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Volumn 23, Issue 12, 2004, Pages 1921-1986

Tutorial in biostatistics: Sample sizes for clinical trials with Normal data

Author keywords

Baselines; Bioequivalence; Cross over trials; Equivalence trials; Non inferiority trials; Parallel group trials; Power; Precision; Sample size; Superiority trials; Type I error; Type II error

Indexed keywords

DRUG;

EID: 4344584922     PISSN: 02776715     EISSN: None     Source Type: Journal    
DOI: 10.1002/sim.1783     Document Type: Review
Times cited : (476)

References (71)
  • 1
    • 77951529251 scopus 로고
    • Streptomycin treatment of pulmonary tuberculosis
    • Medical Research Council
    • Medical Research Council. Streptomycin treatment of pulmonary tuberculosis. British Medical Journal 1948; 2:769-782.
    • (1948) British Medical Journal , vol.2 , pp. 769-782
  • 3
    • 25544449312 scopus 로고    scopus 로고
    • Statistical principals for clinical trials
    • ICH E9. September 1998 [last accessed 23 October]
    • ICH E9. Statistical principals for clinical trials. September 1998. http://www.fda.gov/cder/guidance/ICH_E9fnl.PDF [last accessed 23 October 2003].
    • (2003)
  • 4
    • 0033128807 scopus 로고    scopus 로고
    • Treatment allocation in controlled trials: Why randomize?
    • Altman DG, Bland JM. Treatment allocation in controlled trials: why randomize? British Medical Journal 1999; 318:1209.
    • (1999) British Medical Journal , vol.318 , pp. 1209
    • Altman, D.G.1    Bland, J.M.2
  • 5
    • 0141854700 scopus 로고    scopus 로고
    • Blinding or masking
    • Armitage P, Colton T (eds). John Wiley and Sons: Chichester
    • Day S. Blinding or masking. In Encyclopedia of Biostatistics, vol. 1, Armitage P, Colton T (eds). John Wiley and Sons: Chichester, 1998; 410-417.
    • (1998) Encyclopedia of Biostatistics , vol.1 , pp. 410-417
    • Day, S.1
  • 6
    • 0018974024 scopus 로고
    • Statistics and ethics in medical research III - How large a sample?
    • Altman DG. Statistics and ethics in medical research III - how large a sample? British Medical Journal 1980; 281:1336-1138.
    • (1980) British Medical Journal , vol.281 , pp. 1138-1336
    • Altman, D.G.1
  • 7
    • 25544471083 scopus 로고    scopus 로고
    • Structure and content of clinical study reports
    • ICH E3. July 1996. [last accessed 23 October]
    • ICH E3. Structure and content of clinical study reports. July 1996. http://www.fda.gov/cder/guidance/iche3.pdf [last accessed 23 October 2003].
    • (2003)
  • 10
    • 0033021536 scopus 로고    scopus 로고
    • Estimating sample sizes for continuous, binary and ordinal outcomes in paired comparisons: Practical hints
    • Julious SA, Campbell MJ, Altman DG. Estimating sample sizes for continuous, binary and ordinal outcomes in paired comparisons: practical hints. Journal of Biopharmaceutical Statistics 1999; 9(2):241-251.
    • (1999) Journal of Biopharmaceutical Statistics , vol.9 , Issue.2 , pp. 241-251
    • Julious, S.A.1    Campbell, M.J.2    Altman, D.G.3
  • 11
    • 0028839815 scopus 로고
    • Estimating sample sizes for binary, ordered categorical, and continuous outcomes in two group comparisons
    • Campbell MJ, Julious SA, Altman DG. Estimating sample sizes for binary, ordered categorical, and continuous outcomes in two group comparisons. British Medical Journal 1995; 311:1145-1148.
    • (1995) British Medical Journal , vol.311 , pp. 1145-1148
    • Campbell, M.J.1    Julious, S.A.2    Altman, D.G.3
  • 12
    • 0021632262 scopus 로고
    • Approaches to sample size estimation in the design of clinical trials - A review
    • Donner A. Approaches to sample size estimation in the design of clinical trials - a review. Statistics in Medicine 1983; 3:199 214.
    • (1983) Statistics in Medicine , vol.3 , pp. 199-214
    • Donner, A.1
  • 14
    • 0029930697 scopus 로고    scopus 로고
    • Statistical considerations for multiplicity in confirmatory trials
    • Koch GG, Gansky SA. Statistical considerations for multiplicity in confirmatory trials. Drug Information Journal 1996; 30:523-534.
    • (1996) Drug Information Journal , vol.30 , pp. 523-534
    • Koch, G.G.1    Gansky, S.A.2
  • 15
    • 25544455399 scopus 로고    scopus 로고
    • Points to consider on multiplicity issues in clinical trials
    • CPMP. (CPMP/EWP/908/99) 19 September 2002 (last accessed 21 October)
    • CPMP. Points to consider on multiplicity issues in clinical trials. (CPMP/EWP/908/99) 19 September 2002 http://www.cmea.cu.int/pdfs/human/ewp/090899cn.pdf (last accessed 21 October 2003).
    • (2003)
  • 16
    • 0026543566 scopus 로고
    • Interim analyses for monitoring clinical trials that do not materially effecct the type I error rate
    • Gould AL. Interim analyses for monitoring clinical trials that do not materially effecct the type I error rate. Statistics in Medicine 1992; 11:55-66.
    • (1992) Statistics in Medicine , vol.11 , pp. 55-66
    • