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Activity Number: 263
Type: Contributed
Date/Time: Monday, August 1, 2016 : 2:00 PM to 3:50 PM
Sponsor: Biopharmaceutical Section
Abstract #319381
Title: A Novel Approach to Non-Muscle Invasive Bladder Cancer Trials: Making Lemonade Out of Apples and Oranges
Author(s): Yuqun Luo* and John Scott
Companies: FDA and FDA
Keywords: NMIBC ; Statistical Evidence ; Clinical Endpoint ; Combined p-value ; Heterogeneous Population ; Novel Trial Design
Abstract:

Non-muscle invasive bladder cancer (NMIBC) presents unmet medical needs. Here, we address a trial design challenge in NMIBC submissions to FDA: the conflict between maximizing sample size in a rare disease clinical trial and recruiting from heterogeneous populations where there may not be a common sensible clinical endpoint. Specifically, NMIBC patients are of two types: those with carcinoma-in-situ at baseline (CIS+) and those with only papillary disease (PAP). PAP patients have their tumors resected prior to treatment and, as such, cannot be sensibly monitored for tumor response. CIS+ patients are not resected at baseline, and cannot be monitored for tumor recurrence until their tumors have responded. We propose a novel trial design consisting of two separate components. The CIS+ component treats patients in a single arm fashion, with a primary endpoint of response rate. The PAP component randomizes patients to receive treatment or control, with a primary endpoint of event free survival. The overall p-value combines the two component p-values. We present some example trial designs and simulations, and discuss the assumptions and implications of this trial design.


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

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