Reference:

Cheung, Y. K., Mitsumoto, H. (2021). Evaluating personalized (N-of-1) trials in rare diseases: how much experimentation is enough? HDSR; Under review

Settings and assumptions:

1. Program evaluation of personalized (N-of-1) trials against standard of care (SOC)

2. Two treatments (1 vs -1) are available under either N-of-1 trials or SOC, with -1 as the reference group, i.e., average treatment effect (muB) indicates the difference between mean outcome under treatment 1 and mean under treatment -1

3. Within-subject errors are conditionally independent given the population-level parameters

4. For two-sided test, enter One-sided alpha a value that is half the nominal value

Reference:

Cheung, Y. K., Mitsumoto, H. (2021). Evaluating personalized (N-of-1) trials in rare diseases: how much experimentation is enough? HDSR; Under review

Settings and assumptions:

1. Patient-centric criteria vs length of experimentation phase (m)

2. Two treatments (1 vs -1) are available under either N-of-1 trials or SOC, with -1 as the reference group, i.e., average treatment effect (muB) indicates the difference between mean outcome under treatment 1 and mean under treatment -1

3. Within-subject errors are conditionally independent given the population-level parameters