Improving efficiency in clinical trials using auxiliary information: Application of a multi-state cure model.

ABSTRACT: In clinical trials, an intermediate marker measured after randomization can often provide early information about the treatment effect on the final outcome of interest. We explore the use of recurrence time as an auxiliary variable for estimating the treatment effect on overall survival in phase three randomized trials of colon cancer. A multi-state model with an incorporated cured fraction for recurrence is used to jointly model time to recurrence and time to death. We explore different ways in which the information about recurrence time and the assumptions in the model can lead to improved efficiency. Estimates of overall survival and disease-free survival can be derived directly from the model with efficiency gains obtained as compared to Kaplan-Meier estimates. Alternatively, efficienc

SUBMITTER: Conlon AS 

PROVIDER: S-EPMC4480062 | biostudies-literature | 2015 Jun

REPOSITORIES: biostudies-literature