What is Enzyme Replacement Therapy?
A rare disease is defined as a disease that affects a small percentage of the general population. Drugs used for the treatment of rare diseases are called orphan drugs. Enzyme Replacement Therapy (ERT) is the treatment for rare diseases. According Global genes.org, 80% of rare diseases are of genetic origin. There are approximately 7,000 different types of rare diseases. Approximately 50% of the people affected by rare diseases are children. According to the Kakkis Every life Foundations, FDA-approved drugs are not available for 95% of rare diseases. ERT replaces an enzyme that is absent in the body. ERT is available for diseases such as fabry disease, gaucher disease, mucopolysaccharidosis, Pompe disease, and lysosomal acid lipase deficiency, among others.
The market study is broken down, by Application (Gaucher Disease, Fabry Disease, MPS Disease, Gastrointestinal Disease and Others) and major geographies with country level break-up.
The companies are exploring the market by adopting expansions, investments, new service launches, and collaborations as their preferred strategies. The players are exploring new geographies through expansions and acquisitions across the globe to gain a competitive advantage through combined collaborations. Research Analyst at AMA estimates that Players from United States will contribute to the maximum growth of Global Enzyme Replacement Therapy market throughout the predicted period.
Johnson & Johnson (United States), Sanofi (France), Shire (United States), BioMarin (United States), AbbVie (United States), Alexion (United States), Allergan (Ireland), Horizon Pharma (Dublin), Actelion (Switzerland) and Recordati Rare Diseases (United States) are some of the key players that are part of study coverage. Additionally, the Players which are also part of the research coverage are Digestive Care and Leadiant Biosciences.
AdvanceMarketAnalytics has segmented the market of Global Enzyme Replacement Therapy market by Type, Application and Region.
On the basis of geography, the market of Enzyme Replacement Therapy has been segmented into South America (Brazil, Argentina, Rest of South America), Asia Pacific (China, Japan, India, South Korea, Taiwan, Australia, Rest of Asia-Pacific), Europe (Germany, France, Italy, United Kingdom, Netherlands, Rest of Europe), MEA (Middle East, Africa), North America (United States, Canada, Mexico). If we see Market by Enzyme , the sub-segment i.e. Imiglucerase will boost the Enzyme Replacement Therapy market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth. If we see Market by Route of Administration, the sub-segment i.e. Injectable Agents will boost the Enzyme Replacement Therapy market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth. If we see Market by Distribution Channel, the sub-segment i.e. Hospital Pharmacies will boost the Enzyme Replacement Therapy market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth.
- Advancement in the medical sector
- Increase in healthcare expenditure and prevalence of rare diseases
- Growth in the demand for ERT to improve the treatment of rare diseases
- Increase in government spending on healthcare infrastructure, such as favorable reimbursement for ERT
- Availability of alternative treatment options
- Dearth of skilled healthcare professionals
- lack of regulatory framework
Key Target AudienceNew Entrants/Investors, Analysts and Strategic Business Planners, Enzyme Replacement Therapy Provider, Government Regulatory and Research Organizations and End-Use Industry
About ApproachDuring this study the evaluation and validation of the market size is done through various sources including primary and secondary analysis. AMA Research & Media follows industrial and regulatory standards such as NAICS/SIC/ICB/TRCB, to have the better understanding of the market. The market study is conducted on basis of more than 200 companies dealing in the market regional as well as global areas with purpose to understand companys positioning regarding market value, volume and their market share for regional as well as global.
Further to bring relevance specific to any niche market we set and apply number of criteria like Geographic Footprints, Regional Segments of Revenue, Operational Centres, etc. The next step is to finalize a team (In-House + Data Agencies) who then starts collecting C & D level executives and profiles, Industry experts, Opinion leaders etc. and work towards appointment generation.
The primary research is performed by taking the interviews of executives of various companies dealing in the market as well as using the survey reports, research institute, and latest research reports. Meanwhile, analyst team keeps preparing set of questionnaires and after getting appointee list; the target audience are then tapped and segregated with various mediums and channels that are feasible for making connection that includes email communication, telephonic, skype, LinkedIn Group & InMail, Community Forums, Community Forums, open Survey, SurveyMonkey etc.
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