Clinical Trial Intelligence

Which Endpoints Are Measured Most Often in European Phase II & III Breast Cancer Trials?

11 July 2026

Across 260 unique European CTIS breast cancer Phase II and Phase III trial records first authorized in 2024–2026, safety/tolerability was the most frequent endpoint family overall, appearing in 199 trials (76.5%). Progression-free survival (PFS) was the most frequent primary endpoint family, appearing as a primary endpoint in 76 trials (29.2%), while overall survival (OS) dominated secondary endpoints, appearing as a secondary endpoint in 174 trials (66.9%).

Trials analyzed
260
Unique CTIS trial records
Most common overall
76.5%
Safety/tolerability endpoints
Top primary
29.2%
PFS as primary endpoint family
Top secondary
66.9%
OS as secondary endpoint family

Which endpoint families appear most often across any endpoint role?

Safety/tolerability endpoints appeared in 199 of 260 trials (76.5%), followed by OS in 181 trials (69.6%), objective response or tumor response in 154 trials (59.2%), and PFS in 134 trials (51.5%).

Trial-level endpoint family frequency
Safety / tolerability
76.5%
Overall survival
69.6%
Objective response
59.2%
PFS
51.5%
PRO / QoL
44.6%
Duration of response
36.9%
DCR / CBR
32.7%
Biomarker / molecular
30.4%
Share of 260 trials with at least one endpoint in each family across primary, secondary, or exploratory roles.
Interpretation

The breast cancer Phase II/III endpoint landscape is anchored by safety plus classical oncology efficacy endpoints. OS, ORR, and PFS together form the dominant efficacy cluster, while PRO/QoL endpoints are common enough to be a mainstream endpoint family rather than a niche add-on.

How do endpoint priorities change by primary, secondary, and exploratory role?

Primary endpoints were listed in 249 of 260 trials (95.8%), secondary endpoints in 235 trials (90.4%), and exploratory or other endpoints in 14 trials (5.4%). PFS led the primary layer with 76 trials (29.2% of all trials), OS led the secondary layer with 174 trials (66.9%), and biomarker/molecular endpoints led exploratory endpoints with 11 of 14 exploratory-endpoint trials (78.6%).

Top endpoint families by role
Primary
PFS 29.2%
Safety 23.5%
ORR / response 20.0%
OS 20.0%
DFS / EFS 16.5%
Secondary
OS 66.9%
Safety 65.8%
ORR / response 52.7%
PRO / QoL 44.2%
PFS 41.9%
Exploratory
Biomarker / molecular 78.6%
Safety 50.0%
Imaging / diagnostic 28.6%
OS 21.4%
ORR / DOR 21.4%
Primary and secondary percentages use all 260 trials as denominator; exploratory percentages use 14 trials with exploratory/other endpoints.
Interpretation

Primary endpoints concentrate on a small group of registration-relevant efficacy and safety measures. Secondary endpoints carry most of the broader clinical value story, especially OS, safety, response, PRO/QoL, DOR, and disease control.

How do endpoint patterns differ between Phase II and Phase III breast cancer trials?

Among 152 Phase II trials, safety/tolerability appeared in 115 (75.7%), objective response in 111 (73.0%), OS in 97 (63.8%), and PFS in 88 (57.9%). Among 105 Phase III trials, OS and safety/tolerability each appeared in 82 trials (78.1%), PRO/QoL in 72 (68.6%), PFS in 43 (41.0%), and disease-/event-free survival in 42 (40.0%).

Endpoint frequency by phase
Endpoint family Phase II Phase III
Safety / tolerability
75.7%
78.1%
Overall survival
63.8%
78.1%
Objective response
73.0%
38.1%
Progression-free survival
57.9%
41.0%
PRO / QoL
39.5%
68.6%
Disease-/event-free survival
17.1%
40.0%
Phase comparison uses 152 Phase II and 105 Phase III trials; 3 combined Phase II/III trials remain in overall totals.
Interpretation

Phase II breast cancer trials retain a response-heavy pattern, with ORR or tumor response in 111 of 152 trials. Phase III trials shift toward confirmatory and patient-centered outcomes, especially OS, safety, PRO/QoL, and disease-/event-free survival.

Which breast cancer subgroups drive different endpoint choices?

Advanced or metastatic disease was present in 121 of 260 trials (46.5%) and was strongly associated with PFS, ORR, safety, OS, and DOR. Early/adjuvant/neoadjuvant disease appeared in 46 trials (17.7%) and shifted the endpoint pattern toward disease-/event-free survival in 34 trials (73.9%) and pathologic complete response in 20 trials (43.5%).

