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The Relationship Between Response Rate and Survival Benefits in Randomized Immunotherapy Studies
1Edinburgh Medical School, Biomedical Sciences, The University of Edinburgh, Edinburgh EH8 9YL, UK.
Cancers
|February 13, 2025
Summary
Objective Response Rate (ORR) moderately predicts Progression-Free Survival (PFS) in immuno-oncology trials but weakly predicts Overall Survival (OS), especially with combination therapies. ORR is a limited surrogate for long-term outcomes.
Area of Science:
- Oncology
- Clinical Trials
- Immunotherapy Research
Background:
- Assessing cancer treatment efficacy requires understanding the link between Objective Response Rate (ORR) and survival outcomes like Progression-Free Survival (PFS) and Overall Survival (OS).
- Immuno-oncology (IO) therapies, including immune checkpoint inhibitors (e.g., anti-PD-(L)1, CTLA-4) and combinations, are transforming cancer care.
Purpose of the Study:
- To evaluate the relationship between ORR, PFS, and OS in clinical trials of immuno-oncology regimens.
- To determine the reliability of ORR as a surrogate endpoint for survival outcomes in IO therapy trials.
Main Methods:
- Analysis of data from 55 FDA-submitted clinical trials involving various immunotherapy regimens (IO, anti-PD-(L)1, CTLA-4, and combinations).
- Inclusion criteria required trials to report ORR, PFS, and OS.
- Correlation analysis was performed to assess the relationship between ORR and survival metrics.
Main Results:
- A moderate correlation was observed between ORR and PFS across most IO regimens, suggesting ORR's utility for predicting short-term disease control.
- The correlation between ORR and OS was weaker, particularly in trials with combination therapies, indicating ORR's limited predictive value for long-term survival.
- ORR's predictive power for PFS was stronger in first-line treatments compared to later lines.
Conclusions:
- Objective Response Rate (ORR) serves as a useful surrogate for Progression-Free Survival (PFS) in immuno-oncology (IO) trials but has restricted utility for predicting Overall Survival (OS).
- The predictive accuracy of ORR for survival outcomes varies significantly across different treatment lines and combination strategies.
- Further research is needed to develop improved surrogate endpoints for immunotherapy to enhance clinical trial design and patient outcomes.
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