V. Orthopaedic Surgery
VI. Anti-arrhythmic Agents
VII. Anti-cardiac Failure Medications
VIII. Oral Hypoglycaemic Agents
IX. Other Medications
C. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. Friedreich Ataxia 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. Reata Company overview, Product & Services, Strategies & Financials
B. Agilis Company overview, Product & Services, Strategies & Financials
C. PTC Company overview, Product & Services, Strategies & Financials
D. Apopharma Company overview, Product & Services, Strategies & Financials
E. Minoryx 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 Friedreich Ataxia Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Diagnosis:

Based on the diagnosis, the Friedreich ataxia suspected in an individual based on clinical examination, thus FRDA treatment market can be bifurcated into

  • Nerve conduction studies
  • Echocardiography
  • Magnetic resonance imaging (MRI)
  • Genetic counseling
  • Vitamin E deficiency
  • Specialized blood tests (for estimation of FBC, U&E, and glucose)
  • Others

The FRDA diagnosis is generally confirmed by molecular genetic testing to look for mutations in the FXN gene that cause the disease.

By Treatment:

In terms of treatment type, the Friedreich ataxia treatment market can be segmented into

  • Physical therapy
  • Speech therapy
  • Occupational therapy
  • Gene therapy
  • Orthopedic surgery
  • Anti-arrhythmic agents
  • Anti-cardiac failure medications
  • Oral hypoglycaemic drugs
  • Other medications

The pharmacotherapy segment accounted for a major market share due to better efficacy to treat symptoms, long-lasting effects, and pipeline products. Thus, projected to exhibit strong growth during the forecast period.

By Distribution Channels:

In terms of distribution channels, the Friedreich ataxia treatment market has been classified into

  • Hospital Pharmacies
  • Retail pharmacies
  • Online pharmacies

The hospital pharmacies segment is anticipated to dominate the market during the forecast period, due to the large inventory maintained by hospitals and the availability of essential drugs at hospital pharmacies.

Top Major Players

The UK Friedreich ataxia disorder treatment market is highly competitive and provides a great opportunity for market players to account for the maximum market share. Numerous companies having drugs in the pipeline have received positive results in clinical trials. As Omaveloxolone registered positive results as a potential therapy for Friedreich ataxia. Thus, the prominent players in the Friedreich ataxia treatment market include Reata Pharmaceuticals Inc, Agilis Biotherapeutics, PTC Therapeutics Inc. Apopharma Corporate, and Minoryx Therapeutics 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

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
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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

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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