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Activity Number: 339
Type: Topic Contributed
Date/Time: Tuesday, August 6, 2013 : 10:30 AM to 12:20 PM
Sponsor: Biopharmaceutical Section
Abstract - #308833
Title: Adaptive Sample Size Re-Estimation for Time-to-Event Confirmatory Studies with Application to the Design of a CV/Renal Outcome Study
Author(s): Yili Lu Pritchett*+ and Hui Tang
Companies: Astellas Pharma Global Development, Inc. and AbbVie Inc.
Keywords: Adaptive sample size re-estimation ; Confirmatory study design ; CV/Renal outcome studies

When inevitable limitations exist at the learning phase of drug development, compounds could enter the confirmatory phase with great uncertainty which would directly affect on the estimation of right sample size. In such event designing confirmatory trials with adaptive sample size re-estimation can be an effective approach. In this presentation, such limitation is illustrated by a hypothetical case study where treatment effect on a biomarker is studied at learning phase, and the confirmatory study using CV or renal outcome events as primary endpoint is designed mainly on the predictive property of the biomarker. To ensure adequate study power to detect meaningful treatment effect for the confirmatory study, a design using adaptive sample size re-estimation on the biomarker enriched population is proposed. In this design, sample size up-adjustment will be guided by a pre-specified "promising zone" determined using conditional power, and the strong control of Type I error rate will be carried out using CHW method (Cui et al, 1999). Design operating characteristics obtained through simulations will be presented, and the pros and cons of such a design approach will be discussed.

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