A Fork In The Road: Assessing Diversity In Early-Phase Clinical Trials In A Rapidly Changing Regulatory Environment

Patients from underrepresented populations are often lacking in biomedical research despite having a disproportionate disease burden for certain indications.

The FDA recently removed its draft guidance on diversity in clinical trials issued in June 2024 and due to be finalized this year from its website without public notice or explanation, and then reinstated it. The removal came shortly after an executive order was issued to curtail diversity, equity, and inclusion programs, which were recently reinstated following a federal court order on February 11, 2025. The removal and reinstatement of the guidance has left many uncertain about how to comply with the requirements for the Diversity Action Plans.

Variations in drug absorption, metabolism, excretion, and target sites can significantly impact both the safety of a novel drug and its efficacy. Early-phase clinical studies, although typically small in sample size, can offer important data to understand a drug’s pharmacokinetics, pharmacodynamics, and safety in differing populations. Structuring early-phase trials to address population variation and contributing factors to safe drug administration are essential to a comprehensive drug development plan aimed at ensuring equitable representation of patient subtypes. Understanding the factors that may lead to variable drug concentrations and responses is critical to constructing robust patient trials relevant to a real-world, diverse patient population. These critical data are relevant and scientifically sound, irrespective of the regulatory environment.