Real-World Representation in Prostate Cancer Clinical Trials: Needs and Opportunities

Prostate cancer (PC) care has undergone a significant transformation in the past decade, leading to unprecedented survival outcomes, particularly for patients with newly diagnosed metastatic disease. However, there is a sizable gap in efficacy and toxicity between PC clinical trial populations and those in the real world. Additionally, there has been a persistent lack of adoption of modern PC therapies, particularly in the metastatic hormone-sensitive PC setting. A key reason for slow adoption, despite robust survival data, is that clinicians feel that their patients are not adequately reflected in clinical trial populations. Key populations that have been underrepresented in PC trials include older adults, individuals with comorbidities, rural populations, and people of non-European ancestry. The lack of these patient populations in modern PC clinical trials impacts the broader adoption of modern therapies, limits our understanding of real-world effectiveness and adverse effect profiles, and hinders the discovery of ancestry-related clinical or pharmacogenomic biomarkers. In this review, we propose multiple strategies to help shape clinical trial populations to better reflect the patients we see in the real world, particularly in later-phase studies.