Representation of Race and Ethnicity in Randomized Clinical Trials of Diabetic Macular Edema and Retinal Vein Occlusion Compared to 2010 US Census Data

Abstract

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.

Racial Disparities in Barriers to Care for Patients With Diabetic Retinopathy in a Nationwide Cohort

“…Eye care clinicians can take a proactive role in addressing these disparities in barriers to care and promoting health equity by working to reduce implicit bias and implementing SDoH assessments to identify patients at risk and connect them with resources. On a larger systemic level, other strategies to promote health equity include improving representation in clinical trials (a recent study found that participants identifying as White were overrepresented in diabetic macular edema and retinal vein occlusion clinical trials) 41 and diversifying the ophthalmology workforce. Broader social policy changes, beyond the realm of medicine, may be needed for issues such as affordable child care access.…”

Use of Teleophthalmology to Evaluate the Incidence and Progression of Diabetic Retinopathy

“…According to 2020 US census data, American Indian and Alaska Native individuals represent only 1.1% of the national population, and yet they experience a higher prevalence of diabetes, with younger patients affected more than other patient populations . Notwithstanding the incidence of disease that American Indian and Alaska Native patients experience and the potential importance of their patient data, they remain underrepresented in ophthalmological clinical trials …”

Representation Matters—Diversity in Retina Clinical Trials

“…In this issue of JAMA Ophthalmology, Kaakour et al 1 highlight an important gap in representation in retina clinical trials among participants from racial and ethnic minority groups. In a cross-sectional study comparing the demographics of USbased diabetic mac ular edema (DME) and retinal vein occlusion (RVO) randomized clinical trials with 2010 US Census data, they found that the vast majority of clinical trials for these conditions were not representative of the racial and ethnic diversity of the general population.…”

“…4 Furthermore, while the authors did not explicitly discuss the extent of underrepresentation in the article, detailed review of the eTables in the Supplement reveals that in addition to being underrepresented in the greatest number of trials, the extent of underrepresentation was also the most severe for Hispanic/Latino participants. 1 To illustrate, the magnitude of difference in proportional representation (ie, the difference between the expected proportional representation based on census data and the observed proportional representation in the trial) was 10% or greater in 9 trials (6 DME and 3 RVO) for Hispanic/Latino participants. For Black participants, the difference between observed and expected proportional representation was 10% or greater in 5 trials (4 DME and 1 RVO).…”

“…The representation gap for Hispanic/Latino participants is particularly concerning, given the burden of diabetes and hypertension in this community and the fact that this is the fastest-growing demographic subset in the US . Furthermore, while the authors did not explicitly discuss the extent of underrepresentation in the article, detailed review of the eTables in the Supplement reveals that in addition to being underrepresented in the greatest number of trials, the extent of underrepresentation was also the most severe for Hispanic/Latino participants . To illustrate, the magnitude of difference in proportional representation (ie, the difference between the expected proportional representation based on census data and the observed proportional representation in the trial) was 10% or greater in 9 trials (6 DME and 3 RVO) for Hispanic/Latino participants.…”