Endpoint frequency within disease subgroup
SubgroupSafetyOSORRPFSDFS/EFSpCR
Advanced/metastatic (n=121)81.0%76.0%86.0%87.6%
HR+/HER2-negative (n=69)84.1%79.7%66.7%58.0%
TNBC (n=51)84.3%84.3%74.5%56.9%
HER2-positive (n=41)87.8%78.0%63.4%58.5%
Early/adjuvant/neoadjuvant (n=46)84.8%67.4%30.4%73.9%43.5%
HER2-low (n=11)90.9%90.9%81.8%81.8%
Subgroups are based on CTIS disease labels and are non-exclusive where disease strings include multiple labels.
Interpretation

Advanced/metastatic trials are dominated by time-to-progression and response endpoints, while early-stage trials depend more on curative-setting endpoints such as DFS/EFS and pCR. TNBC and HER2-positive subgroups show broad use of safety, OS, ORR, and PFS rather than a single endpoint family.

Do endpoint patterns differ by drug modality?

Small molecules appeared in 196 of 260 trials (75.4%), monoclonal antibodies in 81 (31.2%), antibody-drug conjugates (ADCs) in 68 (26.2%), and radiopharmaceuticals in 20 (7.7%). ADC-containing trials had particularly high OS (59 of 68; 86.8%), safety (55 of 68; 80.9%), ORR (52 of 68; 76.5%), and PFS (45 of 68; 66.2%) coverage.

Top endpoint families within modality
Small molecule
Safety 80.6%
OS 72.4%
ORR 62.8%
PFS 54.1%
PRO/QoL 49.0%
Monoclonal antibody
Safety 85.2%
OS 81.5%
ORR 72.8%
PFS 55.6%
PRO/QoL 43.2%
ADC
OS 86.8%
Safety 80.9%
ORR 76.5%
PFS 66.2%
PRO/QoL 52.9%
Radiopharmaceutical
Imaging/diagnostic 90.0%
Safety 55.0%
ORR 45.0%
PFS 45.0%
Biomarker 40.0%
Drug modality labels are non-exclusive for combination trials.
Interpretation

ADC and monoclonal-antibody trials show a broad confirmatory endpoint profile with high OS, safety, ORR, and PFS coverage. Radiopharmaceutical breast cancer trials are distinct: imaging or diagnostic endpoints dominate 18 of 20 trials (90.0%).

How much of the endpoint burden sits in secondary endpoints?

The 260 trials contained 2,032 endpoint entries: 378 primary endpoint entries (18.6%), 1,629 secondary endpoint entries (80.2%), and 25 exploratory/other endpoint entries (1.2%). On average, each trial listed 1.45 primary endpoint entries and 6.27 secondary endpoint entries.

Endpoint entry distribution
Primary 18.6%
Secondary 80.2%
Exploratory 1.2%
Distribution of 2,032 endpoint entries across endpoint roles.
Interpretation

The operational endpoint burden is overwhelmingly secondary. Breast cancer trial teams should expect the primary endpoint strategy to be relatively concentrated, while secondary endpoints carry most of the measurement complexity across efficacy, safety, PRO/QoL, pharmacokinetics, biomarkers, and treatment exposure.

What does the CTIS authorization window show for the European breast cancer cohort?

By first CTIS authorization year, 181 of 260 trials (69.6%) were authorized in 2024, 59 (22.7%) in 2025, and 20 (7.7%) in 2026. The median interval from initial CTIS/EU submission to first CTIS authorization was 65 days across 260 trials, with an interquartile range of 32 to 111 days.

First CTIS authorization year
2024
69.6%
2025
22.7%
2026
7.7%
Median
65 days
IQR low
32 days
IQR high
111 days
CTIS timing calculated from initial CTIS submission date to first CTIS authorization date.
Interpretation

The endpoint dataset is heavily weighted toward CTIS-authorized 2024 European submissions, but 2025 and 2026 records add newer trial-design signals. The 65-day median submission-to-authorization interval provides a practical EU planning benchmark for breast cancer Phase II/III trial intelligence.

Endpoint Definitions

Definitions reflect how endpoint abbreviations were used in the CTIS endpoint text.

Definitions
PFS: progression-free survival, usually time to progression or death.
OS: overall survival, usually time to death from any cause.
ORR: objective response rate, typically complete response plus partial response under RECIST criteria.
DOR: duration of response, time from first response to progression or death.
DCR / CBR: disease control rate or clinical benefit rate, commonly response plus stable disease or sustained clinical benefit.
DFS / iDFS / EFS / RFS: disease-, invasive disease-, event-, or recurrence-free survival endpoint families used mainly in early/adjuvant settings.
pCR: pathologic complete response, commonly absence of invasive carcinoma in breast and lymph nodes at surgery.
PRO / QoL: patient-reported outcome and quality-of-life endpoints, including EORTC, FACT, EQ-5D, and symptom scales.
CTIS: Clinical Trials Information System, used here for European clinical trial submission and authorization timing.