Development and Validation of a Rapid Benefit-Risk Assessment Index for Pediatric Off-Label Drug Use.

IF 4.7 2区医学 Q1 MEDICINE, GENERAL & INTERNAL

Journal of Evidence‐Based Medicine Pub Date : 2026-08-30 DOI:10.1111/jebm.70177

Xuejuan Li, Yingna Xu, Guanglei Jin, Wei Jiang, Zebin Chen, Zhihua Zheng, Xiaoling Wang, Xiaolan Mo, Xinyu Liu, Benjie Zhou, Hong Chang, Shaojie Deng, Xiaomei Fan, Jinping Wang, Yuntao Jia, Zhong Li, Jing Miao, Yunzhu Lin, Rui Fang, Zhiling Li, Jianhong Zhu, Zhichao He, Yinghui Jin, Qiru Su, Wudong Guo, Jie Jiang, Sha Li, Jinqiu Yuan, Jun Yang, Sixi Liu, Zhe Su, Yanmin Bao, Dezhi Cao, Zhaoxia Wang, Weiping Tan, Bihong Zhang, Chunxiu Gong, Xiaodong Wang, Zhiyong Zhang, Chunhong Xie, Yushan Ma, Haidong Fu, Xinle Luo, Junyan Wu

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引用次数: 0

Abstract

Objective: To develop and validate a rapid, evidence-based benefit-risk assessment index for off-label drug use in pediatrics, thereby promoting standardized clinical decision-making.

Methods: Under the auspices of the Guangdong Pharmaceutical Association, a multidisciplinary expert panel from pediatrics, pharmacy, methodology, and ethics was convened. Systematic literature searches informed a preliminary index framework. Three Delphi rounds refined indicators. Weights were assigned using the analytic hierarchy process. Expert-based validation involved 20 hematology-oncology experts assessing 10 cases; intraclass correlation coefficient (ICC) and Spearman's correlations evaluated reliability and validity.

Results: The index consists of 2 primary dimensions (benefits: 56 points; risks: 44 points), 10 third-level, and 40 fourth-level indicators, with five prerequisites (e.g., no alternatives, informed consent). The ICC was 0.77 (good reliability); Spearman's correlations were r_s = 0.97 (benefits) and r_s = 0.68 (risks) (both p < 0.05), indicating strong alignment with expert consensus. Junior experts showed lower correlations (benefits r_s = 0.89; risks r_s = 0.56) than seniors (benefits r_s = 0.97; risks r_s = 0.78), highlighting the system's role in standardizing assessments.

Conclusion: This quantitative index provides an evidence-based tool for pediatric off-label drug decisions, supporting clinicians, policymakers, and standardization efforts.

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儿童超说明书用药快速获益-风险评估指标的开发与验证。

目的：建立并验证一种快速、循证的儿科超说明书用药获益-风险评价指标，促进临床规范化决策。方法：在广东省药学会的主持下，召集了儿科、药学、方法学、伦理学等多学科专家小组。系统的文献检索提供了初步的索引框架。三轮德尔菲精炼指标。采用层次分析法分配权重。专家验证涉及20名血液肿瘤学专家评估10例；类内相关系数（ICC）和Spearman相关评估信度和效度。结果：该指标由2个主要维度（获益56分，风险44分）、10个三级指标和40个四级指标组成，有5个先决条件（如无替代方案、知情同意）。ICC为0.77（信度良好）；Spearman相关性rs = 0.97（收益）和rs = 0.68（风险）（均p < 0.05），表明与专家共识高度一致。初级专家的相关性（收益rs = 0.89，风险rs = 0.56）低于高级专家（收益rs = 0.97，风险rs = 0.78），突出了该系统在标准化评估中的作用。结论：该定量指标为儿科超说明书用药决策提供了循证工具，为临床医生、政策制定者和标准化工作提供了支持。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Evidence‐Based Medicine MEDICINE, GENERAL & INTERNAL-

CiteScore

11.20

自引率

1.40%

发文量

期刊介绍： The Journal of Evidence-Based Medicine (EMB) is an esteemed international healthcare and medical decision-making journal, dedicated to publishing groundbreaking research outcomes in evidence-based decision-making, research, practice, and education. Serving as the official English-language journal of the Cochrane China Centre and West China Hospital of Sichuan University, we eagerly welcome editorials, commentaries, and systematic reviews encompassing various topics such as clinical trials, policy, drug and patient safety, education, and knowledge translation.