For over a decade, European regulators have tried to boost the number of clinical trials involving children. Yet progress remains slow, and the continent’s youngest patients continue to face a shortage of properly tested medicines. The core dilemma is stark: testing drugs on minors raises serious ethical questions, but failing to do so leaves paediatricians prescribing treatments without solid evidence of safety or efficacy.
Segolene Gaillard, a clinical project leader in Lyon, France, knows this tension well. She coordinates the national initiative Kids France at the Hospices Civils de Lyon, which brings young people together to review paediatric research projects. “When I tell people what I do, some say: ‘Really? You carry out clinical trials with children? That’s unethical,’” she explains. “I tell them it would be even more unethical to prescribe a drug to a child without knowing if it’s truly effective and safe.”
Legislative progress and persistent gaps
Since 2006, the European Union has required pharmaceutical companies to submit Paediatric Investigation Plans (PIPs) before marketing new adult medications. These plans outline how a treatment will be tested on children, unless a waiver is granted for diseases that do not affect minors. According to the European Commission, this framework has increased the share of paediatric trials in the EudraCT database from 8.25% in 2007 to 12.4% in 2016 — a 50% rise. Yet a report published earlier this year by the Commission concluded that children’s medical needs are still “not sufficiently met”.
One major obstacle is the widespread use of deferrals. Currently, 86% of PIPs include deferrals, meaning companies can delay paediatric trials until after the adult drug receives market authorisation. The European Medicines Agency (EMA) estimates that the average PIP takes 9.18 years to complete. Moreover, a 2021 EMA report found that a number of PIPs and waiver applications were not submitted on time, and that waivers may be granted inappropriately — for instance, when a drug for an adult-only illness could still be relevant for a similar paediatric condition. In the last five years, 60% of new drug applications were required to carry out PIPs, while 40% received exemptions.
Consent, assent, and the child’s voice
The ethical challenges go beyond regulatory compliance. Marcin Waligóra, a professor and vice dean at Jagiellonian University Medical College in Poland, recalls that in the 1970s some ethicists argued children should never be included in trials because they cannot give informed consent. “Basically because children are not able to give informed consent, we somehow would exploit them in clinical trials,” he says. Over time, the medical community has shifted its view, recognising that a lack of testing leads to a lack of safe treatments. But consent remains a thorny issue.
Different EU member states set different age thresholds for medical consent. In Poland, the legal age is 13, though minors under 18 still need parental or guardian agreement. For younger children, researchers rely on a non-legal concept called “assent” — an opinion that, while not binding, should involve the child in the decision-making process as much as possible. “We need to include the child in the decision-making process as fast as possible,” Waligóra stresses. This means designing trials and explanations in a child-friendly way, a challenge that Gaillard’s Kids France project directly addresses by asking young participants what they need and want.
Most children who enter clinical trials are already seriously ill, often with conditions like cancer where standard treatments have failed. In such cases, the potential benefit of an experimental therapy can outweigh the risks. But for non-verbal infants, obtaining any form of assent is impossible, leaving the decision entirely to parents and ethics committees.
Broader implications for European health policy
The struggle to include children in clinical trials is part of a wider conversation about how Europe prioritises paediatric medicine. The EU’s legislative framework has spurred some progress, but the high rate of deferrals and waivers suggests that pharmaceutical companies still view paediatric testing as a burden rather than an opportunity. Meanwhile, initiatives like Kids France show that involving children in the design of research can improve both ethics and outcomes. As Gaillard puts it, “We do this a lot for adults but we haven’t been doing it enough for children.”
For an informed European audience, the question is not whether to test on children, but how to do so responsibly. The continent’s patchwork of national consent laws, coupled with EU-wide regulations, creates a complex environment where progress is possible but uneven. As the European Commission reviews its paediatric legislation, the voices of researchers, ethicists, and young patients themselves will be crucial in shaping a system that truly serves Europe’s children.
