Evaluation of Event-Free Survival as a Surrogate Endpoint for Overall Survival in Muscle-Invasive Bladder Cancer Following Neoadjuvant Treatment: A Systematic Review and Meta-Analysis
Oncology and Therapy, 2025
As novel treatments for muscle-invasive bladder cancer (MIBC) rapidly advance, there is an increasing need for clinical endpoints that allow for more efficient evaluation of survival outcomes. However, surrogate endpoints for overall survival (OS) that can be reached with shorter follow-up times have not been frequently studied. Event-free survival (EFS), a common endpoint in clinical trials of pre-surgery (or neoadjuvant) therapies, has been used in lieu of OS to accelerate the evaluation of MIBC treatments that may reduce clinical burdens associated with the disease. However, using EFS as a surrogate for OS in such settings has yet to be established.
To bridge this gap, an Analysis Group team led by Vice President Yan Song, Manager Aozhou Wu, and Associate Yipeng Gao collaborated with researchers from Merck and Weill Cornell Medicine to evaluate EFS as a surrogate endpoint for OS in neoadjuvant-treated MIBC. The researchers conducted a meta-analysis of eligible neoadjuvant or perioperative clinical trials and assessed the associations between EFS and OS in terms of treatment effects and survival outcomes (i.e., survival rates and median survival times).
In an article on their research, the authors find “robust evidence that EFS is a valid surrogate endpoint for OS in MIBC following neoadjuvant treatment.” These findings indicate that EFS may be used to assist regulatory and reimbursement decision making regarding new and existing MIBC therapies.
Authors
Sternberg CN, Squires P, Song Y, Wu A, Gao Y, Kataria RS, Li H