Table 2.
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.
Implies degenerate design.
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.