Table 3

Trial designs classification

Core designsBiomarker strategyEnrichmentMaster protocolsRandomise-all
Design features
  Disease progression
 Control group
  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
 Treatment tailoring aspects
  Pharmacodynamic biomarker assessment after run-in phase period
  Dynamic treatment regime
  PK/PD modelling
  • PK/PD, Pharmacokinetic/pharmacodynamic.