Optimal marker-strategy clinical trial design to detect predictive markers for targeted therapy.

ABSTRACT: In developing targeted therapy, the marker-strategy design (MSD) provides an important approach to evaluate the predictive marker effect. This design first randomizes patients into non-marker-based or marker-based strategies. Patients allocated to the non-marker-based strategy are then further randomized to receive either the standard or targeted treatments, while patients allocated to the marker-based strategy receive treatments based on their marker statuses. Little research has been done on the statistical properties of the MSD, which has led to some widespread misconceptions and placed clinical researchers at high risk of using inefficient designs. In this article, we show that the commonly used between-strategy comparison has low power to detect the predictive effect and is valid only

SUBMITTER: Zang Y 

PROVIDER: S-EPMC4915612 | biostudies-literature | 2016 Jul

REPOSITORIES: biostudies-literature