The European Medicines Agency (EMA) is a neutral medicine regulator and can therefore not sponsor medicine, or fund research studies for a specific medicine. However, the EMA can express in which areas they see a need for new medicines to encourage pharmaceutical and biotechnology companies to start developing them.
The EU legislation encourages those companies to develop medicines for rare diseases, by offering reductions for scientific advice from the EMA. To explain the procedures on how the EMA supports development of medicines, and how it assesses a medicine’s benefits and risks once it receives an application for marketing authorisation, the EMA published a booklet. This booklet describes the journey of a medicine for human use and how it gets authorised through the EMA, from initial research to discussions on patient access to medicines.