Global Orphan Drug Market Report Highlights: Orphan Designated Drugs in Clinical Phase: 600 Marketed Orphan Designated Drugs: 281 Highest Number of Drugs in Phase-2 Trial: 231 US Dominates Clinical Trial Process: 350 in Pipeline (Research till Registration) Indication for Clinical Trials for Orphan Drug: More than 30% for Cancer Treatment Share of Biological in Orphan Drug: More than 60% Major Market: US (Sales > US$ 40 Billion) Why Shift to Orphan Drugs Global & Regional Orphan Drug Market Overview Orphan Drug Designation Criteria Across US, Europe & Asia Market Specific Reimbursement Policy for US, Europe & Asia Regulatory Framework for US, Europe & Asia Orphan Drug Pipeline by Phase, Orphan Designated Indication & Country

1. Global Orphan Drug Market Drive by High Profitability Rate
Pharmaceutical companies investing in the orphan drugs market have been successful in maximizing their
revenues from the existing drugs by repositioning them into additional rare diseases.
Global Orphan Drug Market Report Highlights:
 Orphan Designated Drugs in Clinical Phase: 600
 Marketed Orphan Designated Drugs: 281
 Highest Number of Drugs in Phase-2 Trial: 231
 US Dominates Clinical Trial Process: 350 in Pipeline (Research till Registration)
 Indication for Clinical Trials for Orphan Drug: More than 30% for Cancer Treatment
 Share of Biological in Orphan Drug: More than 60%
 Major Market: US (Sales > US$ 40 Billion)
 Why Shift to Orphan Drugs
 Global & Regional Orphan Drug Market Overview
 Orphan Drug Designation Criteria Across US, Europe & Asia
 Market Specific Reimbursement Policy for US, Europe & Asia
 Regulatory Framework for US, Europe & Asia
 Orphan Drug Pipeline by Phase, Orphan Designated Indication & Country
Though the developmental costs are very high, the potential to earn significant profits certainly outweigh
such high costs. As an illustration, in 2010, Soliris, which is used to treat paroxysymal nocturnal
hemoglobinuria, a rare life-threatening blood disease was the most expensive drug in the USA in 2012
costing more than $440,000 annually. The developer of this drug, Alexion Pharmaceuticals, earned a total
revenue of USD 1.134 billion in sales. Given that the target population is only 4,000 to 6,000 people, the
revenue earned by Soliris shows the immense potential that this industry holds for the future.
Additionally, the repositioning of orphan drugs as treatment to other rare diseases can increase the sales
volumes. It has been observed that close to 15 percent of the drugs had subsequent launches for other rare
illnesses. Also, it was observed that among the top 10 orphan drugs, six had more than one rare disease
indication with a peak value averaging USD 34.3 billion in overall sales potential, compared with USD 8.1
billion for drugs with only one such indication.
For Report Sample Contact: avinash@kuickresearch.com