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Activity Number: 170 - SPEED: Biopharmaceutical Methods and Application I, Part 1
Type: Contributed
Date/Time: Monday, July 29, 2019 : 10:30 AM to 12:20 PM
Sponsor: Biopharmaceutical Section
Abstract #304365 Presentation
Title: Optimal Design and Analysis of Efficacy Expansion in Phase I Oncology Trials
Author(s): Iris Wu* and Fang Liu and Heng Zhou and Cong Chen
Companies: Merck & Co. and Merck and Merck & Co., Inc and Merck & Co., Inc
Keywords: Basket Trial; Efficacy Expansion; Heterogeneity; Immuno-oncology; Optimal Design

In oncology studies, efficacy expansion is typically conducted in a small single arm study of multiple tumor types. Information from efficacy expansion trial is used to evaluate the anti-tumor activity as well as safety and PK/PD of the investigational drug in order to make a future Go-No-Go decision for larger randomized trials. Efficacy expansion cohorts are usually equally sized and individually analyzed for selected tumor types. Individual analyses are usually under powered due to small sample size, and pooled analysis ignores the heterogeneity of anti-tumor activity across tumor types. We propose an optimal design and analysis strategy for the efficacy expansion to assess the overall effectiveness of the investigational drug. Allowing the heterogeneous anti-tumor effect across tumor indications, we pool the potential active tumor indications to increase the power of claiming overall effectiveness of the study drug. The bar to prune inactive tumor types are optimized under specified type I and type II error rates. Simulation studies were conducted to compare the operating characteristics to other approaches.

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

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