Interval estimation in two-stage, drop-the-losers clinical trials with flexible treatment selection

Statistics in Medicine

Volumn 30, Issue 23, 2011, Pages 2804-2814

  Neal, Dan ^a   Casella, George ^b   Yang, Mark C K ^b   Wu, Samuel S ^a  

^a UNIVERSITY OF FLORIDA   (United States)

^b University of Florida   (United States)

Author keywords

Clinical trial; Drop the losers design; Flexible selection; p value combination; Two stage test

Indexed keywords

ARTICLE; CLINICAL TRIAL (TOPIC); CONFIDENCE INTERVAL; DRUG CHOICE; DRUG EFFICACY; DRUG SAFETY; HUMAN; LOWER CONFIDENCE LIMIT; MATHEMATICAL ANALYSIS; MATHEMATICAL COMPUTING; PATIENT SELECTION; PROCESS DESIGN; PROCESS DEVELOPMENT; SAMPLE SIZE; SIMULATION; STATISTICAL ANALYSIS; STATISTICAL PARAMETERS; TREATMENT OUTCOME; TREATMENT RESPONSE;

CLINICAL TRIALS AS TOPIC; COMPUTER SIMULATION; CONFIDENCE INTERVALS; HUMANS; RESEARCH DESIGN;

EID: 80053179167     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.4308     Document Type: Article

References (26)

1. Staff Report. Study shows clinical trials last longer than expected. Clinical Trials Week, 4 October 2004, p. 20.
2. Thall PF, Simon R, Ellenberg SS. Two-stage selection and testing designs for comparative clinical trials. Biometrika. 1988; 75:303-310.
3. Bischoff W, Miller F. Adaptive two-stage procedures to find the best treatment in clinical trials. Biometrika 2005; 92:197-212.
4. Sampson AR, Sill MW. Drop-the-losers design: normal case. Biometrical Journal 2005; 47:257-268.
5. Wang J. An adaptive two-stage design with treatment selection using the conditional error function approach. Biometrical Journal 2006; 48:679-689.
6. Wu SS, Sun S, Yang G, Yang MCK. Least favorable configuration for two-stage clinical trials, Technical Report 2008-23, Department of Statistics, University of Florida, Gainesville, Florida, USA, 2008.
7. Bauer P, Köhne K. Evaluation of experiments with adaptive interim analyses. Biometrics 1994; 50:1029-1041.
8. Bretz F, Schmidli H, Köhne K, Racine A, Maurer W. Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: general concepts. Biometrical Journal 2006; 48:623-634.
9. Chang M. Adaptive design method based on sum of p-values. Statistics in Medicine 2007; 26:2772-2784.
10. Lehmacher W, Wassmer G. Adaptive sample size calculation in group sequential trials. Biometrics 1999; 55:1286-1290.
11. Tsiatis AA, Mehta C. On the inefficiency of the adaptive design for monitoring clinical trials. Biometrika 2003; 90:367-378.
12. Jennison C, Turnbull BW. Adaptive and nonadaptive group sequential tests. Biometrika 2006; 93:1-21.
13. Stallard N, Todd S. Point estimates and confidence regions for sequential trials involving selection. Journal of Planning and Statistical Inference 2005; 135:402-419.
14. Wu SS, Wang W, Yang MCK. Interval estimation for drop-the-losers designs. Biometrika 2010; 97:405-418.
15. Liu Q, Pledger GW. Phase 2 and 3 combination designs to accelerate drug development. Journal of the American Statistical Association 2005; 100:493-502.
16. Conaway MR, Petroni GR. Designs for phase II trials allowing for a trade-off between response and toxicity. Biometrics 1996; 52:1275-1286.
17. Kieser M. Discussion of the paper by Sampson and Sill. Biometrical Journal 2005; 47:271-273.
18. Kelly PJ, Stallard N, Todd S. An adaptive group sequential design for phase II/III clinical trials that select a single treatment from several. Journal of Biopharmaceutical Statistics 2005; 15:641-658.
19. Stallard N, Friede T. A group-sequential design for clinical trials with treatment selection. Statistics in Medicine 2008; 27:620-627.
20. Thall PF. Some geometric methods for constructing decision criteria based on two-dimensional parameters. Journal of Statistical Planning and Inference 2008; 138:516-527.
21. Chen YY, Smith BJ. Adaptive group sequential design for phase II clinical trials: a Bayesian decision theoretic approach. Statistics in Medicine 2009; 28:3347-3362.
22. Köenig F, Brannath W, Bretz F, Posch M. Adaptive Dunnett tests for treatment selection. Statistics in Medicine 2008; 27:1612-1625.
23. Brannath W, Köenig F, Bauer P. Estimation in flexible two stage designs. Statistics in Medicine 2006; 25:3366-3381.
24. Posch M, Köenig F, Branson M, Brannath W, Dunger-Baldauf C, Bauer P. Testing and estimation in flexible group sequential designs with adaptive treatment selection. Statistics in Medicine 2005; 24:3697-3714.
25. Bretz F, Köenig F, Brannath W, Glimm E, Posch M. Adaptive designs for confirmatory clinical trials. Statistics in Medicine 2009; 28:1181-1217.
26. Li G, Zhu J, Ouyang P, Xie J, Deng L, Law G. Adaptive design for interim dose selection. Statistics in Biopharmaceutical Research 2009; 1:366-376.