Determinants of decision-making and patient participation in paediatric clinical trials: A literature review

Abstract

Background: Studies have shown that many drugs used in children may not have been authorised for use in this age group. This poses significant risks on children. A new EU regulation came into effect in 2007 to ensure that medicinal products that are researched, developed and authorised will also meet the therapeutic needs of children. This will mean an increased demand for children to participate in clinical trials. Objectives: To identify the needs and motivations of children and their families who have participated, might participate or declined to participate in clinical trials. Methods: We did a literature search and reviewed empirical studies which examined eligible outcomes. The findings of these studies were analyzed in a broad qualitative and descriptive bottom-up process. Results: We found that the understanding of advanced concepts of research such as randomisation is often insufficient. The needs of families affected by a serious disease are not addressed explicitly. Personal benefit and altruistic motives were two important reasons for participation. Conclusions: The comprehension of information given in the consent process should be secured by improving the information material. The communication process should be more mutual. Children and their families should be empowered to address their needs in the process of considering participation in a clinical trial.

Paediatric patient family engagement with clinical research at a tertiary care paediatric hospital

“…In fact, suggestions from families to increase participation focused on educating individuals and emphasizing the importance of research for the benefit of all children. This finding is consistent with a recent review by Wulf et al (10) that reported personal benefit and altruistic motives as two essential reasons for participating in paediatric research. Perhaps this explains why incentives, a subject that remains controversial (18), were rarely emphasized by families to increase participation.…”

“…Family engagement with paediatric research stems from the invitation and recruitment process: families must initially be invited to participate. However, many clinical studies fail because they are underpowered and unsuccessful in approaching and recruiting a sufficient number of participants, and while the inclusion of families' views regarding research is a relatively new subject in the literature (10), it may help in elucidating the decision-making process with regard to recruitment.…”

“…Predictably, these difficulties may deter researchers and funding bodies and may have contributed to the dearth of high-quality paediatric research (4,5). As a result, <40% of paediatric medical decisions are supported by sufficient evidence (6), and most of these decisions are based on the results of research conducted with adult populations or no research whatsoever (7)(8)(9)(10).…”

“Watching time tick by…”: Decision making for Duchenne muscular dystrophy trials

“…[4, 8–10] Perceived harms included randomization, time demands, and general inconveniences. [10] Many parents have an insufficient understanding of the distinction between the child’s treatment options and options for clinical trial participation, as well as incomplete understanding of randomization [8, 9]. …”

Why do children decide not to participate in clinical research: a quantitative and qualitative study

“…Reasons for, or against, participation may be influenced by ethnic status, societal factors, and anxiety or stress, and concrete barriers such as time commitment, childcare, and transportation difficulties (9). Providing participants with inadequate information concerning the particulars of the research also interferes with the participants' ability to make well-considered decisions (10). Studies in adolescents, one of which concerned a hypothetical research project, demonstrated that several participants expected direct health benefit from being questioned and examined by the physician (11)(12)(13).…”