" Global Orphan Drug Pipeline & Regulatory Insight 2025" Report Highlights:
Global Orphan Drug Market Overview: US$ 260 Billion Opportunity
Global Orphan Drug market by Segment
Regulatory Landscape: USA, Europe & Asia
Orphan Drug Designation & Reimbursement Policy: USA, Europe & Asia
Global Orphan Drug Clinical Pipeline Insight: 973 Drugs
Marketed Orphan Drug Clinical Insight: 366 Drugs
The global orphan drug market opportunity is expected to surpass US$ 260 Billion by 2025 as per recent research report \"Global Orphan Drug Pipeline & Regulatory Insight 2025\" published by PNS Pharma. The orphan drug segment has emerged as a new growth frontier for pharmaceutical companies faced with issues like patent expiry, competition from generics and biosimilars, exhausting pipelines and stringent regulatory and policy framework. Increasing investment in clinical research and rapid commercialization of orphan drugs has resulted in the paradigm shift in operational strategy of pharmaceutical companies. In past, pharmaceutical companies were more focused on the research and development of essential medicines leading to high competition levels and low financial margins.
The exponential growth in global orphan drug market is driven by favorable regulatory and policy framework, increasing research and developmental activities, high ROI rates , government incentives for drug developmental, market exclusivity along with strong support from FDA and EU Commission in special protocols. The research and developmental activities for orphan drugs have also received impetus thrust from the pharmaceutical companies efforts to overcome the impact of revenue loss due the patent expiry of blockbuster drugs.
In terms of therapeutic application, oncology has been predominant segment in the global orphan drug market landscape followed neurology, rheumatology, hematology, infectious diseases and others. Currently, more than 200 companies are partnering for research and development of more than 300 orphan designated drugs in oncology segment. Oncology, as a therapeutic area has also transformed from evolution to customized or precision medicine in the pharmaceutical industry. Utilizing biomarkers to develop targeted treatments and therapies is an approach which is being increasingly used in recent years, which also further explains the reasons behind this therapeutic area’s majority share of orphan drug approvals.
“Global Orphan Drug Pipeline & Regulatory Insight 2025” report gives comprehensive insight on clinical and non-clinical parameters related to development and commercialization of orphan drug market. As per report findings, there are more than 350 orphan designated drugs commercially available in the market and more than 900 in the clinical pipeline. Most of the orphan drugs in clinical pipeline are in Phase - II trials followed by Phase - III trials. More than 30 new orphan designated drugs are further expected to enter the market in next five years.