Modernizing Clinical Trial Eligibility: Recommendations of the American Society of Clinical Oncology-Friends of Cancer Research Minimum Age Working Group
Modernizing Clinical Trial Eligibility: Recommendations of the American Society of Clinical Oncology-Friends of Cancer Research Minimum Age Working Group
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DOI：
10.1200/jco.2017.74.4144
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发表时间：
2017-11-20
影响因子：
45.3
通讯作者：
Reaman, Gregory
中科院分区：
文献类型：
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作者：
Gore, Lia;Ivy, S. Percy;Reaman, Gregory
目的儿童历来被排除在有前途的新癌症药物的首次人体研究和后期成人临床试验之外。评估的延迟可能导致标签外使用，而没有剂量信息作为获得新药的唯一途径。ASCO和癌症研究之友于2016年5月召开了一次多方利益相关者研讨会，以确定何时在科学上适当扩大试验资格，将18岁以下的儿童纳入首次人体和其他成人癌症临床试验。和行业审查障碍招募儿童和青少年在肿瘤临床试验。我们评估了历史背景，已发表的文献，监管方面的考虑，和无数的风险和好处与降低年龄的入学肿瘤临床试验。ResultsWe的结论是，许多历史上的关注，包括儿童早期肿瘤临床试验不适用于目前的科学和临床环境的儿科肿瘤和药物开发;我们提供了具体的建议，如何纳入儿童在早期阶段的研究性癌症药物试验可能会完成。在评估剂量、安全性、结论在适当设计的成人临床肿瘤学试验中纳入儿童是可行的，并且可以在不影响安全性或安全性的情况下增加儿童对这些药物的获得。发展战略。(C)2017年美国临床肿瘤学会
PurposeChildren have historically been excluded from first-in-human studies of promising new cancer drugs and later phase adult clinical trials. Delays in evaluation may result in off-label use without dosing information as the only access to new drugs. A multistakeholder workshop was convened in May 2016 by ASCO and Friends of Cancer Research to identify opportunities for when it would be scientifically appropriate to expand trial eligibility to include children younger than age 18 years in first-in-human and other adult cancer clinical trials.MethodsThis group convened experts from academia, government, and industry to review barriers to enrolling children and adolescents in oncology clinical trials. We evaluated the historical context, published literature, regulatory considerations, and myriad risks and benefits associated with lowering the age of enrollment on oncology clinical trials.ResultsWe conclude that many of the historical concerns about including children early in oncology clinical trials do not apply in the current scientific and clinical environment of pediatric oncology and drug development; we provide specific recommendations for how the inclusion of children in early-phase investigational cancer drug trials might be accomplished. Automatic inclusion of pediatric patients is appropriate in early-phase trials that assess dose, safety, and pharmacokinetics in a variety of tumor types and later phase trials that assess efficacy in a specific disease that spans adult and pediatric populations.ConclusionIncluding children in appropriately designed adult clinical oncology trials is feasible and can be done in a way that enhances their access to these agents without compromising safety or development strategies. (C) 2017 by American Society of Clinical Oncology