Finally, the study of the pathogenesis of the disease at the genetic level and molecular studies have led to the discovery of new targets for therapy and the production of disease-modifying drugs.
London, United Kingdom. January 29, 2018: Visiongain has launched a new pharmaceutical report Market report of orphan drugs 2018-2028: forecasts and analysis by therapeutic class (oncology, alimentary tract and metabolism, hormonal disorders and hematology, neuroscience, respiratory, cardiovascular, nephrology and ophthalmology), Class drug (biological, small molecule) and by region, plus analysis of the main companies.
An orphan disease is also known as a rare disease, where the health condition affects a smaller number of people that is often serious and life threatening compared to other diseases prevalent in the general population around the world. There are many rare diseases due to genetic abnormalities that may be present throughout the life of the person, even symptoms may not appear immediately. In addition, some rare diseases arise from exposure to toxins, infections or as an adverse reaction to other therapeutic treatments. According to the Global Genes estimate, there are around 7,000 rare diseases worldwide. Approximately 50 percent of people affected by rare diseases are children.
The first act of orphan drugs was approved in the United States in 1983 to facilitate the development of orphan drugs. Under the designation of orphan drug, incentives are given to sponsors to develop a new drug for rare diseases. These incentives include a partial tax credit for clinical trial expenses, marketing exclusivity for years and exempt user fees.
“Orphan drugs are drugs that have been developed to treat rare diseases that affect only a small part of the population.” In the US alone, the FDA reports that nearly 7,000 rare diseases affect approximately 30 million people in 2015 Orphan drugs generally develop faster because their target population is very small and generally has a high level of genetic similarity among patients, and governments generally encourage the development of orphan drugs to encourage production, which includes the exclusivity of patents, along with relaxed regulations for FDA approval and financial advantages.
Companies that see the advantages of patent exclusivity, price protection and less stringent regulations have been interested in producing orphan drugs and may charge higher drug prices to this very specific and small segment of the patient population. In addition, orphan drugs have a higher price due to the various costs associated with the drug development process, such as the cost of research and development and the advanced technologies involved. This in turn acts as a reinforcement for more and more pharmaceutical and biotechnology companies to participate in the orphan drug industry. ”
The global market is highly competitive with many different players entering the market. The report analyzes products from leading companies in the market. The report analyzes products from leading companies in the market. The most important companies in the Orphan Drug market include Hoffmann-La Roche, Celgene, Bristol-Myers Squibb, Novartis, Amgen, Merck and Pfizer, among others.