Presentation (CTI)

OBJECTIVES: To identify methodological considerations in economic evaluations of pediatric populations, focusing on model inputs, health outcome measurement, and value assessment.
METHODS: A targeted literature review was conducted using a predefined PICO-S framework focused on pediatric populations aged 0-18 years and methodological parameters relevant to health economic modeling. Searches were conducted in PubMed/MEDLINE for studies published from 2020-2026 in English, Spanish, or French, supplemented by targeted review of HTA and grey literature sources. Search terms captured pediatric populations, economic evaluation, health economic models, utilities, QALYs, willingness-to-pay thresholds, discounting, time horizons, life tables, and caregiver spillover. Records were screened at title/abstract level and categorized by relevance. From 346 PubMed records, 103 were retained after screening, and 16 methodological studies met criteria for detailed synthesis. Extracted domains included pediatric utility measurement, proxy versus self-report, caregiver/family spillover, time horizon and extrapolation, discounting, mortality assumptions, analytical perspective, WTP thresholds, and limitations of QALYs in pediatric decision-making.
RESULTS: The 16 studies clustered around five methodological domains: utility measurement and valuation, preference elicitation, caregiver spillover effects, societal value assessment, and long-term modeling assumptions. Multiple studies identified limitations in applying adult-derived utility values to pediatric populations and recommended development of pediatric-specific valuation approaches, particularly for infants and young children. Several studies highlighted uncertainty regarding whether child, parent, or adult preferences should be used to value pediatric health states. Caregiver and family spillover effects were recognized as important but inconsistently incorporated into economic evaluations. Studies examining QALY-based frameworks recommended broader consideration of equity, disease severity, risk, and societal preferences. Finally, evaluations of newborn screening, genome sequencing, and gene therapies emphasized challenges associated with extrapolating lifelong benefits from limited short-term evidence and recommended greater transparency around structural uncertainty.
CONCLUSIONS: Greater methodological standardization and pediatric-specific guidance may improve the relevance of economic evaluations for HTA and reimbursement decision-making.