Sample size and power for a stratified doubly randomized preference design. (July 2018)

• Record Type: Journal Article
• Authors: Cameron, Briana; Esserman, Denise A
• Other Names: Dattner Itai guest-editor.; Huppert Amit guest-editor.
• Is Part Of: Statistical methods in medical research. Volume 27:Number 7(2018)
• Page Start: 2168
• Page End: 2184
• Publication Date: 2018-07
• Subjects: Sample size -- power -- two-stage trial -- patient preference -- stratified; Medicine -- Research -- Statistical methods -- Periodicals; Research -- Periodicals; Review Literature -- Periodicals; Statistics -- methods -- Periodicals; Médecine -- Recherche -- Méthodes statistiques -- Périodiques; 610.727
• DOI: 10.1177/0962280216677573
• Languages: English
• ISSNs: 0962-2802

Abstract

The two-stage (or doubly) randomized preference trial design is an important tool for researchers seeking to disentangle the role of patient treatment preference on treatment response through estimation of selection and preference effects. Up until now, these designs have been limited by their assumption of equal preference rates and effect sizes across the entire study population. We propose a stratified two-stage randomized trial design that addresses this limitation. We begin by deriving stratified test statistics for the treatment, preference, and selection effects. Next, we develop a sample size formula for the number of patients required to detect each effect. The properties of the model and the efficiency of the design are established using a series of simulation studies. We demonstrate the applicability of the design using a study of Hepatitis C treatment modality, specialty clinic versus mobile medical clinic. In this example, a stratified preference design (stratified by alcohol/drug use) may more closely capture the true distribution of patient preferences and allow for a more efficient design than a design which ignores these differences (unstratified version).