Barriers and facilitators to implementing adaptive trial designs in paediatric critical care: an international mixed-methods study

Abstract

Aims This study aimed to identify the barriers and facilitators to implementing adaptive trial designs in paediatric intensive care unit (PICU) trials. Methods A mixed-methods approach was used, combining an electronic survey and semi-structured interviews. The survey targeted clinicians and researchers involved in PICU trials, collecting information on their knowledge, and experience with adaptive trial designs. Semi-structured interviews were conducted with experienced international PICU trialists to explore themes related to adaptive trial design development and implementation. Results Eighty-six survey responses were collected from 21 countries, with 62 responses from 20 countries eligible for analysis. Of these, 45% (n = 28) of respondents had experience with adaptive trials, particularly in platform trials (n = 9, 32%) and adaptive randomisation (n = 8, 29%). Barriers to implementation included statistical complexities, infrastructure requirements, and a lack of expertise. Facilitators included increasing understanding of adaptive methodologies and access to statistical expertise. Ten interviews were conducted. Interview themes highlighted optimism about adaptive designs, a lack of formal training, the need for collaboration, the role of high-profile trials, and the potential risks involved. Conclusions Adaptive trial designs in PICU trials hold promise but are underutilised due to substantial barriers, particularly in statistical and infrastructural areas. Addressing these challenges, promoting formal training, enhancing collaboration, and raising awareness of adaptive trial methodologies are critical to increasing their adoption in paediatric critical care.