Global Orphan Drug Pipeline & Regulatory Insight 2015

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Jan 27, 2015, 12:30 ET

DUBLIN, Jan. 27, 2015 /PRNewswire/ --Research and Markets

(http://www.researchandmarkets.com/research/t9qncf/global_orphan) has announced the addition of the "Global Orphan Drug Pipeline & Regulatory Insight 2015" report to their offering.

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, there was a desire to address the unmet treatment needs of orphan diseases, which transformed into the 1983 U.S. Orphan Drug Act, as well as similar Acts in 1991 in Singapore, 1993 in Japan, 1997 in Australia and in 2000 by the European Union. The introduction of these acts, along with high-profile philanthropic funding, has made orphan diseases an attractive segment and encouraged investment in R&D for a number of these destructive conditions.

Historically, the development of 70-75% of orphan drugs was mostly in the realm of smaller biotechnology and specialty pharmaceutical companies, and the remaining 25-30% of the orphan drugs were developed by the pharma giants. However, in the past decade, the share of pharma giants in the orphan drug approvals has increased to close to 40-45%.

The US and European region are the major markets for orphan drugs. It is in these two regions that the popularity of these drugs and diseases is increasing rapidly. The US alone accounts for a dominating share of 55%-60% in the global pie, followed by Europe. As the Asian pharmaceutical markets are still in nascent stage, there opportunities for orphan drugs in Asia are also immense and getting increasingly popular. It is expected that by 2017-18, the Asian orphan drugs market would witness steady growth.

Key Topics Covered:

1. What Are Orphan Drugs?

2. Global Orphan Drug Market Outlook

3. Orphan Drug Designation Criteria

4. Reimbursement Policy For Orphan Drug

5. US (FDA) Regulatory Framework For Orphan Drugs

6. Europe (EMA) Regulatory Framework For Orphan Drugs

7. Asia Regulatory Framework For Orphan Drugs

8. Global Orphan Drug Pipeline by Company, Country, Indication & Phase

9. Marketed Orphan Drug by Company, Country & Indication

10. Discontinued & Suspended Orphan Drugs in Clinical Trials

11. Competitive Landscape

Companies Mentioned: