artcat: Sample-size calculation for an ordered categorical outcome

Stata J. 2023 Mar;23(1):3-23. doi: 10.1177/1536867X231161934. Epub 2023 Apr 5.

Abstract

We describe a new command, artcat, that calculates sample size or power for a randomized controlled trial or similar experiment with an ordered categorical outcome, where analysis is by the proportional-odds model. artcat implements the method of Whitehead (1993, Statistics in Medicine 12: 2257-2271). We also propose and implement a new method that 1) allows the user to specify a treatment effect that does not obey the proportional-odds assumption, 2) offers greater accuracy for large treatment effects, and 3) allows for noninferiority trials. We illustrate the command and explore the value of an ordered categorical outcome over a binary outcome in various settings. We show by simulation that the methods perform well and that the new method is more accurate than Whitehead's method.

Keywords: artcat; categorical variable; clinical trial; evaluation; noninferiority trial; power; proportional-odds model; randomized controlled trial; sample size; st0700; substantial-superiority trial.