Predictive power to assist phase 3 go/no go decision based on phase 2 data on a different endpoint

Statistics in Medicine

Volumn 31, Issue 9, 2012, Pages 831-843

  Hong, Shengyan ^a   Shi, Li ^a  

^a MEDIMMUNE   (United States)

Author keywords

Hazard ratio; Overall survival; Phase 3 go no go decision; Predictive power; Progression free survival

Indexed keywords

ARTICLE; BOOTSTRAPPING; COMPARATIVE EFFECTIVENESS; DECISION MAKING; DRUG DESIGN; HUMAN; INTERMETHOD COMPARISON; MATHEMATICAL COMPUTING; MATHEMATICAL MODEL; MULTIVARIATE ANALYSIS; OUTCOME ASSESSMENT; OVERALL SURVIVAL; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PREDICTION; PREDICTIVE POWER; PROBABILITY; PROCESS DEVELOPMENT; PROCESS MODEL; PROCESS OPTIMIZATION; PROGRESSION FREE SURVIVAL; RISK ASSESSMENT; RISK FACTOR; SAMPLE SIZE; SENSITIVITY ANALYSIS; STATISTICAL DISTRIBUTION; STATISTICAL PARAMETERS; STRATEGIC PLANNING; SYSTEMATIC ERROR; VALIDATION PROCESS;

CLINICAL TRIALS, PHASE III AS TOPIC; COMPUTER SIMULATION; DECISION MAKING; DISEASE-FREE SURVIVAL; ENDPOINT DETERMINATION; HUMANS; MONTE CARLO METHOD; PREDICTIVE VALUE OF TESTS; SURVIVAL ANALYSIS;

EID: 84859590881     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.4476     Document Type: Article

References (20)

1. Kola I, Landis J. Can the pharmaceutical industry reduce attrition rates? Nature Reviews Drug Discovery 2004; 3:711-715.
2. Ballman KV, Buckner JC, Brown PD, Giannini C, Flynn PJ, LaPlant BR, Jaeckie KA. The relationship between six-month progression-free survival and 12-month overall survival end points for phase II trials in patients with glioblastoma multiforme. Neuro-oncology 2007; 9:29-38.
3. Buyse M, Burzykowski T, Michiels S, Carroll K. Individual- and trial-level surrogacy in colorectal cancer. Statistical Methods in Medical Research 2008; 17:467-475.
4. Buyse M, Burzykowski T, Carroll K, Michiels S, Sargent DJ, Miller LL, Elfring GL, Pignon JP, Piedbois P. Progression-free survival is a surrogate for survival in advanced colorectal cancer. Journal of Clinical Oncology 2007; 25:5218-5224.
5. Mandrekar SJ, Hillman SL, Ziegler AKL, Reuter NF, Rowland KM, Marks RS, Schild SE, Adjei AA. Comparison of progression-free survival (PFS) with best or confirmed response (BR, CR) as an endpoint for overall survival (OS) in advanced non small cell lung cancer (A-NSCLC): A North Central Cancer Treatment Group (NCCTG) investigation. Journal of Clinical Oncology 2008; 26:abstr 8021.
6. Wilkerson J, Fojo J. Progression-free survival is simply a measure of a drug's effect while administered and is not a surrogate for overall survival. Cancer Journal 2009; 15:379-385.
7. Zhuang SH, Xiu L, Elsayed YA. Overall survival: a gold standard in search of a surrogate: the value of progression-free survival and time to progression as end points of drug efficacy. Cancer Journal 2009; 15:395-400.
8. Foster NR, Qi YW, Shi Q, Krook JE, Kugler JW, Jett JR, Molina JR, Schild SE, Adjei AA, Mandrekar SJ. Tumor response and progression-free survival as potential surrogate endpoints for overall survival in extensive stage small-cell lung cancer. Cancer 2011; 117:1262-1271.
9. Bast RC, Thigpen JT, Arbuck SG, Basen-Engquist K, Burke LB, Freedman R, Horning SJ, Ozols R, Rustin GJ, Spriggs D, Wenzel LB, Pazdur R. Clinical trial endpoints in ovarian cancer: Report of an FDA/ASCO/AACR Public Workshop. Gynecologic Oncology 2007; 107:173-176.
10. Miller K, Wang M, Gralow J, Dickler M, Cobleigh M, Perez EA, Shenkier T, Cella D, Davidson NE. Paclitaxel plus bevacizumab versus paclitaxel alone for metastatic breast cancer. New England Journal of Medicine 2007; 357:2666-2676.
11. Jennison C, Turnbull BW. Group Sequential Methods with Applications in Clinical Trials. Chapman and Hall/CRC: Boca Raton, Florida, 2000.
12. Schmidli H, Bretz F, Racine-Poon A. Bayesian predictive power for interim adaptation in seamless phase II/III trials where the endpoint is survival up to some specified timepoint. Statistics in Medicine 2007; 26:4925-4938.
13. Inoue LYT, Thall PF, Berry DA. Seamlessly expanding a randomized phase II trial to phase III. Biometrics 2002; 58:823-831.
14. Engel B, Walstra P. Increasing precision or reducing expense in regression experiments by using information from a concomitant variable. Biometrics 1991; 47:13-20.
15. Galbraith S, Marschner IC. Interim analysis of continuous long-term endpoints in clinical trials with longitudinal effects. Statistics in Medicine 2003; 22:1787-1805.
16. Todd S, Stallard N. A new clinical trial design combining phase II and III: sequential designs with treatment section and a change of endpoint. Drug Information Journal 2005; 39:109-118.
17. Stallard N. A confirmatory seamless phase II/III clinical trial design incorporating short-term endpoint information. Statistics in Medicine 2010; 29:959-971.
18. O'Hagan A, Stevens JW, Campbell MJ. Assurance in clinical trial design. Pharmaceutical Statistics 2005; 4:187-201.
19. Hildebrand FB. Introduction to Numerical Analysis. McGraw-Hill: New York, 1956.
20. Wei LJ, Lin DY, Weissfeld L. Regression analysis of multivariate incomplete failure time data by modeling marginal distributions. JASA 1989; 84:1065-1073.