Generating family centred prioritised outcomes in paediatric trials

Introduction:
A key component of good health research is that the outcomes measured or variables that are monitored during the study have therapeutic importance and matter to the people affected by the findings of the study. However, few paediatric clinical trials report that there chosen outcomes were developed in consultation with children or parents. This means that research may not be providing answers to questions that mater to families.

Goals and Methods:
The aim of this research was to develop a rigorous method of identifying outcomes that matter to parents and children to inform the design of family centred paediatric clinical trials.
While conducting eight mixed-method paediatric feasibility and pilot trials (2015-2023), the UK Qualitative Patient Centred Trials team undertook multiple Action Research Cycles (Plan, act, observe and reflect) to develop a methodology for identifying parent and child prioritised outcomes. All the trials included a mixed methods study comprising, but not limited to, 200 parent interviews, two focus groups involving 15 parents, and two focus groups with 19 young people.

Results
A worked example from the seventh feasibility study ‘Timing of Stoma Closure in Neonates’ study of a five stage outcomes prioritisation method including: 1, Generating an initial outcomes list, 2. Interview/focus group conduct detailing topic guide design, 3. Thematic, ranked content analysis, 4. Data triangulation and 5. Application.

Conclusions
Our approach to outcome prioritisation through qualitative research had been used to identify trial outcomes that provide answers to questions that matter to children and families. This methodology could be applied in wider in non-paediatric settings.