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.
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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.
The global enzyme replacement therapy market has been segmented on the basis of:
- Therapeutic Conditions
- Route of Administration
- Distribution Channels
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:
- North America
- Latin America
- Western Europe
- Eastern Europe
- Asia Pacific excluding China & Japan (APECJ)
- Middle East & Africa (MEA)
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:
- Fabry Disease
- Gaucher Disease
- MPS I
- MPS II (Hunter syndrome)
- MPS IVA (Morquio syndrome, type A)
- MPS VI (Maroteaux-Lamy syndrome)
- MPS VII (Sly syndrome)
- Pompe Disease
- 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:
- Hospital Pharmacies
- Specialty Treatment Pharmacies
- Retail Pharmacies