Minimization of sample size when comparing two small probabilities in a non-inferiority safety trial

Statistics in Medicine

Volumn 23, Issue 11, 2004, Pages 1683-1699

  de Boo, Theo M ^a   Zielhuis, Gerhard A ^a  

^a RADBOUD UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

Binomial probabilities; ML methods; Non inferiority; Safety; Sample size

Indexed keywords

ARTICLE; CALCULATION; CLINICAL RESEARCH; COMPARATIVE STUDY; HYPOTHESIS; MAXIMUM LIKELIHOOD METHOD; PROBABILITY; RISK ASSESSMENT; SAFETY; SAMPLE SIZE; STATISTICAL PARAMETERS; TREATMENT OUTCOME; VALIDATION PROCESS;

CLINICAL TRIALS, PHASE II; DRUG EVALUATION; HUMANS; RESEARCH DESIGN; SAMPLE SIZE;

EID: 2942659222     PISSN: 02776715     EISSN: None     Source Type: Journal    
DOI: 10.1002/sim.1760     Document Type: Article

References (13)

1. Fleming RF. One-sample multiple testing procedure for phase II clinical trials. Biometrics 1982; 38:143-151.
2. A'Hern RP. Sample size tables for exact single-stage phase II designs. Statistics in Medicine 2001; 20: 859-866.
3. Simon R. Optimal two-stage designs for phase II clinical trials. Controlled Clinical Trials 1989; 10:1-10.
4. Chen TT. Optimal three-stage designs for phase II cancer clinical trials. Statistics in Medicine 1997; 16: 2701-2711.
5. Roebruck P, Kühn A. Comparison of tests and sample size formulas for proving therapeutic equivalence based on the difference of binomial probabilities. Statistics in Medicine 1995; 14:1583-1594.
6. Miettinen OS, Nurminen M. Comparative analysis of two rates. Statistics in Medicine 1985; 4:213-226.
7. Farrington CP, Manning G. Test statistics and sample size formulas for comparative binomial trials with null hypothesis of non-zero difference or non-unity relative risk. Statistics in Medicine 1990; 9:1447-1454.
8. Blackwelder WC. Proving the null hypothesis in clinical trials. Controlled Clinical Trials 1982; 3:345-353.
9. Makuch R, Simon R. Sample size requirements for evaluating a conservative therapy. Cancer Treatment Reports 1978; 62:1037-1040.
10. Brittain E, Schlesselman JJ. Optimal allocation for the comparison of proportions. Biometrics 1982; 38: 1003-1009.
11. Chan ISF. Exact tests of equivalence and efficacy with a non-zero lower bound for comparative studies. Statistics in Medicine 1998; 17:1403-1413.
12. Kang S-H, Chen JJ. An approximate unconditional test of non-inferiority between two proportions. Statistics in Medicine 2000; 19:2089-2100.
13. Fleiss JL. Statistical Methods for Rates and Proportions. Wiley: New York, 1981; 44-46.