Lastly, the study of the pathogenesis of disease at genetic and molecular level studies have led to the discovery of new targets for therapy and producing disease modifying drugs.
LONDON, UK. 29th January 2018: Visiongain has launched a new pharma report Orphan Drugs Market Report 2018-2028: Forecasts and Analysis By Therapeutic Class (Oncology, Alimentary Tract & Metabolism, Hormonal Disorders & Haematology, Neuroscience, Respiratory, Cardiovascular, Nephrology and Ophthalmology), Drug Class (Biologic, Small Molecule) & By Region Plus Analysis of Leading Companies
An orphan disease is also known as rare disease where the health condition affects a smaller number of people that is often serious and life threatening as compared with other prevalent diseases in general population around the world. Many rare diseases exist due to genetic abnormalities that can be present throughout the person’s life even the symptoms may not appear immediately. In addition, some rare diseases arise from exposure to toxin, infections or as an adverse reaction to other therapeutic treatments. As per the Global Genes estimation, around 7,000 rare diseases exist worldwide. Approximately 50 percent of the people affected by rare diseases are children.
The first Orphan drug act was passed in the US in 1983 to facilitate the development of orphan drugs. Under the orphan drug designation incentives are given to the sponsors to develop a new drug for rare disease. These incentives includes a partial tax credit for clinical trial expenditures, marketing exclusivity for years and waived user fees.
“Orphan drugs are drugs that have been developed to treat rare diseases which affect only a small portion of the population. In the US alone, the FDA reports that nearly 7000 rare diseases are affecting approximately 30 million people in 2015. Orphan drugs are generally faster to develop as their target population is very small and usually has a high level of genetic similarity between patients. Governments usually incentivise the development of orphan drugs in order to encourage production and this includes exclusivities on patents, along with relaxed regulations for approval from the FDA and financial advantages.
Companies who see the advantages of patent exclusivity, price protection, and less strict regulations have become interested in producing orphan drugs and can charge higher drug prices to this very specific and small segment of the patient population. In addition, Orphan drug are priced at higher rate because of the various cost associated with the drug development process such as R&D cost and advanced technologies involved. This in turn acts as a booster for more and more pharmaceutical and biotech companies to get involved in the orphan drug industry.”
The Global market is highly competitive with many different players entering the market. The report discusses products from leading companies in the market. The report discusses products from leading companies in the market. Notable companies in the Orphan Drugs market include Hoffmann-La Roche, Celgene, Bristol-Myers Squibb, Novartis, Amgen, Merck and Pfizer amongst others.
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