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