Impact on: paediatric medicine

Medical innovation has often been slow to trickle down to children and adolescents. IMI's paediatric research portfolio is changing that. 

 

What's the problem?

For a long time, paediatric medicine development has largely been neglected, meaning that drugs used in children have not been appropriately tested in paediatric populations. However, European legislation introduced more than 10 years ago, as well as equivalent legislation in the United States, have stimulated research efforts in this area. Yet despite these developments, there are still many challenges and unmet medical needs to address, including childhood cancer; a fraction of the number of anti-cancer medicines for kids are authorised compared to those for adults, for example. 

 

What is IMI doing about it?

We're setting up a sustainable infrastructure for delivering paediatric clinical trials, transforming the way clinical trials are conducted particularly for paediatric rare diseases, building a platform to identify promising molecules to fight paediatric cancer, studying genetic newborn screening for rare diseases, and tackling infectious diseases that plague children, all while placing kids and their families at the centre of research. 

 

IMI research is...

...addressing childhood cancer

The ITCC-P4 project is creating hundreds of solid and liquid childhood tumour models in order to explore the biology of paediatric cancers, identify sub-groups of patients that might respond better to certain treatments, and carry out tests on potential new drugs. Meanwhile, the HARMONY Alliance projects aim to improve care for children with cancer by creating a unique big data platform with approx. 8,000 anonymised child patient records to accelerate paediatric blood cancer research.

... building clinical trial networks and improving on their design

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.

...identifying biomarkers and driving personalised medicine

Clinical research needs to address the needs of patients. HARMONY are analysing anonymised patient data from different sources to find out about patients’ priority outcomes. PIONEER is carrying out the same big data analysis to make interventions more efficient and patient-centred in men diagnosed with prostate cancer, while BigData@Heart’s real-world data-driven research platform is providing insights that are helping usher in treatments tailored to individual patients.

…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. 

...putting parents and children at the centre of research

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.