A proof of concept phase II non-inferiority criterion

• Published in: Statistics in medicine Vol. 30; no. 13; pp. 1618 - 1627
• Main Authors: Neuenschwander, Beat, Rouyrre, Nicolas, Hollaender, Norbert, Zuber, Emmanuel, Branson, Michael
• Format: Journal Article
• Language: English
• Published: Chichester, UK John Wiley & Sons, Ltd 15.06.2011; Wiley Subscription Services, Inc
• Subjects: Antineoplastic Agents - therapeutic use; Bayes Theorem; Bayesian; Bayesian analysis; Carcinoma, Renal Cell - drug therapy; Clinical trials; Clinical Trials, Phase II as Topic - methods; Comparative analysis; Confidence Intervals; Decision making; Everolimus; Humans; Indoles - therapeutic use; Kidney Neoplasms - drug therapy; level of proof; Medical treatment; Oncology; progression-free survival; proof of concept; Pyrroles - therapeutic use; Research Design; Sirolimus - analogs & derivatives; Sirolimus - therapeutic use; time-to-event

Traditional phase III non‐inferiority trials require compelling evidence that the treatment vs control effect bfθ is better than a pre‐specified non‐inferiority margin θNI. The standard approach compares this margin to the 95 per cent confidence interval of the effect parameter. In the phase II sett...