Decision theoretic designs for phase II clinical trials with multiple outcomes

Biometrics

Volumn 55, Issue 3, 1999, Pages 971-977

  Stallard, Nigel ^a   Thall, Peter F ^b   Whitehead, John ^a  

^a UNIVERSITY OF READING   (United Kingdom)

^b UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

Author keywords

Backward induction; Clinical trial design; Optimal stopping; Safety and efficacy monitoring; Sequential procedure

Indexed keywords

MEDICAL APPLICATIONS; PATIENT TREATMENT; STEM CELLS;

BACKWARD INDUCTION; BAYESIAN DECISION; CLINICAL TRIAL; CLINICAL TRIAL DESIGNS; DECISION-THEORETIC; GAIN FUNCTION; OPTIMAL STOPPING; PHASE II; SAFETY AND EFFICACY MONITORING; SEQUENTIAL PROCEDURES;

DECISION THEORY;

ARTICLE; CLINICAL TRIAL; EXPERIMENTAL TEST; HUMAN; MATHEMATICAL ANALYSIS; MEDICAL RESEARCH; METHODOLOGY; MULTIPLE MYELOMA; PHASE 2 CLINICAL TRIAL; STEM CELL TRANSPLANTATION;

EID: 0032885857     PISSN: 0006341X     EISSN: None     Source Type: Journal    
DOI: 10.1111/j.0006-341X.1999.00971.x     Document Type: Article

References (31)

1. Bellisant, E., Benichou, J., and Chastang, C. (1990). Application of the triangular test to phase II cancer trials. Statistics in Medicine 9, 907-917.
2. Berger, J. O. (1985). Statistical Decision Theory and Bayesian Analysis. New York: Springer-Verlag.
3. Berry, D. A. and Ho, C.-H. (1988). One-sided sequential stopping boundaries for clinical trials: A decision-theoretic approach. Biometrics 44, 219-227.
4. Berry, D. A. and Stangl, D. K. (1996). Bayesian methods in health-related research. In Bayesian Biostatistics, D. A. Berry and D. K. Stangl (eds), 3-66. New York: Dekker.
5. Berry, D. A., Wolff, M. C., and Sack, D. (1994). Decision making during a phase III randomized controlled trial. Controlled Clinical Trials 15, 360-378.
6. Brunier, H. C. and Whitehead, J. (1994). Sample sizes for phase II clinical trials derived from Bayesian decision theory. Statistics in Medicine 13, 2493-2502.
7. Bryant, J. and Day, R. (1995). Incorporating toxicity considerations into the design of two-stage phase II clinical trials. Biometrics 51, 1372-1383.
8. Conaway, M. R. and Petroni, G. R. (1995). Bivariate sequential designs for phase II trials. Biometrics 51, 656-664.
9. Conaway, M. R. and Petroni, G. R. (1996). Designs for phase II trials allowing for a trade-off between response and toxicity. Biometrics 52, 1375-1386.
10. Cressie, N. and Biele, J. (1994). A sample-size-optimal Bayesian decision procedure for sequential pharmaceutical trials. Biometrics 50, 700-711.
11. DeGroot, M. H. (1970). Optimal Statistical Decisions. New York: McGraw-Hill.
12. Efron, B. (1986). Why isn't everyone a Bayesian? American Statistician 40, 1-5.
13. Ensign, L. G., Gehan, E. A., Kamen, D. S., and Thall, P. F. (1994). An optimal three-stage design for phase II clinical trials. Statistics in Medicine 13, 1727-1736.
14. Fisher, L. D. (1996). Comments on Bayesian and frequentist analysis and interpretation of clinical trials. Controlled Clinical Trials 17, 423-434.
15. Fleming, T. R. (1982). One-sample multiple testing procedure for phase II clinical trials. Biometrics 38, 143-151.
16. Heitjan, D. F. (1997). Bayesian interim analysis of phase II cancer clinical trials. Statistics in Medicine 16, 1791-1802.
17. Hilden, J. (1990). Corrected loss calculation for phase II trials. Reader reaction. Biometrics 46, 535-538.
18. Lewis, R. J. and Berry, D. A. (1994). Group sequential clinical trials: A classical evaluation of Bayesian decision-theoretic designs. Journal of the American Statistical Association 89, 1528-1534.
19. Raiffa, H. and Schlaifer, R. (1961). Applied Statistical Decision Theory, Cambridge, Massachusetts: MIT Press.
20. Rosner, G. L. and Berry, D. A. (1995). A Bayesian group sequential design for a multiple-arm randomized clinical trial. Statistics in Medicine 14, 381-394.
21. Simon, R. (1989). Optimal 2-stage designs for phase-II clinical trials. Controlled Clinical Trials 10, 1-10.
22. Spiegelhalter, D. J. and Freedman, L. S. (1988). Bayesian approaches to clinical trials. In Bayesian Statistics 3, J. M. Bernado, M. H. DeGroot, D. V. Lindley, and A. F. M. Smith (eds), 453-477. Oxford: Oxford University Press.
23. Stallard, N. (1998). Sample size determination for phase II clinical trials based on Bayesian decision theory. Biometrics 54, 279-294.
24. Staquet, M. J. and Sylvester, R. J. (1977). A decision theory approach to phase II clinical trials. Biomedicine 26, 262-266.
25. Sylvester, R. J. (1988). A Bayesian approach to the design of phase II clinical trials. Biometrics 44, 823-836.
26. Thall, P. F. and Simon, R. (1994a). Practical Bayesian guidelines for phase IIB clinical trials. Biometrics 50, 337-349.
27. Thall, P. F. and Simon, R. (1994b). A Bayesian approach to establishing sample size and monitoring criteria for phase II clinical trials. Controlled Clinical Trials 15, 463-481.
28. Thall, P. F., Simon, R., and Estey, E. H. (1995). Bayesian sequential monitoring designs for single-arm clinical trials with multiple outcomes. Statistics in Medicine 14, 357-379.
29. Thall, P. F., Simon, R., and Estey, E. H. (1996). New statistical strategy for monitoring safety and efficacy in single- arm clinical trials. Journal of Clinical Oncology 14, 296-303.
30. Thall, P. F. and Sung, H.-G. (1998). Some extensions and applications of a Bayesian strategy for monitoring multiple outcomes in clinical trials. Statistics in Medicine 17, 1563-1580.
31. Wald, A. (1947). Sequential Analysis. New York: John Wiley.