This article summarises key insights and learnings gathered by the Little Journey research team from published research and interviews with caregivers who have lived through trials. All quotes have been anonymised for participant privacy.
At Little Journey, our purpose is to change the future of paediatric care, one empowered family at a time. Improving enrolment, adherence, and retention in paediatric clinical trials begins with understanding families’ real-world experiences including what makes participation difficult, and what helps.
By combining evidence from published research with interviews from caregivers who have lived through trials, we’ve identified consistent challenges, motivators, and opportunities to create a more compassionate and efficient experience that benefits families, research sites, and sponsors alike.
Clinical trials are the foundation of medical progress. They generate the evidence needed to understand whether new medicines, vaccines, and interventions are safe and effective. However, for children, many treatments prescribed today are still based on adult data. This approach, while well-intentioned, can lead to both ineffective and sometimes harmful outcomes [1].
Children are not simply smaller versions of adults. Their developing bodies process medicines differently; their metabolism, organ function, and responses to treatment change rapidly as they grow. As Sandeep [2] highlights, only around six per cent of adult data can be safely applied to children. Without research designed specifically for paediatric populations, we risk limiting improvements in children’s health outcomes and slowing medical progress for future generations.
Paediatric clinical trials must also navigate complex ethical, logistical, and emotional challenges. Researchers need to secure both parental consent and child assent, ensuring understanding without coercion [2]. It is therefore unsurprising that many studies struggle to recruit or retain participants. Nearly one in five paediatric trials are discontinued before completion, most often due to difficulties enrolling or keeping families engaged [3]. Yet, there remains limited high-quality evidence on how to improve retention [4].
Behind those statistics are real families facing the everyday logistical challenges of trial participation, such as travel, time off work, and the emotional toll of managing complex care alongside daily life. They are often balancing hope, fear, and fatigue:
“It has been hard at times – the amount of travelling, juggling work, home life, [son’s] education and appointments has meant there have been periods of exhaustion and mental health has suffered, but we do this for the sake of our kids so we kind of suck it up.” - P5
Children and adolescents often want to be involved in the consent or assent process and to have their voices respected. Yet studies show that young participants sometimes feel pressured or unheard [5,6].
Families do their best to try and be honest with their child:
“We’ve always been honest with him. I always say, ‘your body, your choice,’ because he has to have so many blood tests.” - P5
Listening to these lived experiences is essential to ensure that paediatric research is not only scientifically rigorous but also ethical, empathetic, and feasible for the children and families it seeks to serve.
Many parents and young people are driven by altruism: a desire to help others, advance science, and improve care for the next family [6,7,8]. For some, deciding to join a study restores a sense of agency at a difficult time [9].
“They see how difficult life is for their sister… they wanted to make a difference for other children who have got eczema.” - P7
Others are motivated by the chance to access innovative treatments or specialist care:
“This drug could potentially be a game changer in my son’s future. Even if they asked me to do 500 visits, I’d do it.” - P5
Trust also plays a huge role. When a trusted clinician recommends participation and communicates clearly, families are far more likely to enrol.
“They told us what was going on at every step and said if we ever felt uncomfortable, we could pull out without question. That gave us confidence.” - P7
Understanding what motivates families to participate is important, as it highlights that joining a trial is often a complex family decision. Paediatric studies need to be designed to reflect these motivations, focusing on:
This approach not only supports families more effectively but also strengthens recruitment, engagement, and long-term retention.
Clear communication consistently emerged as a determinant of a positive trial experience. Families value honesty, transparency, and accessible information.
“At the start it was very informative. They told us exactly what would happen every week. We were prepared for it.” - P4
However, many families find the clinical trial written materials overwhelming or overly technical.
“It was just all black and white paper… it looked like a mortgage document.” - P9
Lengthy, jargon-filled consent forms can deter participation and increase anxiety [10]. Dense, technical participant information can also alienate families.
