Race/ethnicity reporting and representation in US clinical trials: a cohort study.
Ontology highlight
ABSTRACT: Background
Systemic progress in improving trial representation is uncertain, and previous analyses of minority trial participation have been limited to small cohorts with limited exploration of driving factors.Methods
We analyzed detailed trial records from all US clinical trials registered in ClinicalTrials.gov from March 2000 to March 2020. Minority enrollment was compared to 2010 US Census demographic estimates using Wilcoxon test. We utilized logistic regression and generalized linear regression with a logit link to assess the association of possible drivers (including trials' funding source, size, phase, and design) with trials' disclosure of and amount of minority enrollment respectively.Findings
Among 20,692 US-based trials with reported results (representing ~4·76 million enrollees), only 43% (8,871/20,692) reported any race/ethnicity data. The majority of enrollees were White (median 79·7%; interquartile range [IQR] 61·9-90·0%), followed by Black (10·0%; IQR 2·5-23·5%), Hispanic/Latino (6·0%; IQR 0·43-15·4%), Asian (1·0%; IQR 0·0-4·1%), and American Indian (0·0%; IQR 0·0-0·2%). Median combined enrollment of minority race/ethnicity groups (Black, Hispanic/Latino, Asian, American Indian, Other/Multi) was below census estimates (27·6%) (p<0·001) however increased at an annual rate of 1·7%. Industry and Academic funding were negatively associated with race/ethnicity reporting (Industry adjusted odds ratio [aOR]: 0·42, 95% confidence interval [CI]: 0·38 to 0·46, p<0.0001; Academic aOR: 0·45, CI: 0·41 to 0·50, p<0.0001). Industry also had a negative association with the proportion of minority ethnicity enrollees (aOR: 0·69, CI: 0·60 to 0·79) compared to US Government-funded trials.Interpretation
Over the past two decades, the majority of US trials in ClinicalTrials.gov do not report race/ethnicity enrollment data, and minorities are underrepresented in trials with modest improvement over time.Funding
Stanford Medical Scholars Research Funding, the National Heart, Lung, and Blood Institute, NIH (1K01HL144607) and the American Heart Association/Robert Wood Johnson Medical Faculty Development Program.
SUBMITTER: Turner BE
PROVIDER: S-EPMC9302767 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:ObjectiveVulnerable populations, including women and racial and ethnic minorities, have been historically underrepresented in clinical trials. We, therefore, studied the demographics of patients enrolled in pivotal endovascular aortic device trials in the United States.MethodsWe queried the Food and Drug Administration (FDA) medical devices database for all FDA-approved endografts for the treatment of aortic aneurysms, transections, and dissections from September 1999 to November 2021. These included abdominal endovascular aortic repair (EVAR), thoracic EVAR (TEVAR), fenestrated EVAR (FEVAR) devices, and dissection stents. Multiple cases of approval for expanded indications were included separately. The primary outcomes included the proportion of trials reporting participant sex, race, and ethnicity and the proportion of enrolled participants across sex, racial, and ethnic groups.ResultsThe FDA provided 29 approvals from 29 trials of 24 devices: 15 EVAR devices (52%), 12 TEVAR devices (41%), 1 FEVAR device (3.4%), and 1 dissection stent (3.4%). These trials had included 4046 patients. Of the 29 trials, all had reported on the sex of the participants, and the median female enrollment was 21% (interquartile range [IQR], 11%-34%). The EVAR trials had the lowest female enrollment (11%; IQR, 8.7%-13%) compared with 41% (IQR, 27%-45%) in the TEVAR trials, 21% in the FEVAR trial, and 34% in the dissection stent trial (P < .01 for the difference). Only 52% of the trials had reported the three most common racial groups (White, Black, Asian), and only 48% had reported Hispanic ethnicity. The TEVAR trials were the most likely to report all three racial groups and Hispanic ethnicity (92% and 75%, respectively), while the EVAR trials had the lowest reporting rates (13% and 20%, respectively). Where reported, the median enrollment of racial and ethnic groups across the trials was as follows: Black patients, 9.8% (FEVAR, 0%; EVAR, 1.9%; TEVAR, 12%; dissection stent, 25%; P = .01); Asian patients, 2.4% (EVAR, 0.6%; FEVAR, 2.4%; TEVAR, 2.5%; dissection stent, 11%; P = .24); and Hispanic patients, 3.8% (EVAR, 1.3%; FEVAR, 2.4%; TEVAR, 3.9%; dissection stent, 4.1%; P = .75).ConclusionsRacial and ethnic minority groups were underrepresented and underreported in pivotal aortic device trials that led to FDA approval. Female patients were also underrepresented in these aortic trials, especially for EVAR. These data suggest the need for standardization of reporting practices and minimum thresholds for minority and female participation in pivotal trials to promote equitable representation.
