Navigating the Evolution of Urothelial Carcinoma Treatment: From Chemotherapy to Immunotherapy
Ben Li, Ziqi Yang, Yihao Pei, Yan Wang, Yumin ChenABSTRACT
Background
The urothelial carcinoma (UC) trial landscape has expanded over two decades, alongside a shift from chemotherapy to immunotherapy and targeted approaches. We characterized temporal patterns in trial phase, interventions, sponsorship, geographic footprint, and the uptake of biomarker specification and artificial intelligence (AI) terminology.
Methods
We conducted a trial registry‐based analysis of 823 interventional UC studies first posted on
Results
Annual registrations increased after 2015, peaking in 2018. Early‐phase studies predominated overall, with expansion of Phase 3 activity in 2015–2024. Drug interventions remained dominant as immunotherapy displaced chemotherapy; pembrolizumab was the leading agent in the later period. The sponsor mix shifted toward industry, and the geographic footprint broadened: the United States/Canada remained the largest contributor, with growth in multinational activity and increased participation from East Asia. Biomarker‐specified language rose to roughly one quarter of registrations in 2015–2024, whereas explicit AI terminology was rare. Early‐phase termination rates were notable.
Conclusions
UC trials have scaled and globalized while consolidating immunotherapy and advancing genomically directed strategies. Priorities include precise preregistration of biomarker and computational endpoints, inclusive multinational accrual, and adequately powered comparative trials against current standards to accelerate durable clinical benefit.