Randomized Clinical Trials With Biomarkers: Design Issues

摘要: Clinical biomarker tests that aid in making treatment decisions will play an important role achieving personalized medicine for cancer patients. Definitive evaluation of the clinical utility these biomarkers requires conducting large randomized trials (RCTs). Efficient RCT design is therefore crucial timely introduction medical advances into practice, and a variety designs have been proposed this purpose. To guide interpretation RCTs evaluating biomarkers, we present in-depth comparison advantages disadvantages commonly used designs. Key aspects discussion include efficiency comparisons special interim monitoring issues arise because complexity RCTs. Important ongoing completed are as examples. We conclude that, most settings, biomarker-stratified (ie, use to analysis but not assignment) should be obtain rigorous assessment utility.