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Endpoint Governance and Reporting Transparency in Biomarker-Guided Oncology Trials: A Systematic Review, 2015 to 2024
Fidelis Nwachukwu1, Mary Obimma2
1Roseman University College of Medicine, Las Vegas, NV.
American Journal of Clinical Oncology
|March 30, 2026
Summary
Biomarker-guided cancer trials often use surrogate endpoints like objective response rate (ORR) instead of overall survival (OS). Many studies lack endpoint transparency, hindering evaluation of evidence sufficiency.
Area of Science:
- Oncology
- Clinical Trial Design
- Biostatistics
Background:
- Biomarker-guided oncology trials frequently use surrogate endpoints for efficient development.
- Overall survival (OS) data often mature later than surrogate endpoints.
Purpose of the Study:
- To evaluate endpoint governance and transparency in biomarker-guided oncology trials.
- To assess the prevalence of surrogate endpoints and the maturity of OS data at primary publication.
Main Methods:
- Systematic review of prospective interventional biomarker-guided oncology trials (2015-2024) following PRISMA 2020 guidelines.
- Analysis of endpoint transparency, primary endpoint selection (OS, ORR, PFS), and OS reporting/maturity.
- Penalized logistic regression to identify predictors of ORR primacy and OS immaturity.
Main Results:
- 166 out of 242 (68.6%) trials were endpoint-opaque.
- In 70 transparent trials, objective response rate (ORR) and progression-free survival (PFS) were primary endpoints in 32.9% and 47.1% respectively; OS was primary in only 2.9%.
- OS was reported in 88.6%, but mature in only 49.2% of cases; ORR primacy correlated with less immature OS (OR: 0.58).
Conclusions:
- Surrogate endpoints are common primary outcomes in biomarker-guided oncology.
- Endpoint opacity is prevalent, limiting transparent evaluation of trial evidence.
- While OS is frequently reported, it is often immature at primary publication, highlighting the need for explicit endpoint hierarchy reporting.
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