“The protocols are clearly designed by people who don’t have children with medical needs. There’s no consideration for the day-to-day life of a family.” - P5
Our findings are consistent with patterns being found across the clinical research space - giving participants the right information, at the right time is key to a study's success. In a recent write up of the SCOPE Europe Summit, our Lead Product Manager, Laura, shared how the right patient support can increase retention through to trial completion by 40%.
Families called for shorter, layered information, visuals, and child-friendly explanations to help children feel informed and reassured. Many caregivers developed creative strategies to prepare their children, such as play and storytelling.
“We role-played at home about lying really still, waving my arms over him pretending it was the MRI machine making all the buzzy noises.” - P5
These personal efforts highlight families’ commitment and the opportunity for digital tools to bridge that preparation gap.
While families are often motivated, trial participation can carry significant logistical, emotional and financial strain [7]. Frequent hospital visits, travel, and schedule disruptions often weigh heavily, particularly for parents balancing work and other caring responsibilities.
“The amount of travelling, juggling work, home life, and school… there have been periods of exhaustion, but we do this for our kids.” - P5
Practical barriers like distance to trial sites, time demands and loss of income are among the common reasons for withdrawal [11].
“I don’t think there was anything that we missed, other than money from work. We did an entire shift in work schedule so that our two days off were actually travel days to and from appointments.” - P8
Evidence shows that logistical pressures, including travel time, frequency of visits, and disruption to daily life contribute to attrition in paediatric studies [12,13]. Families also weigh their child’s health, the perceived stress of procedures, and their understanding of the study when deciding whether to stay involved [14,15]
Even when motivated by altruism or access to new treatments, families often face exhausting practical realities that test their resilience. Recognising and addressing these pressures is essential to designing studies that truly work for families.
Across both our literature review and interviews, a clear message emerged: research design must fit into families’ lives, not the other way around.
Families suggested simple, high-impact improvements:
“There was a lot of preparation (we did) around accommodation, like ‘Where were we going to stay?’, ‘Is it wheelchair accessible?’, ‘Can we get in the bathroom?’ Wherever we were staying, I think that was the biggest barrier.” - P8
“It would have been good to have the key dates and milestones written down… like a timeline you could follow.” - P2
“A timeline would be great. It doesn’t have to be all bells and whistles… just something that moves along to show what’s done and what’s to come.” - P3
These recommendations from families guided the development of our My Next Visit module, which gives families an at-a-glance guide to their appointments throughout the trial. It includes custom reminders, timelines, and more. You can read more about the co-design process for My Next Visit in this article.
“Having that personal touch — calls, messages, knowing someone cares — was reassuring.” - P1
P1: “Trying to even it out with excitement and fun really helped to counterbalance his nerves.” - P1
“I made him a bravery certificate… after the cannula, that’s what he opted for — ice cream and a sticker.” - P1
Our findings confirm that successful paediatric clinical research relies not only on scientific rigour, but on compassionate, family-centred design.
By embedding these insights into Little Journey’s digital family retention platform, we’re helping research teams deliver trials that are more accessible, empathetic, and effective — supporting families from enrolment through to follow-up, and helping sites improve enrolment, retention, and adherence.
When families are listened to and supported, research becomes a partnership, one that benefits children today and advances care for those yet to come.
Speak to our team about the full capabilities of our platform
[1] Caldwell PH, Murphy SB, Butow PN, Craig JC. Clinical trials in children. Lancet. 2004 Aug 28-Sep 3;364(9436):803-11.
[2] Sandeep (2013) Paediatric clinical Trials: Perspectives in Clinical Research 4(1):p 89-99, Jan–Mar 2013
[3] Pica N, Bourgeois F. Discontinuation and Nonpublication of Randomized Clinical Trials Conducted in Children. Pediatrics. 2016 Sep;138(3)
[4] Gaunt et al (2023) Participant retention in paediatric randomised controlled trials published in six major journals 2015–2019: systematic review and meta-analysis. Trials (2023) 24:403
[5] Carter, B., Bray, L., al-Najjar, N., Tort Piella, A., Tudur-Smith, C., Spowart, C., Collingwood, A., Crudgington, H., Currier, J., Hughes, D. A., Wood, E., Martin, R., Morris, C., Roberts, D., Rouncefield-Swales, A., Sutherland, H., Watson, V., Cook, G., Wiggs, L., Gringras, P., & Pal, D. (2023). The impact of parent treatment preference and other factors on recruitment: lessons learned from a paediatric epilepsy randomised controlled trial. Trials, 24(1), 83.
