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. 2023 Jan 10;32(3):572–592. doi: 10.1177/09622802221146305

Table 2.

Concordance rates and equity values in alternative trial designs.

Design
Parallel group and fully randomised Two-stage Partially randomised Zelen single consent, treatments concealed Zelen single consent, treatments revealed Zelen double consent, treatments concealed Zelen double consent, treatments revealed
Concordance rate for patients who prefer treatment A ρ θ + (1 − θ)ρ 1 θφ θ 1 − φ(1 − θ) 1
Concordance rate for patients who prefer treatment B 1 − ρ θ + (1 − θ)(1 − ρ) 1 1 − θφ 1 1 − φθ 1
Concordance rate αρ + β(1 − ρ) + γ θ + (1 − θ) [αρ + β(1 − ρ) + γ] 1 αθφ + β(1 − θφ) + γ αθ + β + γ 1 − φ[α(1 − θ) + βθ] 1
Scenarios to maximise concordance (C = 1) 1) γ = 1a
2) α = 1, ρ = 1a
3) β = 1, ρ = 0a
1) γ = 1a
2) θ = 1a
3) α = ρ = 1a
4) β = 1, ρ = 0a
Always 1) γ = 1a
2) α = 1, θ = φ = 1a
3) β = 1, θ = φ = 0a
1) γ = 1a
2) β = 1a
3) θ = 1a
γ = 1a
φ = 0a
α = θ = 0a
β = θ = 1a
Always
Scenarios where concordance is conditionallyb maximised ρ = 1/2(1) ρ = 1/2(2) Always θφ = 1/2(3) θ = 1a(4) φ = 0a(5)
θ = 1/2
Always
Conditional maximum concordance (1 + γ)/2 θ + (1 − θ)(1 + γ)/2 1 (1 + γ)/2 1 1 (if φ = 0)
1 − 1/2 (1 − γ) (if θ = 1/2)
1
Equity (A vs. B) 2ρ − 1 (1 − θ)(2ρ − 1) 0 2θφ − 1 θ − 1 φ(2θ − 1) 0
Scenarios to optimise equity ρ = 1/2 ρ = 1/2
θ = 1a
Always θφ = 1/2 θ = 1a φ = 0a
θ = 1/2
Always
Conditional optimal equity 0 0 0 0 0 0 0

ρ = proportion randomised to treatment A in standard design, and in a random arm of 2-stage design; (α, β) = preference rates for treatment A or B; γ = undecided rate; θ = proportion randomised to choice arm in two-stage design, or to A-consent arms in Zelen designs; φ = proportion of patients who consent to randomised treatment in Zelen concealed designs.

a

Implies degenerate design.

b

Maximised over definitive treatment preference rates (α and β), conditional on fixed parameters (1) γ, ρ; (2) γ, ρ, θ; (3) γ, θφ; (4) γ, θ; (5) γ, φ, θ.