ImportanceThe Best Pharmaceuticals for Children Act states that in issuing a written request (WR), the US Food and Drug Administration (FDA) shall consider the adequate representation (eg, proportionate to the disease population) of children from racial and ethnic minority populations. If the terms of the WR are fulfilled, the FDA may grant an additional 6 months of exclusivity for any unexpired patents and exclusivities attached to approved indications.ObjectiveTo report on the race and ethnicity of participants enrolled in pediatric studies conducted in response to WRs for which pediatric exclusivity was granted between 2001 and 2021.Design, Setting, and ParticipantsThis retrospective review examines pediatric exclusivity request submissions for oncologic drugs that received pediatric exclusivity between December 2001 and January 2021 based on fulfillment of the requirements of a WR that were identified using the FDA’s Document Archiving Reporting and Regulatory Tracking System. Demographic data were manually abstracted from supporting study reports, and data were pooled across submissions for the analysis. Data were analyzed throughout 2022 and 2023.Main Outcomes and MeasuresRepresentation by race, sex, and ethnicity in pediatric studies conducted in response to WRs.ResultsA total of 22 pediatric exclusivity requests were identified, comprising 40 studies and 2025 patients. Most trials (26 [65%]) in the analysis were cooperative group studies. Representation by race was as follows: American Indian/Alaska Native (13 [0.6%]), African American/Black (228 [11.3%]), Asian (92 [4.6%]), Native Hawaiian/other Pacific Islander (33 [1.6%]), White (1303 [64.3%]), other (194 [9.6%]), and unknown/not reported (162 [8.0%]). Representation by sex was female individuals (41.2%) and male individuals (58.8%). Ethnicity was as follows: Hispanic (226 [5.7%]), non-Hispanic (910 [22.5%]), unknown/not reported ethnicity (2800 [69.1%]), and other ethnicity (114 [2.8%]).Conclusions and RelevanceThe study results suggest that overall, representation of participants of racial and ethnic minority groups in studies supporting pediatric exclusivity requests appear comparable with the racial distribution of childhood cancers in the US based on data from the National Childhood Cancer Registry Explorer. However, fewer Hispanic participants were enrolled in the trials we reviewed (8%) compared with the representation of Hispanic patients in the National Childhood Cancer Registry (28%). This discrepancy may be partially explained by the large proportion of participants with unknown information regarding ethnicity. Further research into the reasons for the large proportion of participants with missing ethnicity information is needed.

中文翻译：

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对通过 FDA 书面请求进行的肿瘤药物儿科临床试验参与者的种族和民族代表性进行审查

重要性《儿童最佳药品法案》规定，在发出书面请求 (WR) 时，美国食品和药物管理局 (FDA) 应考虑少数民族儿童的充分代表性（例如，与疾病人口成比例）。如果 WR 的条款得到履行，FDA 可能会为任何未过期的专利和已批准适应症所附的独占权授予额外 6 个月的独占权。 目的报告参加针对 WR 进行的儿科研究的参与者的种族和民族2001 年至 2021 年期间授予儿科独占权。 设计、环境和参与者这项回顾性审查审查了 2001 年 12 月至 2021 年 1 月期间获得儿科独占权的肿瘤药物提交的儿科独占权请求，该申请基于满足使用 FDA 确定的 WR 要求文件归档报告和监管跟踪系统。人口统计数据是从支持研究报告中手动提取的，并且汇总了提交的数据以进行分析。对 2022 年和 2023 年的数据进行了分析。主要结果和措施在针对 WR 进行的儿科研究中按种族、性别和民族进行表示。结果共确定了 22 项儿科独占请求，其中包括 40 项研究和 2025 名患者。分析中的大多数试验（26 [65%]）都是合作小组研究。按种族划分的代表性如下：美洲印第安人/阿拉斯加原住民 (13 [0.6%])、非裔美国人/黑人 (228 [11.3%])、亚洲人 (92 [4.6%])、夏威夷原住民/其他太平洋岛民 (33 [ 1.6%]）、白人（1303 [64.3%]）、其他（194 [9.6%]）和未知/未报告（162 [8.0%]）。按性别划分，女性（41.2%）和男性（58.8%）。种族如下：西班牙裔 (226 [5.7%])、非西班牙裔 (910 [22.5%])、未知/未报告的种族 (2800 [69.1%]) 和其他种族 (114 [2.8%])。 结论研究结果表明，总体而言，根据国家儿童癌症登记浏览器的数据，支持儿科独家请求的研究中种族和族裔群体参与者的代表性似乎与美国儿童癌症的种族分布相当。然而，与国家儿童癌症登记处西班牙裔患者的代表性 (28%) 相比，参加我们审查的试验的西班牙裔参与者较少 (8%)。这种差异的部分原因可能是大部分参与者的种族信息未知。需要进一步研究大部分参与者缺少种族信息的原因。