Considerations for pooling real-world data as a comparator cohort to a single arm trial: a simulation study on assessment of heterogeneity.

ABSTRACT:

Background

Novel precision medicine therapeutics target increasingly granular, genomically-defined populations. Rare sub-groups make it challenging to study within a clinical trial or single real-world data (RWD) source; therefore, pooling from disparate sources of RWD may be required for feasibility. Heterogeneity assessment for pooled data is particularly complex when contrasting a pooled real-world comparator cohort (rwCC) with a single-arm clinical trial (SAT), because the individual comparisons are not independent as all compare a rwCC to the same SAT. Our objective was to develop a methodological framework for pooling RWD focused on the rwCC use case, and simulate novel approaches of heterogeneity assessment, especially for small datasets.

Methods

We present a framewor

SUBMITTER: Backenroth D 

PROVIDER: S-EPMC10464044 | biostudies-literature | 2023 Aug

REPOSITORIES: biostudies-literature