Rare Disease Medications: Incentives and Regulations in the European Union

Abstract

This comprehensive review explores the intricate regulatory framework surrounding orphan drugs in     the European Union, emphasizing the challenges and strategic considerations for pharmaceutical companies. From obtaining orphan designation to addressing pre-clinical development challenges and navigating payer requirements, the article delves into the complexities of rare disease drug development. The conclusion highlights the imperative for collaborative efforts in overcoming these challenges to facilitate successful market access for orphan drugs in the European Union.