The European Paediatric Regulation mandated the European Commission to fund research on off-patent medicines with demonstrated therapeutic interest for children. Responding to this mandate, five FP7 project calls were launched and 20 projects were granted. This paper aims to detail the funded projects and their preliminary results. Publicly available sources have been consulted and a descriptive analysis has been performed. Twenty Research Consortia including 246 partners in 29 European and non-European countries were created (involving 129 universities or public-funded research organisations, 51 private companies with 40 SMEs, 7 patient associations). The funded projects investigate 24 medicines, covering 10 therapeutic areas in all paediatric age groups. In response to the Paediatric Regulation and to apply for a Paediatric Use Marketing Authorisation, 15 Paediatric Investigation Plans have been granted by the EMA-Paediatric Committee, including 71 studies of whom 29 paediatric clinical trials, leading to a total of 7,300 children to be recruited in more than 380 investigational centres. Conclusion: Notwithstanding the EU contribution for each study is lower than similar publicly funded projects, and also considering the complexity of paediatric research, these projects are performing high-quality research and are progressing towards the increase of new paediatric medicines on the market. Private–public partnerships have been effectively implemented, providing a good example for future collaborative actions. Since these projects cover a limited number of off-patent drugs and many unmet therapeutic needs in paediatrics remain, it is crucial foreseeing new similar initiatives in forthcoming European funding programmes.

PedCRIN is a Horizon 2020 project aimed to develop a paediatric component of ECRIN (European Clinical Research Infrastructure Network) including tools supporting the conduct of neonatal and paediatric trials.

Introduction: In paediatrics, medicines repurposing is a particularly advantageous approach, offering a route to address unmet medical needs and turn off-label use into evidence-based treatments for paediatric populations. This study analysed the effects of funds provided under the Seventh Framework Programme for Research (FP7-FRP), issued by the European Commission from 2007 to 2013 according to the European Paediatric Regulation, in terms of new paediatric marketing authorisations (MAs) including paediatric Use Marketing Authorisations (PUMAs). Additionally, we investigated which funded projects included repurposing initiatives.Methods: Data was collected on paediatric Investigation Plans (PIPs), new MAs, and MAs variations from the EMA website, national medicine registers, and final project reports. A survey to project coordinators was also conducted to explore the challenges faced during paediatric drug development plans.Results: The 20 FP7-funded projects studied 24 off-patent active substances. Eighteen substances had agreed PIPs with the European Medicines Agency paediatric Committee (PDCO). Positive compliance checks were granted for three PIPs, resulting in three new PUMAs. According to the adopted definition, 22 out of 24 (91.6%) paediatric development plans could be classified as repurposing. New conditions were proposed for eight substances, while 16 aimed to extend existing indications to broader paediatric populations. Additionally, 18 development plans included new age-appropriate formulations. The survey revealed that primary challenges in paediatric development plans included budgeting, lengthy regulatory processes, and recruitment.Discussion: Taken together, these results highlighted on one hand that the FP7 programme had a positive impact, as three new PUMAs were effectively obtained, representing one third of the nine PUMAs obtained since the paediatric Regulation entered into force, and three out of 18 agreed PIPs were successfully completed within 3–10 years. In addition, repurposing existing drugs for paediatric use significantly contributed to addressing unmet medical needs in paediatrics. On the other hand, the gap between the number of agreed PIPs and those that have led to PUMAs is still considerable, due to regulatory barriers and financial constraints. This underscores the need for continued support and further initiatives to streamline public-private partnerships for paediatric drug development, ensuring that off-patent medicines can be safely and effectively repurposed for paediatric use.

In pediatric clinical research, it is essential to implement ethical and regulatory requirements, training, and facilities to grant the proper management of specimens, considering that blood sampling may be difficult, the number of specimens is usually limited, and all efforts should be made to minimize sample volumes. In the context of the Pediatric Clinical Research Infrastructure Network (PedCRIN) project, an easy‐to‐use tool has been developed to guide investigators and sponsors in managing specimens and associated data in compliance with the applicable European rules in the context of pediatric clinical trials. Key topics and research questions to properly manage biosamples and related data in the context of pediatric trials were identified by PedCRIN partners; the current European regulatory/ethical and legal resources were searched for and analyzed; the items/measures/procedures to ensure regulatory compliance of a pediatric trial with regards to biosamples were defined. A checklist of the key items to be considered for the management of biological samples in pediatric clinical trials in compliance with the European applicable rules and legislation, was prepared. It is publicly available on the PedCRIN website https://ecrin.org/projects/pedcrin. Five different topics were covered: consent and assent; minimizing harm and maximizing welfare; sampling volume; skills, training and facilities required for sampling; and long‐term storage of biological material. This exercise addressed a specific need in the field of pediatric research to implement ad hoc procedures for specimen handling. In fact, specific guidance on the management of biosamples in pediatrics is not available.

Background: Sickle cell disease (SCD) is an autosomal recessive haemoglobin disorder, affecting about 7.74 million individuals worldwide, but it is more prevalent among Africans and Asians. SCD is characterised by many complications, and it is a major health issue in Nigeria, the country with the largest burden of the disease globally. This work aims to present the design and implementation of electronic registries (ER) for SCD in a tertiary hospital in Nigeria. Methods: Registry design was initiated during a staff exchange programme within the ARISE initiative (EU grant agreement no. 824021). Ethical approval was obtained, and paper records were retrieved and transferred into one adult and one paediatric database, developed with Microsoft Access. Results: Data from 2659 SCD patients were entered in the ERs, including 698 (26.3%) adults and 1961 (73.7%) children. There were 287 (41%) male adults, 404 (58%) female and 7 (1%) patients whose gender was missing. There were 1041 (53.1%) male children, 906 (46.2%) female and 14 (0.7%) whose gender was missing. Information on phenotype was available for 2385 subjects, and most of them (2082, 87.3%) were SS. The most prevalent SCD-related complication was painful events (26.6% in adults and 68.7% in children, considering valid cases). Conclusions: About 60% of SCD patients in the centre were included in the ERs providing useful, hands-on recommendations for future ER design in SCD. These ERs might be an appropriate tool for collecting and analysing SCD patients’ data.