Evaluation of Early Efficacy Endpoints for Proof-of-Concept Trials

REFERENCES

• Biomarker Definitions Working Group . ( 2001 ). Biomarkers and surrogate endpoints: preferred definitions and conceptual framework . Clinical Pharmacology and Therapeutics 69 : 89 – 95 .
   PubMed Web of Science ®Google Scholar
• Burzykowski , T. , Molenberghs , G. , Buyse , M. ( 2005 ). The Evaluation of Surrogate Endpoints . New York , NY : Springer .
   Google Scholar
• Buyse , M. , Molenberghs , G. ( 1998 ). The validation of surrogate endpoints in randomized experiments . Biometrics 54 : 1014 – 1029 .
   PubMed Web of Science ®Google Scholar
• Buyse , M. , Piedbois , P. ( 1996 ). On the relationship between response to treatment and survival time . Statistics in Medicine 15 : 2797 – 2812 .
   PubMed Web of Science ®Google Scholar
• et al. , ( 2000 ). The validation of surrogate endpoints in meta-analyses of randomized experiments . Biostatistics 1 : 49 – 67 .
   PubMedGoogle Scholar
• Chen , C. , Beckman , B. ( 2009a ). Optimal cost-effective designs of proof of concept trials and associated Go–No Go decisions . Journal of Biopharmaceutical Statistics 19 ( 2 ): 424 – 436 .
   PubMed Web of Science ®Google Scholar
• Chen , C. , Beckman , R. A. (2009b). Optimal cost-effective Go–No Go decisions in late-stage oncology drug development. Statistics in Biopharmaceutical Research 1:159–169.
   Web of Science ®Google Scholar
• Chen , C. , Wang , H. W. ( 2005 ). Proportion of treatment effect . Encyclopedia of Biopharmaceutical Statistics 1 ( 1 ): 1 – 5 .
   Google Scholar
• Fleming , T. R. ( 2005 ). Objective response rate as a surrogate endpoint: A commentary . Journal of Clinical Oncology 23 : 4845 – 4846 .
   PubMed Web of Science ®Google Scholar
• Mackenzie , T. , Abrahamowicz , M. ( 2005 ). Using categorical markers as auxiliary variables in log-rank test and hazard ratio estimation . Canadian Journal of Statistics 33 : 201 – 219 .
   Web of Science ®Google Scholar
• et al. ( 2002 ). Statistical challenges in the evaluation of surrogate endpoints in randomized trials . Controlled Clinical Trials 23 : 607 – 625 .
   PubMedGoogle Scholar
• Prentice , R. L. ( 1989 ). Surrogate endpoints in clinical trials: Definitions and operational criteria . Statistics in Medicine 8 : 431 – 440 .
   PubMed Web of Science ®Google Scholar
• Robins , J. M. ( 1993 ). Information recovery and bias adjustment in proportional hazards regression analysis of randomized trials using surrogate markers. Proceedings of Biopharmaceutical Section, American Statistical Association. pp. 24–33 .
   Google Scholar
• Robins , J. M. , Finkelstein , D. M. ( 2000 ). Correcting for noncompliance and dependent censoring in an AIDS clinical trial with inverse probability of censoring weighted (IPCW) log-rank tests . Biometrics 56 : 779 – 788 .
   PubMed Web of Science ®Google Scholar
• Sargent , D. , Wieand , S. , et al. ( 2005 ). Disease-free survival (DFS) vs. overall survival (OS) as a primary endpoint for adjuvant colon cancer studies: Individual patient data from 20, 898 patents on 18 randomized trials . Journal of Clinical Oncology 23 : 8664 – 8670 .
   PubMed Web of Science ®Google Scholar
• Tang , P. A. , Bentzen , S. M. , Chen , E. X. , Siu , L. L. ( 2007 ). Surrogate end points for median overall survival in metastatic colorectal cancer: Literature-based analysis from 39 randomized controlled trials of first-line chemotherapy . Journal of Clinical Oncology 25 : 4562 – 4568 .
   PubMed Web of Science ®Google Scholar
• Whitehead , A. ( 2002 ). Meta-analysis of Controlled Clinical Trials . New York , NY : Wiley .
   Google Scholar