The global orphan drugs market size is projected to reach USD 340.84 billion by the end of 2027. Increasing number of regulatory approvals will have a positive impact on the growth of the overall market in the coming years according to a new report from Fortune Business Insights
An orphan drug is a medical product
intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare. A disease is defined as rare if fewer than five in 10,000 people have it (EMA
). The European Medicines Agency estimates that 30 million people in Europe are affected by a rare disease. Currently there are
over 6000 identified rare diseases and developing treatments is challenging. Clinical trials to find new treatments face significant obstacles including:
At Cromos Pharma we have built up extensive expertise in orphan drug research and developed solutions to overcome some of key challenges in this area. Our expert team has contributed to the analysis, design, management and/or conduct of 20+ studies in rare diseases, covering Phases II to IV. You can learn more by clicking here.
- Limited pool of eligible patients;
- Wide geographical spread of study subjects and Investigators;
- Large heterogeneity in patient populations with different phenotypes and various disease pathophysiology;
- Lack of preceding clinical trials to establish a baseline standard for study execution;
- Large heterogeneity in treatment effects;
- Uncertainty in regulatory practice and various regulatory requirements in each country of operations.
We would be pleased to discuss our experience and opportunities for collaboration at the upcoming World Orphan Drug Congress 2-5 November. You can register here to connect via the event’s dedicated partnering program or email us at firstname.lastname@example.org to set up a virtual meeting.
You can follow all the latest at the event via Twitter @orphan_drugs and post using