Comparison of single-arm vs. Randomized phase II clinical trials: A bayesian approach

Journal of Biopharmaceutical Statistics

Volumn 25, Issue 3, 2015, Pages 474-489

  Sambucini, Valeria ^a  

^a SAPIENZA UNIVERSITY OF ROME   (Italy)

Author keywords

Bayesian approach; historical control rate; phase II clinical trials; randomization.

Indexed keywords

ANALYTICAL PARAMETERS; ARTICLE; BAYES THEOREM; COMPARATIVE STUDY; HUMAN; PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROBABILITY; QUANTITATIVE STUDY; RANDOMIZED CONTROLLED TRIAL (TOPIC); RESPONSE VARIABLE; SAMPLE SIZE; SINGLE ARM PHASE II CLINICAL TRIAL (TOPIC); BIOASSAY; COMPUTER SIMULATION; PROCEDURES; STATISTICAL ANALYSIS; STATISTICS AND NUMERICAL DATA; TREATMENT OUTCOME;

ANTINEOPLASTIC AGENT;

ANTINEOPLASTIC AGENTS; BAYES THEOREM; CLINICAL TRIALS, PHASE II AS TOPIC; COMPUTER SIMULATION; DATA INTERPRETATION, STATISTICAL; ENDPOINT DETERMINATION; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SAMPLE SIZE; TREATMENT OUTCOME;

EID: 84928125073     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543406.2014.920856     Document Type: Article

References (24)

1. Baey, C. and Le Deley, M.C. (2011). Effect of a misspecification of response rates on type I and type II errors, in a phase II Simon design. European Journal of Cancer 2(1):5-12.
2. Bernardo, J.M. and Smith, A.F.M. (1994). Bayesian Theory. New Yo rk: Wiley.
3. Gan, H.K., Grothey, A., Pond, G.R., Moore, M.J., Siu, L.L. and Sargent, D. (2010). Randomized Phase II Trials: Inevitable or Inadvisable? Journal of Clinical Oncology 28(15):2641-2647.
4. Jung, S-H. (2013). Randomized Phase II Cancer Clinical Trials. Boca Raton, FL: Chapman and Hall/CRC Biostatistics Series.
5. Lee J.J. and Feng, L. (2005). Randomized phase II designs in cancer clinical trials: Current status and future directions. Journal of Clinical Oncology 23(19):4450-4457.
6. Mandrekar S.J. and Sargent, D.J. (2010). Randomized phase II trials: Time for a new era in clinical trial design. Journal of Thoracic Oncology 5(7):932-934.
7. Pham-Gia, T. (2000). Distributions of the ratios of independent beta variables and applications. Communications in Statistics Theory and Methods 29:2693-2715.
8. Pond G.R. and Abbasi S. (2011). Quantitative evaluation of single-arm versus randomized phase II cancer clinical trials. Clinical Trials 8(3):260-269.
9. R Core Team(2013): R: A Language and Environment for Statistical Computing. Vienna, Austria http://www.R-project.org.
10. Ratain, M.J. and Sargent , D.J. (2009). Optimising the design of phase II oncology trials: The importance of randomisation. European Journal of Cancer 45(2):275-280.
11. Redman, M. and Crowley, J. (2007). Small randomized tri als. Journal of Thoracic Oncology 2:1-2.
12. Rubinstein, L.V., Korn, E.L., Freidlin, B., Hunsberger, S., Ivy, S.P. and Smit h, M.A. (2005). Design issues of randomized phase II trials and a proposal for phase II screening trials. Journal of Clinical Oncology 23(28):7199-7206.
13. Rubinstein, L., Crowley, J., Ivy, P., LeBlanc, M. and Sargent, D. (2009). Randomized phase II designs. Clinical Cancer Research 15:1883-1890.
14. Rubinstein, L., LeBlanc, M. and Smith, M.A. (2011). More randomization in phase II trials: Necessary but not sufficient. Journal of the National Cancer Institute 103(14):1075-1077.
15. Sambucini, V. (2008). A Bayesian predictive two-stage design for phase II clinical trials. Statistics in Medicine 27(8):1199-1224.
16. Sambucini, V. (2010). A Bayesian predictive strategy for an adaptive two-stage design in phase II clinical trials. Statistics in Medicine 29(13):1430-1442.
17. Sharma, M.R., Stadler, W.M. and Ratain, M.J. (2011). Randomized phase II trials: A longterm investment with promising returns. Journal of the National Cancer Institute 103(14):1093-1100.
18. Simon, R. (1989). Optima l two-stage designs for phase II clinical trials. Controlled Clinical Trials 10:1-10.
19. Snapinn, S., Chen, M.G., Jiang, Q. and Koutsoukos, T. (2006). Assessment of futility in clinical trials. Pharmaceutical Statistics 5(4):273-281.
20. Stewart, D.J. (2010). Randomized phase II trials: Misleading and unreliable. Journal of Clinical Oncology 28(31):649-651.
21. Tan S.B. and Machin D. (2002). Bayesian two-stage designs for phase II clinical trials. Statistics in Medicine 21:1991-2012.
22. Tang, H., Foster, N.R., Grothey, A., Ansell, S.M., Goldberg, R.M. and Sargent, D.J. (2010 ). Comparison of error rates in single-arm versus randomized phase II cancer clinical trials. Journal of Clinical Oncology 28(11):1936- 1941.
23. Taylor, J.M., Braun, T.M. and Li Z. (2006). Compari ng an experimental agent to a standard agent: Relative merits of a one-arm or randomized two-arm phase II design. Clinical Trials 3(4):335-348.
24. Wang, Y.G., Leung D.H., Li M., Tan S.B. (2005). Bayes ian designs with frequentist and Bayesian error rate considerations. Statistical Methods in Medical Research 14(5):445-456.