Trial designs classification
| Core designs | Biomarker strategy | Enrichment | Master protocols | Randomise-all |
| Design features | ||||
| Framework | ||||
| Bayesian | ||||
| Frequentist | ||||
| Model | ||||
| Disease progression | ||||
| Longitudinal | ||||
| Hierarchical | ||||
| Control group | ||||
| Common/shared | ||||
| Contemporaneous | ||||
| Historical | ||||
| Randomisation | ||||
| With treatment randomisation in both biomarker-positive and biomarker-negative subgroups | ||||
| Without treatment randomisation in the biomarker-negative subgroup | ||||
| Only for patients with discordant clinical and genomic risk evaluation | ||||
| Randomisation in the non-biomarker based strategy arm | ||||
| With treatment randomisation | ||||
| Without treatment randomisation | ||||
| Reverse biomarker strategy | ||||
| Subgroup specific | ||||
| Sequential subgroup specific | ||||
| Parallel subgroup specific | ||||
| Biomarker positive and overall strategies | ||||
| With sequential assessment | ||||
| With parallel assessment | ||||
| With fall-back analysis | ||||
| Marker sequential test | ||||
| Biomarker assessment | ||||
| With biomarker assessment in the entire population | ||||
| Without biomarker assessment in the control arm | ||||
| Personalised medicine specific adaptive aspects | ||||
| Adaptive enrichment | ||||
| Adaptive signature | ||||
| Threshold determination | ||||
| Generic adaptive aspects | ||||
| Adding a new arm | ||||
| Early stopping | ||||
| Interim analysis | ||||
| Outcome-based adaptive randomisation | ||||
| Sample size reassessment | ||||
| Seamless | ||||
| Treatment tailoring aspects | ||||
| Pharmacodynamic biomarker assessment after run-in phase period | ||||
| Dynamic treatment regime | ||||
| PK/PD modelling |
PK/PD, Pharmacokinetic/pharmacodynamic.