When Does a Team Start the Discussion on Inclusion and Diversity in Early-phase Clinical Trials?

Abstract

Purpose: The purpose of a clinical trial is to determine whether a new drug is safe, efficacious, and has fewer side effects than products that may already be on the market. They are essential for ensuring that new potential therapies will advance patient outcomes in some way. This may represent a therapy for a previously untreatable condition, or it may represent an improvement in the efficacy or safety compared with an existing treatment. A new therapy should improve patients' quality of life in a meaningful way, and it should do so for as many potential patients as possible.

Methods: To meet this goal, companies should consider the lived experiences and exposures of different populations that are included in the target population for therapies in development. This is why it is generally beneficial to advocate diversity in trials as early as possible in the drug development (Phase I and II trials), as doing so expands the number of trials having inclusivity of racial and ethnic minority groups and are inclusive of more women. Although some improvements in diversity in early-phase trials have been observed in recent years, the overall participation of minority populations in these types of trials continues to be disproportionately lower than their representation in the overall population. In 2023, the minority makeup of the US population was 18.7% Latino and 12.1% Black. In comparison, data reported by the National Cancer Institute in 2023 found the racial makeup in early-phase trials to be 6.9% Latino and 7.1% Black, well below their representation in the larger population.

Findings: The authors, who have been involved in numerous clinical trial study design discussions, understand that people from different backgrounds can experience the same disease differently, and thus, it is important to explore these differences in all communities. These differences represent an important consideration in addition to the acknowledged benefit diversity plays to ensure therapies are appropriately tested in the target patient population.

Implications: This article explores the need for earlier collaboration/discussion on including diversity in the earlier clinical studies. Our commentary calls for key opinion leaders, study Investigators, study leads, study site recruiters, and coordinators to be open to going beyond the current paradigm of diversity only in pivotal trials, generally Phase III, and explore remedies for inclusion of diversity in early trials.