Do single-arm trials have a role in drug development plans incorporating randomised trials?

ABSTRACT: Often, single-arm trials are used in phase II to gather the first evidence of an oncological drug's efficacy, with drug activity determined through tumour response using the RECIST criterion. Provided the null hypothesis of 'insufficient drug activity' is rejected, the next step could be a randomised two-arm trial. However, single-arm trials may provide a biased treatment effect because of patient selection, and thus, this development plan may not be an efficient use of resources. Therefore, we compare the performance of development plans consisting of single-arm trials followed by randomised two-arm trials with stand-alone single-stage or group sequential randomised two-arm trials. Through this, we are able to investigate the utility of single-arm trials and determine the most efficient d

SUBMITTER: Grayling MJ 

PROVIDER: S-EPMC4855632 | biostudies-literature | 2016 Mar-Apr

REPOSITORIES: biostudies-literature