Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols

Table 1 PICOS (population, intervention, comparator, outcomes, study design) criteria

Category	Inclusion criteria
Population	Humans
Interventions	No restrictions
Comparator	No restrictions
Outcomes	No restrictions
Study design	Master protocols were defined as a single overarching protocol that has been designed to be divided into multiple sub-studies that could allow for evaluation of multiple interventional hypotheses. These included: - Basket trials - Umbrella trials - Platform trials
Other	Peer-reviewed publications and conference abstracts with results or published protocols in the English language

‘Basket trials’ were defined as any prospective clinical trials that tested the utility (e.g., effectiveness, dosage, and safety) of intervention(s) in a study population of multiple diseases with common predictive biomarkers and/or other common predictive patient characteristics that can be used to predict whether a patient will respond to a specific intervention as the unifying eligibility criteria
‘Umbrella trials’ were defined as any prospective clinical trials that tested the utility of targeted interventions based on predictive biomarkers or other patient characteristics or both; in umbrella trials, the single disease study population is stratified into multiple subgroups on predictive biomarkers or other characteristics or both
‘Platform trials’ were defined as any clinical trials that allowed the intervention arm(s) to be dropped and the flexibility of introducing new intervention(s) during the trial. Platform trials are sometimes referred to as multi-arm, multi-stage (MAMS) designs, but the MAMS designs that do not allow flexibility of adding new arms during the trial are not truly platform trials

ISSN: 1745-6215