A strengthened focus on unmet medical needs, regulatory processes more suited to support innovation and an increased alignment of data requirements between decision-makers are among the top priorities addressed in the past four years by European regulators in order to increase the efficiency of paediatric regulatory processes. The so obtained achievements are reported in the closing report of the EMA and European Commission (EC) action plan on paediatrics, available at EMA’s website.
Areas of particular need for paediatric medicines have been jointly identified by the Agency and relevant stakeholder, so to optimise the research agenda. Paediatric tumors and inflammatory bowel disease are among the main topics addressed by multi-stakeholder strategy fora, those results have been taken into account by EMA during discussions on paediatric investigation plans (PIPs).
Simplification and adaptation of regulatory processes also occurred to facilitate the establishment of PIPs. The pilot phase for a ‘stepwise PIP’ agreement, for example, is focused in assessing the possibility to agree, in certain cases, on a partial development programme, conditional on the development of a full PIP once evidence becomes available over time. Collaboration with international partners has also been strengthened in order to facilitate the compatibility of paediatric requirements across different regulatory geographic areas.