Children's Mercy Hospital and University of Missouri-Kansas City School of Medicine, Kansas City, Missouri;
Penn State College of Medicine, Hershey, Pennsylvania.
Pediatrics. 2021 May;147(5). doi: 10.1542/peds.2020-042903. Epub 2021 Apr 12.
The Best Pharmaceuticals for Children Act (BPCA) incentivizes the study of on-patent medicines in children and mandates that the National Institutes of Health sponsor research on off-patent drugs important to pediatric therapeutics. Failing to enroll cohorts that reflect the pediatric population at large restricts the generalizability of such studies. In this investigation, we evaluate racial and ethnic minority representation among participants enrolled in BPCA-sponsored studies.
Data were obtained for all participants enrolled in 33 federally funded studies of drugs and devices conducted from 2008 through June 2020. Observed racial and ethnic distributions were compared with expected distributions by sampling Census data at the same geographic frequency as in the studies. Racial and ethnic enrollment was examined by demography, geography, study type, study burden, and expected bias. Standard descriptive statistics, χ, generalized linear models, and linear regression were applied.
A total of 10 918 participants (51% male, 6.6 ± 8.2 years) were enrolled across 46 US states and 4 countries. Studies ranged from treatment outcome reviews to randomized, placebo-controlled trials. Minority enrollment was comparable to, or higher than, expected (+0.1% to +2.6%) for all groups except Asian Americans (-3.7%, < .001). American Indian and Alaskan Native and multiracial enrollment significantly increased over the evaluation period ( < .01). There were no significant differences in racial distribution as a function of age or sex, although differences were observed on the basis of geography, study type, and study burden.
This study revealed no evidence of racial and ethnic bias in enrollment for pediatric studies conducted with funding from BPCA, fulfilling the legislation's expectation to ensure adequate representation of all children.
《最佳儿童用药法案》（BPCA）鼓励对专利药品进行儿童研究，并要求美国国立卫生研究院赞助对儿科治疗学重要的非专利药物研究。未能招募反映总体儿科人群的队列限制了此类研究的普遍性。在这项研究中，我们评估了在 BPCA 赞助的研究中入组的参与者中的种族和少数民族代表性。
从 2008 年到 2020 年 6 月，我们获取了所有参加 33 项联邦资助的药物和器械研究的参与者的数据。通过在与研究相同的地理频率上抽样人口普查数据，将观察到的种族和族裔分布与预期分布进行比较。根据人口统计学、地理位置、研究类型、研究负担和预期偏差来检查种族和族裔的入组情况。应用了标准描述性统计、χ²检验、广义线性模型和线性回归。
共有 10918 名参与者（51%为男性，6.6±8.2 岁）来自美国 46 个州和 4 个国家。研究范围从治疗结果审查到随机、安慰剂对照试验。除了亚裔美国人（-3.7%，<0.001）外，所有组别的少数民族入组率均与预期相符或高于预期（+0.1%至+2.6%）。美洲印第安人和阿拉斯加原住民以及多种族入组率在评估期间显著增加（<0.01）。种族分布与年龄或性别无显著差异，但在地理位置、研究类型和研究负担方面存在差异。
这项研究没有发现 BPCA 资助的儿科研究在入组方面存在种族和民族偏见的证据，这符合该立法确保所有儿童都有足够代表性的期望。
