Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR Good Research Practices for the Assessment of Patient-Reported Outcomes in Children and Adolescents Task Force

Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR Good Research Practices for the Assessment of Patient-Reported Outcomes in Children and Adolescents Task Force Abstract. Patient-reported outcome (PRO) instruments for children and adolescents are often included in clinical trials with the intention of collecting data to support claims in a medical product label. The purpose of the current task force report is to recommend good practices for pediatric PRO research that is conducted to inform regulatory decision making and support claims made in medical product labeling. The recommendations are based on the consensus of an interdisciplinary group of researchers who were assembled for a task force associated with the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). In those areas in which supporting evidence is limited or in which general principles may not apply to every situation, this task force report identifies factors to consider when making decisions about the design and use of pediatric PRO instruments, while highlighting issues that require further research. Five good research practices are discussed: 1) Consider developmental differences and determine age-based criteria for PRO administration: Four age groups are discussed on the basis of previous research

Comparison of the web-based and digital questionnaires of the Spanish and Catalan versions of the KIDSCREEN-52

Background: The objectives of the study were to develop web-based Spanish and Catalan versions of the KIDSCREEN, and to compare scores and psychometric properties with the paper version. Methods: Internet and paper Spanish and Catalan versions of the KIDSCREEN-52 were included in a cross-sectional study in school-age children. Web-based and paper Spanish or Catalan versions of the KIDSCREEN-52 were administered to students aged 8 to 18 years from primary and secondary schools in Palafolls (Barcelona, Spain, n5923). All students completed both web-based and paper versions during school time with an interval of at least 2 hours between administrations. The order of administration was randomized. The KIDSCREEN-52, the Strengths and Difficulties Questionnaire (SDQ), and sociodemographic variables were collected. Missing values, floor and ceiling effects, and internal consistency were compared between both versions, as well as mean score differences, level of agreement, and known groups and construct validity. Results: Participation rate was 77% (n5715). Web-based and paper versions showed low percentage of missing values and similar high ceiling effect (range 0 to 44%). Mean score differences showed an effect size (ES) lower than 0.2 in all dimensions. Internal consistency ranged from 0.7 to 0.88, and degree of agreement was excellent (Intraclass correlation coefficient [ICC] range 0.75 to 0.87). Expected differences were seen by sex, age, socioeconomic status and mental health status. Conclusions: The web-based KIDSCREEN-52 showed similar scale score and reliability and validity than the paper version. It will incorporate the child population in the assessment of quality of life providing a more attractive format

Internal consistency and reliability of Primary Care Assessment Tool (PCATool-Brasil) for child health services

Health strategies based on primary health care have been expanding in Brazil. An instrument applied to users, the Primary Care Assessment Tool (the PCATool), which measures the extent of primary care, has been validated in the United States. We sought to adapt the PCATool to Brazil and analyze its validation and reliability through a cross-sectional validation study of the Child PCATool.Validation included: translation, back-translation, adaptation, debriefing, content and construct validate, internal consistency, and reliability analysis. The questionnaire was applied to 468 parents or guardians of children registered with 18 primary health services in Porto Alegre, representing the services’ normal pediatric population. Using factor analysis, 8 domains were identified, with Cronbach’s α ranging from 0.74 to 0.88.Validation resulted in a 45-item scale, divided into 8 attributes (Access to First Contact, Continuity, Coordination, 3 attributes of Comprehensiveness, Community Orientation, and Family Orientation). These results showed that the PCATool-Brasil has adequate validity and reliability and could be used as a national instrument to evaluate primary health care after its application to other population settings in the country.Fundação de Amparo à Pesquisa, Estado do Rio Grande do Sul e Escola de Saúde Pública, Rio Grande do Su