Precision oncology needs precision trial design: lessons from FGFR2 -rearranged cholangiocarcinoma

The phase III FIGHT-302 trial showed that the FGFR inhibitor pemigatinib improves progression-free survival and response rates versus first-line chemotherapy in patients with advanced FGFR2-rearranged cholangiocarcinoma, but the trial accrued slowly and closed early after chemoimmunotherapy became the new standard of care. Two similarly terminated trials underscore the challenges of evaluating targeted therapies in rare, biomarker-defined cancer populations. The authors argue that innovative trial designs and more flexible regulatory frameworks are essential to generate robust evidence for precision therapies in small patient subgroups.