The clinical landscape of bladder cancer treatment: a brief report on new developments and future perspectives in pharmaceutical interventions

Abstract

This study systematically delineates the clinical research status and trends in bladder cancer (Bca) therapeutics through an analysis of 1024 Bca drug treatment clinical trials identified from a harmonised search of the Trialtrove database, ClinicalTrials.gov, and the World Health Organization International Clinical Trials Registry Platform (WHO ICTRP) database as of December 31, 2025. The results demonstrate that the overall number of clinical trials has tended to increase, with a subsequent decline observed in the post-COVID-19 period; the United States has led research efforts, followed by China, the United Kingdom, Canada, and Australia, which together account for 68.6% of all registered trials; the therapeutic focus has shifted from traditional chemotherapy toward immunotherapy (38.4% of trials, primarily targeting PD-1, PD-L1 and ICIs) and targeted therapies [24.7% of trials, e.g., FGFR inhibitors and Nectin-4-targeting antibody–drug conjugates (ADCs)]. Among validated clinical advances, the EV-302 trial established enfortumab vedotin plus pembrolizumab as the first-line standard of care for locally advanced or metastatic urothelial carcinoma. However, the high trial registration frequency does not uniformly translate into clinical benefit, as exemplified by the subsequent withdrawal of atezolizumab’s bladder cancer indications following negative confirmatory phase III results. In the future, molecular subtype-based personalized treatment strategies, including circulating tumor DNA (ctDNA)-guided adjuvant therapy, novel ADC combination strategies, and artificial intelligence (AI)-driven multiomics integration, will be pivotal for enhancing therapeutic outcomes in Bca.