Barriers and Enablers to Trial Optimization in the Neonatal Intensive Care Unit

Abstract

For years, neonates have been "therapeutic orphans" and denied the benefits of clinical research because most therapeutic options are usually tested in the adult population. Most treatments and interventions have not been explored, and there is room potential rigorous, evidence-based clinical trials towards diseases specific to this population. Regenerative medicine holds great promise by potentially offering new ways of treating incurable diseases. However, bench to bedside translations often fail due to low recruitment rate. Thus, there is a need for effective interventions to increase trial participation and execution to help accelerate neonatal research. Behaviour theories could help to better understand trial participation, screening and recruiting behaviours, inform fit-for-purpose interventions, and assist in building cumulative evidence. There is a lack of clarity on the barriers and enablers to clinical trial participation from important stakeholder groups; NICU parents and research staff members. Study 1 reports findings on identified barriers and enablers that might affect parents' decision-making to participate in an early phase mesenchymal stromal cell (MSC) therapy trial including concerns with safety, efficacy and expected outcomes, but were willing to consider consenting to the trial after watching the animated video and having altruistic consideration. Study 2 reports findings on identified barriers and enablers that might affect research personnel recruitment to a multi-centre MSC trial which include: having cautious hope about the trial, importance of coordination with the clinical staff and study team and optimizing the study flow. Due to the challenging context of the study, the participants prefer to have clinicians involved with the recruitment. Lasty, Study 3 reports findings on identified barriers and enablers to screening potential patients for an adeno- associated viral vector gene therapy trial. Physicians were optimistic about the treatment but were concerned about the safety, feasibility, the expected outcomes of the treatment, and available resources (personnel, equipment, funding). Many expressed the need for support from clinical professionals prior to approaching parents and highlighted variability in screening roles. The resulting comprehensive set of factors helps to identify priorities for future research and provide insights towards developing novel interventions for neonatal research.