Across 92 European CTIS-authorized Phase II and Phase III bladder/urothelial cancer trials, safety and tolerability endpoints are the most frequently measured overall, appearing in 70 trials (76.1%). Overall survival (OS) follows in 58 trials (63.0%), while response and time-to-event endpoints cluster closely: duration of response (DOR) and progression-free survival (PFS) each appear in 39 trials (42.4%), and objective response rate (ORR) appears in 38 trials (41.3%). Primary endpoints are less dominated by one measure: recurrence/event-free survival leads at 25 trials (27.2%), followed by ORR in 24 trials (26.1%) and safety in 23 trials (25.0%).
The most common endpoint family is safety/tolerability, measured in 70 of 92 trials (76.1%). Overall survival is measured in 58 trials (63.0%), while DOR, PFS, ORR and recurrence/event-free survival each appear in roughly 38–39 trials (41.3%–42.4%).
European bladder/urothelial cancer protocols remain safety-heavy even in Phase III, but the core efficacy language is clearly oncology-standard: OS, PFS, ORR, DOR and recurrence/EFS together form the dominant endpoint backbone.
Primary endpoint fields were non-empty in 88 of 92 trials (95.7%), secondary endpoint fields in 73 trials (79.3%), and other/exploratory endpoint fields in 2 trials (2.2%). The most frequent primary endpoint family was recurrence/event-free survival in 25 trials (27.2%), followed by ORR in 24 trials (26.1%) and safety in 23 trials (25.0%).
Other or exploratory endpoint fields were rare: 2 of 92 trials (2.2%) had non-empty other-endpoint lists. Within that small subset, immunogenicity, biomarker/molecular, QoL/PRO, recurrence/EFS, time to response and complete-response/local-clearance measures each appeared in 1 trial (1.1%).
The primary endpoint strategy is not uniform across bladder/urothelial trials: it splits between local-control/time-to-event endpoints, response endpoints and safety. Secondary endpoint packages are much more standardized, with OS and safety each appearing in 55 trials (59.8%).
The dataset includes 56 Phase II and 36 Phase III trials. Safety/tolerability appears in 48 Phase II trials (85.7%) versus 22 Phase III trials (61.1%). ORR appears in 28 Phase II trials (50.0%) but only 10 Phase III trials (27.8%), while QoL/PRO appears more often in Phase III: 18 of 36 trials (50.0%) versus 16 of 56 Phase II trials (28.6%).
| Endpoint family | Phase II | Phase III |
|---|---|---|
| Safety / tolerability | 48 / 56 · 85.7% | 22 / 36 · 61.1% |
| Overall survival | 38 / 56 · 67.9% | 20 / 36 · 55.6% |
| Objective response / ORR | 28 / 56 · 50.0% | 10 / 36 · 27.8% |
| DOR | 26 / 56 · 46.4% | 13 / 36 · 36.1% |
| PFS | 25 / 56 · 44.6% | 14 / 36 · 38.9% |
| Recurrence / EFS | 20 / 56 · 35.7% | 18 / 36 · 50.0% |
| Quality of life / PRO | 16 / 56 · 28.6% | 18 / 36 · 50.0% |
| Pharmacokinetics / exposure | 18 / 56 · 32.1% | 9 / 36 · 25.0% |
Phase II protocols carry heavier safety, response and PK packages, consistent with dose-optimization and early efficacy screening. Phase III protocols shift toward confirmatory durability and patient-benefit measures, with recurrence/EFS and QoL/PRO each present in 18 of 36 trials (50.0%).
The largest disease-setting groups were non-muscle-invasive bladder cancer (20 trials), muscle-invasive bladder cancer (19), broad/unspecified bladder or urothelial cancer (18), advanced/metastatic urothelial cancer (16), solid-tumour basket studies including urothelial cancer (15), and upper tract urothelial carcinoma (4). Recurrence/EFS dominates NMIBC and MIBC, while ORR dominates advanced/metastatic and basket studies.