Abstract
BackgroundHealth-related quality of life (HRQOL) measurement has emerged as an important health outcome in clinical trials, clinical practice improvement strategies, and healthcare services research and evaluation. While pediatric patient self-report should be considered the standard for measuring perceived HRQOL, there are circumstances when children are too young, too cognitively impaired, too ill or fatigued to complete a HRQOL instrument, and reliable and valid parent proxy-report instruments are needed in such cases. Further, it is typically parents' perceptions of their children's HRQOL that influences healthcare utilization. Data from the PedsQL™ DatabaseSM were utilized to test the reliability and validity of parent proxy-report at the individual age subgroup level for ages 2–16 years as recommended by recent FDA guidelines.MethodsThe sample analyzed represents parent proxy-report age data on 13,878 children ages 2 to 16 years from the PedsQL™ 4.0 Generic Core Scales DatabaseSM. Parents were recruited from general pediatric clinics, subspecialty clinics, and hospitals in which their children were being seen for well-child checks, mild acute illness, or chronic illness care (n = 3,718, 26.8%), and from a State Children's Health Insurance Program (SCHIP) in California (n = 10,160, 73.2%).ResultsThe percentage of missing item responses for the parent proxy-report sample as a whole was 2.1%, supporting feasibility. The majority of the parent proxy-report scales across the age subgroups exceeded the minimum internal consistency reliability standard of 0.70 required for group comparisons, while the Total Scale Scores across the age subgroups approached or exceeded the reliability criterion of 0.90 recommended for analyzing individual patient scale scores. Construct validity was demonstrated utilizing the known groups approach. For each PedsQL™ scale and summary score, across age subgroups, healthy children demonstrated a statistically significant difference in HRQOL (better HRQOL) than children with a known chronic health condition, with most effect sizes in the medium to large effect size range.ConclusionThe results demonstrate the feasibility, reliability, and validity of parent proxy-report at the individual age subgroup for ages 2–16 years. These analyses are consistent with recent FDA guidelines which require instrument development and validation testing for children and adolescents within fairly narrow age groupings and which determine the lower age limit at which reliable and valid responses across age categories are achievable. Even as pediatric patient self-report is advocated, there remains a fundamental role for parent proxy-report in pediatric clinical trials and health services research.
Highlights
Health-related quality of life (HRQOL) measurement has emerged as an important health outcome in clinical trials, clinical practice improvement strategies, and healthcare services research and evaluation
Even as pediatric patient self-report is advocated, there remains a fundamental role for parent proxy-report in pediatric clinical trials and health services research
The Role for parent proxy-report While pediatric patient self-report should be considered the standard for measuring perceived HRQOL, there may be circumstances when the child is too young, too cognitively impaired, too ill or fatigued to complete a HRQOL instrument, and parent proxy-report may be needed in such cases [16]
Summary
Health-related quality of life (HRQOL) measurement has emerged as an important health outcome in clinical trials, clinical practice improvement strategies, and healthcare services research and evaluation. While pediatric patient selfreport should be considered the standard for measuring perceived HRQOL, there are circumstances when children are too young, too cognitively impaired, too ill or fatigued to complete a HRQOL instrument, and reliable and valid parent proxy-report instruments are needed in such cases It is typically parents' perceptions of their children's HRQOL that influences healthcare utilization. The U.S Food and Drug Administration (FDA) published draft guidance for industry in which the FDA describes how it evaluates patient-reported outcome (PRO) instruments as efficacy outcomes in clinical trials [4] In this draft document, the FDA articulated the importance of patient-reported outcomes in clinical trials, including those for pediatric patients [4]. Even as pediatric patient self-report is advocated, there remains a fundamental role for parent proxy-report in pediatric clinical trials and health services research
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