Paediatric Unmet Medical Needs (UMNs) is a major concern in the management of diseases in children not only by the paediatric community but also by both the EC and the EMA which released a joint action plan in November 2018, subsequently updated in 2020.

In 2020, important changes for the regulatory framework occurred, with the EC publishing major documents relating to the revision of the Paediatric and of the Orphan Regulation: the Staff Working Document on the Joint Evaluation of these two regulations and the Inception Impact Assessment. The revision of these two legislations is also one of the actions of the Pharmaceutical Strategy for Europe released in November 2020. As a result, legislative and/or non-legislative proposals to amend these two regulations are expected by the end of 2021/early 2022.

In this context, and as a follow-up to the 2019 EFGCP-EFPIA workshop, a second workshop is now proposed with the objective through stakeholders’ perspectives and needs, to identify criteria to help define paediatric UMNs using four conditions as case examples: Asthma, NASH, Duchenne Muscular Dystrophy and Haemophilia. These conditions were selected as representing all possible paediatric development options. Moreover, workshop outputs are expected to inform the revision of the European Paediatric and Orphan Regulations.

Join the discussion on the 28 and 30 June and support us with your insight, experience and connections in this initiative which is crucial for the future of our paediatric patients.