Biomarker-informed trial enrollment appears straightforward for identifying responders and optimizing efficiency, but failures to account for demographic variation in cut-off selection, causality versus correlation, and composite biomarker complexity risk excluding viable patients and exacerbating disparities.
Biomarker-informed trial enrollment can optimize trial efficiency and accuracy, but it can also exclude viable patient populations or jeopardize approval. Particularly for indications beyond oncology, there is limited explicit regulatory guidance for biomarker-informed trial enrollment.1 However, methodological insights have emerged from prior successes and failures.2
