The impact of parent treatment preference and other factors on recruitment: lessons learned from a paediatric
Bernie Carter1, Lucy Bray2, Nadia Al-Najjar3
1Faculty of Health, Social Care and Medicine, Edge Hill University, Ormskirk, UK. bernie.carter@edgehill.ac.uk.
Insights
Recruiting children with Rolandic epilepsy to clinical trials is challenging due to parental preferences for antiseizure medications (ASMs) and limited patient involvement. Novel trial designs are needed to address these recruitment barriers.
Area of Science:
- Pediatric Neurology
- Clinical Trial Design
- Epilepsy Research
Background:
- Evidence for antiseizure treatment effectiveness in pediatric epilepsy is often inconclusive.
- Paediatric clinical trials face challenges that can compromise external validity.
- Rolandic epilepsy in children presents specific recruitment difficulties for treatment trials.
Purpose of the Study:
- To critically reflect on factors impacting recruitment for a pilot phase of a pediatric epilepsy trial.
- To identify reasons for insufficient recruitment in a phase IV trial of antiseizure medications and a sleep intervention.
- To inform future trial designs by analyzing recruitment challenges in pediatric epilepsy.
Main Methods:
- Analysis of recruitment processes for a pilot phase of a randomized controlled trial.
- Examination of public and patient involvement and engagement (PPIE) strategies.
- Review of recruitment targets, figures, and reasons for non-consent in children with Rolandic epilepsy.
Main Results:
- Parental treatment preferences for or against antiseizure medications (ASMs) significantly impacted randomization willingness.
- Insufficient engagement with children regarding their treatment preferences hindered recruitment.
- Potential factors for non-consent included infrequent seizures, natural remission, and unclear clinical equipoise communication.
Conclusions:
- Diligent consultation with parents and patients is crucial for future pediatric epilepsy trials.
- Novel methods like parent/patient preference trials and discrete choice experiments should be considered.
- Addressing parental and patient preferences is essential for successful recruitment in ASM effectiveness studies.
Background:
In paediatric epilepsy, the evidence of effectiveness of antiseizure treatment is inconclusive for some types of epilepsy. As with other paediatric clinical trials, researchers undertaking paediatric epilepsy clinical trials face a range of challenges that may compromise external validity MAIN BODY: In this paper, we critically reflect upon the factors which impacted recruitment to the pilot phase of a phase IV unblinded, randomised controlled 3×2 factorial trial examining the effectiveness of two antiseizure medications (ASMs) and a sleep behaviour intervention in children with Rolandic epilepsy. We consider the processes established to support recruitment, public and patient involvement and engagement (PPIE), site induction, our oversight of recruitment targets and figures, and the actions we took to help us understand why we failed to recruit sufficient children to continue to the substantive trial phase. The key lessons learned were about parent preference, children's involvement and collaboration in decision-making, potential and alternative trial designs, and elicitation of stated preferences pre-trial design. Despite pre-funding PPIE during the trial design phase, we failed to anticipate the scale of parental treatment preference for or against antiseizure medication (ASMs) and consequent unwillingness to be randomised. Future studies should ensure more detailed and in-depth consultation to ascertain parent and/or patient preferences. More intense engagement with parents and children exploring their ideas about treatment preferences could, perhaps, have helped predict some recruitment issues. Infrequent seizures or screening children close to natural remission were possible explanations for non-consent. It is possible some clinicians were unintentionally unable to convey clinical equipoise influencing parental decision against participation. We wanted children to be involved in decisions about trial participation. However, despite having tailored written and video information to explain the trial to children we do not know whether these materials were viewed in each consent conversation or how much input children had towards parents' decisions to participate. Novel methods such as parent/patient preference trials and/or discrete choice experiments may be the way forward.
Conclusion:
The importance of diligent consultation, the consideration of novel methods such as parent/patient preference trials and/or discrete choice experiments in studies examining the effectiveness of ASMs versus no-ASMs cannot be overemphasised even in the presence of widespread clinician equipoise.
