Thanks to advances in neonatal care and technology, babies born as early as 23 weeks preterm survive at higher rates than ever before. This group of very early preterm babies encounter many challenges concerning their health and development which require special needs and demands. However, to manage health complications, up to 90% of drugs used in newborn and preterm babies are used off-label, meaning drugs without official approval for this specific age group that can come with unknown optimal dosage, unexpected side effects or a lack of positive impact on the baby’s condition.

Clinical trials, targeted at newborn and preterm babies, provide an opportunity to understand the impact on families and what they consider optimal outcomes, and these might not necessarily be the same as for medical professionals. Their involvement also helps to tailor medical treatments, improve babies’ outcomes and reduce off-label medication and side effects. Nevertheless, involving this group in clinical trials is challenging. The enrolment of babies depends on the parents’ consent and their adherence to follow-up care. Understandably, parents often feel uncomfortable with trials being carried out on their babies and find themselves left alone with their decision. Additionally, follow-up as part of the data collection is lacking due to gaps in platforms, gaps in standardised protocols or simply due to participant drop-out. In particular, very preterm babies are severely underrepresented in clinical trials.

A key step towards resolving low enrolment and greater acceptance or normalisation of clinical trials in this patient group is through cooperation and support of parent organisations to guarantee public and patient involvement (PPI). In this case, the involvement of parents of preterm babies and the participation of adults born preterm during the early stages of clinical trial study design is important. Only by allowing those directly impacted to express their opinions and concerns can researchers better plan to achieve optimal outcomes, greater acceptance and the desired enrolment rates.

The c4c Proof of Viability study, TREOCAPA, a randomised European clinical trial on the use of paracetamol to close the ductus arteriosus, integrates the voices of parents/families in throughout the study. The European Foundation for the Care of Newborn Infants (EFCNI) is the Public and Patient Involvement (PPI) partner in the TREOCAPA clinical trial, ensuring that the public, patients and caregivers are heard. With the establishment of a parental advisory board (PAB), EFCNI’s goal is also to ensure the aspects important to parents are included in the trial and to create understandable information materials for them.

Acknowledging that one in every ten babies is born preterm, it is also essential that the public recognises the importance of the involvement of preterm babies and their families in research projects, and awareness campaigns like World Prematurity Day are an excellent way to start. EFCNI generally and also within the c4c project strives to advance neonatal research and comprehend the needs of preterm and very preterm babies, with patients and families at the centre of their approach. Let us offer every baby the best start in life.