The European Medicines Agency (EMA) has recently published a question-and-answer document to dispel any doubt on the meaning of orphan designation and other aspects related to orphan medicines Regulation in EU.
The EU legislation foresees giving orphan designation for substances that could be used for treating, preventing or diagnosing a rare and serious condition. Orphan designation can help the medicine’s developer advance the medicine to the stage where it can be authorised to be put on the market. A formal approval is needed before a medicine can legally be marketed.
The document also includes questions and answers related to significant benefits and market exclusivity of orphan medicines.
This document is really important because it helps patients and their families to understand what are the procedures and meaning of orphan designation. This is even more essential in case of paediatric rare diseases.
Children suffering from a rare disease can be considered orphan twice because most of rare diseases are paediatric and also because the difficulties are doubled in studying rare paediatric diseases. As a matter of fact, many drugs on the market have not been studied in the pediatric population. That’s why children are classified as “therapeutic orphans”.
So, such a document from EMA can be a first and easy-to-understand user manual for those who are approaching the rare disease world and in particular the orphan designation aspects.