Presentation (CTI)

OBJECTIVES: To develop and demonstrate a conceptual PICO (Population, Intervention, Comparator, Outcomes)-to-economic evaluation translation framework enabling health technology developers (HTDs) to prospectively map JCA PICO elements to national cost-effectiveness (CE) modelling requirements, thereby reducing submission burden and improving clinical and economic evidence alignment.
METHODS: A two-stage approach was employed. First, a mapping matrix was developed by cross-referencing JCA PICO dimensions with CE submission requirements for five EU jurisdictions (Germany, France, England/Wales, Italy, Poland), using published guidelines from G-BA, HAS, NICE, AIFA, and AOTMiT. Requirements related to comparators, outcome measures (QALYs, LYGs, and clinical endpoints), analytical perspective, time horizon, and discount rate were extracted and aligned with JCA outputs to identify areas requiring supplementary evidence. Second, the framework was applied to the first completed JCA report (tovorafenib for pediatric low-grade glioma, approved in April 2026) to demonstrate how a single JCA PICO scope generates divergent national CE requirements and to quantify the translation burden per member state.
RESULTS: The matrix identified systematic divergence across PICO dimensions and economic evaluations. Comparator alignment emerged as a key challenge, reflecting differences in nationally relevant standards of care and reimbursement pathways. Outcome expectations also varied, with Germany focusing on patient-relevant clinical benefit, while France, England/Wales, Italy, and Poland required or accepted QALY-based assessments, often supplemented by LYGs or disease-specific outcomes. Perspective, time horizon, and discounting were not routinely addressed within the JCA. The framework generated country-specific evidence translation pathways, highlighting JCA outputs that could be leveraged directly and which required local adaptation, modelling or additional evidence.
CONCLUSIONS: The PICO-to-economic evaluation framework suggests that gaps between JCA outputs and national CE requirements are systematic and predictable. Early application may support proactive evidence planning, reduce downstream submission burden, and improve alignment with national HTA expectations. As this was an initial application, further validation across additional therapeutic areas and JCAs is warranted.