On March 2020, the European Medicines Agency released its annual overview of the involvement of patients and healthcare professionals in the work of the Agency. The report underlined a high percentage (90%) of cases, in which coordinators of scientific advice procedures considered patient involvement as a key action in their decision-making process. Several events have been organised involving the patients and patient organisation such as the “Regulatory Science to 2025: multi-stakeholder workshop” and the “Methotrexate containing medicines- stakeholder meeting”. In particular, this last meeting involved mainly patients representing rheumatoid arthritis, psoriasis and Crohn’s disease groups, healthcare professional specialists and medication error experts and was aimed to discuss new measures to prevent serious and potentially fatal errors with the dosing of methotrexate. Despite the higher number of initiatives increasing awareness of importance of patient engagement, the actions foreseeing the involvement of paediatric patients and children are still rare. Nevertheless, these initiatives could be very useful in overcoming challenges and issues of paediatric clinical trials as lack of patients’ recruitment and retention.