Work

Proper inference from Simon's two‐stage designs

Year: 2007

Type: article

Abstract: Simon's two-stage designs are very popular for phase II clinical trials. A literature review revealed that the inference procedures used with Simon's designs almost always ignore the actual sampling plan used. Reported P-values, point estimates and confidence intervals for the response rate are not usually adjusted for the design's adaptiveness. In addition, we found that the actual sample size for the second stage is often different from that planned. We present here a method for inferences using both the planned and the actual sample sizes. The conventional and the preferred inference procedures usually yield similar P-values and confidence intervals for the response rate. The conventional inference, however, may contradict the result of the corresponding hypothesis testing.

Source: Statistics in Medicine

DOI: 10.1002/sim.3123

Authors: Tatsuki Koyama, Heidi Chen

Institution: Vanderbilt University

Language: English

Topic: Statistical Methods in Clinical Trials

Subfield: Statistics and Probability

Field: Mathematics

Domain: Physical Sciences

Open Access status: green

Funder: National Cancer Institute

Award: G725763919