A comparison of methods for treatment selection in seamless phase II/III clinical trials incorporating information on short-term endpoints

Journal of Biopharmaceutical Statistics

Volumn 25, Issue 1, 2015, Pages 170-189

  Kunz, Cornelia Ursula ^a   Friede, Tim ^b   Parsons, Nicholas ^a   Todd, Susan ^c   Stallard, Nigel ^a  

^a UNIVERSITY OF WARWICK   (United Kingdom)

^b UNIVERSITY MEDICAL CENTER GÖTTINGEN   (Germany)

^c UNIVERSITY OF READING   (United Kingdom)

Author keywords

Adaptive seamless design; Multi arm multi stage trial; Surrogate endpoints

Indexed keywords

ARTICLE; CHRONIC OBSTRUCTIVE LUNG DISEASE; DECISION MAKING; DRUG DESIGN; HUMAN; INTERMETHOD COMPARISON; MATHEMATICAL MODEL; MEDICAL INFORMATION; METHODOLOGY; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROBABILITY; BIOASSAY; COMPARATIVE STUDY; COMPUTER SIMULATION; PATIENT SELECTION; STATISTICAL MODEL; STATISTICS AND NUMERICAL DATA; TIME FACTOR; TREATMENT OUTCOME;

CLINICAL TRIALS, PHASE II AS TOPIC; CLINICAL TRIALS, PHASE III AS TOPIC; COMPUTER SIMULATION; ENDPOINT DETERMINATION; HUMANS; MODELS, STATISTICAL; PATIENT SELECTION; TIME FACTORS; TREATMENT OUTCOME;

EID: 84921511772     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543406.2013.840646     Document Type: Article

References (38)

