Exposure–response (E–R) modeling provides a quantitative tool to leverage adult data to support pediatric trial design and drug approval. The pediatric E–R studies submitted to US Food and Drug Administration (FDA) between 2007 and 2018 were surveyed in the context of various types of trial designs supporting drug approval in the pediatric population. The applications of E–R evaluation in pediatric drug development programs are mainly focused on three areas: (i) supporting pediatric extrapolation when the E–R relationships are similar between the pediatric and adult populations; (ii) dose selection to balance the risk–benefit profile based on the change in efficacy and safety response with different exposure levels; and (iii) approval of a new formulation, new dosing regimen, or new route of administration, where E–R evaluation helps quantify the change in clinical response between the old and new strategies. E–R modeling will continue to play an expanded role in pediatric drug development in the future.

中文翻译：

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提交美国食品和药物管理局的儿科药物开发研究中的暴露-反应评估。

暴露-反应（ER）建模提供了一种定量工具，可以利用成人数据来支持儿科试验设计和药物批准。在2007年至2018年间提交给美国食品药品监督管理局（FDA）的儿科E-R研究中，对支持儿科人群药物批准的各种类型的试验设计进行了调查。E-R评估在儿科药物开发计划中的应用主要集中在三个领域：（i）当儿科和成年人口之间的E-R关系相似时，支持儿科外推；（ii）选择剂量，以根据不同暴露水平下药效和安全性反应的变化来平衡风险-收益状况；（iii）批准新的制剂，新的给药方案或新的给药途径，E–R评估有助于量化新旧策略之间临床反应的变化。未来，ER模型将继续在儿科药物开发中发挥更大的作用。