Office of the Commissioner, US Food and Drug Administration, Silver Spring, Maryland, USA.
Center for Biologic Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
J Clin Pharm Ther. 2021 Dec;46(6):1576-1581. doi: 10.1111/jcpt.13532. Epub 2021 Sep 20.
The discussion about health equity in the United States frequently involves concerns over racial and ethnic minority under-representation in clinical trials and particularly in trials conducted in support of product approvals. The FDA has long worked to encourage diverse participation in clinical trials and through its Drug Trials Snapshots (DTS) program, the U.S. Food and Drug Administration (FDA) has moved to make trial demographic data more accessible and transparent. We conducted a demographic study of U.S. participants in clinical trials for FDA-approved new drugs (new molecular entities [NMEs], and original Biologics License Applications [BLAs]) from 2015 to 2019, as reported in DTS database with a purpose of understanding the extent to which U.S.-based trials used to support product approvals represent the racial and ethnic diversity of the U.S. population by therapeutic area.
Participant-level trial data were collected by accessing the FDA electronic common technical document (eCTD), for the applications used to publish each Snapshot. The therapeutic area (TA) for each drug was determined by review division assignment. The demographic data were analysed and compared to U.S. census data.
We examined 102,596 U.S. participants in trials of new drugs that were approved and presented in Drug Trials Snapshots between 2015 and 2019. White participation ranged from 51% in psychiatric trials to 90% in cardiovascular (CV) trials; Black or African American participation ranged from 5% in medical imaging to 45% in psychiatric trials; Asian participation ranged from 0.75% in CV to 4% in dermatologic trials; and Hispanic or Latino participation ranged from 1% in medical imaging to 22% in infectious diseases and gastroenterology trials.
Our data showed variable representation of racial and ethnic minorities across therapeutic areas at the U.S. sites. Blacks or African Americans were represented at or above U.S. census estimates across most therapeutic areas, while Asians and American Indian or Alaska Natives were consistently underrepresented. Hispanic or Latino participation across most therapeutic areas was below U.S. census estimates, however, more variable, and a sizable proportion of data was missing. The next step is a comparison of trial participation based on disease prevalence and epidemiology, which is a more accurate assessment of trial diversity.
在美国，关于卫生公平性的讨论经常涉及到对临床试验中少数族裔代表性不足的担忧，尤其是在支持产品批准的试验中。美国食品和药物管理局（FDA）长期以来一直致力于鼓励临床试验中的多样性参与，并通过其药物试验快照（DTS）计划，FDA 已努力使试验人口统计学数据更易于获取和透明。我们对 2015 年至 2019 年期间在美国进行的 FDA 批准的新药（新分子实体[NME]和原始生物制品许可申请[BLA]）临床试验中的美国参与者进行了一项人口统计学研究，这些数据来自 DTS 数据库，旨在了解用于支持产品批准的基于美国的试验在多大程度上代表了美国人口的种族和民族多样性按治疗领域划分。
通过访问 FDA 电子通用技术文件（eCTD）获取用于发布每个快照的应用程序，收集参与者级别的试验数据。每个药物的治疗领域（TA）通过审查部门分配确定。对人口统计学数据进行了分析，并与美国人口普查数据进行了比较。
我们检查了 2015 年至 2019 年期间在 DTS 中批准和展示的新药试验中的 102596 名美国参与者。白人参与度从精神病学试验的 51%到心血管（CV）试验的 90%不等；黑人或非洲裔美国人的参与度从医学影像学的 5%到精神病学试验的 45%不等；亚洲人的参与度从心血管的 0.75%到皮肤科的 4%不等；西班牙裔或拉丁裔的参与度从医学影像学的 1%到传染病和胃肠病学试验的 22%不等。
我们的数据显示，在美国试验点，不同治疗领域的少数族裔代表情况各不相同。在大多数治疗领域，黑人或非洲裔美国人的代表人数与美国人口普查估计数持平或高于估计数，而亚洲人和美国印第安人或阿拉斯加原住民的代表人数一直不足。西班牙裔或拉丁裔的参与度在大多数治疗领域都低于美国人口普查估计数，但更为多变，而且相当一部分数据缺失。下一步是根据疾病流行率和流行病学进行试验参与情况的比较，这是对试验多样性的更准确评估。
