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.
方法:我们分多个阶段构建了基于MCDA的模型,以创建儿童健康技术综合评估(CATCH)框架。使用改进的德尔菲过程,利益相关者具有广泛的学科和地理差异(n=23),我们改进了先前生成的标准,并开发了基于等级的权重.我们建立了一个与标准相关的评分标准,用于评估新药的增量益处。三名临床医生通过对九种药物进行飞行员评分来独立评估理解力。然后,我们通过专家小组的结构化审议(n=10)验证了CATCH的两种儿童癌症疗法,获得个人分数,共识分数和口头反馈。分析包括描述性统计,专题分析,探索性分歧指数(DI),和敏感性分析。
结果:修改后的Delphi过程产生了10个标准,基于绝对重要性/相关性和商定重要性(中位数DI=0.34):有效性,儿童特定健康相关的生活质量,疾病严重程度,未满足的需求,治疗安全,Equity,家庭影响,生命历程发展,Rarity,公平分享生活。试点评分导致调整的标准定义和更精确的分数缩放指南。验证小组成员认可框架的关键值修饰符。他们的个人预分模式与审议性共识得分密切相关。
结论:我们迭代地开发了一个价值评估框架,该框架涵盖了儿童特定健康和非健康收益的维度。CATCH可以提高HTA决策对加拿大儿童和可比卫生系统的丰富性和相关性。