Endpoints and benchmark rationale
Define outcomes and comparison basis
A success threshold, an aggregate historical benchmark and participant-level external controls are different comparison bases. Record why the benchmark is applicable; none creates randomized exchangeability.
Flow diagram
Steps
Define study objectives
Choose estimation, benchmark testing or another justified objective.
Primary endpoints
Define outcome scale, assessment window and success criteria.
Secondary endpoints
Define secondary and exploratory measures.
Safety endpoints
Specify safety outcomes and ascertainment.
Justify comparison basis
Distinguish a threshold, historical estimate and patient-level external comparator.
Specify estimand and missingness
Define population, outcome, event strategies, summary and incomplete-data approach.
Endpoints and benchmark rationale
End flow
Tables and supporting information
Benchmark evidence
External-comparator data require additional selection and confounding flows.
Endpoint register
Support multiple primary and secondary endpoints.
Comparison rationale
Attach sources and limitations for the proposed comparison.