Adaptive sample size modification in clinical trials: Start small then ask for more?

Statistics in Medicine

Volumn 34, Issue 29, 2015, Pages 3793-3810

  Jennison, Christopher ^a   Turnbull, Bruce W ^b  

^a University of Bath   (United Kingdom)

^b Cornell University   (United States)

Author keywords

Adaptive design; Clinical trial; Group sequential test; Optimal design; Promising zone; Sample size re estimation

Indexed keywords

ADAPTIVE SAMPLE SIZE; ARTICLE; CLINICAL STUDY; CLINICAL TRIAL (TOPIC); ERROR; HUMAN; PHASE 3 CLINICAL TRIAL (TOPIC); PROBABILITY; SAMPLE SIZE; SEQUENTIAL ANALYSIS; STATISTICAL ANALYSIS; STUDY DESIGN; TREATMENT OUTCOME; TREATMENT RESPONSE; METHODOLOGY; PROCEDURES; SCHIZOPHRENIA; STANDARDS; STATISTICAL BIAS; STATISTICS AND NUMERICAL DATA;

NEUROLEPTIC AGENT;

ANTIPSYCHOTIC AGENTS; BIAS (EPIDEMIOLOGY); CLINICAL TRIALS AS TOPIC; CLINICAL TRIALS, PHASE III AS TOPIC; HUMANS; RESEARCH DESIGN; SAMPLE SIZE; SCHIZOPHRENIA;

EID: 84945978676     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.6575     Document Type: Article

References (26)

1. US Food and Drug Administration Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics (draft). FDA: Silver Spring, MD, 2010. (Available at http://www.fda.gov/downloads/Drugs/ GuidanceComplianceRegulatoryInformation/Guidances/UCM201790.pdf), [Accessed 1 May 2015].
2. Bauer P, Kohne K. Evaluation of experiments with adaptive interim analyses. Biometrics 1994; 50: 1029-1041. Correction 52:380.
3. Fisher LD. Self-designing clinical trials. Statistics in Medicine 1998; 17: 1551-1562.
4. Cui L, Hung HMJ, Wang S-J. Modification of sample size in group sequential clinical trials. Biometrics 1999; 55: 853-857.
5. Jennison C, Turnbull BW. Group Sequential Methods with Applications to Clinical Trials. Chapman & Hall/CRC: Boca Raton, 2000.
6. Jennison C, Turnbull BW. Mid-course sample size modification in clinical trials based on the observed treatment effect. Statistics in Medicine 2003; 22: 971-993.
7. Jennison C, Turnbull BW. Adaptive and nonadaptive group sequential tests. Biometrika 2006; 93: 1-21.
8. Jennison C, Turnbull BW. Efficient group sequential designs when there are several effect sizes under consideration. Statistics in Medicine 2006; 25: 917-932.
9. Tsiatis AA, Mehta C. On the inefficiency of the adaptive design for monitoring clinical trials. Biometrika 2003; 90: 367-378.
10. Fleming TR. Standard versus adaptive monitoring procedures: a commentary. Statistics in Medicine 2006; 25: 3305-3312.
11. Lokhnygina Y, Tsiatis AA. Optimal two-stage group-sequential designs. Journal of Statistical Planning and Inference 2008; 138: 489-499.
12. Banerjee A, Tsiatis AA. Adaptive two-stage designs in phase II clinical trials. Statistics in Medicine 2006; 25: 3382-3395.
13. Mehta CR, Pocock SJ. Adaptive increase in sample size when interim results are promising: a practical guide with examples. Statistics in Medicine 2011; 30: 3267-3284.
14. Hampson LV, Jennison C. Group sequential tests for delayed responses (with discussion). Journal of the Royal Statistical Society: Series B 2013; 75: 3-54.
15. Meretoja A, Churilov L, Campbell BC, Aviv RI, Yassi N, Barras C, Mitchell P, Yan B, Nandurkar H, Bladin C, Wijeratne T, Spratt NJ, Jannes J, Sturm J, Rupasinghe J, Zavala J, A Lee, Kleinig T, Markus R, Delcourt C, Mahant N, Parsons MW, Levi C, Anderson CS, Donnan GA, Davis SM. The Spot sign and Tranexamic acid On Preventing ICHgrowth - AUStralasia Trial (STOP-AUST): Protocol of a phase II randomized, placebo-controlled, double-blind, multicenter trial. International Journal of Stroke 2014; 9: 519-524.
16. Chen YHJ, DeMets DL, Lan GKK. Increasing the sample size when the unblinded interim result is promising. Statistics in Medicine 2004; 23: 1023-1038.
17. Glimm E. Comments on 'Adaptive increase in sample size when interim results are promising: a practical guide with examples'. Statistics in Medicine 2012; 31: 98-99.
18. Proschan MA, Hunsberger SA. Designed extension of studies based on conditional power. Biometrics 1995; 51: 1315-1324.
19. Gao P, Ware JH, Mehta C. Sample size re-estimation for adaptive sequential design in clinical trials. Journal of Biopharmaceutical Statistics 2008; 18: 1184-1196.
20. Emerson SS, Levin GP, Emerson SC. Comments on 'Adaptive increase in sample size when interim results are promising: a practical guide with examples'. Statistics in Medicine 2011; 30: 3285-3301.
21. Faldum A, Hommel G. Strategies for including patients recruited during interim analysis of clinical trials. Journal of Biopharmaceutical Statistics 2007; 17: 1211-1225.
22. Fisher RA. Statistical Methods for Research Workers 5th edition. Oliver and Boyd: Edinburgh, 1934.
23. Müller HH, Schäfer H. Adaptive group sequential designs for clinical trials: combining the advantages of adaptive and of classical group sequential approaches. Biometrics 2001; 57: 886-891.
24. Burman CF, Sonesson C. Are flexible designs sound?Biometrics 2006; 62: 664-669.
25. Wang SJ, Brannath W, Brückner M, Hung JHM, Koch A. Unblinded adaptive statistical information design based on clinical endpoint or biomarker. Statistics in Biopharmaceutical Research 2013; 5: 293-310.
26. Golub H, Mehta C, Tsiatis B, Temple R, D'Agostino R, Fleming T, Siegel J, Ellenberg S, Hung J, Chuang-Stein C, et al. Papers from the 2004 Harvard-MIT Division of Health Science and Technology Workshop, 'Adaptive Clinical Trial Designs: Ready for Prime Time?' group discussion. Statistics in Medicine 2006; 25: 3326-3347.