ArticleStatistics in medicine2018
Relative efficiency of precision medicine designs for clinical trials with predictive biomarkers.
Article in Statistics in medicine, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
8 citing papers in PubMed.
- Article
- Two-stage Adaptive Enrichment Designs with Survival Outcomes and Adjustment for Misclassification in Predictive Biomarkers.Statistics in biopharmaceutical research · 2025Article
- Two-stage stratified designs with survival outcomes and adjustment for misclassification in predictive biomarkers.Statistics in medicine · 2024Article
- Confirmatory prediction-driven RCTs in comparative effectiveness settings for cancer treatment.British journal of cancer · 2023Article
- Defining Clinical Utility of Germline Indicators of Toxicity Risk: A Perspective.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2022Article
- A twelve-gene signature for survival prediction in malignant melanoma patients.Annals of translational medicine · 2020Article
- Two-stage enrichment clinical trial design with adjustment for misclassification in predictive biomarkers.Statistics in medicine · 2019Article
- An alternative method to analyse the biomarker-strategy design.Statistics in medicine · 2018Article
Corrections and comments
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Authors and funding
2 authors.
Funding
Abstract
Prospective randomized clinical trials addressing biomarkers are time consuming and costly, but are necessary for regulatory agencies to approve new therapies with predictive biomarkers. For this reason, recently, there have been many discussions and proposals of various trial designs and comparisons of their efficiency in the literature. We compare statistical efficiencies between the marker-stratified design and the marker-based precision medicine design regarding testing/estimating 4 hypotheses/parameters of clinical interest, namely, treatment effects in each marker-positive and marker-negative cohorts, marker-by-treatment interaction, and the marker's clinical utility. As may be expected, the stratified design is more efficient than the precision medicine design. However, it is perhaps surprising to find out how low the relative efficiency can be for the precision medicine design. We quantify the relative efficiency as a function of design factors including the marker-positive prevalence rate, marker assay and classification sensitivity and specificity, and the treatment randomization ratio. It is interesting to examine the trends of the relative efficiency with these design parameters in testing different hypotheses. We advocate to use the stratified design over the precision medicine design in clinical trials with predictive biomarkers.
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Registered trials
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