What's the problem?

For a long time, children were treated like miniature adults when it came to medication, meaning that drugs were not tested on children. It was previously assumed that children would react similarly to adults (adjusting dosage for weight), however in fact they process, excrete and metabolise medications completely differently. The European Union’s Paediatric Regulation came into force in 2007 and aims to ensure that medicines for children are of high quality, ethically researched and authorised appropriately. This policy change has led to an increase in the amount of research into child-specific medication, 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. An additional problem is posed by the clinical trial process – as children are growing constantly, new designs for trials must be implemented.

What are we doing about it?

There are a number of Innovative Medicines Initiative (IMI) projects in the paediatric field. 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.

IHI / 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. Due to finish by end of 2023, the project has created a legal entity to continue its work past the project’s end. 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. They developed a pilot expert advice network, composed of clinicians, researchers, parents and patients, through which drug developers can easily access a wide array of important information when designing clinical trials for children’s medication. EU-PEARL was working out the best practices for platform trials, wherein more than one intervention was tested at a time, and setting up the infrastructure to carry them out, using the paediatric rare disease neurofibromatosis – a genetic condition that involves tumours growing on nerves – 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 focuses on autism, which is often diagnosed in childhood, and is building a clinical trials network to ensure the safety, integrity, and quality of autism research.

…tackling infectious diseases in children

The RESCEU team used data analysis to model how the transmission of respiratory syncytial virus (RSV) – a disease that claims millions of mostly infant victims every year – varies from month to month, as well as the efficacy and cost-effectiveness of different approaches to immunising infants.

PERISCOPE is generating technologies and infrastructure for the 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.