Orphan diseases are also referred to as rare diseases. Despite the low market interest, treatment demand continues to grow for orphan diseases. Orphan diseases represent a very small percentage of the global population. However, prevalence rate has increased considerably in recent years. Most orphan diseases are genetic and may remain in patient throughout their life. While many orphan diseases are low-risk and do not threaten life, some may have severe impact on the health. Approximately 7000 different rare diseases are listed, however, no exact figure is available. 

The Orphan Diseases Market Size is growing swiftly. Rising level of awareness and increased funding is reflecting favourably on the market. According to a study conducted by market research future, The global Orphan Drugs Market size is projected to reach 4,35,686.9 million at a CAGR of 11.8% from 2022 to 2030.

Demand for orphan drugs has grown substantially in recent years. Many governments all around the world are financially incentivising drugs through regulations. A data released by National Organization for Rare Disorders (NORD), reveals that in 2018, nearly 30 Mn Americans suffer from 7,000 rare diseases. More and more initiatives are taken towards improving orphan disease care. This is notable for advanced countries such as the U.S., Japan and EU countries. Over the past couple years, orphan drug sales have doubled. 

Factors such as increased prevalence and rising healthcare expenditure are making the market more attractive. Commercial interest in orphan diseases drugs have improved in recent years. Advanced are likely to display a higher enthusiasm. When it comes of research and development smaller firms and start-ups are more active, however some of the bigger players are also positioning themselves to enter the segment. In the comings, collaborations, partnerships and MA activities are expected to increase in the global orphan diseases market. Big pharmaceutical companies are expected to focus on improving their market position during the forecast period. 

Competitive Landscape

Some of the top-notch companies operating in the global orphan diseases market include F. Hoffmann-La Roche Ltd (Switzerland), Vertex Pharmaceuticals Incorporated (US), CELGENE CORPORATION (US), Merck Co., Inc (US), Novartis AG (Switzerland), Bristol-Myers Squibb Company (US), AbbVie Inc. (US), Sanofi (French), Alexion (US), GlaxoSmithKline plc. (UK) and Pfizer Inc. (US).

Global Orphan Diseases Market: Segmental Overview 

MRFR’s report includes a detailed segmental analysis of the market based on disease, treatment and end user. 

On the basis of disease, the market has been segmented into genetic disorders, cancer, cardiovascular diseases, respiratory disorders, autoimmune disorders, neurological disorders, blood disorders, eye disorders, digestive disorders, growth disorder and others. On the basis of treatment, the market has been segmented into drug therapy, cell therapy, gene therapy and others. Based on end user, the market has been segmented into research laboratory, hospital and clinics and others. 

Global Orphan Diseases Market: Regional Segmentation 

The regions covered in the market include Asia Pacific (APAC), North America, Europe, Latin America, and the Middle East Africa (MEA). Globally, North America represents the largest market for orphan diseases. Widespread awareness, strong government support and presence of robust healthcare system supports the market growth in the region. North Rare Disease and Orphan Drugs is expected to post a sound growth during the forecast period. Europe also holds significant market potentials and second largest contributor to the global market for orphan diseases in terms of revenue. North America and Europe are followed by Asia Pacific (APAC). In fact, the APAC orphan diseases market is likely to witness an impressive growth over the next couple of years. 

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