Statistical inference for extended or shortened phase II studies based on Simon¿s two-stage designs

摘要: Simon’s two-stage designs are popular choices for conducting phase II clinical trials, especially in the oncology trials to reduce number of patients placed on ineffective experimental therapies. Recently Koyama and Chen (2008) discussed how conduct proper inference such studies because they found that procedures used with almost always ignore actual sampling plan used. In particular, proposed an method when second stage sample sizes differ from planned ones. We consider alternative based likelihood ratio. we order permissible paths under using their corresponding conditional likelihood. this way, can calculate p-values common definition: probability obtaining a test statistic value at least as extreme observed null hypothesis. addition providing couple scenarios where Chen’s be difficult apply, resulting estimate our appears have certain advantage terms properties many numerical simulations. It generally led smaller biases narrower confidence intervals while maintaining similar coverages. also illustrated two methods real data setting. Inference plan. Reported P-values, point estimates response rate not usually adjusted design’s adaptiveness. Proper statistical should