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- Title
Randomized Clinical Trials With Biomarkers: Design Issues.
- Authors
Freidlin, Boris; McShane, Lisa M.; Korn, Edward L.
- Abstract
Clinical biomarker tests that aid in making treatment decisions will play an important role in achieving personalized medicine for cancer patients. Definitive evaluation of the clinical utility of these biomarkers requires conducting large randomized clinical trials (RCTs). Efficient RCT design is therefore crucial for timely introduction of these medical advances into clinical practice, and a variety of designs have been proposed for this purpose. To guide design and interpretation of RCTs evaluating biomarkers, we present an in-depth comparison of advantages and disadvantages of the commonly used designs. Key aspects of the discussion include efficiency comparisons and special interim monitoring issues that arise because of the complexity of these RCTs. Important ongoing and completed trials are used as examples. We conclude that, in most settings, randomized biomarker-stratified designs (ie, designs that use the biomarker to guide analysis but not treatment assignment) should be used to obtain a rigorous assessment of biomarker clinical utility.
- Subjects
BIOMARKERS; CLINICAL trials; CANCER treatment; CANCER patients; MEDICAL experimentation on humans
- Publication
JNCI: Journal of the National Cancer Institute, 2010, Vol 102, Issue 3, p152
- ISSN
0027-8874
- Publication type
Article
- DOI
10.1093/jnci/djp477