The mission of the European Medicines Agency (EMA) is to foster scientific excellence in the evaluation and supervision of medicines, for the benefit of public health in the European Union (EU). Particularly EMA aims to:

• facilitate the development and the access to medicines
• evaluate the applications for marketing authorisation
• monitor the safety of medicines across their lifecycle
• provide information to healthcare professionals and patients

EMA has recently published an overview of its key recommendations in 2019 on the authorisation and safety monitoring of medicines for human use.

In 2019, EMA recommended 66 medicines for marketing authorisation. Of these, 30 had a new active substance which had never been authorised in the EU before. Innovative medicines are essential to advancing public health as they bring new opportunities to treat certain diseases.

Once a medicine is authorised by the European Commission, EMA and the EU Member States continuously monitor its quality and benefit-risk balance and take regulatory action when needed. Measures can include a change to the product information, the suspension or withdrawal of a medicine, or a recall of a limited number of batches.

Regarding the orphan drugs, the EU framework aims to encourage the development and marketing of medicines for patients with rare diseases by providing incentives for developers. Orphan designations are reviewed by EMA’s Committee for Orphan Medicinal Products (COMP) at the time of approval to determine whether the information available allows maintaining the medicine’s orphan status and granting the medicine ten years of market exclusivity. Among the medicines recommended for marketing authorisation in 2019, 7 had their orphan designation confirmed by the end of the year.

While a specific section on the orphan drugs is included, no section has been dedicated to the paediatric medicines. However, the document describes 6 approved medicines with a paediatric indication.

Discover more about the EMA recommendations in the whole document that you can read here.