Smith Zachary, Botto Emily, Johnson Otis, Rudo Todd, Getz Kenneth
Tufts Center for the Study of Drug Development, Tufts University, Boston, MA, USA.
Trial Equity, Philadelphia, PA, USA.
Ther Innov Regul Sci. 2024 Jan;58(1):143-152. doi: 10.1007/s43441-023-00579-1. Epub 2023 Sep 29.
A lack of diversity and representation in clinical trials is an established issue in drug development, and the COVID-19 pandemic increased awareness of the problem among the general public. This awareness has led to increased pressure on drug development sponsors, as well as additional attention and regulation from federal bodies, to improve the diversity of clinical trials. This study updates existing baselines regarding demographic disparities, as well as detecting early signs that the situation may be starting to improve.
Building on an existing dataset, this study collected and analyzed pivotal trial demographic data for drugs and biologics approved by the FDA between 2007 and 2021. Demographic data were collected from applications on the FDA website and clinicaltrials.gov, and compared to indication-specific demographic data when available, or US census estimates when they were not. Regression analyses were used to test for significant trends in reporting of demographic data and representation in pivotal trials, as well as the effect of representation on clinical trial duration and FDA review.
Reporting of demographic data has improved significantly for all three demographic categories (sex, racial identity, and ethnic identity) over the observed time period (p < 0.0001). During this time period, overrepresentation of white participants has decreased significantly (p < 0.0001), and representation of Black participants has increased (p = 0.0003). Other racial and ethnic identities did not show significant trends. Representation of demographic subgroups was not significant predictors of trial duration except for the representation of Black participants, which was a negative correlation, indicating that as representation of Black participants increases, trial duration decreases (p = 0.0350).
临床试验中缺乏多样性和代表性是药物研发中一个既定的问题，而新冠疫情提高了公众对这一问题的认识。这种认识给药物研发赞助商带来了更大压力，同时也引起了联邦机构更多关注并加强了监管，以提高临床试验的多样性。本研究更新了有关人口统计学差异的现有基线，并检测情况可能开始改善的早期迹象。
本研究在现有数据集的基础上，收集并分析了2007年至2021年期间美国食品药品监督管理局（FDA）批准的药物和生物制品的关键试验人口统计学数据。人口统计学数据从FDA网站和ClinicalTrials.gov上的申请中收集，并在有可用数据时与特定适应症的人口统计学数据进行比较，若无可用数据，则与美国人口普查估计数进行比较。回归分析用于检验人口统计学数据报告和关键试验代表性方面的显著趋势，以及代表性对临床试验持续时间和FDA审评的影响。
在观察期内，所有三个人口统计类别（性别、种族身份和族裔身份）的人口统计学数据报告都有显著改善（p < 0.0001）。在此期间，白人参与者的过度代表性显著下降（p < 0.0001），黑人参与者的代表性有所增加（p = 0.0003）。其他种族和族裔身份未显示出显著趋势。除黑人参与者的代表性呈负相关外，人口统计亚组的代表性并非试验持续时间的显著预测因素，这表明随着黑人参与者代表性的增加，试验持续时间会缩短（p = 0.0350）。
