OBJECTIVES: Health technology assessments (HTAs) to determine precision oncology drug funding do not systematically account for the circumstances and needs of children and youth. In an era of exceptionally-priced drugs, “value” and health system sustainability are also being challenged. To inform and complement traditional HTA, we aimed to develop and test a child-tailored value assessment framework based on a multi-criteria decision analysis (MCDA) model.

METHODS: We initiated construction of an additive MCDA model by using deliberative public engagement of Canadians (n=43) to generate preliminary assessment criteria. Through a modified-Delphi process with stakeholders having broad disciplinary and geographic variation (n=24), we refined the criteria and developed associated weights through ranking and deliberation. We developed a 4-point scoring rubric, accompanied by guidelines. We grouped total scores into four prioritization levels for funding recommendations. Three clinical specialists independently assessed the framework’s usability by test-scoring nine precision therapies with pediatric indications. Their feedback was used to further refine the framework. Analyses included descriptive statistics, thematic analysis, and exploratory disagreement indices (DI) and intraclass correlation coefficients.

RESULTS: Public engagement yielded 16 candidate criteria; the 14 highest-ranked proceeded to the modified-Delphi stage. Criteria were refined to the following 10, based on absolute importance/relevance and agreed importance (median DI=0.34): Effectiveness, Child-specific Health-related Quality-of-Life (hrQOL), Disease Severity, Unmet Need, Therapeutic Safety, Equity, Family Impacts, Life-course Development, Rarity, Fair-Share-of-Life. Test-scoring resulted in adjusted criteria definitions and scoring guidelines, including additional guidelines for qualitative hrQOL data. Four drugs were scored as “high priority” or “priority” for funding by all reviewers (dinutuximab, larotrectinib, blinatumomab, dabrafenib.)

CONCLUSIONS: We developed a societally responsive, transparent, and coherent value assessment framework, child-tailored to inform evaluation of child health technologies. Refinement and application of this framework in real-world HTA contexts could improve the relevance and quality of assessments of novel technologies for children in Canada and comparable health systems.