IMI-funded researchers are inviting children and young people to help decide how clinical trials with child participants should be run.
Most medicines given to kids are used off-label, meaning they have never been tested in children. “The main reason for that is because clinical research is adult-driven, and most of these trials have inclusion criteria that says they must be over 18 to participate,” says Joana Claverol, clinical research manager at the Hospital San Joan de Déu in Barcelona, and member of the IMI-funded project c4c (conect4children).
An EU law that entered into force in 2007 requires pharmaceutical companies to undertake mandatory research for drugs that are intended for use in paediatric populations. “It changes the landscape dramatically,” says Ms. Claverol, “because all the companies that are developing treatments for adult diseases now have to perform paediatric clinical trials to get data on the safety and efficacy for their use in kids with the same disease. And it’s mandatory, it’s not an option.”
The mission of the IMI-funded c4c project is to build a network of hospitals in multiple countries that can undertake high-quality clinical trials for children, across all paediatric age groups and diseases in children. They are developing standards and procedures and training clinical trial site staff. In 2020, the first four trials will start and will be used to test the c4c processes.
Children as advisors
Children taking part in a clinical trial, depending on their maturity and understanding, have to sign an assent form declaring that they understand the purpose of the study and its benefits and risks, and that they agree to participate. Ms. Claverol and her colleagues at Hospital San Joan de Déu in Barcelona set up a European network of advisory groups made up of children and young people with different diseases to get their help to improve the different research projects developed at the hospital.
“We want these children to participate not only as participants in the trial, but to act as advisors on trial design, to review protocols, to assess patient documentation, and to talk about paediatric and medical needs,” she says.
“The information that we give to these kids to make this important decision is written by adults and it is not kid-friendly. It might be 15 to 16 pages long with words they don’t understand. So one of the things that these young people can help us with is reviewing the documents and making sure they are understandable for the kids who will receive them.”
Ms. Claverol and her team think that technology could be helpful: “What about, for example, an electronic consent application on an iPad with videos, that shows all of the assessments the children will have – like three electrocardiograms, three blood samples, and so on. We can use this new technology to help empower the patients by really helping them understand better.”
What matters to kids?
“Another issue in the design of clinical trials is that we always ask the patient to adapt their life. We don’t tend to adapt the clinical trial to their life,” says Ms. Claverol. “We want to ask young people: what matters to you? What is your reason for not wanting to take part in the trial? Is it because of the number of visits? Is it because you have to skip school? You have to imagine, some trials last up to three years, and you’re asking children to come in every month or even every week. The impact that has on their lives is huge.”
Another big difference between clinical trials for adults and those for children is that children are always accompanied to the hospital by an adult. This means major disruption for parents and other family members who have to adapt their own lives, too.
“The family are really embedded in the clinical trials, so we have to ask the parents what is feasible for them. We focus on both the parents and the children, their views are very important to the success of the trials. We probably can’t change the route of administration of a drug, but we might be able to set up home visits, so that nobody has to skip school or miss work.”
Joana Claverol is a clinical research manager at the Hospital San Joan de Déu in Barcelona and member of the Patient Public Involvement cross-cutting team in the IMI-funded project conect4children (c4c).