Synthetic data as external control arms in scarce single-arm clinical trials.

ABSTRACT: An external control arm based on health registry data can serve as an alternative comparator in single-arm drug development studies that lack a benchmark for comparison to the experimental treatment. However, accessing such observational healthcare data involves a lengthy and intricate application process, delaying drug approval studies and access to novel treatments. Clinical trials typically comprise only a few hundred patients usually with high-cardinality features, which makes individual data instances more exposed to re-identification attacks. We examine whether synthetic data can serve as a proxy for the empirical control arm data by providing the same research outcomes while reducing the risk of information disclosure. We propose a reversible data generalization procedure to address

SUBMITTER: Elvatun S 

PROVIDER: S-EPMC11756779 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature