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Quantifying Clinical Trial Diversity in Pivotal Registration Trials of FDA Novel Drug Approvals

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Abstract

Background

Health care inequity includes the lack of adequate representation of various populations in clinical trials. Government, academic and industry organizations have highlighted these issues and committed to actions to improve. In order to assess the current status and future success of these initiatives a quantitative objective measure to assess the state of clinical trial diversity is needed.

Methods

FDA review documents for all novel drug approvals from January 2022 through March 2023 were assessed using a scorecard that considers diversity across different demographic subgroups including age (≥ 65 years old), sex (female), race (Black and Asian) and ethnicity (Hispanic/Latino). The scorecard assigns each drug a letter grade, between A and F, for each subgroup (and overall) based on (1) the percent of each sub-population included in the trials and grades relative to the percent of the US population per the 2020 Census, (2) the number of participants from each subpopulation that received the novel new drug in the trials, (3) the incidence or prevalence of the disease/condition in each of the sub-populations.

Results

The FDA approved 49 novel new drugs for 50 indications (one drug was simultaneously approved for two indications). There was good representation of elderly and females with only two drugs receiving a D grade in either of these sub-populations. In contrast, Black (5 F grades) and Hispanic (4 F grades) representation was often inadequate. There were 10 drugs (20.0%) where there were no Black participants receiving the novel new drug and 16 (32.0%) approvals where there were 1–9 Black participants receiving the novel drug. In the Hispanic/Latino population there were 4 (8.0%) approvals with no Hispanic participants receiving the novel drug and 15 (30.0%) approvals where there were 1–9 Hispanic participants receiving the drug.

Conclusions

This scorecard provides an objective quantitative approach to assess the current state of diversity in clinical trials supporting new drug approvals. Substantial improvement in racial and ethnic representation is needed. Meaningful change will require actions and cooperation among all stakeholders to address this multifaceted issue and will take commitment, perseverance, and appropriate incentives.

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Funding

The author(s) received no financial support for the research, authorship, and/or publication of this article.

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Authors and Affiliations

  1. Department of Medicine and Clinical Research Center, Morehouse School of Medicine, Atlanta, GA, USA

    Muhammed Y. Idris PhD & Priscilla Pemu MD

  2. Colleges of Pharmacy and Medicine, University of Illinois at Chicago, 833 S. Wood St., Chicago, IL, 60607, USA

    William E. Fitzsimmons PharmD, MS

  3. CARER Group, Catalyzing Access to Research Equity in Representation, San Diego, CA, USA

    Muhammed Y. Idris PhD & William E. Fitzsimmons PharmD, MS

Authors
  1. Muhammed Y. Idris PhD
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  2. William E. Fitzsimmons PharmD, MS
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  3. Priscilla Pemu MD
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Contributions

WEF collected, analyzed, and interpreted the data and contributed to the description of the protocol and writing of this article. MYI and WEF wrote the original draft of this manuscript contributed to data collection and analysis methods. PP provided feedback and assisted with editing the manuscript.

Corresponding author

Correspondence to William E. Fitzsimmons PharmD, MS.

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Idris, M.Y., Fitzsimmons, W.E. & Pemu, P. Quantifying Clinical Trial Diversity in Pivotal Registration Trials of FDA Novel Drug Approvals. Ther Innov Regul Sci 58, 175–183 (2024). https://doi.org/10.1007/s43441-023-00583-5

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