Abstract
In oncology clinical trials, the primary endpoint is often time to an event of clinical interest, e.g., time to disease progression or time to death. As a result, progression-free survival (PFS: the time from initiation of treatment till disease progression or death whichever occurs first) and overall survival (OS: the time from initiation of treatment till death) are the focus of statistical analysis in comparison of two treatment arms. It is often argued that PFS may serve as a surrogate endpoint for OS, while the validity of such surrogacy is still under debates in different types of cancer. In practice, one may observe a significant difference in PFS but no significant difference in OS; or vice versa. We provide a concordance index (C-index) to measure the degree of concordance between PFS and OS, and elaborate on the PFS vs OS discrepancies using the C-index using simulation studies and real trial analysis.
Original language | English |
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Article number | 106353 |
Journal | Contemporary Clinical Trials |
Volume | 104 |
DOIs | |
Publication status | Published - May 2021 |
Externally published | Yes |
Keywords
- Concordance index
- Oncology trials
- Overall survival
- Progression-free survival
- Surrogate
- Survival analysis