| S-EPMC9613501 | biostudies-literature
Project description:BackgroundIt is of paramount importance that clinical trials are designed with adequate health equity considerations to prevent disproportionate analyses of specific demographics.ObjectiveIn this study, we investigated the representation of sex, race, and ethnicity in pivotal clinical trials for drugs with dermatological disease indications approved by the U.S. Food and Drug Administration between 1995 and 2019.MethodsThirty-six novel drugs with indications to treat dermatological diseases, approved by the U.S. Food and Drug Administration between January 1995 and December 2019 were abstracted from Drugs@FDA. The drug approval label, statistical review, official record, and trial publication were reviewed for data on disease indication, approval year, pathway, number of participants, participant demographics (sex, race, and ethnicity), location, and sponsor type.ResultsThe overall female representation was 45.6% (n = 17,492 of 38,320). Adequate female representation was noted for five of six disease indications. Caucasians were predominantly overrepresented (80.4%; n = 28,065 of 34,890); Blacks (9.8%; n = 3242 of 33,240) and Asians (5.5%; n = 1535 of 27,696) were consistently underrepresented. Across sponsor types, there was a significant difference in the distribution of women (χ2 = 6.332; p = .042), as well as Caucasians (χ2 = 12.813; p = .002), Blacks (χ2 = 13.002; p = .002), and Hispanics/Latinos (χ2 = 7.747; p = .021).ConclusionPersistence of disparities disproportionately affect the quality of data behind therapies for certain demographics; as such, enrollment practices must continue to address the issue of underrepresentation. Efforts to facilitate demographic equity among clinical trial participants must be supported to ensure that safety and efficacy conclusions are drawn from representative population samples.
| S-EPMC8484973 | biostudies-literature
Project description:Importance:Representative 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. Objective:To 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 Participants:Database 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 Measures:Primary 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 ?2 tests were used to analyze the data. Proportions and odds ratios (OR) with 95% CIs were reported. Results:Among 230 trials with a total of 112?293 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 Relevance:Race 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.
| S-EPMC6696743 | biostudies-literature
Project description:ImportanceDiverse enrollment and adequate representation of racial and ethnic minority groups in randomized clinical trials (RCTs) are valuable to ensure external validity and applicability of results.ObjectiveTo compare the distribution of race and ethnicity in RCTs of diabetic macular edema (DME) and macular edema from retinal vein occlusion (RVO) to that of US Census data.Design, setting, and participantsThis was a cross-sectional retrospective analysis comparing racial and ethnic demographic characteristics of US-based RCTs of DME and RVO between 2004 and 2020 with 2010 US Census data. PubMed and ClinicalTrials.gov were searched to screen for completed phase 3 RCTs with published results. Of 169 trials screened, 146 were excluded because they were incomplete, did not report race and ethnicity, or were not based in the US, and 23 trials were included (15 DME and 8 RVO). The number and percentage of American Indian or Alaska Native, Asian, Black, Hispanic, Native Hawaiian or Other Pacific Islander, and White participants was recorded in each RCT. The demographic distribution and proportion was compared to the reported distribution and proportion in the 2010 US Census using the χ2 test.Main outcomes and measuresOverrepresentation, underrepresentation, or representation commensurate with 2010 US Census data in the racial and ethnic populations of RCTs of retinal vascular disease.ResultsIn 23 included RCTs of DME and RVO, there were a total of 38 participants (0.4%) who identified as American Indian or Alaska Native and Native Hawaiian or Other Pacific Islander (groups combined owing to small numbers), 415 Asian participants (4.4%), 904 Black participants (9.6%), 954 Hispanic participants (10.1%), and 7613 White participants (80.4%). By comparison, the 2010 US Census data indicated that 1.1% of the US population self-reported as American Indian or Alaska Native and Native Hawaiian or Other Pacific Islander (groups combined for comparison in this study), 4.8% self-reported as Asian, 12.6% as Black or African American, 16.3% as Hispanic, and 63.7% as White. American Indian or Alaska Native and Hawaiian or Other Pacific Islander participants were underrepresented in 2 trials, neither overrepresented nor underrepresented in 20, and not overrepresented in any of the included trials. Asian participants were underrepresented in 10 trials, overrepresented in 4, and neither overrepresented nor underrepresented in 8. Black participants were underrepresented in 9 trials, overrepresented in 2, and neither overrepresented nor underrepresented in 11. Hispanic participants were underrepresented in 15 trials, overrepresented in 2, and neither overrepresented nor underrepresented in 5. White participants were underrepresented in 2 trials, overrepresented in 14, and neither overrepresented nor underrepresented in 7. The χ2 values comparing RCT demographic distribution to US 2010 Census data were significantly different in 22 of 23 included RCTs.Conclusions and relevanceThe findings in this study indicated a discrepancy between racial and ethnic demographic data in RCTs of DME and RVO and the US population according to the 2010 Census. White study participants were most frequently overrepresented, and Hispanic study participants were most frequently underrepresented. These findings support the need for more efforts to recruit underrepresented racial and ethnic minorities to improve external validity in trial findings.
