Development of a Value Assessment Framework for Pediatric Health Technologies Using Multicriteria Decision Analysis: Expanding the Value Lens for Funding Decision Making

Cindy L Gauvreau; Leighton Schreyer; Paul J Gibson; Alicia Koo; Wendy J Ungar; Dean Regier; Kelvin Chan; Robin Hayeems; Jennifer Gibson; Antonia Palmer; Stuart Peacock; Avram E Denburg

doi:10.1016/j.jval.2024.03.012

Development of a Value Assessment Framework for Pediatric Health Technologies Using Multicriteria Decision Analysis: Expanding the Value Lens for Funding Decision Making

Value Health. 2024 Mar 27:S1098-3015(24)00127-X. doi: 10.1016/j.jval.2024.03.012. Online ahead of print.

Authors

Cindy L Gauvreau¹, Leighton Schreyer², Paul J Gibson³, Alicia Koo⁴, Wendy J Ungar⁵, Dean Regier⁶, Kelvin Chan⁷, Robin Hayeems⁵, Jennifer Gibson⁸, Antonia Palmer⁹, Stuart Peacock¹⁰, Avram E Denburg¹¹

Affiliations

¹ Child Health Evaluative Sciences, The Hospital for Sick Children (SickKids) Research Institute, Toronto, ON, Canada.
² Temerty Faculty of Medicine, University of Toronto, Toronto, ON, Canada.
³ McMaster Children's Hospital, Hamilton, ON, Canada; Pediatric Oncology Group of Ontario, Toronto, ON, Canada.
⁴ Department of Pharmacy, The Hospital for Sick Children, Toronto, ON, Canada.
⁵ Child Health Evaluative Sciences, The Hospital for Sick Children (SickKids) Research Institute, Toronto, ON, Canada; Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, ON, Canada.
⁶ BC Cancer Research Institute, Vancouver, BC, Canada.
⁷ Sunnybrook Health Sciences Centre, Toronto, ON, Canada.
⁸ Joint Centre for Bioethics, University of Toronto, Toronto, ON, Canada.
⁹ Ac4orn: Advocacy for Canadian Childhood Cancer Research Network, Toronto, ON, Canada.
¹⁰ Faculty of Health Sciences, Simon Fraser University, Vancouver, BC, Canada; Cancer Control Research, BC Cancer, Vancouver, BC, Canada.
¹¹ Child Health Evaluative Sciences, The Hospital for Sick Children (SickKids) Research Institute, Toronto, ON, Canada; Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, ON, Canada; Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, ON, Canada. Electronic address: avram.denburg@sickkids.ca.

PMID: 38548179
DOI: 10.1016/j.jval.2024.03.012

Abstract

Objectives: A 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 multicriteria decision analysis approach.

Methods: We constructed a multicriteria-decision-analysis-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 pilotscoring 9 drugs. We then validated CATCH for 2 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, and sensitivity analysis.

Results: The modified Delphi process yielded 10 criteria, based on absolute importance/relevance and agreed importance (median disagreement indices = 0.34): Effectiveness, Child-specific Health-related Quality of Life, Disease Severity, Unmet Need, Therapeutic Safety, Equity, Family Impacts, Life-course Development, Rarity, and Fair Share of Life. Pilot scoring resulted in adjusted criteria definitions and more precise score-scaling guidelines. Validation panelists endorsed the framework's key modifiers of value. Modes of their individual prescores aligned closely with deliberative consensus scores.

Conclusions: We iteratively developed a value assessment framework that captures dimensions of child-specific health and nonhealth gains. CATCH could improve the richness and relevance of HTA decision making for children in Canada and comparable health systems.

Keywords: HTA; child health technologies; deliberative processes; multicriteria decision analysis; value assessment.