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Orphan drugs, also called niche busters, are intended to treat patients suffering from very serious diseases for which no treatment or at least a satisfactory one, has so far been available. These diseases, often referred to as orphan diseases or rare diseases, affect only a small portion of the population. The number of diseases for which no treatment is currently available is estimated to be between 4,000 to 5,000, worldwide. The patent expiry of blockbuster drugs and increased competition from generics has shifted the focus of pharmaceutical industry from the essential medicines to orphan drugs. Incentives for drug development provided by governments, as well as support from the regulatory bodies, are a further boost for the companies developing orphan drugs all over the world.
 
According to the report “Global Orphan Drugs Market Outlook 2020”, the orphan drug market is anticipated to reach around US$ 180 Billion by 2020. The report provides a detailed analysis of the market for these orphan drugs. An estimation of the market potential of these drugs has been done keeping in mind that many more of these drugs are being tested in clinical trials for various indications, apart from the ones that are already approved. Furthermore, the report also provides the revenue generated by top 10 orphan drugs in 2014.

The report provides information about the segments of the orphan drug market on the basis of type, geography, and therapeutic application. It also highlights orphan drugs at various stages of clinical development. In addition, the report also provides insight about the major drivers and challenges, latest trends and developments, and strategic collaborations impacting the industry growth. The regulatory scenario of orphan drug industry across different geographic regions of the world has also been discussed in the report.

The later part of the report discusses some of the prominent players in the global orphan drug market. A brief business overview of each player has been provided along with their business segments, product portfolios, and recent developments. Moreover, analysis of their strengths and weaknesses has also been done. Overall, the report will prove as a complete source of knowledge and analysis for clients and potential investors.