Table 1.
Formulae for calculating without baseline covariates the ITT, AT, PP, and IV treatment effect estimates under two different clinical trial designs: 1) when adherence to the experimental treatment is not measured in the control group; and 2) when adherence is measured in the control group, e.g., adherence to treatment guidelines. We first define the character symbols used for the formulae.
Design | Estimated Treatment Effect | |||
---|---|---|---|---|
Intention to Treat |
As Treated | Per Protocol |
Instrumental Variable |
|
Adherence not measured in controls |
A1−A0 = (A11+A10) − A0 |
A11 − (A10+A0) |
A11−A0 | ITT/P11 = (A1− A0)/P11 |
Adherence measured in controls |
A1−A0 = (A11+A10) − (A01+A00) |
(A11+A01) − (A10+A00) |
A11−A00 | ITT/(P11−P01) = (A1−A0)/ (P11−P01) |
A1 = observed average for randomized to treatment group
A0 = observed average for randomized to control group
A11 = observed average for participants who receive treatment in the randomized to treatment group
A10 = observed average for participants who do not receive treatment in the randomized to treatment group
A00 = observed average for participants who do not receive the treatment in the randomized to control group
A01 = observed average for participants who do receive the treatment in the randomized to control group (e.g., take the medication in the usual care group)
P11 = Proportion receiving treatment in the randomized to treatment group
P01 = Proportion receiving treatment in the randomized to control group