DINART, Derek; RONDEAU, Virginie; BELLERA, Carine

doi:10.1002/pst.2407

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DINART, Derek

Bordeaux population health [BPH]

RONDEAU, Virginie

Bordeaux population health [BPH]

BELLERA, Carine
Bordeaux population health [BPH]

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Pharmaceutical Statistics. 2024-07-16

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Biomarker-guided therapy is a growing area of research in medicine. To optimize the use of biomarkers, several study designs including the biomarker-strategy design (BSD) have been proposed. Unlike traditional designs, the emphasis here is on comparing treatment strategies and not on treatment molecules as such. Patients are assigned to either a biomarker-based strategy (BBS) arm, in which biomarker-positive patients receive an experimental treatment that targets the identified biomarker, or a non-biomarker-based strategy (NBBS) arm, in which patients receive treatment regardless of their biomarker status. We proposed a simulation method based on a partially clustered frailty model (PCFM) as well as an extension of Freidlin formula to estimate the sample size required for BSD with multiple targeted treatments. The sample size was mainly influenced by the heterogeneity of treatment effect, the proportion of biomarker-negative patients, and the randomization ratio. The PCFM is well suited for the data structure and offers an alternative to traditional methodologies.< Réduire

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Biomarker‐Strategy

Frailty Model

Heterogeneity

Randomized

Sample Size

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Sample Size Estimation Using a Partially Clustered Frailty Model for Biomarker-Strategy Designs With Multiple Treatments

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