Researchers', Regulators', and Sponsors' Views on Pediatric Clinical Trials: A Multinational Study
Researchers', Regulators', and Sponsors' Views on Pediatric Clinical Trials: A Multinational Study
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DOI：
10.1542/peds.2016-1171
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发表时间：
2016-10-01
期刊：
影响因子：
8
通讯作者：
Caldwell, Patrina H. Y.
中科院分区：
文献类型：
--
作者：
Joseph, Pathma D.;Craig, Jonathan C.;Caldwell, Patrina H. Y.
背景和目标：过去十年，监管环境发生了巨大变化，以支持更多涉及儿童的试验，但针对儿童的挑战和跨收入地区的不公平行为仍然存在。本研究的目的是描述利益相关者对儿童试验的态度和意见，为在全球范围内促进更多高质量、相关儿科试验提供更多策略。方法：对有目的地从低收入到中等收入国家和高收入国家抽样的利益相关者（研究人员、监管者和申办者）进行关键知情人半结构化访谈。对记录进行了主题分析。 结果：来自 10 个国家的 35 名利益相关者接受了采访。确定了五个主要主题：解决普遍存在的不平等问题（缺乏安全性和有效性数据、知识差距、不稳定的环境、双重标准、背景相关性、市场驱动力、行业赞助偏见和高昂的成本）；应对基础设施障碍（资源限制、缺乏儿科试验专业知识以及后勤复杂性）；应对复杂的道德和监管框架（“严厉”的监督、模糊的要求、剥削、过度的家长作风以及强制与志愿服务的不稳定）；尊重儿童的独特性（儿科研究范式、适合儿童的方法和以家庭为中心的赋权）；结论：利益相关者承认，监管环境的变化鼓励了更多的儿童试验，但他们认为不平等以及政治、监管和资源障碍仍然存在。建议将试验作为常规临床护理的一部分，满足儿童的独特需求，并简化监管审批。利益相关者建议加强国际合作，建立集中试验基础设施，并使研究与儿童健康优先事项保持一致，以鼓励满足全球儿童保健需求的试验。
BACKGROUND AND OBJECTIVE: The last decade has seen dramatic changes in the regulatory landscape to support more trials involving children, but child-specific challenges and inequitable conduct across income regions persist. The goal of this study was to describe the attitudes and opinions of stakeholders toward trials in children, to inform additional strategies to promote more high-quality, relevant pediatric trials across the globe.METHODS: Key informant semi-structured interviews were conducted with stakeholders (researchers, regulators, and sponsors) who were purposively sampled from low-to middle-income countries and high-income countries. The transcripts were thematically analyzed.RESULTS: Thirty-five stakeholders from 10 countries were interviewed. Five major themes were identified: addressing pervasive inequities (paucity of safety and efficacy data, knowledge disparities, volatile environment, double standards, contextual relevance, market-driven forces, industry sponsorship bias and prohibitive costs); contending with infrastructural barriers (resource constraints, dearth of pediatric trial expertise, and logistical complexities); navigating complex ethical and regulatory frameworks ("draconian" oversight, ambiguous requirements, exploitation, excessive paternalism and precariousness of coercion versus volunteerism); respecting uniqueness of children (pediatric research paradigms, child-appropriate approaches, and family-centered empowerment); and driving evidence-based child health (advocacy, opportunities, treatment access, best practices, and research prioritization).CONCLUSIONS: Stakeholders acknowledge that changes in the regulatory environment have encouraged more trials in children, but they contend that inequities and political, regulatory, and resource barriers continue to exist. Embedding trials as part of routine clinical care, addressing the unique needs of children, and streamlining regulatory approvals were suggested. Stakeholders recommended increasing international collaboration, establishing centralized trials infrastructure, and aligning research to child health priorities to encourage trials that address global child health care needs.