AbstractRandomised controlled trials (RCTs) rely on effective recruitment and retention for successful completion. Potential trial participants’ preference for a treatment (trial intervention) can affect recruitment, post randomisation drop-out and adherence to intervention groups in adult RCTs, but little is known about how they may affect paediatric trials. Communication of trial information in paediatric trial settings is complex as it needs to accommodate the parent’s as well as young person’s perspective, whilst at the same time maintaining high standards of trial conduct. This PhD explored how treatment preferences influenced recruitment and participation in paediatric RCTs by undertaking a systematic review of the literature and embedding qualitative research in four paediatric trials.
The systematic literature review focused on paediatric RCTs and qualitative studies that reported the treatment preferences of children and young people aged 0-17 years, and their parents. Fifty-two papers were identified, twelve of which contained qualitative data. CONSORT figures reporting decline or withdrawal from trials due to treatment preference were tabulated and discussed descriptively. Techniques of meta-ethnography were drawn on to evaluate qualitative data. The systematic review showed treatment preferences acting as a barrier to recruitment to paediatric RCTs, particularly from a parental perspective. Parents’ understanding of trial processes and perceptions of the benefits and risks associated with treatments promoted discussion of preference. Few RCT papers reported the views of young people in relation to preference for treatment.
Qualitative methods were embedded in three chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME) trials, and one surgical trial for acute, uncomplicated appendicitis. The QuinteT Recruitment Intervention (QRI) has been embedded successfully in adult RCTs to identify and address recruitment difficulties with the intention to optimise informed decision-making and recruitment. Methods and approaches from the QRI (audio-recorded recruitment consultations, interviews, recruiter training) were employed in the present research to explore the treatment preferences of young people, their parents, and to discuss issues of equipoise with recruiting health professionals. Data analyses drew on techniques of constant comparison, content and thematic analysis. All four RCTs were able to successfully recruit paediatric participants, but preference for treatment was a consistent reason for trial decline, post randomisation drop-out and discontinued treatment in the four trials under investigation. Young people and their parents expressed treatment preferences when considering RCT participation in all four trials. However, young people were less likely to express preferences than their parents. The views and equipoise of those recruiting and treating patients influenced families at all stages of recruitment, and during trial participation. Providing training for recruiters and wider clinical teams that promoted communicating equipoise, and the exploration of preference during discussions with families, had a positive effect on observed recruitment practices. More efforts are now needed to understand preference for treatment in paediatric RCT settings, particularly in relation to the impact on trial retention and the treatment outcomes under investigation.
|Date of Award||28 Nov 2019|
|Supervisor||Esther M Crawley (Supervisor), Nicola J Mills (Supervisor) & Bridget Young (Supervisor)|
- Randomised Controlled Trial
- Treatment preference
A qualitative exploration of treatment preference in paediatric randomised controlled trials
Beasant, L. (Author). 28 Nov 2019
Student thesis: Doctoral Thesis › Doctor of Philosophy (PhD)