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Original Investigation
April 22, 2021

Racial/Ethnic Disparities in Ophthalmology Clinical Trials Resulting in US Food and Drug Administration Drug Approvals From 2000 to 2020

Author Affiliations
  • 1Vanderbilt University School of Medicine, Nashville, Tennessee
  • 2Vanderbilt University Medical Center, Vanderbilt Eye Institute, Nashville, Tennessee
JAMA Ophthalmol. 2021;139(6):629-637. doi:10.1001/jamaophthalmol.2021.0857
Key Points

Question  Were clinical trials leading to US Food and Drug Administration (FDA) ophthalmology drug approvals representative of the racial/ethnic distribution in the US from 2000 to 2020?

Findings  In this cohort study of 31 clinical trials of 13 drugs with 18 410 participants, the racial/ethic distribution of trial participants was different from the expected distribution for 12 drugs, with Black, Hispanic or Latinx, and other non-White participants being underrepresented.

Meaning  In this cohort study, racial/ethnic distribution of participants in clinical trials leading to FDA ophthalmology drug approvals differed from the expected disease burden and racial/ethnic distribution in the US, suggesting that further efforts to increase enrollment of minority groups in clinical trials is warranted.

Abstract

Importance  Diverse, representative enrollment in pivotal clinical trials is vital to sufficiently power subgroup analyses and ensure equity and validity of trial results.

Objective  To evaluate the racial/ethnic representation, trends, and disparities in clinical trials leading to US Food and Drug Administration (FDA) ophthalmology drug approvals from 2000 to 2020.

Design, Setting, and Participants  This cohort study used data from participants in clinical trials of drugs for neovascular age-related macular degeneration (AMD), open-angle glaucoma (OAG), and expanded indications for diabetic retinopathy (DR) from January 1, 2000, to December 31, 2020. Trial data were sourced from FDA reviews, ClinicalTrials.gov, and relevant linked studies. National expected racial/ethnic proportions were sourced from public National Eye Institute prevalence data as well as published rates scaled using US Census Bureau data.

Main Outcomes and Measures  The primary outcome measures were the distribution of and change over time in the racial/ethnic proportion of participants in clinical trials leading to FDA approval of drugs for AMD, OAG, and DR.

Results  During the 20-year period, 31 clinical trials were identified for 13 medications with 18 410 participants. The distribution of trial participants was different from the expected trial distribution for most approvals with regard to race/ethnicity (12 drugs) and sex (10 drugs). Compared with the first decade (2000-2010), trials conducted in the second decade (2011-2020) showed increases in enrollment of Asian (odds ratio [OR], 2.30; 95% CI, 1.97-2.68; P < .001) and Hispanic or Latinx participants (OR, 1.74; 95% CI, 1.49-2.03; P < .001) for AMD, Asian participants (OR, 2.21; 95% CI, 1.46-3.42; P < .001) for DR, and Black (OR, 1.60; 95% CI, 1.43-1.78; P < .001) and Hispanic or Latinx participants (OR, 10.31; 95% CI, 8.05-13.35; P < .001) for OAG. There was a decrease in Black participants in DR trials (OR, 0.58; 95% CI, 0.42-0.79; P < .001). Based on these trends, the enrollment incidence ratio is expected to worsen by 2050, with overrepresentation of white participants vs underrepresentation of Black and Hispanic or Latinx participants in trials of drugs for AMD (1.08 vs 0.04 vs 0.77), DR (1.83 vs 0.87 vs 0.59), and OAG (1.62 vs 0.90 vs 0.37).

Conclusions and Relevance  In this cohort study, Black, Hispanic or Latinx, and other non-White participants were underrepresented in clinical trials leading to FDA ophthalmology drug approvals compared with the expected disease burden and racial/ethnic distribution in the US. Although there was meaningful improvement from 2000 to 2020, further efforts to increase minority enrollment in clinical trials seem to be warranted.

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    1 Comment for this article
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    Investigator bias.
    mandes kates, PhD, MD | Private Practice
    1. This investigation, which reads more like an editorial, looks at 31 clinical trials performed over a 20 year period, during which time hundreds, if not thousands, of clinical trials about AMD, glaucoma and diabetic retinopathy were performed. How can I be sure there was no bias in the selection of the included studies?

     

    2. The principle result of this study is that racial and ethnic disparities are decreasing! Nevertheless, the authors recommend additional efforts to match the ethnic and racial distribution of clinical study participants to that of the national population. It seems to me to be not a good use of time, effort and resources, inasmuch as the trend is already toward greater representation of formerly under-represented groups in clinical trials.

     

    3. The authors have not identified or suggested that any harm has come to various groups because of under-representation in clinical trials

     

    4. Should authors of studies like these take steps to insure racial and ethnic representation among all investigators?




    CONFLICT OF INTEREST: None Reported
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