In order to incentivise the potentially uneconomical development and marketing of a medicinal product for rare diseases, the European Commission has launched a voluntary and cost-free procedure for orphan designation(Regulation 141/2000), which can be used to apply for benefits for medicinal product developments. Orphan designation (OD) is a multi-stage process with initial designation and confirmation of the criteria at the time of authorisation or major extension of the indication.

Orphan drug status can be applied for from the Committee for Orphan Medicinal Products ( COMP)
on the basis of disease models or early clinical data as early as the beginning of drug development, i.e. in some cases years before marketing authorisation.

The authorisation recommendation for medicinal products with an orphan designation is issued by the CHMP in the centralised authorisation procedure.