Addressing the statistical analysis dilemma that exists when analyzing clinical trial results with full efficacy using the Kaplan Meier survival analysis method

Abstract

The use of a Kaplan–Meier (K–M) survival time approach is generally considered appropriate to report antimalarial efficacy trials. However, when a treatment arm has 100% efficacy, confidence intervals may not be computed. Furthermore, methods that use probability rules to handle missing data for instance by multiple imputation, encounter perfect prediction problem when a treatment arm has full efficacy, in which case all imputed values are either treatment success or all imputed values are failures. The use of a survival K–M method addresses this imputation problem in estimating the efficacy estimates also referred to as cure rates. We discuss the statistical challenges and propose a potential way forward.

Keywords

• efficacy
• intention to treat
• Kaplan–Meier
• missing data
• multiple imputation