Global Orphan Drug Pipeline & Regulatory Insight 2015

Mar 30, 2015- Mumbai, India : Bharatbook.com announces a report,on “Global Orphan Drug Pipeline & Regulatory Insight 2015”. The Therapeutic Substances Regulations in Australia defines rare drugs as those which should not be intended for use in more than 2000 patients annually

Rare diseases are also known as Orphan diseases. These diseases are generally categorized as chronic, degenerative, life threatening in nature and normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders. As per the official definition, rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. These two regions form a dominant portion of the global market for orphan drugs.

In Japan, any disease with less than 50,000 prevalent cases is defined as a rare disease. In Australia, the perspective is different. The Therapeutic Substances Regulations in Australia defines rare drugs as those which should not be intended for use in more than 2000 patients annually. In Taiwan, the official definition of rare diseases is that these diseases would be termed “rare” if the prevalence rate is 1:10,000 people. For drugs to get an orphan designation in Korea, less than 20,000 people in Korea should suffer from the disease/condition, or no other alternative treatment should be available for the disease in the country.

Owing to the pharmaceutical companies’ inadequate interest in this category of drugs, the term “orphan” was coined for drugs targeted to treat rare diseases. Almost 222 orphan designated drugs in US and 132 in Europe are marketed till date. However, in spite of the increased efforts in this market, there is still a shortage in the number of treatments for most rare disease indications. Thus, the market for rare diseases in the United States, Europe and other regions continue to offer significant potential for growth owing to enormous unmet medical need.

Globally,...