Cindy L. Gauvreau PhD , Leighton Schreyer BSc , Paul J. Gibson MD , Alicia Koo PharmD , Wendy J. Ungar PhD , Dean Regier PhD , Kelvin Chan MD, PhD , Robin Hayeems PhD , Jennifer Gibson PhD , Antonia Palmer MSc , Stuart Peacock DPhil , Avram E. Denburg MD, PhD
{"title":"利用多标准决策分析为儿科保健技术制定价值评估框架：为资金决策拓展价值视角。","authors":"Cindy L. Gauvreau PhD , Leighton Schreyer BSc , Paul J. Gibson MD , Alicia Koo PharmD , Wendy J. Ungar PhD , Dean Regier PhD , Kelvin Chan MD, PhD , Robin Hayeems PhD , Jennifer Gibson PhD , Antonia Palmer MSc , Stuart Peacock DPhil , Avram E. Denburg MD, PhD","doi":"10.1016/j.jval.2024.03.012","DOIUrl":null,"url":null,"abstract":"<div><h3>Objectives</h3><p>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.</p></div><div><h3>Methods</h3><p>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.</p></div><div><h3>Results</h3><p>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.</p></div><div><h3>Conclusions</h3><p>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.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9000,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Development of a Value Assessment Framework for Pediatric Health Technologies Using Multicriteria Decision Analysis: Expanding the Value Lens for Funding Decision Making\",\"authors\":\"Cindy L. Gauvreau PhD , Leighton Schreyer BSc , Paul J. Gibson MD , Alicia Koo PharmD , Wendy J. Ungar PhD , Dean Regier PhD , Kelvin Chan MD, PhD , Robin Hayeems PhD , Jennifer Gibson PhD , Antonia Palmer MSc , Stuart Peacock DPhil , Avram E. 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Development of a Value Assessment Framework for Pediatric Health Technologies Using Multicriteria Decision Analysis: Expanding the Value Lens for Funding Decision Making
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.
期刊介绍：
Value in Health contains original research articles for pharmacoeconomics, health economics, and outcomes research (clinical, economic, and patient-reported outcomes/preference-based research), as well as conceptual and health policy articles that provide valuable information for health care decision-makers as well as the research community. As the official journal of ISPOR, Value in Health provides a forum for researchers, as well as health care decision-makers to translate outcomes research into health care decisions.
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