Fostering Collaboration to Advance Shared Goals for Representativeness in Clinical Trials

Clinical trials conducted within the United States (U.S.) face structural and systemic complexities that can result in failure to address critical research questions accurately, equitably, and efficiently across a range of treatment and disease areas. Data collection burdens on providers, sometimes onerous study protocols, and pressure to enroll narrowly defined patient populations, can limit the location of clinical trials to well-resourced centers, such as large academic medical centers, due to concerns around feasibility. Additionally, trial participants often encounter significant burdens beyond the confines of trial design, including logistical challenges (e.g., transportation), financial obstacles (e.g., fair compensation for participation), and stigmatization (e.g., implicit bias), which further exacerbate disparities in trial enrollment and participation rates. These factors complicate the breadth, quality, and generalizability of clinical trial evidence due to the lack of representation within the trials, which can increase inequities. In addition, under- or unrepresented populations often experience systemic exclusion and harbor institutional mistrust. As needs for rapid, rigorous, and generalizable evidence grow along improved technology supports to enable that evidence generation, a growing impetus exists to reimagine clinical trial conduct and advance trial representation without compromising vital research standards.

To address these issues, the Duke-Margolis Institute for Health Policy conducted a multiphase research study encompassing a landscape review of existent literature and published materials, multiple convenings, and structured stakeholder interviews with key experts across the clinical trial enterprise including researchers, pharmaceutical representatives, and patient advocacy organizations. This work was conducted between October 2022 and Spring 2024 with interviews taking place primarily in Spring 2023. Public workshops were hosted in July 2023 and March 2024. This paper endeavors to highlight evolving policies and recent initiatives in the clinical trial space and explore strategies for fostering greater representativeness in recruited patient populations, through shared stakeholder goals.

By harnessing insights from diverse stakeholders, and identifying avenues for collaborative action and strategic intervention, we encourage measurable improvements in the clinical trial landscape, ensuring clinical trial design and conduct help advance health care for all.

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