“Global Orphan Drug Clinical Trials, Patent & Guidelines Insight 2026” Report Highlights:
- Global Orphan Drug Market Opportunity: US$ 300 Billion
- US Dominates Global Orphan Drug Market: 50% Market Share
- US Orphan Drug Opportunity To Surpass: US$ 150 Billion
- Global Orphan Drug Clinical Insight: More Than 900 Drugs
- Clinical Insight on Marketed Orphan Drugs: More Than 400 Drugs
- Oncology To Dominate Orphan Drug Development: 35% Share
- FDA & EMA Regulations For Orphan Drugs
- Orphan Drug Designation Criteria & Reimbursement Policy by Country
The orphan drugs enjoy significant competitive advantages in the market owing to the market exclusivity period after the drug has been authorized. Through this clause, the manufacturer of orphan drugs is given a monopoly status in the market because according to the law, no other company is allowed to market the orphan drugs during the exclusivity period. Additionally, this monopolistic power is further strengthened with the fact that no other alternative health technology exists for many orphan drugs.
In the orphan drugs segment, oncology had the highest share of 34%, followed by neurology, rheumatology, haematology, infectious diseases and others. In the segment for non-biological orphan drugs, the most dominating therapeutic area was oncology, followed by neurology, and fibrosis. At present there are more than 200 companies partnering with other parties to develop 300 orphan drugs in oncology drugs segment. Oncology represents the biggest disease category for new orphan drugs.