IMI impact on paediatric medicine

The connect4children project is putting in place the infrastructure needed to speed up and facilitate high-quality clinical testing of new treatments for the entire paediatric population via an extensive pan-European network of paediatric clinical trial sites. EU-PEARL is working out the best practices for platform trials, wherein more than one intervention is tested at a time, and setting up the infrastructure to carry them out, using the paediatric rare disease neurofibromatosis as a case study. INNODIA is also setting up a network of clinical centers and initiating clinical intervention trials involving adolescents and younger children with type one diabetes.  The AIMS-2-TRIALS project is also building a clinical network.

The AIMS-2-TRIALS project, in its quest to study how autism develops from before birth into adulthood, is looking at biomarkers that could be used for diagnosis ,to predict who may develop co-occurring health symptoms, and to select which interventions, supports or treatments will work best for an individual. The INNODIA projects are doing the same for type one diabetes. The U-BIOPRED project made a major step forward in understanding severe asthma, a disease that affects millions of children, by uncovering a number of subtypes of the disease, and PERISCOPE is identifying  biomarkers of protective immunity to pertussis infection, (also known as whooping cough), a disease that mainly affects babies and children.

…tackling infectious diseases in children 

RESCEU put together the most comprehensive cache of evidence to date on the true burden of respiratory syncytial virus (RSV), a disease that claims millions of mostly infant victims every year, and this data will ultimately feed public health strategies. PERISCOPE is generating technologies and infrastructure for the future development of improved pertussis vaccines, while increasing our scientific understanding of immunity to pertussis induced by vaccines and infections.

EBOVAC studies have shown that Johnson & Johnson's two-dose Ebola vaccine regimen is well-tolerated and produces a strong immune response in children over the age of one. This is a major success as children under five are at higher risk of death from Ebola. Further studies are being carried out in Sierra Leone and Guinea to investigate immune responses in infants aged under one year.

... studying the safety of medications during pregnancy and breastfeeding 

 The CONCEPTION project is investigating different ways in which medicines make their way into breastmilk, working on improving in vitro and in silico models, studying the most human-like animal models, and combining all three, enabling medicines developers to predict an infant's exposure to medicines the mother takes while breastfeeding. 

Children, young people and families play a central role in connect4children, from providing input to better design trials, to reviewing patient documentation and sharing their needs. RESCEU has a patient network run by parents of children who have been hospitalised with RSV, in order to raise public awareness that what looks like a common cold in babies can develop into serious disease. INNODIA's patient advocacy group includes parents advocating for their children with diabetes, and the EU-PEARL project has partnered with the Children's Tumor Foundation in order to incorporate the needs and wishes of children and their families in the neurofibromatosis case study.