Fondazione Gianni Benzi (FGB) provided its contribution to the public consultation on the revision of the European legislation on medicines for children and rare diseases launched by the European Commission (EC- Inception Impact Assessment – Ref. Ares (2020)7081640 – 25/11/2020), based on the outcome of the evaluation of the medicines for rare diseases and children legislation.

The comments were prepared within the European Paediatric Translational Research Infrastructure – EPTRI, an initiative aimed to accelerate and expand the paediatric drug discovery and development processes by focusing on basic, preclinical, and translational research methods, services and technologies, carried out in collaboration between 112 research Institutions.

The comments, underline that both the two Regulations (Paediatric and Rare Diseases) have only partially met the expectations of patients and that there is the need to advance in that direction. Considering that the majority of rare diseases are paediatric, the implementation of harmonised joint solutions is promoted by the European Commission.

The EPTRI document, to which FGB has strongly contributed, suggested the following urgent actions:

• To make the orphan and paediatric markets more attractive for developers and researchers
• To update the “Inventory of paediatric, including orphan, therapeutic needs” asking to the European Medicines Agency Paediatric Committee to work in collaboration with experts, company, other stakeholders
• hear from the patients’ voice about their real needs and recognise their active role in the decision-making process on research and access to treatments
• secure public funds for paediatric and orphan disease research in the framework of the next HORIZON EUROPE Research Framework.

Based on the feedback received during the consultation period, the EC will examine the impacts of future policy options to update the legal framework on medicines for rare diseases and paediatric medicines.

Discover more about the EPTRI considerations here!

Moreover, Fondazione Benzi contributed to the comments provided by the European Network of Excellence for Paediatric Research – TEDDY.