Choosing Timing and Boundary for Futility Analysis Based on Cost-Effectiveness Assessment. (3rd April 2018)

• Record Type: Journal Article
• Authors: Deng, Qiqi; Lu, Xiaoqi
• Is Part Of: Statistics in biopharmaceutical research. Volume 10:Number 2(2018)
• Page Start: 104
• Page End: 111
• Publication Date: 2018-04-03
• Subjects: Benefit-cost -- Cost-effectiveness -- Futility analysis -- Utility; Pharmacy -- Statistical methods -- Periodicals; Pharmaceutical biotechnology -- Statistical methods -- Periodicals; Biopharmaceutics -- Periodicals; Biometry -- Periodicals; Pharmacy -- Statistical methods; Periodicals; 615.190727
• DOI: 10.1080/19466315.2018.1456483
• Languages: English
• ISSNs: 1946-6315

Abstract

ABSTRACT: When including a futility analysis in a trial, it is important to choose the correct timing for the interim analysis and an appropriate futility boundary such that the trial may be stopped when the interim data suggest that a reasonable treatment effect likely does not exist. This approach is appealing from an ethical perspective because it can reduce the exposure of patients to an ineffective treatment. It is also appealing from an economic perspective because phase III trials are among the most significant financial investments in drug development. However, the design may become inefficient if the timing and boundary of the futility analysis are not chosen carefully. In this article, we will use a cost-effectiveness analysis to assess the performance of different futility rules, and we will introduce a graphical tool to guide the selection of design parameters. In addition, we will discuss how prior information or prior belief of the treatment effect and other factors may influence the futility decision.