Journal of evidence-based medicine · 2026
Development and Validation of a Rapid Benefit-Risk Assessment Index for Pediatric Off-Label Drug Use.
СтатьяВалидационное исследованиеНазвание и аннотация: Перевод в очереди
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Li Xuejuan, Xu Yingna, Jin Guanglei, Jiang Wei, Chen Zebin, Zheng Zhihua, Wang Xiaoling, Mo Xiaolan, Liu Xinyu, Zhou Benjie, Chang Hong, Deng Shaojie, Fan Xiaomei, Wang Jinping, Jia Yuntao, Li Zhong, Miao Jing, Lin Yunzhu, Fang Rui, Li Zhiling, Zhu Jianhong, He Zhichao, Jin Yinghui, Su Qiru, Guo Wudong, Jiang Jie, Li Sha, Yuan Jinqiu, Yang Jun, Liu Sixi, Su Zhe, Bao Yanmin, Cao Dezhi, Wang Zhaoxia, Tan Weiping, Zhang Bihong, Gong Chunxiu, Wang Xiaodong, Zhang Zhiyong, Xie Chunhong, Ma Yushan, Fu Haidong, Luo Xinle, Wu Junyan
Аннотация
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 rs = 0.97 (benefits) and rs = 0.68 (risks) (both p < 0.05), indicating strong alignment with expert consensus. Junior experts showed lower correlations (benefits rs = 0.89; risks rs = 0.56) than seniors (benefits rs = 0.97; risks rs = 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.
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 rs = 0.97 (benefits) and rs = 0.68 (risks) (both p < 0.05), indicating strong alignment with expert consensus. Junior experts showed lower correlations (benefits rs = 0.89; risks rs = 0.56) than seniors (benefits rs = 0.97; risks rs = 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.