What aspects of outcome measurement instruments are important to parents and caregivers in child health trials: Survey protocol

Introduction

Health research involving children can include exploring their experiences of illness and healthcare and/or interventions that seek to prevent ill health and/or improve health outcomes. Often interventions targeting child health outcomes are examined using trial methods, and findings from trials of interventions to improve child health outcomes and/or prevent illness can influence healthcare, research, and policy (Naka et al., 2017). Such trials can include examination of pharmacologic, non-pharmacologic, behavioural and/or lifestyle interventions, to determine impacts on child health outcomes including prevention/reduction of illness and symptomatology and improvement/maintenance of good health (Naka et al., 2017). The findings of these trials play a critical role in informing age-appropriate and evidence-based care in children. This is because, for instance, drugs often metabolize differently in children than adults (Joseph et al., 2015; Rieder, 2018); certain diseases, such as hyaline membrane disease or neonatal respiratory distress syndrome in newborns, exist only in pediatric populations (Bavdekar, 2013; Dyer, 2019); and interventions may have varying levels of effectiveness in children (Field et al., 2004; Methods for Child Health Reviews | Cochrane Child Health, n.d.). As such, it is vital that trials of interventions involving children up to 12 years of age, as well as their caregivers responsible for their care and illness management, are conducted to the highest standard. Caregivers often play a more active role in health-decision making for children under 12. From 12 years on children are suggested to have the capacity for more autonomy in “decision-making situations” (Grootens-Wiegers et al., 2017), which may include the decision to engage with health research.

Within trials of child health interventions, the recruitment and retention of children and their parents and caregivers to health studies can pose unique challenges (Cohen et al., 2010; Kim et al., 2020). Perceived risks and benefits associated with a trial (Hoberman et al., 2013), trust in the study team (Greenberg et al., 2017), and participant burden (Hein et al., 2015; Tromp & van de Vathorst, 2019) have been identified as factors impacting caregivers’ decisions to consent or not consent to their child’s participation in health research. Despite evidence for factors such as these (Greenberg et al., 2017; Hoberman et al., 2013), the impact of outcome measurement instruments (OMIs) used in trials on recruitment, retention, and engagement is largely under-researched. Characteristics of outcome measurement instruments, such as measurement properties, acceptability, feasibility, and burden, may impact involvement in trials of child health interventions. Given that trial findings are only as good as the data underlying them, and the type and quality of data collected are only as good as the tools used to collect them (Coster, 2013; Tugwell et al., 2007) it is important to understand how best to select and utilize OMIs that are appropriate for children and their caregivers in trials.

This recognition of the importance of considering appropriateness and feasibility for caregivers and children can be seen in studies such as the ‘PARENT’ trial (Vanderloo et al., 2021). This trial found that engaging with a parent panel early in trial design, specifically focusing on recruitment, engagement, and the intervention, contributed to notable improvements in the trial protocol (Vanderloo et al., 2021). Though not explicitly focused on OMIs, these findings highlight the integral role of caregiver perspectives, and reinforce that the priorities and focus of caregivers may be different than that of trialists. For instance, in a survey of families of extremely preterm infants, behavioural problems, emotional health, and hyperactivity were identified as ‘major’ long-term outcomes of concern, despite often being considered ‘mild by the medical literature’ (Jaworski et al., 2022). Such findings further underscore the value and need to consider perspectives of those stakeholders who engage with and potentially benefit from trials. While research is currently being undertaken to explore trialists’ perspectives on what and how to choose OMIs when designing a trial (Matvienko-Sikar et al., 2024), there is little to no evidence about these perspectives and preferences for caregivers in the context of trials of child health interventions and child health research more broadly. Caregivers’ perspectives and preferences for OMIs may influence whether they and their children are likely to take part in, and contribute meaningfully to, trials that have the potential to make an impact on future child health research, policy, and practice.

The choice and characteristics of OMIs have the potential to influence the amount and quality of data collected in trials of child health interventions through impacting participant recruitment and engagement in data collection. Understanding if and what characteristics of OMIs influence caregiver engagement with data collection in child health research, can inform choice and use of OMIs in future trials of child health interventions and broader child health research. This can maximise the quantity and quality of data provided while ensuring that data collection approaches are appropriate, acceptable, and feasible for children and their caregivers.

Aim

The aim of this project is to examine what characteristics of OMIs are important in caregivers’ decisions to engage with and participate in trials of interventions for child health outcomes.

Methods

Discussion

The aim of this study is to evaluate what characteristics of OMIs are important to caregivers of children participating in health research. To date, there is little to no research on the impact of the characteristics of OMIs, such as type, length, burden, or how, where or by whom they are collected, on recruitment and retention in child health trials. The results of this study will inform the selection of OMIs in future trials of child health interventions and can potentially improve recruitment to, and the experience of both caregivers and children engaging in research.

Ethics and consent

All research activities will be conducted following the University College Cork (UCC) Code of Research Conduct, the Declaration of Helsinki, and in accordance with General Data Protection Regulations (GDPR). Ethical approval was obtained from University College Cork’s Social Research Ethics Committee on July 15^th, 2024, (ID Log No. 2024–148), Participants will be provided with full study information leaflet (please see extended data). The information leaflet will include the study aims, what is involved in participating, their ethical rights, how data will be managed in the study, and contact information for the study team. It will be made clear in the participant information leaflet that participation is voluntary, and participants can withdraw from the study any time up to the point of submitting their survey responses (at which point it will not be possible to identify individual participants due to the anonymous nature of the survey). Following reading this information leaflet, participants will be taken to an electronic consent form and must provide electronic informed consent (please see extended data) via the Qualtrics platform before gaining access to the survey. Following survey completion, the study aims and approach to data management will be restated; participants will also be provided with the team’s contact information upon completion. The anonymous aggregated dataset in this study will be stored in accordance with the UCC Code of Research Conduct, the UCC data protection policy and the European General Data Protection Regulations (GDPR). Any potentially identifying data provided by participants in ‘free response’ text boxes will be anonymised, with any potentially identifying features removed, in the final paper. Data will also be stored and managed in line with the Findability, Accessibility, Interoperability, and Reuse of digital assets (FAIR) Principles. Data will be stored for a minimum of ten years on the UCC-supplied OneDrive for Business using Microsoft Teams.

Dissemination

Findings from this study will be disseminated as a peer-review publication and as presentations at seminars/webinars and academic conferences. An infographic summary of anonymous aggregated study findings will also be shared on social media (i.e., Twitter/X) and on the EPOCH-Translate (Translating Early Prevention of Obesity in Childhood) website.

Study status

Data collection is currently underway.