Children 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.

This 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.

We 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.

Including 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.

从历史上看，儿童已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中。评估的延迟可能导致标签外使用，而没有作为新药的唯一获得途径而提供剂量信息。ASCO和癌症研究之友于2016年5月召集了一个多方利益相关者研讨会，以确定在科学上适当的时候扩大试验资格的机会，以便将18岁以下的儿童纳入首次人类和其他成人癌症临床试验中。

该小组召集了来自学术界，政府和行业的专家，以审查在肿瘤临床试验中招收儿童和青少年的障碍。我们评估了历史背景，已发表的文献，法规方面的考虑因素以及与降低肿瘤临床试验入组年龄相关的无数风险和收益。

我们得出的结论是，关于将儿童包括在肿瘤临床试验的早期阶段的许多历史问题在当前的儿科肿瘤学和药物开发的科学和临床环境中并不适用。我们提供了有关如何实现将儿童纳入早期研究性癌症药物试验的具体建议。在评估各种肿瘤类型的剂量，安全性和药代动力学的早期试验和评估跨越成人和儿童人群的特定疾病的疗效的晚期试验中，将儿童患者自动纳入是合适的。

将儿童纳入经过适当设计的成人临床肿瘤试验中是可行的，并且可以在不损害安全性或发展策略的前提下，增加他们获得这些药物的途径。