Irma M. Hein

Accuracy of the MacArthur competence assessment tool for clinical research (MacCAT-CR) for measuring children's competence to consent to clinical research

IMPORTANCE An objective assessment of childrens competence to consent to research participation is currently not possible. Age limits for asking childrens consent vary considerably between countries, and, to our knowledge, the correlation between competence and childrens age has never been systematically investigated. OBJECTIVES To test a standardized competence assessment instrument for children by modifying the MacArthur Competence Assessment Tool for Clinical Research (MacCAT-CR), to investigate its reliability and validity, and to examine the correlation of its assessment with age and estimate cutoff ages. DESIGN, SETTING, AND PARTICIPANTS This prospective study included children and adolescents aged 6 to 18 years in the inpatient and outpatient departments of allergology, gastroenterology, oncology, ophthalmology, and pulmonology from January 1, 2012, through January 1, 2014. Participants were eligible for clinical research studies, including observational studies and randomized clinical trials. EXPOSURES Competence judgments by experts aware of the 4 relevant criteria-understanding, appreciation, reasoning, and choice-were used to establish the reference standard. The index test was the MacCAT-CR, which used a semistructured interview format. MAIN OUTCOMES AND MEASURES Interrater reliability, validity, and dimensionality of the MacCAT-CR and estimated cutoff ages for competence. RESULTS Of 209 eligible patients, we included 161 (mean age, 10.6 years; 47.2% male). Good reproducibility of MacCAT-CR total and subscale scores was observed (intraclass correlation coefficient range, 0.68-0.92). We confirmed unidimensionality of the MacCAT-CR. By the reference standard, we judged 54 children (33.5%) to be incompetent; by the MacCAT-CR, 61 children (37.9%). Criterion-related validity of MacCAT-CR scores was supported by high overall accuracy in correctly classifying children as competent against the reference standard (area under the receiver operating characteristics curve, 0.78). Age was a good predictor of competence on the MacCAT-CR (area under the receiver operating characteristics curve, 0.90). In children younger than 9.6 years, competence was unlikely (sensitivity, 90%); in those older than 11.2 years, competence was probable (specificity, 90%). The optimal cutoff age was 10.4 years (sensitivity, 81%; specificity, 84%). CONCLUSIONS AND RELEVANCE The MacCAT-CR demonstrated strong psychometric properties. In children aged 9.6 to 11.2 years, consent may be justified when competence can be demonstrated in individual cases by the MacCAT-CR. The results contribute to a scientific underpinning of regulations for clinical research directed toward children.

Assessing children’s competence to consent in research by a standardized tool: a validity study

BackgroundCurrently over 50% of drugs prescribed to children have not been evaluated properly for use in their age group. One key reason why children have been excluded from clinical trials is that they are not considered able to exercise meaningful autonomy over the decision to participate. Dutch law states that competence to consent can be presumed present at the age of 12 and above; however, in pediatric practice children’s competence is not that clearly presented and the transition from assent to active consent is gradual. A gold standard for competence assessment in children does not exist. In this article we describe a study protocol on the development of a standardized tool for assessing competence to consent in research in children and adolescents.Methods/designIn this study we modified the MacCAT-CR, the best evaluated competence assessment tool for adults, for use in children and adolescents. We will administer the tool prospectively to a cohort of pediatric patients from 6 to18 years during the selection stages of ongoing clinical trials. The outcomes of the MacCAT-CR interviews will be compared to a reference standard, established by the judgments of clinical investigators, and an expert panel consisting of child psychiatrists, child psychologists and medical ethicists. The reliability, criterion-related validity and reproducibility of the tool will be determined. As MacCAT-CR is a multi-item scale consisting of 13 items, power was justified at 130–190 subjects, providing a minimum of 10–15 observations per item. MacCAT-CR outcomes will be correlated with age, life experience, IQ, ethnicity, socio-economic status and competence judgment of the parent(s). It is anticipated that 160 participants will be recruited over 2 years to complete enrollment.DiscussionA validity study on an assessment tool of competence to consent is strongly needed in research practice, particularly in the child and adolescent population. In this study we will establish a reference standard of children’s competence to consent, combined with validation of an assessment instrument. Results can facilitate responsible involvement of children in clinical trials by further development of guidelines, health-care policies and legal policies.

Medical decision-making in children and adolescents: developmental and neuroscientific aspects

BackgroundVarious international laws and guidelines stress the importance of respecting the developing autonomy of children and involving minors in decision-making regarding treatment and research participation. However, no universal agreement exists as to at what age minors should be deemed decision-making competent. Minors of the same age may show different levels of maturity. In addition, patients deemed rational conversation-partners as a child can suddenly become noncompliant as an adolescent. Age, context and development all play a role in decision-making competence. In this article we adopt a perspective on competence that specifically focuses on the impact of brain development on the child’s decision-making process.Main bodyWe believe that the discussion on decision-making competence of minors can greatly benefit from a multidisciplinary approach. We adopted such an approach in order to contribute to the understanding on how to deal with children in decision-making situations. Evidence emerging from neuroscience research concerning the developing brain structures in minors is combined with insights from various other fields, such as psychology, decision-making science and ethics. Four capacities have been described that are required for (medical) decision-making: (1) communicating a choice; (2) understanding; (3) reasoning; and (4) appreciation. Each capacity is related to a number of specific skills and abilities that need to be sufficiently developed to support the capacity. Based on this approach it can be concluded that at the age of 12 children can have the capacity to be decision-making competent. However, this age coincides with the onset of adolescence. Early development of the brain’s reward system combined with late development of the control system diminishes decision-making competence in adolescents in specific contexts. We conclude that even adolescents possessing capacities required for decision-making, may need support of facilitating environmental factors.ConclusionThis paper intends to offer insight in neuroscientific mechanisms underlying the medical decision-making capacities in minors and to stimulate practices for optimal involvement of minors. Developing minors become increasingly capable of decision-making, but the neurobiological development in adolescence affects competence in specific contexts. Adequate support should be offered in order to create a context in which minors can make competently make decisions.

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

Background:More pediatric drug trials are needed, but although specific pediatric regulations warrant safety, recruitment of children for these trials remains one of the main difficulties. Therefore, we investigated potential determining factors of nonparticipation in clinical research, in order to optimize research participation of children by recommending improved recruitment strategies.Methods:Between 1 January 2012 and 1 January 2014, we performed a prospective study among161 pediatric patients, aged 6 to 18 y, who were eligible for clinical research. We quantitatively analyzed the association of potential explanatory variables (e.g., age, cognitive development, experience, ethnicity) with nonparticipation and qualitatively analyzed interviews on reasons for nonparticipation.Results:Sixty percent of the children did not participate in the research project on offer (39% decided not to participate, 21% were indecisive). Lower age, less disease experience, and less complex research with lower risk were predictive for not participating. Time constraint and extra burden were expressed as decisive reasons for not participating.Conclusions:Strategies to optimize research participation should be aimed at younger children and their families, who are logistically challenged and unfamiliar with health care and research. Recommendations include informing pediatric patients and their families of the value of research; minimizing logistic burdens; and improving accessibility.