Abstract
In the past decade, adaptive design methods in clinical research have attracted much attention because it offers the principal investigators (1) potential flexibility for identifying clinical benefit of a test treatment under investigation, but efficiency for speeding up the development process. One of the most commonly considered adaptive designs is probably a two-stage seamless (e.g., phase I/II or phase II/III) adaptive design. The two-stage seamless adaptive designs can be classified into four categories depending upon study objectives and study endpoints at different stages. These categories include (I) design with same study objectives and study endpoints at different stages, (II) designs with same study objectives but different study endpoints at different stages, (III) designs with different study objectives but same study endpoints at different stages, and (IV) designs with different study objectives and different study endpoints at different stages. In this article, an overview of statistical methods for analysis of these different types of two-stage designs is provided. In addition, a case study concerning the evaluation of a test treatment for treating hepatitis C infected patients utilizing type (IV) trial design is presented.
Highlights
In the past decade, adaptive design methods in clinical research have attracted much attention because it offers the principal investigators (1) potential flexibility for identifying clinical benefit of a test treatment under investigation, but efficiency for speeding up the development process
It is often of interest to determine whether the typical O’Brien-Fleming type of boundaries is feasible. Another challenge is “How to perform a valid analysis that combines data collected from different stages?” to address these questions, Cheng and Chow [5] proposed the concept of a multiple-stage transitional seamless adaptive design which takes into consideration of different study objectives and study endpoints
Two-stage seamless adaptive designs can be classified into four categories depending upon the study objectives and endpoints used at different stages
Summary
Adaptive design methods in clinical research have attracted much attention because it offers the principal investigators (1) potential flexibility for identifying clinical benefit of a test treatment under investigation, but efficiency for speeding up the development process. The FDA adaptive design draft guidance defines an adaptive design as a clinical study that includes a prospectively planned opportunity for modification of one or more specified aspects of the study design and hypotheses based on analysis of data (usually interim data) from subjects in the study [1]. As it is recognized by many investigators/researchers, the use of adaptive design methods in clinical trials may allow the researchers to correct assumptions used at the planning stage and select the most promising option early. A two-stage adaptive seamless phase II/III design only refers to a study containing an exploratory phase II stage (stage 1) and a confirmatory phase III stage (stage 2) while data collected at both phases (stages) will be used for final analysis
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