The paucity of evidence-based data on formulation characteristics preferred by the children is known to limit the design of tailored paediatric dosage forms. The European Paediatric Translational Research Infrastructure (EPTRI) commissioned a study to evaluate children’s dosage forms perceived preferences in some European countries and explore the feasibility of using the young persons advisory groups (YPAGs) to involve children in formulation research. An online, age-adapted survey was developed and translated into six languages. The survey link was disseminated across seven European countries: Albania, Italy, the Netherlands, and Dutch-speaking part of Belgium, Romania, Spain, and the United Kingdom. Respondents’ (n = 1172) perceived preferences for oral dosage forms primarily differed based on age, health status, and experience. Conventional dosage forms, i.e., liquid (35%), tablets (19%), and capsules (14%), were the most selected. Liquid was widely selected by children less than 12 years and by those healthy and taking medicines rarely. Monolithic solid forms were mostly chosen by adolescents and by children with a chronic disease taking medicines frequently. There was a clear lack of familiarity with more novel dosage forms (e.g., orodispersible films and granules). Noteworthy, granules were not appreciated, particularly by adolescents (52.8%). To rationalise the creation of paediatric formulations, it is important to involve children as active stakeholders and to apply tools assessing children’s perspectives on medicines to inform acceptable dosage form development from the start.

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.

The lack of paediatric medicines, including innovative and advanced ones, is a long-lasting and well-known problem at European and international levels. Despite the existing legal frameworks and incentives, children remain deprived of many kinds of therapy because of challenges faced in appropriately study and tailoring medicinal and other products for them. In this context, the necessity to foster paediatric research addressing unsolved and uncovered issues within a ‘translational approach’ has appeared. This article, after having clarified the concept of translational research in the perspective of the establishment of a European paediatric research infrastructure (RI), will identify and point out ethical, legal and regulatory issues particularly relevant in a children’s rights perspective. It concludes asking for the setting up of an adequate model of governance within a future RI, including adequate and independent ethical oversight and a pluridisciplinary common service dealing with ethical, legal and societal issues relevant for children.

Background The key role played by research nurses in coordinating clinical trials in a paediatric setting has developed in line with increasing complexities of trial design. A questionnaire-based survey was conducted to investigate the training of research nurses involved in paediatric trials across Europe, to identify potential training needs and compare roles across specialties and countries. Methods A structured, cross-sectional questionnaire survey was used, with the aim of describing and quantifying research nurse experiences. The questionnaire was designed to cover four main areas of interest: demographics, training, clinical trial experience and research nurse roles/activities. Results The questionnaire was completed by 341 respondents across 45 different specialties in 20 European countries. A higher percentage of research nurses within 3 years of taking up post were dissatisfied with the level of training received (16%), as compared with those in post for 3–6 years (8%) and >6 years (6%). There was a trend towards a higher percentage of respondents receiving self-funded training in mainland Europe, with reported values of 15%–20%, as compared with <5% in the UK and Ireland. Only 3% of research nurses prescribed investigational medicinal products in a clinical trial setting, with contrasting roles observed between countries. Conclusions While high levels of training satisfaction were observed, 67% of respondents felt they would benefit from additional training in line with frequently changing practices. Currently, low levels of nurse prescribing are observed in a paediatric clinical trial setting across Europe. Appropriate research nurse training programmes should be promoted through national networks across Europe.

Over the last years, a Europe-wide trend toward a patient-focused approach is developing and is influencing the decision-making process related to the clinical research. This new vision aims to draw on patient knowledge and experience in order to deliver benefits for all stakeholders of the drug development process, optimizing the clinical study design. In this context, the “patient empowerment” concept has been developed as an approach encouraging the active participation and self-determination of the patients in the caring procedure. For this reason, in 2016, European Patients’ Academy (EUPATI) launched a public consultation that ended in September 2016 with the release of the EUPATI guidance for patient involvement in the medicine research and development process. Likewise, the recommendations on the “Summaries of Clinical Trial Results for Laypersons” for the Implementation of Regulation (EU) No 536/2014 recommended a clear and comprehensible communication of the clinical trial results to the patients. However, rarely, all these attempts for the patient involvement pay attention to the pediatric population needs. An innovative approach for the patients’ involvement in pediatric clinical research is represented by the Young Persons Advisory Groups, an organization composed of youths, patients, and carers, actively participating in clinical research and advising researchers and their teams.