Improvement of assessment in surrogate endpoint and safety outcome of single-arm trials for anticancer drugs

Abstract

ABSTRACT Introduction Single-arm trials (SATs) and surrogate endpoints were adopted as pivotal evidence for accelerated approval of anticancer drugs for more than 30 years. However, concerns regarding clinical evidence quality in trials, particularly in the SATs of anticancer drugs have increasingly been raised. SAT may not always provide strong evidence due to the lack of control and endpoint of overall survival that is typically present in randomized controlled trials. Areas covered Clinical trial endpoint adjudication is a crucial factor in surrogate outcome measurement to ensure the data quality of the clinical trial of anticancer drugs. In this review, we systematically discuss the characteristics of adjudications in assessments in surrogate endpoint and safety outcome respectively, which are essential for ensuring reliable and transparent outcomes. Endpoint adjudication effectively reduces potential bias and mitigates variance that may be introduced by investigators when analyzing the medical records for the surrogate endpoints. We analyze the advantages and disadvantages of each type of adjudicator and provide a summary of the roles of adjudicators. Expert opinion By suggestion of improving data reliability and transparency in pivotal trials, this review aims to supply a strategy for better clinical investigation for anticancer drugs, ultimately leading to better patient outcomes.