Designation of orphan conditions in Europe: regulatory observations and considerations after implementation of regulation 141/2000

ABSTRACT Introduction In the first 18 years of the implementation of the orphan medicinal products regulation, it has been noted that every year a steady number of orphan designations refer to new conditions, not designated previously. This is important because it offers documented evidence that research and development is ongoing for many areas of rare diseases. These newly designated rare conditions reflect drug development opportunities in areas of limited regulatory knowledge. Authors carried out a literature search via pubmed and Google as well as refering to previous articles they have been involved in as authors. Areas covered The aim of this paper is to review the outcomes associated with new rare conditions designation by the COMP. With over 2000 designations made since its creation data specific to conditions designation collected by the European Medicines Agency (EMA) after each monthly plenary session is presented here. The data is observational and has been grouped into therapeutic criteria based on ATC codes. Expert opinion Regulators should continue to engage in constructive dialogue with stakeholders so that the regulatory requirements are less of a hurdle and more of an opportunity to speed up drug development in areas of unmet medical need. The designation of new conditions further supports the utility, need, and meaning of the orphan regulation as a catalyst of drug development.