| S-EPMC9539735 | biostudies-literature
Project description:ObjectiveTo assess if authors of randomised clinical trials convey the fact that they have used surrogate outcomes and discussed their validity.DesignCohort study.SettingSix major general medical journals.ParticipantsRandomised clinical trials published in 2005 and 2006 that used a surrogate as a primary outcome.ResultsOf 626 published randomised clinical trials, 109 (17%) used a surrogate as a primary outcome. Of these trials, 62 (57%, 95% confidence interval 47% to 67%) clearly reported that the primary outcome was a surrogate. Only 38 (35%, 26% to 45%) also discussed the validity of the surrogate.ConclusionOnly about one third of authors of randomised clinical trials that used a surrogate as a primary outcome reported adequately on the surrogate. Better reporting is needed.
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Project description:ImportanceRepresentativeness of populations within neonatal clinical trials is crucial to moving the field forward. Although racial and ethnic disparities in research inclusion are well documented in other fields, they are poorly described within neonatology.ObjectiveTo describe the race and ethnicity of infants included in a sample of recent US neonatal clinical trials and the variability in this reporting.Evidence reviewA systematic search of US neonatal clinical trials entered into Cochrane CENTRAL 2017 to 2021 was conducted. Two individuals performed inclusion determination, data extraction, and quality assessment independently with discrepancies adjudicated by consensus.FindingsOf 120 studies with 14 479 participants that met the inclusion criteria, 75 (62.5%) included any participant race or ethnicity data. In the studies that reported race and ethnicity, the median (IQR) percentage of participants of each background were 0% (0%-1%) Asian, 26% (9%-42%) Black, 3% (0%-12%) Hispanic, 0% (0%-0%) Indigenous (eg, Alaska Native, American Indian, and Native Hawaiian), 0% (0%-0%) multiple races, 57% (30%-68%) White, and 7% (1%-21%) other race or ethnicity. Asian, Black, Hispanic, and Indigenous participants were underrepresented, while White participants were overrepresented compared with a reference sample of the US clinical neonatal intensive care unit (NICU) population from the Vermont Oxford Network. Many participants were labeled as other race or ethnicity without adequate description. There was substantial variability in terms and methods of reporting race and ethnicity data. Geographic representation was heavily skewed toward the Northeast, with nearly one-quarter of states unrepresented.Conclusions and relevanceThese findings suggest that neonatal research may perpetuate inequities by underrepresenting Asian, Black, Hispanic, and Indigenous neonates in clinical trials. Studies varied in documentation of race and ethnicity, and there was regional variation in the sites included. Based on these findings, funders and clinical trialists are advised to consider a 3-point targeted approach to address these issues: prioritize identifying ways to increase diversity in neonatal clinical trial participation, agree on a standardized method to report race and ethnicity among neonatal clinical trial participants, and prioritize the inclusion of participants from all regions of the US in neonatal clinical trials.
| S-EPMC10739112 | biostudies-literature
Project description:BackgroundWe examined associations between suicidality and genotypes that predict plasma efavirenz exposure among AIDS Clinical Trials Group study participants in the United States.MethodsFour clinical trials randomly assigned treatment-naive participants to efavirenz-containing regimens; suicidality was defined as reported suicidal ideation or attempted or completed suicide. Genotypes that predict plasma efavirenz exposure were defined by CYP2B6 and CYP2A6 polymorphisms. Associations were evaluated with weighted Cox proportional hazards models stratified by race/ethnicity. Additional analyses adjusted for genetic ancestry and selected covariates.ResultsAmong 1833 participants, suicidality was documented in 41 in exposed analyses, and 34 in on-treatment analyses. In unadjusted analyses based on 12 genotype levels, suicidality increased per level in exposed (hazard ratio, 1.11; 95% confidence interval, .96-1.27) and on-treatment 1.16; 1.01-1.34) analyses. In the on-treatment analysis, the association was strongest among white but nearly null among black participants. Considering 3 metabolizer levels (extensive, intermediate and slow), slow metabolizers were at increased risk. Results were similar after baseline covariate-adjustment for genetic ancestry, sex, age, weight, injection drug use history, and psychiatric history or recent psychoactive medication.ConclusionsGenotypes that predict higher plasma efavirenz exposure were associated with increased risk of suicidality. Strength of association varied by race/ethnicity.
