Presentation (CTI)

OBJECTIVES: To systematically review how PRO and ObsRO data are collected and incorporated into economic evaluations of pediatric oncology therapies, and to assess alignment with regulatory and HTA guidance.
METHODS: A systematic literature review was conducted using MEDLINE, Embase, and EconLit for studies published between January 2010 and December 2024. Eligible studies included pediatric oncology trials and HTA reports enrolling children aged 2-18 years that reported PRO or ObsRO data and discussed economic or value-related outcomes. Regulatory guidance from FDA, EMA, NICE, CADTH, and ICER was reviewed in parallel. Data extraction focused on instrument type, respondent (child or caregiver), recall period, and use of outcomes in cost-effectiveness or budget impact models.
RESULTS: Of 1,142 records screened, 47 studies met the inclusion criteria. Pediatric-specific quality-of-life instruments were used in 31 studies (66%), most commonly the Pediatric Quality of Life Inventory (PedsQL). Proxy- or observer-reported outcomes were reported in 19 studies (40%), primarily for children under the age of seven. Only 13 studies (28%) incorporated PRO or ObsRO data into formal economic models, and fewer than one-fifth reported mapping outcomes to utility values suitable for quality-adjusted life-year estimation. Short recall periods (≤7 days), recommended for capturing acute treatment effects, were inconsistently applied.
CONCLUSIONS: While PRO and ObsRO data are infrequently collected in pediatric oncology studies, and also their integration into economic evaluation remains limited. Greater methodological consistency and alignment with regulatory and HTA expectations are needed to ensure pediatric patient experiences meaningfully inform value assessment.