TL48: Sensitivity Analyses for Progression-free Survival in Supporting Labeling Claim
*Yun Wang, FDA/CDER 

Keywords:

Progression-free survival (PFS) is often used as a surrogate primary endpoint to demonstrate clinical benefit in Oncology clinical trials. However, PFS is subject to ascertainment bias and any imbalance in assessment dates or a substantial amount of missing data could undermine confidence in the PFS results of the trial and may prevent a labeling claim on PFS. In this roundtable discussion, we will explore the role of sensitivity analyses of PFS in supporting the effectiveness claim based on primary endpoint of PFS.

Key questions for discussion: 1. What kinds of sensitivity analyses should be performed for PFS under different situations, such as asymmetric tumor assessments, missing data, subjective symptomatic progression? 2. How these sensitivity analyses can be used to reduce the bias and demonstrate robustness in PFS estimates.