Across 89 European CTIS melanoma Phase II and III trial records, safety/tolerability endpoints are the most frequent overall, appearing in 60 trials (67.4%), followed by objective response rate in 59 trials (66.3%) and overall survival in 56 trials (62.9%). Phase II trials are more response- and biomarker-heavy, while Phase III trials shift toward time-to-event endpoints such as progression-free survival, relapse-free survival, and overall survival.
Safety/tolerability, objective response rate (ORR), and overall survival (OS) form the dominant endpoint set. Progression-free survival (PFS) appears in 43 of 89 trials (48.3%), while biomarker/translational endpoints appear in 38 of 89 trials (42.7%).
European melanoma trials are still anchored in classical oncology efficacy and safety: ORR, OS, PFS, and adverse-event outcomes dominate. Biomarker endpoints are also common, appearing in 38 of 89 trials, reflecting continued biomarker-driven melanoma development.
ORR is the leading primary endpoint category, appearing as a primary endpoint in 30 of 89 trials (33.7%). Secondary endpoints are broader: ORR appears in 54 trials (60.7%), safety in 51 trials (57.3%), and OS in 49 trials (55.1%). Exploratory endpoints are concentrated, with 3 of 89 trials (3.4%) reporting exploratory biomarker/translational endpoints.
| Endpoint category | Primary | Secondary | Exploratory |
|---|---|---|---|
| Objective response / ORR | 30 / 89 · 33.7% | 54 / 89 · 60.7% | 1 / 89 · 1.1% |
| Safety / tolerability | 21 / 89 · 23.6% | 51 / 89 · 57.3% | 1 / 89 · 1.1% |
| Progression-free survival / progression | 19 / 89 · 21.3% | 35 / 89 · 39.3% | 0 / 89 · 0.0% |
| Overall survival | 11 / 89 · 12.4% | 49 / 89 · 55.1% | 0 / 89 · 0.0% |
| Biomarker / translational | 10 / 89 · 11.2% | 32 / 89 · 36.0% | 3 / 89 · 3.4% |
| Duration of response | 0 / 89 · 0.0% | 32 / 89 · 36.0% | 0 / 89 · 0.0% |
Primary endpoint strategy is more selective, led by ORR, safety, and PFS. Secondary endpoint strategy is more comprehensive, usually layering survival, response durability, disease control, safety, pharmacokinetics, and patient-reported outcomes around the main efficacy question.
The dataset supports comparison across five disease groups: melanoma not otherwise specified (22/89), solid-tumour basket trials including melanoma (22/89), advanced/metastatic cutaneous melanoma (19/89), resectable or stage II–III cutaneous melanoma (14/89), and uveal/ocular melanoma (12/89).
Endpoint choice follows clinical setting. Advanced/metastatic trials emphasize ORR and PFS, resectable/stage II–III trials emphasize RFS and surgery-linked outcomes, and uveal/ocular melanoma trials show heavier safety, disease-control, and visual/ocular outcome orientation.
Phase II records account for 56 of 89 trials (62.9%), Phase III for 27 of 89 (30.3%), and mixed Phase II/III designs for 6 of 89 (6.7%). Phase II is led by ORR in 43 of 56 trials (76.8%), while Phase III is more time-to-event oriented, with safety and OS each in 13 of 27 trials (48.1%) and PFS/RFS more prominent as primary endpoint choices.