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 report classifies the global ERT market by therapeutic condition, route of administration, distribution channels, and regions.
Market Value and Forecast
The global ERT market is expected to grow at a CAGR of 6.5% over the forecast period of 2018–2028 to reach US$ 13,776.2 Mn by 2028. By country, the U.S. dominated North America ERT market and is expected to be the most lucrative market in the region.
Increase in healthcare expenditure and prevalence of rare diseases are among prime factors driving the ERT market. Growth in the demand for ERT to improve the treatment of rare diseases is anticipated to drive the market over the forecast period. Increase in government spending on healthcare infrastructure, such as favorable reimbursement for ERT, is expected to boost the market. In addition, socioeconomic factors such as increase in the prevalence of rare diseases, are among factors driving the market globally.
However, availability of alternative treatment options, dearth of skilled healthcare professionals, and lack of regulatory framework in emerging economics are some of the factors expected to restrain market growth over the forecast period.
Market Segmentation by Therapeutic Condition
Based on therapeutic condition, global ERT market has been segmented into fabry disease, gaucher disease, mucopolysaccharidosis, Pompe disease, lysosomal acid lipase deficiency, and others. Among these, the lysosomal acid lipase deficiency segment is anticipated to register the highest CAGR of 7.5% over the forecast period. The mucopolysaccharidosis segment is expected to register a market share of 24.0% in 2028.
Market Segmentation by Route of Administration
Route of administration of ERT covered in this report includes oral and injectable. The injectable segment held the highest market share and this trend is expected to continue over the forecast period owing to increase in the demand for injectable ERT drugs. In addition, this segment is expected to register a market share of 86.6% in 2028.
Market Segmentation by Distribution Channel
Distribution channels of ERT covered in this report include hospital pharmacies, specialty treatment pharmacies, and retail pharmacies. The specialty treatment pharmacies segment held the largest market share and this trend is expected to continue over the forecast period. The hospital pharmacies segment is also anticipated to occupy a significant market share over the forecast period.
Geographically, the global ERT market has been segmented into eight major regions: North America, Latin America, Western Europe, Eastern Europe, Asia Pacific excluding China and Japan (APEJ), China, Japan, and Middle East & Africa (MEA). In terms of revenue, North America has been estimated to account for the highest share of the global ERT market with approximately 25%-30% by the end of 2017. On the other hand, revenue from the market in Western Europe is expected to grow significantly at a CAGR of 5.4% over the forecast period.
This Future Market Insights report examines the ‘Enzyme Replacement Therapy Market’ for the period 2013–2017 and provides forecasts for 2018–2028. The primary objective of the report is to offer updates and in-depth analysis of the global market opportunities for Enzyme Replacement Therapy (ERT).
Increase in the global demand for ERT for the treatment of rare diseases is anticipated to drive the market over the forecast period. Increase in government spending on healthcare infrastructure such as favourable reimbursement for ERT is expected to boost the global market. Growth in the number of research initiatives with the help of non-profit organisations and increase in government support are also among factors expected to fuel market growth.
However, the availability of alternative treatment options and high treatment cost are some of the factors that are expected to hamper the growth of the global market. Dearth of skilled healthcare professionals and lack of regulatory framework in emerging economics are also likely to restrain the global market over the forecast period.
The global enzyme replacement therapy market has been segmented on the basis of:
Route of Administration
The report analyses the global ERT market in terms of value (US$ Mn). The report begins with the definition of the market and explains different therapeutic conditions. The market viewpoint section includes FMI’s analysis on key trends, drivers, restraints, opportunities and macro factors that influence the global market. Opportunity analysis provided in this section equips clients with crystal clear decision-making insights.
The report analyses the market based on regions and presents the forecast in terms of value for the next 10 years. Regions covered in the report include:
Asia Pacific excluding China & Japan (APECJ)
Middle East & Africa (MEA)
Each of these sections analyse regional markets by therapeutic condition, route of administration, distribution channels and country. The regional market dynamics section provides key growth trends pertaining to each region. The representative market participants section consists of a list of companies that operate in different regions, competitive landscape and intensity map of their presence in each region.
Weighted average selling price is considered to estimate the market size of various devices mentioned in the scope of the study. The country price is captured with the local currency. The local currency figures are converted into USD to offer forecasts in a consistent currency standard. Furthermore, FMI also considers the latest annual exchange rate to reflect the impact of the most recent economic conditions of country. Prices considered in these models are standardised based on the average cost per ERT.
The forecast presented in the report provides total revenue of approved ERT over 2018–2028. In addition, we have taken into consideration the year-on-year growth to understand the predictability of the market and to identify the right growth opportunities in the global market.
In the next section of the report, a ‘Competitive Landscape’ is included to provide a dashboard view and to access the key differentiators among competitor firms. This section is primarily designed to provide clients an objective and detailed comparative assessment of product offerings and strategies of key providers specific to market segments. Detailed profiles of market players are also provided in the report, which highlight company description, overview, product/segment overview, SWOT analysis, financial information, key developments related to the market and strategic overview.
The next section of report analyses the market based on therapeutic conditions and presents the forecast in terms of value for the next 10 years. Therapeutic conditions covered in the report include:
MPS II (Hunter syndrome)
MPS IVA (Morquio syndrome, type A)
MPS VI (Maroteaux-Lamy syndrome)
MPS VII (Sly syndrome)
Lysosomal Acid Lipase Deficiency
The next section of report analyses the market based on the route of administration of ERT and provides forecast in terms of value for the next 10 years. On the basis of route of administration, the market is segmented into:
The next section of report analyses the market based on the distribution channels of ERT and provides forecast in terms of value for the next 10 years. On the basis of distribution channel, the market is segmented into:
Specialty Treatment Pharmacies
Another key feature of this report is the analysis of key segments in terms of absolute dollar opportunity. This is traditionally overlooked while forecasting the market. However, absolute dollar opportunity is critical for assessing the level of opportunity that a provider can look to achieve as well as to identify potential resources from a sales and delivery perspective of products. To understand key growth segments, Future Market Insights provides the ‘Attractiveness Index’ analysis that helps clients identify real market opportunities.