Disparity of Race Reporting and Representation in Clinical Trials Leading to Cancer Drug Approvals From 2008 to 2018
Disparity of Race Reporting and Representation in Clinical Trials Leading to Cancer Drug Approvals From 2008 to 2018
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DOI：
10.1001/jamaoncol.2019.1870
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发表时间：
2019-10-01
期刊：
影响因子：
28.4
通讯作者：
Raghav, Kanwal
中科院分区：
文献类型：
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作者：
Loree, Jonathan M.;Anand, Seerat;Raghav, Kanwal
在导致美国食品和药物管理局（FDA）批准肿瘤药物的具有里程碑意义的试验中，种族/族裔群体的报告和代表情况如何？在过去十年中，FDA批准的230项肿瘤药物试验中，只有145项（63%）试验报告了种族。与白人（98%的预期比例）相比，黑人（22%的预期比例）和西班牙裔（44%的预期比例）在这些试验中的代表性低于他们在美国癌症人群中的比例。意义：在具有里程碑意义的肿瘤试验中，经常出现种族报告和代表性不佳的情况（尤其是黑人和西班牙裔），需要加大努力，提高少数族裔的代表性，消除这些差异。本数据库研究评估了支持FDA肿瘤药物批准的试验中种族报告的频率和比例种族代表。在研究中，特别是在建立护理标准的临床试验中，有代表性的种族/族裔参与是必要的，以尽量减少结果的差异，并维护保健方面的社会公平。目的评价支持美国食品和药物管理局（FDA）肿瘤药物批准的试验中种族报告的频率和比例种族代表性。设计、设置和参与者数据库研究支持2008年7月至2018年6月期间FDA肿瘤药物批准的所有报告试验。试验的主要报告来自PubMed和ClinicalTrials.gov。食品和药物管理局的批准是使用FDA档案确定的。根据美国国家癌症研究所的监测、流行病学和最终结果以及美国人口普查数据库，计算了美国人口基于种族的癌症估计。主要结局和测量方法主要结局是报道种族的试验比例和种族患者参与试验的比例。次要结果包括种族亚组分析报告和试验中种族比例与美国人口之间的差距。采用描述性统计、Fisher精确检验和chi(2)检验对数据进行分析。报告了95% ci的比例和优势比（OR）。结果在230项试验中，共有112293名受试者，145项（63.0%）报告了至少1个种族，18项（7.8%）记录了美国的4个主要种族（白人、亚洲人、黑人和西班牙人），58项（25.2%）报告了种族亚组分析。分别有144项（62.6%）、110项（47.8%）、88项（38.2%）和23项（10.0%）试验报道了白人、亚洲人、黑人和西班牙裔人种。在2008年7月至2013年6月与2013年7月至2018年6月之间，报告种族的试验数量（45项[56.6%]对100项[67.1%]；OR, 1.63; 95% CI, 0.93-2.87； P= 0.09）和种族亚组分析（13项[16.1%]对45项[30.2%]；OR, 2.26, 95% CI, 1.16-4.67； P= 0.03）变化最小，且在不同种族之间存在差异。白人、亚洲人、黑人和西班牙裔分别占试验参与者的76.3%、18.3%、3.1%和6.1%，从2008年7月到2013年6月，从2013年7月到2018年6月，每个种族的入组比例名义上发生了变化（黑人3.6% vs 2.9%，西班牙裔5.3% vs 6.7%）。与他们在美国癌症发病率中的比例相比，黑人（占预期的22%）和西班牙裔（占预期的44%）的比例低于白人（占预期的98%）和亚洲人（占预期的438%）。结论和相关性种族亚组分析报告很少发生，在FDA肿瘤药物批准的里程碑式试验中，黑人和西班牙裔种族的癌症发病率负担一直不足。在试验中需要加强少数群体的参与，以确保结果的有效性和对所有人的可靠益处。
Key PointsQuestionWhat is the status of reporting and representation of racial/ethnic groups in landmark trials leading to US Food and Drug Administration (FDA) approval of oncology drugs? FindingsIn 230 trials leading to FDA oncology drug approvals over the past decade, race was reported in only 145 (63%) trials. Compared with whites (98% of expected proportion), blacks (22% of expected proportion) and Hispanics (44% of expected proportion) were underrepresented in these trials relative to their proportion among the US cancer population. MeaningSuboptimal race reporting and representation (especially in blacks and Hispanics) occurs regularly in landmark oncology trials and increased efforts are needed to enhance minority representation and eliminate these disparities.This database study assesses the frequency of race reporting and proportional race representation in trials that support FDA oncology drug approvals.ImportanceRepresentative racial/ethnic participation in research, especially in clinical trials that establish standards of care, is necessary to minimize disparities in outcomes and to uphold societal equity in health care. ObjectiveTo evaluate the frequency of race reporting and proportional race representation in trials supporting US Food and Drug Administration (FDA) oncology drug approvals. Design, Setting, and ParticipantsDatabase study of all reported trials supporting FDA oncology drug approvals granted between July 2008 and June 2018. Primary reports of trials were obtained from PubMed and ClinicalTrials.gov. Food and Drug Administration approvals were identified using the FDA archives. The US population-based cancer estimates by race were calculated using National Cancer Institute-Surveillance, Epidemiology, and End Results and US Census databases. Main Outcomes and MeasuresPrimary outcomes were the proportion of trials reporting race and the proportion of patients by race participating in trials. Secondary outcomes included race subgroup analyses reporting and gaps between race proportion in trials and the US population. Descriptive statistics, Fisher exact, and chi (2) tests were used to analyze the data. Proportions and odds ratios (OR) with 95% CIs were reported. ResultsAmong 230 trials with a total of 112293 participants, 145 (63.0%) reported on at least 1 race, 18 (7.8%) documented the 4 major races in the United States (white, Asian, black, and Hispanic), and 58 (25.2%) reported race subgroup analyses. Reporting on white, Asian, black, and Hispanic races was included in 144 (62.6%), 110 (47.8%), 88 (38.2%), and 23 (10.0%) trials, respectively. Between July 2008 and June 2013 vs July 2013 and June 2018, the number of trials reporting race (45 [56.6%] vs 100 [67.1%]; OR, 1.63; 95% CI, 0.93-2.87; P=.09) and race subgroup analysis (13 [16.1%] vs 45 [30.2%]; OR, 2.26, 95% CI, 1.16-4.67; P=.03) changed minimally and varied across races. Whites, Asians, blacks, and Hispanics represented 76.3%, 18.3%, 3.1% and 6.1% of trial participants, respectively, and the proportion for each race enrolled over time changed nominally (blacks, 3.6% vs 2.9% and Hispanics, 5.3% vs 6.7%) from July 2008 to June 2013 vs July 2013 to June 2018. Compared with their proportion of US cancer incidence, blacks (22% of expected) and Hispanics (44% of expected) were underrepresented compared with whites (98% of expected) and Asians (438% of expected). Conclusions and RelevanceRace and race subgroup analysis reporting occurs infrequently, and black and Hispanic races are consistently underrepresented compared with their burden of cancer incidence in landmark trials that led to FDA oncology drug approvals. Enhanced minority engagement is needed in trials to ensure the validity of results and reliable benefits to all.