Work

Testing for efficacy in adaptive clinical trials with enrichment

Year: 2014

Type: article

Abstract: Adaptive design of clinical trials has attracted considerable interest because of its potential of reducing costs and saving time in the clinical development process. In this paper, we consider the problem of assessing the effectiveness of a test treatment over a control by a two-arm randomized clinical trial in a potentially heterogenous patient population. In particular, we study enrichment designs that use accumulating data from a clinical trial to adaptively determine patient subpopulation in which the treatment effect is eventually assessed. A hypothesis testing procedure and a lower confidence limit are presented for the treatment effect in the selected patient subgroups. The performances of the new methods are compared with existing approaches through a simulation study.

Source: Statistics in Medicine

DOI: 10.1002/sim.6127

Authors: Samuel S. Wu, Yi-Hsuan Tu, Ying He

Institutions: University of Florida, National Cheng Kung University

Language: English

Topic: Statistical Methods in Clinical Trials

Subfield: Statistics and Probability

Field: Mathematics

Domain: Physical Sciences

SDG: Good health and well-being

Open Access status: green

Funders: National Institute on Aging, National Institute of General Medical Sciences, National Institute of Arthritis and Musculoskeletal and Skin Diseases