IMI Impact: transforming the lives of children (Guest blog)

Children represent 20% of the EU population yet many of them live with significant unmet medical need. Less than half of all medicines used by children today are approved for paediatric use. And, for many therapies, there is little or no information about dosing in children.

Due to lack of sufficient pre-clinical information on paediatric diseases, many clinical studies fail, wasting precious resources and delaying access to better treatment options for children. Trials fail for a range of reasons, including a lack of acceptability to children, adolescent patients or their families.

This is the legacy of an era where children are not included in clinical trials and, in the absence of alternatives, their healthcare professionals have prescribed medicines which are not tailored to their patients’ needs.

Playing catch-up
Patients, paediatricians, regulators and researchers agree that this is no longer acceptable. Medicines used to treat children should be developed for – and with – children and their families. Because children were excluded from trials for so long, there is a lot of catching up to do. Building the capacity to conduct paediatric research requires investment and collaboration from public and private partners.

That is why the Innovative Medicines Initiative (IMI), financed by the European Union and EFPIA, has stepped up to fill the gap. IMI partners are developing new ways to plan and conduct clinical trials that facilitate the inclusion of children in the design of these studies.

Building networks
IMI 2 has supported the creation of a €150 million clinical trial network across Europe, helping to increase young patients’ access to the latest therapies and supporting innovation in paediatric care.

In addition, the IMI-funded C4C project will support multinational paediatric clinical trials across Europe. This network includes 33 academic and 10 industry partners from 20 European countries, as well as patient and parent organisations and approximately 500 affiliated partners.
Together they are working towards the shared goal of developing innovative trial designs and rapid dissemination of results via direct interaction with, and training of, study sponsors and children taking part in trials. IMI projects are at the cutting edge of patient engagement, pioneering the use of apps and devices adapted to the needs of children.

A further €337 million has been invested in paediatric research projects between 2012 and 2024. This represents a significant contribution to Europe’s ability to develop new medicines for all children. 

This includes those who have in the past been overlooked, such as those with rare diseases and complex conditions. For example, while autism spectrum disorders (ASD) affect around 1% of the population, there are no medicines specifically designed to treat ASD. The EU-AIMS and AIMS 2 projects have mobilised a large and diverse consortium to fill the gap.

Paediatric cancer
Cancer kills 6 000 young people in Europe every year, making it the leading cause of disease-related death in the under-19s. Today, 20% of childhood cancers remain incurable. To help change this for good, IMI is working to address unmet need in paediatric oncology.
Rising to the challenge is a complex task. Paediatric cancers are diverse and different from adult tumours. For some adult cancers, pre-clinical testing helps to identify promising treatment options. However, the tests that are currently available are designed for use in adults. This leads many children to be treated with drugs not tailored to their tumour type.
To address this, IMI 2 created ITCC-P4 project, a new platform composed of 21 partners from 8 countries. It has established 400 new patient-derived pre-clinical models of paediatric tumours. These new models, together with standardised preclinical testing procedures, can be used in regulatory filings across the EU. This is an important step in translating research into products that reach patients.
There has also been increased focus on how clinical trial protocols could research studies to better meet the needs of patients. In platform trials, for example, several therapies are studied in a single disease.  In practice, this means fewer participants are needed for the placebo arm of the trial – and thus more children have access to innovative therapies.
Through the EU-PEARL project, the Children’s Tumour Foundation has led work on a master protocol for neurofibromatosis which could be adapted for platform trials in other diseases.
Reaching potential
Young people are pure potential. Society benefits from good medicines for children because their benefit is measured in decades of improved life. However, some children do not reach their potential due to ill-health arising from lack of access to diagnosis and medicines.

IMI is pushing the boundaries of science to develop faster, better and more personalised treatments for childhood illnesses. The impact of this work will be felt by children, their families and wider society for decades to come.

Heidrun Hildebrand

Heidrun Hildebrand is Pediatric Development Alliance Manager, Pediatric Development; 
Research and Development...
Read Morechevron_right