Discussion on the issue of sample size determination for a targeted to an untargeted and to a mixed effect model-based clinical trial design

ABSTRACT

More and more studies have shown that genetic determinants may mediate variability among persons in the response to a drug. In other words, some therapeutics benefit only a subset of treated patients. Genomic technologies – such as DNA sequencing, mRNA transcript profiling, and comparative genomic hybridization – are providing biomarkers that can be used to predict which patients are most likely to respond to a given drug. In this paper, the sample size determination of a targeted clinical trial, an untargeted clinical trial and a random effect model is conducted. Treatment effect for the responder and non-responder patients, the assay specificity and sensitivity, and the proportion of the population for non-responder can affect sample size determination of the experimental design.

KEYWORDS:

• Clinical trial
• cost issue
• genetic polymorphism
• heterogeneity
• random effect design

Disclosure statement

No potential conflict of interest was reported by the author.