Sample Size Reestimation for Confirmatory Clinical Trials

摘要: This chapter discusses the benefits and limitations of adaptive sample size reestimation for phase 3 confirmatory clinical trials. Comparisons are made with more traditional fixed group sequential designs. It is seen that real benefit approach arises through ability to invest resources into trial in stages. The starts a small up-front commitment. Additional committed only if promising results obtained at an interim analysis. strategy be advantageous than or approaches certain settings. discussion illustrated help three case studies actual trials, one neurology continuous endpoint cardiology binomial endpoint, oncology time event endpoint. Methodological, regulatory operational issues examined.