[6] Dobra, R., Wilson, G., Matthews, J., Boeri, M., Elborn, S., Kee, F., Davies, J. C., & Madge, S. (2023). A systematic review to identify and collate factors influencing patient journeys through clinical trials. JRSM Open, 14(6), 1–11.
[7] The Centre for Information and Study on Clinical Research Participation (CISCRP, 2020) Pediatric Perceptions & Insights study: Survey findings.
[8] Joseph, P. D., Craig, J. C., & Caldwell, P. H. Y. (2015). Clinical trials in children. British Journal of Clinical Pharmacology, 79(3), 357–369.
[9] Shilling, V., Williamson, P. R., Hickey, H., Sowden, E., Beresford, M. W., Smyth, R. L., & Young, B. (2011). Communication about children’s clinical trials as observed and experienced: Qualitative study of parents and practitioners. PLoS ONE, 6(7),
[10] Tait, A. R., Voepel-Lewis, T., & Malviya, S. (2003). Do they understand? (Part I): Parental consent for children participating in clinical anesthesia and surgery research. Anesthesiology, 98(3), 603–608.
[11] van der Stijl, W., van den Berg, E., van der Velden, R. M. J., van den Berg, M. H., van der Heijden, A. J., & van Spronsen, F. J. (2018). Factors influencing participation and dropout in paediatric clinical trials: A mixed-methods study. European Journal of Pediatrics, 177(7), 1081–1090.
[12] Driscoll, K. A., Johnson, S. B., Hogan, J., Gill, E., Wright, N., Deeb, L. C., & Geller, D. H. (2009). Predictors of study completion and withdrawal in a randomized clinical trial of a pediatric diabetes adherence intervention. Contemporary Clinical Trials, 30(3), 212–220.
[13] Karlson, C. W., & Rapoff, M. A. (2008). Attrition in randomized controlled trials for pediatric chronic conditions.Journal of Pediatric Psychology, 34(7), 782–793.
[14] Elliott, V., Morgan, K., Setchell, J., Priebe, I. K., Wright, A., Davies, K., & McDonagh, J. E. (2019). Patient and caregiver engagement in research: Factors that influence co-enrollment in research. Pediatric Rheumatology, 17, Article 85.
[15] Dobra, R., Wilson, G., Matthews, J., Boeri, M., Elborn, S., Kee, F., Davies, J. C., & Madge, S. (2023). A systematic review to identify and collate factors influencing patient journeys through clinical trials. JRSM Open, 14(6), 1–11.
Elliot et al (2019) Patient and caregiver engagement in research: factors that influence co-enrollment in research Pediatric Rheumatology volume 17, Article number: 85 (2019)
[16] Mandel, L. A., O’Donnell, E., Canenguez, K., Castro-Mendoza, P. B., Lotze, T., Waubant, E., Weinstock-Guttmann, B., & Chitnis, T. (2021). Family perspectives on clinical research for pediatric multiple sclerosis: Enhancing equity. Journal of Patient Experience, 8.
[17] Shen, A. K., O’Brien, K., Figueiredo, A., Schwartz, J. L., Piltch-Loeb, R., & DiMaggio, C. (2022). Factors influencing parental and individual COVID-19 vaccine decision making in a pediatric network. Vaccines, 10(8), 1277.
[18] Corneli, A., Cho, M. K., Ramos, S. R., Yarbrough, D., & Henderson, G. E. (2021). Evidence-based strategies for honoring research participants and communicating study results: Perspectives from a multi-stakeholder workshop. Journal of Empirical Research on Human Research Ethics, 16(4), 335–347.