Many activities focused on paediatric research are carried out by Fondazione Gianni Benzi that is strongly engaged in the promotion of scientific research for populations with unmet medical needs such as children.

Lack of authorised paediatric medicines and the consequent off-label use is a relevant problem for the paediatric population due to the difficulty in conducting clinical trials, the relatively low patient numbers and the generally smaller size of the market.

However, the Paediatric Regulation sets up a system of obligations, rewards and incentives to encourage manufacturers to research and develop medicines for children’s specific therapeutic needs and since 2007 new medicines for use in children and new pharmaceutical forms appropriate for children were authorised in the EU.

Nevertheless, a gap in the paediatric medicines development still exists. As a consequence, more efforts should be made by all the stakeholders to discover, develop and deliver innovative and effective medicines that meet the paediatric needs!

The European Federation of Pharmaceutical Industries and Associations (EFPIA) and the European Forum for Good Clinical Practice (EFGCP) collaborated to organise a multi-stakeholder workshop to explore how to best define and tackle unmet needs in children, in order to provide with a basis for strategic decision making on paediatric medicine development.

The objective of the workshop was to find a broadly accepted approach to define unmet medical needs (UMN) among paediatric patients, in order to make better and safer medicines for children and to make sure they are available and affordable.

By reading the report you can figure out that the outcome of the workshop is an outline of potential guiding principles for defining UMN in children and a programme for testing the principles in selected therapeutic areas over the course of 2020.

Among the key recommendations it was highlighted that, since no one-size-fits-all approach can work at present, progresses should be made through addressing UMN by speciality, with particular reference to treatment categories or gaps, but not on products.

Defining unmet medical needs in children is one of the building blocks in meeting the needs of children across Europe and beyond. This is one of the ongoing activities to address the European Commission and EMA action plan for better implementation of the Paediatric Regulation.

More research focused on unmet paediatric needs, more medicines for children, more interest in paediatrics!

Discover more about the workshop by reading the report.

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