On 2 and 3 November 2024, the first face-to-face meeting of the INVENTS Paediatric Patient Expert Group (PEG) titled “Innovative methodologies based on in silico models and the secondary use of health data” was held in Bari.
Under the European INVENTS project, TEDDY network, partner of the project, has established a PEG to support the researchers participating in this EU project with their opinions and suggestions to improve the conditions of thousands of children worldwide suffering from a rare disease. The purpose of this PEG is to ensure the proper collection of patients' health data, including methodological considerations for data collection, reporting, management, and analysis, along with meaningful patient input.
The event saw the active participation of eight girls between 11 and 19 years old from Italy, France, Greece, Norway and the United Kingdom. The girls, affected by chronic diseases, such as multiple sclerosis and juvenile rheumatoid arthritis, learned key concepts about clinical research, data collection, in silico models, and use real-world data for research, which support faster drug development. Eight teachers and facilitators explained several topics. The first lesson “What is paediatric research, children’s rights in health and research” was held by our researcher Silvia Torretta. She provided general concepts about rare and paediatric diseases, the rights of children, and the ethical-regulatory aspects of data collection and use. Silvia highlighted the importance of pharmacological research for developing new treatments and described the steps to obtain safe drugs through clinical studies. She emphasized the need to conduct these studies rigorously, especially for drugs intended for children, ensuring their informed consent and protection. Furthermore, she reiterated that children’s rights must be prioritized at every stage of the research, including principles such as informed consent, protection, confidentiality, and listening to their opinions.
The training was complemented by practical activities and tools to enthusiastically engage the PEG members.
The event concluded on Sunday, 3 November, with a farewell until the next training courses, online or in-person, which will be organized during the project to further empower children and adolescents about the progress of biomedical research, including the latest advancements in health data and in clinical research methodologies and their relevance to paediatric rare diseases.