1. Barnes, P., Pocock, S., Magnussen, H., Iqbal, A., Kramer, B., Higgins, M., Lawrence, D. (2010). Integrating indacaterol dose selection in a clinical study in COPD using an adaptive seamless design. Pulmonary Pharmacology and Therapeutics 23:165-171.
2. Bauer, P., Kieser, M. (1999). Combining different phases in the development of medical treatments within a single trial. Statistics in Medicine 18:1833-1848.
3. Brannath, W., Posch, M., Bauer, P. (2002). Recursive combination tests. Journal of the American Statistical Association 97:236-244.
4. Bretz, F., Koenig, F., Brannath, W., Glimm, E., Posch, M. (2009). Tutorial in biostatistics: Adaptive designs for confirmatory clinical trials. Statistics in Medicine 28:1181-1217.
5. Bretz, F., Schmidli, H., König, F., Racine, A., Maurer, W. (2006). Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: General concepts. Biometrical Journal 48:623-634.
6. Chataway, J., Nicholas, R., Todd, S., Miller, D., Parsons, N., Valdés-Márquez, E., Stallard, N., Friede, T. (2011). A novel adaptive design strategy increases the efficiency of clinical trials in secondary progressive multiple sclerosis. Multiple Sclerosis 17:81-88.
7. Chow, S.-C., Chang, M., Pong, A. (2005). Statistical consideration of adaptive methods in clinical development. Journal of Biopharmaceutical Statistics 15:575-591.
8. Cook, R., Farewell, V. (1996). Incorporating surrogate endpoints into group sequential trials. Biometrical Journal 38:119-130.
9. Dragalin, V. (2011). An introduction to adaptive designs and adaptation in CNS trials. European Neuropsychopharmacology 21(2):153-158.
10. Dunnett, C. W. (1955). A multiple comparison procedure for comparing several treatments with a control. Journal of the American Statistical Association 50:1096-1121.
11. Engel, B., Walstra, P. (1991). Increasing precision or reducing expense in regression experiments by using information from a concomitant variable. Biometrics 47(1):13-20.
12. European Medicines Agency (EMEA) - Committee for Medicinal Products for Human Use (CHMP) (2007). CHMP reflection paper on methodological issues in confirmatory clinical trials planned with an adaptive design. http://www.emea.europa.eu/docs/en-GB/document-library/Scientific-guideline/2009/09/WC500003616.pdf (accessed July 13, 2012).
13. Food and Drug Administration (FDA) (2010). Guidance for industry - adaptive design clinical trials for drugs and biologics. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm201790.pdf (accessed July 13, 2012).
14. Friede, T., Parsons, N., Stallard, N., Todd, S., Valdés-Márquez, E., Chataway, J., Nicholas, R. (2011). Designing a seamless phase II/III clinical trial using early outcomes for treatment selection: an application in multiple sclerosis. Statistics in Medicine 30:1528-1540.
15. Friede, T., Stallard, N. (2008). A comparison of methods for adaptive treatment selection. Biometrical Journal 50:767-781.
16. Galbraith, S., Marschner, I. (2003). Interim analysis of continuous long-term endpoints in clinical trials with longitudinal outcomes. Statistics in Medicine 22:1787-1805.
17. Gallo, P., Chuang-Stein, C., Dragalin, V., Gaydos, B., Krams, M., Pinheiro, J. (2006). Adaptive designs in clinical drug development - an executive summary of the pharma working group. Journal of Biopharmaceutical Statistics 16:275-283.
18. Genz, A., Bretz, F., Miwa, T., Mi, X., Leisch, F., Scheipl, F., Bornkamp, B., Hothorn, T. (2012). Package 'mvtnorm'. URL http://CRAN.R-project.org, R package version 0.9-9992.
19. Hampson, L. V., Jennison, C. (2013). Group sequential tests for delayed responses. Journal of the Royal Statistical Society: Series B (Statistical Methodology) 75:1-37.
20. Jennison, C., Turnbull, B. (1993). Group sequential tests for bivariate response: Interim analyses of clinical trials with both efficacy and safety endpoints. Biometrics 49:741-752.
21. Julious, S. A., Mullee, M. A. (2008). Issues with using baseline in last observation carried forward analysis. Pharmaceutical Statistics 7:142-146.
22. Kelly, P. J., Stallard, N., Todd, S. (2005). An adaptive group sequential design for phase II/III clinical trials that select a single treatment from several. Journal of Biopharmaceutical Statistics 15:641-658.
23. Kimani, P. K., Stallard, N., Hutton, J. L. (2009). Dose selection in seamless phase ii/iii clinical trials based on efficacy and safety. Statistics in Medicine 28:917-936.
24. Koenig, F., Brannath, W., Bretz, F., Posch, M. (2008). Adaptive dunnett tests for treatment selection. Statistics in Medicine 27:1612-1625.
25. Lee, S. J., Kim, K., Tsiatis, A. A. (1996). Repeated significance testing in longitudinal clinical trials. Biometrika 83:779-789.
26. Lehmacher, W., Wassmer, G. (1999). Adaptive sample size calculations in group sequential trials. Biometrics 55(4):1286-1290.
27. Marcus, R., Peritz, E., Gabriel, K. R. (1976). On closed testing procedures with special reference to ordered analysis of variance. Biometrika 63(3):655-660.
28. Marschner, I., Becker, S. (2001). Interim monitoring of clinical trials based on long-term binary endpoints. Statistics in Medicine 20:177-192.
29. Posch, M., König, F., Branson, M., Brannath, W., Dunger-Baldauf, C., Bauer, P. (2005). Testing and estimation in flexible group sequential designs with adaptive treatment selection. Statistics in Medicine 24:3697-3714.
30. Schmoll, H., Cunnighmam, D., A., S., Krapetis, C., Rougier, P., Koski, S., P., B., Mookerjee, B., Robertson, J., van Cutsem, E. (2010). mFOLFOX6 + cediranib vs mFOLFOX6 + bevacizumab in previously untreated metastatic colorectal cancer (mcrc): A randomised, doubleblind, phase II/III study (HORIZON III). Ann. Oncol. 21(Supplement 8): vii189-vii224.
31. Sooriyarachchi, M., Whitehead, J., Whitehead, A., Bolland, K. (2006). The sequential analysis of repeated binary responses: A score test for the case of three time points. Statistics in Medicine 25:2196-2214.
32. Spiessens, B., Lesaffre, E., Verbeke, G., Kim, K., DeMets, D. L. (2000). An overview of group sequential methods in longitudinal clinical trials. Statistical Methods in Medical Research 19:497-515.
33. Stallard, N. (2010). A confirmatory seamless phase II/III clinical trial design incorporating short-term endpoint information. Statistics in Medicine 29:959-971.
34. Stallard, N., Todd, S. (2003). Sequential designs for phase III clinical trials incorporating treatment selection. Statistics in Medicine 22:689-703.
35. Stallard, N., Todd, S. (2011). Seamless phase II/III designs. Statistical Methods in Medical Research 20:623-634.
36. Todd, S., Stallard, N. (2005). A new clinical trial design combining phases II and III: Sequential designs with treatment selection and a change of endpoint. Drug Information Journal 39:109-118.
37. Westfall, P. H., Tobias, R. D., Wolfinger, R. D. (2011). Multiple Comparisons and Multiple Tests Using SAS. Cary, NC: SAS Institute Inc.
38. Whitehead, A., Sooriyarachchi, M., Whitehead, J., Bolland, K. (2008). Incorporating intermediate binary responses into interim analyses of clinical trials: A comparison of four methods. Statistics in Medicine 27:1646-1666.