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 market for orphan diseases 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 Orphan diseases market share is anticipated to reach USD 328.5 billion by 2030 at 10.70% of CAGR during the forecast timeframe 2022-2030.
The orphan diseases market has witnessed significant growth in recent years, driven by advancements in genetic research and a growing understanding of rare and often life-threatening conditions. Orphan diseases, also known as rare diseases, affect a small percentage of the population, making them a niche but crucial focus for pharmaceutical companies. This market presents unique challenges and opportunities, as regulatory incentives such as orphan drug designations have encouraged drug development in this space. Patients with orphan diseases have long faced limited treatment options, but the orphan diseases market is changing that narrative by fostering innovation and providing hope for those who once had none.
Understanding Orphan Diseases
Orphan diseases are characterized by their rarity, typically affecting fewer than 1 in 2,000 individuals in the United States. These conditions can be genetic, autoimmune, infectious, or degenerative in nature. What sets orphan diseases apart is the significant unmet medical need they represent. Patients with orphan diseases often face delayed diagnoses, limited treatment options, and a lack of research into their conditions.
Challenges in the Orphan Diseases Market
- Limited patient populations: The small number of patients affected by each orphan disease makes it economically challenging for pharmaceutical companies to invest in research and development. The potential return on investment is typically lower compared to more common diseases.
- High development costs: Developing treatments for orphan diseases can be cost-intensive due to the need for specialized research, clinical trials, and manufacturing processes. These costs can deter pharmaceutical companies from pursuing orphan drug development.
- Regulatory hurdles: Regulatory agencies, such as the U.S. Food and Drug Administration (FDA), have established specific pathways and incentives to encourage orphan drug development. However, navigating these regulatory processes can still be complex and time-consuming.
- Access and affordability: Even when orphan drugs receive approval, patients may struggle to access these treatments due to their high costs. Health systems and insurers may be reluctant to cover the expenses associated with orphan disease treatments.
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 America orphan diseases market 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.
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).
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