Flexible design clinical trial methodology in regulatory applications

Statistics in Medicine

Volumn 30, Issue 13, 2011, Pages 1519-1527

  Hung, H M James ^a   Wang, Sue Jane ^a   O'Neill, Robert ^a  

^a CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

Adaptive patient selection; Sample size planning; Sample size reassessment; Statistical bias

Indexed keywords

ADAPTIVE SELECTION DESIGN; ARTICLE; CALCULATION; CLINICAL TRIAL (TOPIC); DRUG EFFICACY; EFFECT SIZE; HEART FAILURE; HUMAN; HYPERTENSION; METHODOLOGY; SAMPLE SIZE PLANNING; SAMPLE SIZE REASSESSMENT; STATISTICAL ANALYSIS; TREATMENT RESPONSE;

ANTIHYPERTENSIVE AGENTS; BIAS (EPIDEMIOLOGY); BLOOD PRESSURE; CLINICAL TRIALS AS TOPIC; HUMANS; HYPERTENSION; PATIENT SELECTION; SAMPLE SIZE; UNITED STATES;

EID: 79956105768     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.4021     Document Type: Article

References (19)

1. Bauer P, Köhne K. Evaluations of experiments with adaptive interim analyses. Biometrics 1994; 50:1029-1041.
2. Hung HMJ, O'Neill R, Wang SJ, Lawrence J. A regulatory view on adaptive/flexible clinical trial design (with Rejoinder). Biometrical Journal 2006; 48:565-573, 613-615. DOI: 10.1002/bimj.200610229, DOI: 10.1002/bimj.200610258.
3. Chow SC, Chang M. Adaptive Design Methods in Clinical Trials. Chapman and Hall/CRC Press, Taylor and Francis: New York, 2006.
4. Hung HMJ, O'Neill RT. Utilities of the P-value distribution associated with effect size in clinical trials. Biometrical Journal 2003; 45:659-669.
5. Burman CF, Sonnesson C. Are flexible designs sound? Biometrics 2006; 62:664-683.
6. Bauer P, König F. The reassessment of trial perspectives from interim data-a critical view. Statistics in Medicine 2006; 25:23-36. DOI: 10.1002/sim.2180.
7. Tsiatis AA, Mehta CR. On the inefficiency of the adaptive design for monitoring clinical trials. Biometrika 2003; 90:367-378.
8. Jennison C, Turnbull BW. Mid-course sample size modification in clinical trials. Statistics in Medicine 2003; 22:971-993. DOI: 10.1002/sim.1457.
9. O'Connor CM, Dunne MW, Pfeffer MA, Muhlestein JB, Yao L, Cupta S, Benner RJ, Fisher MR, Cook TD. Azithromycin for the secondary prevention of coronary heart disease events. The WIZARD Study: a randomized controlled trial. Journal of American Medical Association 2003; 290:1459-1466.
10. Taylor A, Ziesche RN, Yancy C, Carson P, D'Agostino R, Ferdinand K, Taylor M, Adams K, Sobolinski M, Worcel M, Cohn J. Combination of isosorbide dinitrate and hydralazine in blacks with heart failure. New England Journal of Medicine 2004; 351:2049-2057.
11. Hung HMJ, Wang SJ, O'Neill R. Statistical considerations for testing multiple endpoints in group sequential or adaptive clinical trials. Journal of Biopharmaceutical Statistics 2007; 17:1201-1210. DOI: 10.1080/10543400701645405.
12. Chen YHJ, DeMets DL, Lan KKG. Increasing the sample size when the unblinded interim result is promising. Statistics in Medicine 2004; 23:1023-1038. DOI: 10.1002/sim.1688.
13. Posch M, König F, Branson M, Brannah W, Dunger-Baldauf C, Bauer P. Testing and estimation in flexible group sequential design with adaptive treatment selection. Statistics in Medicine 2005; 24:3697-3714. DOI: 10.1002/sim.2389.
14. König F, Brannah W, Bretz F, Posch M. Adaptive Dunnett tests for treatment selection. Statistics in Medicine 2008; 27:1612-1625. DOI: 10.1002/sim.3048.
15. Wang SJ, O'Neill RT, Hung HMJ. Approaches to evaluation of treatment effect in randomized clinical trials with genomic subset. Pharmaceutical Statistics 2007; 6:224-227. DOI: 10.1002/pst.300.
16. Brannath W, Zuber E, Branson M, Bretz F, Gallo P, Posch M, Racine-Poon A. Confirmatory adaptive designs with Bayesian decision tools for a targeted therapy in oncology. Statistics in Medicine 2009; 28:1445-1463. DOI: 10.1002/sim.3559.
17. Wang SJ, Hung HMJ, O'Neill RT. Adaptive patient enrichment designs in therapeutic trials. Biometrical Journal 2009; 51:358-374. DOI: 10.1002/bimj.200900003.
18. 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.
19. Wang SJ, Hung HMJ. Sample size adaptation in combination drug products. Presented in the Statistics Annual Meeting of Drug Information Association and Food and Drug Administration, 14 March 2006.