Journal
BRITISH JOURNAL OF CANCER
Volume 110, Issue 8, Pages 1950-1957Publisher
SPRINGERNATURE
DOI: 10.1038/bjc.2014.156
Keywords
adaptive design; biomarker strategy design; concordance; personalised medicine; two-stage design
Categories
Funding
- Medical Research Council [U.1052.00.014]
- National Institute for Health Research Health Technology Assessment (NIHR HTA) Programme [10/34/01]
- National Institute for Health Research University College London Hospitals Biomedical Research Centre
- Medical Research Council [MR/L004933/1, MC_UP_1302/2] Funding Source: researchfish
- National Institute for Health Research [10/34/01] Funding Source: researchfish
- MRC [MR/L004933/1, MC_UP_1302/2] Funding Source: UKRI
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Background: The Biomarker Strategy Design has been proposed for trials assessing the value of a biomarker in guiding treatment in oncology. In such trials, patients are randomised to either receive the standard chemotherapy treatment or a biomarker-directed treatment arm, in which biomarker status is used to guide treatment. Methods: Motivated by a current trial, we consider an adaptive design in which two biomarkers are assessed. The trial is conducted in two stages. In the first stage, patients in the biomarker-guided arm are assessed using a standard and an alternative cheaper biomarker, with the standard biomarker guiding treatment. An analysis comparing biomarker results is then used to choose the biomarker to use for the remainder of the trial. The new biomarker is used if the results for the two biomarkers are sufficiently similar. Results: We show that in practical situations the first-stage results can be used to adapt the trial without type I error rate inflation. We also show that there can be considerable cost gains with only a small loss in power in the case where the alternative biomarker is highly concordant with the standard one. Conclusions: Adaptive designs have an important role in reducing the cost and increasing the clinical utility of trials evaluating biomarker-guided treatment strategies.
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