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Activity Number: 417 - Recent advancement on life time data analysis
Type: Contributed
Date/Time: Thursday, August 12, 2021 : 2:00 PM to 3:50 PM
Sponsor: Lifetime Data Science Section
Abstract #319015
Title: Impact on Type I Error and Power Due to Mixed Interval-Censoring in Single-Arm Phase II Clinical Trial Designs
Author(s): Fang-Shu Ou* and Martin Heller and Phillip Schulte
Companies: Mayo Clinic and Sole proprietor and Mayo Clinic
Keywords: Phase II; clinical trial; single-arm; interval-censoring

Single-arm phase II trials remain relevant in drug development, requiring smaller sample size compared to randomized phase II trials, and are practical when recruiting small target populations (e.g. molecularly selected patents). Historically, objective response (binary endpoint measuring tumor shrinkage) has been the endpoint of choice in single-arm phase II; however, modern therapies may extend progression-free survival (PFS) and overall survival (OS) without tumor shrinkage. Single-arm trials with PFS endpoints are typically designed using standard methodology assuming right-censored survival data despite PFS being mixed interval-censored, i.e. progression is interval-censored and death is right-censored. Ignoring the interval-censoring nature of progression leads to overestimation of time to progression and potentially introducing bias and inflating type I error in treatment comparisons with historic cohorts. We evaluate and compare methods applied to mixed interval-censored endpoints in terms of type I error and study power.

Authors who are presenting talks have a * after their name.

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