Development of a value assessment framework for pediatric health technologies using multi-criteria decision analysis: expanding the value lens for funding decision-making

Objectives Health technology assessment (HTA) does not systematically account for the circumstances and needs of children and youth. To supplement HTA processes, we aimed to develop a child-tailored value assessment framework using a multi-criteria decision analysis (MCDA) approach. Methods We constructed an MCDA-based model in multiple phases to create the Comprehensive Assessment of Technologies for Child Health (CATCH) framework. Using a modified Delphi process with stakeholders having broad disciplinary and geographic variation (n=23), we refined previously generated criteria and developed rank-based weights. We established a criterion-pertinent scoring rubric for assessing incremental benefits of new drugs. Three clinicians independently assessed comprehension by pilot-scoring nine drugs. We then validated CATCH for two childhood cancer therapies through structured deliberation with an expert panel (n=10), obtaining individual scores, consensus scores and verbal feedback. Analyses included descriptive statistics, thematic analysis, exploratory disagreement indices (DI), and sensitivity analysis. Results The modified Delphi process yielded 10 criteria, based on absolute importance/relevance and agreed importance (median DI=0.34): Effectiveness, Child-specific Health-related Quality-of-Life, Disease Severity, Unmet Need, Therapeutic Safety, Equity, Family Impacts, Life-course Development, Rarity, Fair Share of Life. Pilot scoring resulted in adjusted criteria definitions and more precise score-scaling guidelines. Validation panellists endorsed the framework’s key modifiers of value. Modes of their individual pre-scores aligned closely with deliberative consensus scores. Conclusions We iteratively developed a value assessment framework that captures dimensions of child-specific health and non-health gains. CATCH could improve the richness and relevance of HTA decision-making for children in Canada and comparable health systems.