Orphan Drug designation – securing the significant benefit

Carima Andrady, senior regulatory writer at PharmaLex, provides an Expert View on a key consideration when seeking to secure an Orphan Drug designation (ODD) in Europe.

There are key eligibility criteria to consider when preparing an  European Union (EU) ODD application (different to that of the USA); one of which is justifying the “significant benefit” of one's product over existing products for an orphan condition. Demonstrating significant benefit contributes considerably in securing a successful ODD application if the correct approach is taken, as described in two recent publications by the European Medicines Agency (EMA) in collaboration with the  Committee for Orphan Medicinal Products (COMP) ( 1,  2). The COMP is in charge of reviewing applications for orphan designation.

What is “significant benefit” and why is it important?