Progression-free survival (PFS) as a primary endpoint for clinical studies in advanced neuroendocrine tumors (NETs) Abstract #158

Introduction: Successfully testing new antitumor agents requires primary study endpoints that are clinically important, and also provide sufficient statistical power within a timeframe and population size that is feasible for the tumor type. Using overall survival (OS) as the primary endpoint in NET studies is challenging because of the low incidence (5.25/100,000 annually), disease heterogeneity, extended survival time (Yao 2008), and confounding effects of multiple therapies. These inherent aspects of NETs make OS an extremely difficult and challenging endpoint to evaluate clinical efficacy.
Aim(s): The rationale for employing PFS as the primary endpoint in studies of NETs is presented.
Materials and methods: Results from NET studies were examined in light of findings from systematic reviews of surrogate/alternative endpoints in several other, i.e. non-NET tumor types.
Conference: 7th Annual ENETS Conference (2010)
Category: Clinical
Presenting Author: Simron Singh
Authors: Singh S, Law C

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