Adaptive increase in sample size when interim results are promising: A practical guide with examples

Statistics in Medicine

Volumn 30, Issue 28, 2011, Pages 3267-3284

  Mehta, Cyrus R ^a,b   Pocock, Stuart J ^c  

^a Cytel Corporation   (United States)

^b HARVARD SCHOOL OF PUBLIC HEALTH   (United States)

^c LONDON SCHOOL OF HYGIENE AND TROPICAL MEDICINE   (United Kingdom)

Author keywords

Adaptive design; Conditional power; Flexible clinical trials; Real examples; Sample size re estimation; Two stage designs

Indexed keywords

ADAPTIVE SAMPLE SIZE REESTIMATION; ANALYTICAL ERROR; ARTICLE; BINOMIAL DISTRIBUTION; CASE STUDY; CONTROLLED STUDY; INFERENTIAL STATISTICS; INTERMETHOD COMPARISON; MATHEMATICAL COMPUTING; MATHEMATICAL VARIABLE; NULL HYPOTHESIS; PHASE 3 CLINICAL TRIAL (TOPIC); PREDICTIVE VALUE; PROBABILITY; QUANTITATIVE ANALYSIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); RECEIVER OPERATING CHARACTERISTIC; REPRODUCIBILITY; RISK REDUCTION; SAMPLE SIZE; SEQUENTIAL ANALYSIS;

ACUTE CORONARY SYNDROME; ALGORITHMS; BIAS (EPIDEMIOLOGY); CLINICAL TRIALS, PHASE III AS TOPIC; COMPUTER SIMULATION; ENDPOINT DETERMINATION; EPIDEMIOLOGIC RESEARCH DESIGN; HUMANS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SAMPLE SIZE; SCHIZOPHRENIA; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 82055200248     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.4102     Document Type: Article

References (23)

1. Mehta CR, Tsiatis AA. Flexible sample size considerations using information-based interim monitoring. Drug Information Journal 2001; 35:1095-1112.
2. Bauer P, Kohne K. Evaluation of experiments with adaptive interim analyses. Biometrics 1994; 50:1029-1041.
3. Lehmacher W, Wassmer G. Adaptive sample size calculations in group sequential trials. Biometrics 1999; 55:1286-1290.
4. Cui L, Hung HM, Wang SJ. Modification of sample size in group sequential clinical trials. Biometrics 1999; 55:853-857.
5. Muller HH, Schafer H. Adaptive group sequential designs for clinical trials: combining the advantages of adaptive and of classical group sequential approaches. Biometrics 2001; 57:886-891.
6. Chen YH, DeMets DL, Lan KK. Increasing the sample size when the unblinded interim result is promising. Statistics in Medicine 2004; 23:1023-1038.
7. 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.
8. Posch M, Bauer P, Brannath W. Issues in designing flexible trials. Statistics in Medicine 2003; 22:953-969.
9. Brannath W, Koenig F, Bauer P. Multiplicity and flexibility in clinical trials. Pharmaceutical Statistics 2007; 6:205-216.
10. Proschan MA, Hunsberger SA. Designed extension of studies based on conditional power. Biometrics 1995; 51:1315-1324.
11. Bauer P, Koenig F. The reassessment of trial perspectives from interim data-a critical view. Statistics in Medicine 2006; 25:23-36.
12. Jennison C, Turnbull BW. Group Sequential Methods with Applications to Clinical Trials. Chapman & Hall/CRC: NY London, 2000.
13. Hwang IK, Shih WJ, Decani JS. Group sequential designs using a family of type 1 error spending functions. Statistics in Medicine 1990; 9:1439-1445.
14. Pocock SJ. When (not) to stop a clinical trial for benefit. JAMA 2005; 294(17).
15. O'Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics 1979; 35:549-556.
16. Mehta C, Gao P, Bhatt DL, Harrington RA, Skerjanec S, Ware JH. Optimizing trial design: sequential, adaptive, and enrichment strategies. Circulation 2009; 119:597-605.
17. Lan KKG, Demets DL. Discrete sequential boundaries for clinical trials. Biometrika 1983; 70:659-663.
18. FDA. Guidance for industry: adaptive design clinical trials for drugs and biologics, 2010.
19. PhRMA. White paper of the pharma adaptive working group. DIA Journal 2007.
20. Tsiatis A, Mehta C. On the inefficiency of the adaptive design for monitoring clinical trials. Biometrika 2003; 90:367-378.
21. Jennison C, Turnbull BW. Mid-course sample size modification in clinical trials based on the observed treatment effect. Statistics of Medicine 2003; 22:971-993.
22. Mehta CR, Bauer P, Posch M, Brannath W. Repeated confidence intervals for adaptive group sequential trials. Statistics in Medicine 2007; 26:5422-5433.
23. Brannath W, Mehta CR, Posch M. Exact confidence bounds following adaptive group sequential tests. Biometrics 2009; 65:539-546.