| S-EPMC5853681 | biostudies-literature
Project description:ImportanceThe description of racial and ethnic data in human trials is relatively unregulated, leading to potential confusion with and inconsistent reporting of these data.ObjectiveTo determine the frequency and use of racial and ethnic data in the ophthalmology literature in 2019.Design, setting, and participantsThis cross-sectional study included all full-length human studies published between January and December 2019 in the American Journal of Ophthalmology, JAMA Ophthalmology, and Ophthalmology.Main outcomes and measuresWhether race or ethnicity was included in the data or analysis, how the categorization was described in the methods and results, specific racial and ethnic categories used, and whether and how the categories were determined.ResultsA total of 547 articles were identified during the study period, 484 (88% ± 2.7%) of which reported background demographic information, including patient age and sex. Only 233 (43% ± 4.1%) reported race and/or ethnicity. Of those articles, 72 (31% ± 5.9%) categorized it as race, 51 (22% ± 5.3%) ethnicity, 56 (24% ± 5.4%) race/ethnicity, 21 (9% ± 3.7%) race and ethnicity, 12 (5% ± 3.0%) descent, 12 (5% ± 2.9%) population, and 5 (2% ± 2.0%) ancestry; in 21 (9% ± 3.8%) it was not defined. Only 30 studies (13% ± 4.4%) described how it was determined in the methods or results, most frequently by self-report (77% ± 3.9%). A total of 78 races and/or ethnicities were defined.Conclusions and relevanceWhile most articles during the study period reported background demographic information, few included race and ethnicity and only a fraction of those described how these data were determined. The categories used were heterogeneous and often inconsistent. These findings suggest the need for increased and more standardized reporting of ethnic and racial demographic data in the ophthalmology literature.
| S-EPMC7303899 | biostudies-literature
Project description:ObjectiveTo produce reliable and informative health plan performance data by race/ethnicity for the Medicare beneficiary population and to consider appropriate presentation strategies.Data sourcesPatient experience data from the 2008-2009 Medicare Advantage (MA) and fee-for-service (FFS) CAHPS surveys and 2008-2009 HEDIS data (MA beneficiaries only).Study designMixed effects linear (and binomial) regression models estimated the reliability and statistical informativeness of CAHPS (HEDIS) measures.Principal findingsSeven CAHPS and seven HEDIS measures were reliable and informative for four racial/ethnic subgroups-Whites, Blacks, Hispanics, and Asian/Pacific Islanders-at sample sizes of 100 beneficiaries (200 for prescription drug plans). Although many plans lacked adequate sample size for reporting group-specific data, reportable plans contained a large majority of beneficiaries from each of the four racial/ethnic groups.ConclusionsStatistically reliable and valid information on health plan performance can be reported by race/ethnicity. Many beneficiaries may have difficulty understanding such reports, however, even with careful guidance. Thus, it is recommended that health plan performance data by subgroups be reported as supplemental data and only for plans meeting sample size requirements.
| S-EPMC3626354 | biostudies-literature
Project description:ObjectiveTo investigate new metrics to improve the reporting of patient race and ethnicity (R/E) by hospitals.Data sourcesCalifornia Patient Discharge Database (PDD) and birth registry, 2008-2009, Healthcare and Cost Utilization Project's State Inpatient Database, 2008-2011, cancer registry 2000-2008, and 2010 US Census Summary File 2.Study designWe examined agreement between hospital reported R/E versus self-report among mothers delivering babies and a cancer cohort in California. Metrics were created to measure root mean squared differences (RMSD) by hospital between reported R/E distribution and R/E estimates using R/E distribution within each patient's zip code of residence. RMSD comparisons were made to corresponding "gold standard" facility-level measures within the maternal cohort for California and six comparison states.Data collectionMaternal birth hospitalization (linked to the state birth registry) and cancer cohort records linked to preceding and subsequent hospitalizations. Hospital discharges were linked to the corresponding Census zip code tabulation area using patient zip code.Principal findingsOverall agreement between the PDD and the gold standard for the maternal cohort was 86 percent for the combined R/E measure and 71 percent for race alone. The RMSD measure is modestly correlated with the summary level gold standard measure for R/E (r = 0.44). The RMSD metric revealed general improvement in data agreement and completeness across states. "Other" and "unknown" categories were inconsistently applied within inpatient databases.ConclusionsComparison between reported R/E and R/E estimates using zip code level data may be a reasonable first approach to evaluate and track hospital R/E reporting. Further work should focus on using more granular geocoded data for estimates and tracking data to improve hospital collection of R/E data.
| S-EPMC4545337 | biostudies-literature