Online Program

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All Times EDT

Thursday, September 24
Thu, Sep 24, 1:30 PM - 2:45 PM
Virtual
Biomarkers and Surrogate Endpoints in Clinical Trials: Current Status and Future Directions

Surrogate Endpoints in Oncology Clinical Trials: Are We There Yet? (301274)

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*Qian Shi, Mayo Clinic 

Keywords: Surrogate endpoints, regulatory approvals, meta-analytic surrogacy evaluation

The choice of an appropriate clinical outcome is the most critical and fundamental design consideration in clinical trials which are aimed to advance the treatment developments in any disease settings. Blooming biologic and genetic research brought oncology into a biomarker-driven treatment development time era. Efficient trials which can facilitate drug developments fast with affordable sizes are ever needed. Surrogate endpoints measured sooner, more frequently, at less cost, or less invasively than “gold-standard” primary endpoints can facilitate rapid treatment development and benefit patients in a timeliness fashion. In real life applications, the well accepted surrogate endpoint validation method is a meta-analytic two-level surrogacy evaluation, namely the trial-level and individual patient-level R2, developed by Buyse and colleagues. Within this framework, the trial-level surrogacy is considered to be of greater importance than high individual patient level surrogacy for a candidate surrogate endpoint to be useful in future trials to replace the clinical true endpoints. In this talk, we discuss practical challenges and debates in evaluating and validating surrogate endpoints in oncology trials. We illustrate the differences in surrogate endpoint evaluation practices between academic research and formal qualification through regulatory agencies based on real examples. We open debates of impacts from data availabilities, qualities, especially the heterogeneities in measurements and collections on true utility of "validated" surrogate endpoints in future trials. We propose principles of selecting potential surrogate endpoint candidates which are clinically meaningful and statistically feasible. We call for long-term and large scale of collaborations on development of biomarkers which are the basis to form potential surrogate endpoints.