Global Orphan Diseases Market - Overview
An Orphan Diseases Market is defined as a disorder that affects fewer than 200,000 people in America annually. This includes diseases as familiar as cystic fibrosis, Lou Gehrig's disease, and Tourette's syndrome, and as unaware as Hamburger disease, Job syndrome, and acromegaly, or "gigantism." Some diseases have patient populations of less than a hundred. Mutually, however, they affect as many as 25 million Americans every year, according to the National Institutes of Health (NIH), and that makes the diseases--and finding treatments for them-a serious public health concern.
New rare diseases are revealed every year. Most are hereditary and caused by adjustments or defects in genes (mutations). Others can be developed as a result of environmental and toxic conditions. Genes are pieces of DNA, part of the code that regulates the traits and discrete characteristics of all living things. Each human cell contains around 30,000 genes. Besides impelling features such as eye and hair colour, genes also can play a role in the development of diseases and in their transmission from parent to child.
As distinct as rare diseases are, patients share many common frustrations. For example, for one-third of people with a rare disease, getting a precise diagnosis can take one to five years. And people often are so isolated that they may never know anyone else with the same disease. Patients often must travel long distances to visit the few doctors knowledgeable about their diseases, and the costs involved with diagnosis, treatment, and other related expenses can be inflated
Before the passage of uncommon diseases laws in the United States, patients diagnosed with a rare disease were deprived of access to effective medicines because prescription drug manufacturers hardly could make a profit from marketing drugs to such small groups. Subsequently, the prescription drug industry did not effectively fund research for orphan product development. Other potential sources, such as research hospitals and universities, also required the capital and business proficiency to develop treatments for small patient groups. In spite of the urgent health need for these medicines, they came to be known as orphans because companies were not concerned in adopting them.
This changed in 1983 when Congress passed the Orphan Drug Act (ODA). The ODA bent financial incentives for drug and biologics manufacturers, comprising of tax credits for costs of clinical research, government grant funding, assistance for clinical research, and a seven-year period of élite marketing given to the first sponsor of an orphan-designated product who achieves market approval from the Food and Drug Administration for the same indication. At the same time, federal programs at the FDA and the NIH initiated encouraging product development, as well as clinical research for products directing rare diseases
Further With a robust pipeline in place, it is analysed where the industry’s current vision lies and highlighted a number of efforts which have been taken by pharmaceutical companies, healthcare organizations and other industry stakeholders. Precisely, our market potential analysis outlines key patient demographics and underscores potential areas estimated to emerge as major revenue initiators for the pharmaceutical companies.
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.
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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