(M1330-10-65) Quantifying Clinical Trial Diversity of New Drugs Approved by the FDA

Purpose: Diversity representation in pivotal Phase 3 registration trials for new drugs is critical to ensuring safety and efficacy in under- served segments of the population. Underrepresentation by any specific population group not only has economic implications, but more importantly, it perpetuates health inequality. We introduce a Clinical Trial Diversity (CTD) Scorecard, an instrument designed to evaluate diversity in the registrational clinical trial for new molecular entities and new therapeutic biological products approved by US FDA.

Methods: FDA review documents for all novel drug approvals from January 2022 through February 16, 2023 were assessed using a scorecard that considers diversity across different demographic subgroups including age ( >65 yo) , 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, 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 43 novel new drugs for 44 indications (one drug was simultaneously approved for two indications). The three drugs with A Grades reflecting the best diversity in their registration trials were tapinarof (Vtama from Dermavant), daprodustat (Jesduvroq from GlaxoSmithKline) and eflapegrastim (Rolvedon from Spectrum Pharmaceuticals.) 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 and Hispanic representation was often inadequate with 4 drugs receiving F grades for the specific subgroup participation. There were 9 drugs (20%) where there were no Black participants receiving the novel new drug and an additional 14 approvals where there were < 10 Black participants receiving the novel drug. The median number of Black participants receiving the investigational drug was 9 for these 43 drugs. In the Hispanic/Latino population there were 2 approvals with no Hispanic participants receiving the novel drug and 14 approvals where there were < 10 Hispanic participants receiving the drug. The median number of Hispanic participants receiving the novel drug was 12.5.

Conclusion: This newly developed 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 amongst all stakeholders to address this multifaceted issue and will take commitment, perseverance, and appropriate incentives.

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Clinical Trial Diversity Score Card of Year 2022- Highest Grade