Key elements of phase 2 studies.
| Study design |
| One-stage |
| Multi-stage |
| Randomized |
| Contemporaneous control design |
| Selection (“pick the winner”) design |
| A prioridefinition of endpoints |
| Response rate |
| Time to event analysis |
| Selection criteria |
| Inclusion |
| Disease |
| Disease status |
| Exclusion |
| Statistical considerations |
| Type I error (α) |
| Type II error (β) |
| Number of patients needed |
| Stopping rules |
| Other issues to be reported |
| Number of patients enrolled |
| Number of patients analyzed |
| Expected results for the endpoint |
| Method used to estimate expected results |
| Comparison group (if used) |
| Historical vs concurrent |
| Study design |
| One-stage |
| Multi-stage |
| Randomized |
| Contemporaneous control design |
| Selection (“pick the winner”) design |
| A prioridefinition of endpoints |
| Response rate |
| Time to event analysis |
| Selection criteria |
| Inclusion |
| Disease |
| Disease status |
| Exclusion |
| Statistical considerations |
| Type I error (α) |
| Type II error (β) |
| Number of patients needed |
| Stopping rules |
| Other issues to be reported |
| Number of patients enrolled |
| Number of patients analyzed |
| Expected results for the endpoint |
| Method used to estimate expected results |
| Comparison group (if used) |
| Historical vs concurrent |