A bootstrapping method to optimize go/no-go decisions from single-arm, signal-finding studies in oncology.

ABSTRACT: Phase Ib trials are common in oncology development but often are not powered for statistical significance. Go/no-go decisions are largely driven by observed trends in response data. We applied a bootstrapping method to systematically compare tumor dynamic end points to historical control data to identify drugs with clinically meaningful efficacy. A proprietary mathematical model calibrated to phase Ib anti-PD-1 therapy trial data (KEYNOTE-001) was used to simulate thousands of phase Ib trials (n = 30) with a combination of anti-PD-1 therapy and four novel agents with varying efficacy. A redacted bootstrapping method compared these results to a simulated phase III control arm (N = 511) while adjusting for differences in trial duration and cohort size to determine the probability that the no

SUBMITTER: Dutta R 

PROVIDER: S-EPMC11330177 | biostudies-literature | 2024 Aug

REPOSITORIES: biostudies-literature