Improving clinical trial efficiency by biomarker-guided patient selection

Table 1 Parameters and parameter values that define the various simulation scenarios

Scenario	r	Δ	β ₁	β ₂	β ₃	β ₄
IA	0.7	0.5	0.0	0.0	0.0	−0.6
IB	0.7	0.5	0.0	0.0	−0.1	0.0
IC	0.7	0.5	0.0	0.0	−0.1	−0.6
IIA	0.7	0.5	−0.3	0.0	0.0	−0.6
IIB	0.7	0.5	−0.3	0.0	−0.1	0.0
IIC	0.7	0.5	−0.3	0.0	−0.1	−0.6
IIIA	0.7	0.5	0.0	0.5	0.0	−0.6
IIIB	0.7	0.5	0.0	0.5	−0.1	0.0
IIIC	0.7	0.5	0.0	0.5	−0.1	−0.6

r is the correlation between A₀ and A₁. ∆ is the assumed mean improvement on the marker on treatment in the first design stage. β ₁ is the primary effect of treatment in the second stage, β ₂ is the main effect of baseline marker level, β ₃ is the treatment status by baseline marker level interaction and β ₄ is the treatment status by first-stage marker improvement interaction.

ISSN: 1745-6215