Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR PRO Good Research Practices for the Assessment of Children and Adolescents Task Force
Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR PRO Good Research Practices for the Assessment of Children and Adolescents Task Force
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DOI：
10.1016/j.jval.2013.04.004
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发表时间：
2013-06-01
期刊：
影响因子：
4.5
通讯作者：
Bullinger, Monika
中科院分区：
文献类型：
--
作者：
Matza, Louis S.;Patrick, Donald L.;Bullinger, Monika
背景资料：儿童和青少年的患者报告结局（PRO）工具通常包含在临床试验中，旨在收集数据以支持医疗产品标签中的声明。目的：当前工作组报告的目的是为儿科PRO研究推荐良好实践，该研究旨在为监管决策提供信息，并支持医疗产品标签中的声明。这些建议是基于一个跨学科研究小组的共识，该小组是为国际药物经济学和结局研究学会（ISPOR）相关的一个工作组聚集的。在支持证据有限或一般原则可能不适用于每种情况的领域，本工作组报告确定了在决定儿科PRO仪器的设计和使用时需要考虑的因素，同时强调了需要进一步研究的问题。良好研究实践：讨论了五个良好的研究实践：1）考虑发育差异，并确定基于年龄的PRO管理标准：在以往研究的基础上讨论了四个年龄组（
Background: Patient-reported outcome (PRO) instruments for children and adolescents are often included in clinical trials with the intention of collecting data to support claims in a medical product label. Objective: The purpose of the current task force report is to recommend good practices for pediatric PRO research that is conducted to inform regulatory decision making and support claims made in medical product labeling. The recommendations are based on the consensus of an interdisciplinary group of researchers who were assembled for a task force associated with the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). In those areas in which supporting evidence is limited or in which general principles may not apply to every situation, this task force report identifies factors to consider when making decisions about the design and use of pediatric PRO instruments, while highlighting issues that require further research. Good Research Practices: Five good research practices are discussed: 1) Consider developmental differences and determine age-based criteria for PRO administration: Four age groups are discussed on the basis of previous research (