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.
By 2016, 101 paediatric medicines and 99 new paediatric indications had been authorised by regulators (see figures) and there has been an increase of almost 50% in the number of clinical trials that include child participants. 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?
Medical innovation has often been slow to trickle down to children and adolescents, and IMI's paediatric research portfolio hopes to change that: 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 organised an event on IMI impact on paediatric medicine. You can view the recording and presentations here.
IMI's paediatric medicine-specific projects
IMI is advancing paediatric medicine research by…
|...addressing childhood cancer||
|... 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||
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.
|...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.
ITCC-P4 are creating a platform of hundreds of childhood cancer tumour models that can be used to test promising molecules
Studies of medicine safety in breastfeeding often involve animals. Now IMI’s CONCEPTION project has a plan that could deliver better results while also using fewer animals.
connect4children (c4c) aims to enable hospitals and clinical sites across Europe to become ‘trial ready’ for paediatric clinical trials. We asked the team about the proof of viability studies that will determine if the network is working as planned.
RESCEU have put together the most comprehensive cache of evidence to date on the true burden of a disease that still claims millions of mostly infant victims every year.
UBIOPRED's classification of severe asthma types using different types of biological data will aid drug development and effective treatment.
The results support the use of the EBOVAC/IMI-funded Johnson & Johnson vaccine regimen for Ebola virus disease prevention in people over the age of one. Additional studies are ongoing to study the regiment in children under one year old.
Agreement is reached on the most important manifestations of neurofibromatosis to select for clinical drug trials, based on the opinions of both NF experts and patient representatives.
Video: stop protecting children from research