Optimal predictive probability designs for randomized biomarker-guided oncology trials.

ABSTRACT:

Introduction

Efforts to develop biomarker-targeted anti-cancer therapies have progressed rapidly in recent years. With efforts to expedite regulatory reviews of promising therapies, several targeted cancer therapies have been granted accelerated approval on the basis of evidence acquired in single-arm phase II clinical trials. And yet, in the absence of randomization, patient prognosis for progression-free survival and overall survival may not have been studied under standard of care chemotherapies for emerging biomarker subpopulations prior to the submission of an accelerated approval application. Historical control rates used to design and evaluate emerging targeted therapies often arise as population averages, lacking specificity to the targeted genetic or immunophenotypic profi

SUBMITTER: Zabor EC 

PROVIDER: S-EPMC9763994 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature