Can a propensity score matching method be applied to assessing efficacy from single-arm proof-of-concept trials in oncology?

ABSTRACT: As a result of the escalating number of new cancer treatments being developed and competition among pharmaceutical companies, decisions regarding how to proceed with phase III trials are frequently based on findings from either single-arm phase I expansion cohorts or phase II studies that compare the efficacy of the study drug to a standard-of-care benchmark derived from historical data. However, even when eligibility criteria are matched, differences in the distribution of baseline patient features may influence the outcome of single-arm trials in real-world scenarios. Therefore, novel methods are needed to enhance the accuracy of efficacy prediction from current cohorts relative to historical data. In this study, we demonstrated the feasibility of using the propensity score matching (PSM

SUBMITTER: Teng SW 

PROVIDER: S-EPMC10508568 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature