Black and Hispanic or Latinx participants remained underrepresented from 2000 to 2020 in clinical trials that led to approvals of drugs for neovascular age-related macular degeneration (AMD), open-angle glaucoma (OAG), and diabetic retinopathy (DR) from the US Food and Drug Administration (FDA), researchers found in a study published in JAMA Ophthalmology.
While the FDA has addressed the inclusion of a diversity of races and ethnicities in clinical trials, some studies indicate that inclusivity remains limited in ophthalmic research.
“Barriers to research enrollment are well studied in medicine and may be structural, clinical, attitudinal, demographic, and/or socioeconomic,” according to the study. “It is critical to promote equitable participation in trials for emerging therapeutics. This is especially important for diseases with strong racial/ethnic components of presentation and severity, such as OAG.”
Investigators analyzed 31 clinical trials conducted between 2000 and 2020 with 18,410 participants regarding the approval of 13 medications. They included 10 trials that supported approval of a new molecular entities (NME) for neovascular AMD, 16 trials that supported approval of an NME for OAG, and 5 trials that supported expanded approval of existing NMEs for sequelae of DR.
They compared the expected racial/ethnic and sex distribution that National Eye Institute population data would project with the actual distribution of trial participants.
The researchers found that the enrollment of Asian participants and Hispanic or Latinx participants increased for trials for approvals by disease category in each decade (287 [5.19%] vs 472 [11.16%]; OR 2.30, 95% CI, 1.97-2.68; P <.001; 315 [5.69%] vs 402 [9.51%]; OR 1.74, 95% CI, 1.49 to 2.03; P <.001) for AMD NMEs.
Inclusion of Asian participants in DR trials also increased (32 [4.22%] vs 112 [8.86%]; OR 2.21, 95% CI, 1.46 to 3.42; P <.001), but participation of individuals who are Black in DR trials decreased (93 [12.25%] vs 94 [7.44%]; OR 0.58, 95% CI, 0.42-0.79; P <.001).
Participation of Black (682 [14.55%] vs 930 [21.38%]; OR 1.60, 95% CI, 1.43- 1.78; P <.001) and Hispanic or Latinx participants (74 [1.58%] vs 617 [14.19%], OR 10.31, 95% CI, 8.05-13.35, P <.001) in OAG trials increased.
Enrollment incidence disparity (EID) and enrollment incidence ratio (EIR) projected increased overrepresentation of White participants and underrepresentation of Black and Hispanic or Latinx participants for 2030 and 2050. Projected EIDs for trials of drugs for AMD are 5.03% vs -4.67% vs -0.24%, respectively, in 2030 and 6.97% vs -4.36% vs -1.89%, respectively, in 2050. For DR, they are projected to be 23.73% vs -1.25% vs -4.44%, respectively, in 2030 and 36.20% vs -1.39% vs -14.64%, respectively, in 2050. EIDs between participants who are White, Black, and Hispanic or Latinx for OAG drug trials are anticipated to be 21.50% vs -2.34% vs -6.50% for 2030 and 29.83% vs -1.90% vs -13.12% for 2050, according to the study.
EIRs for AMD in 2030 are projected to be 1.06 vs 0.04 vs 0.96 and 1.08 vs 0.04 vs 0.77 in 2050. For DR, they are projected to be 1.42 vs 0.88 vs 0.83 in 2030 and 1.83 vs 0.87 vs 0.59 in 2050. OAG projects are 1.38 vs 0.88 vs 0.54 for 2030 and 1.62 vs 0.90 vs 0.37 for 2050.
Limitations of the study include inconsistency in race/ethnic category reporting and the fact that comparison demographic data came from various datasets.
“The most concerning explanation for the underrepresentation of certain demographic groups is an underlying systemic barrier to enrollment in rigorous clinical studies. Financial resources, transportation, employment, and other factors may additionally prevent the consistent follow-up required for clinical trial protocols. These many obstacles to successful participation in clinical research may in part account for the suboptimal outcomes seen in real-world settings,” researchers explain.
Disclosure: One study author declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.
Berkowitz ST, Growth SL, Gangaputra S, Patel S. Racial/ethnic disparities in ophthalmology clinical trials resulting in US Food and Drug Administration drug approvals from 2000 to 2020. JAMA Ophthalmol. Published online April 22, 2021. doi:10.1001/jamaophthalmol.2021.0857