PENDING U.S. LEGISLATION

肿瘤学治疗学的开发商紧密遵循研究，以加速儿童法案（也称为《儿童竞赛法》），分别在众议院和参议院中引入了两个相同的法案，H.R.58581和S.3239,22016年7月14日。

This legislation would apply to new drug applications (NDAs) and biologic license applications (BLAs) submitted 18 months after enactment, and would impact many therapies in development today. The bills have potential to dramatically alter the pediatric oncology landscape by expanding Pediatric Research Equity Act (PREA) requirements for oncology therapeutics. For drugs being developed for an indication that affects children, PREA requires pediatric trials for new active ingredients, indications, dosage forms, dosing regimens, and routes of administration. For oncology drugs, RACE would remove the requirement that the indication studied in adults affects children; instead, the obligation to conduct pediatric trials would be triggered if the drug or biologic is “directed at a molecular target present in one or more cancers in one or more pediatric populations.” Also, orphan designation would no longer exempt such products from PREA requirements.

种族的规定之一是，除了在提交初始儿科研究计划（IPSP）的90天内提供的评论评论外，FDA还将在第一阶段结束时与赞助商会面，以讨论IPSP的准备。对于与小儿癌相关的分子靶标的药物。在颁布的一年内，将针对研究设计和可能的分子目标发布指导。在种族下，国会将在2021年7月12日之前收到有关该立法实施的报告。

This insight brief, authored by IQVIA Pediatric and Oncology Center of Excellence experts, provides a summary of the proposed legislation and potential impacts on oncology development programs if adopted. The insight brief also examines specific considerations and planning for pediatric oncology trials, and the changing landscape in pediatric oncology drug development.

每年在美国，约有16,000名儿童被诊断出患有癌症，每年有4个诊断约30万名。5各种形式的白血病几乎占所有儿童癌症的三分之一。儿童期癌症被归类为罕见疾病，在临床试验中识别，招募和保留患者方面，与其他罕见疾病相关的挑战。

近几十年来，各种形式的小儿癌的结果大大改善。这样做的原因很多，包括在学术研究人员之间具有强烈的国家和国际合作精神，合作团体推动了研究议程。此外，在临床试验环境中，经常照顾儿科患者的心态。迄今为止的大多数进展是通过对标准细胞毒性剂的给药，组合和调度的增量调整而获得的。

Relative pediatric cancer survival rates are shown in. Many of these survival rates have now started to plateau. This coincides with an era of discovery that is delivering an improved understanding of the molecular basis of many childhood cancers. The challenge is to understand how to leverage this explosion in scientific knowledge to further improve outcomes.

The first challenge is the need for targeted agents for pediatric cancers. These will come from repurposed targeted agents developed for adult tumors or from the development of novel agents against novel targets defined in pediatric cancers. Advances in genomic technologies are expanding understanding of how pediatric tumors can be targeted. Potential legislative changes under RACE may also expand the range of adult agents studied in pediatric populations.

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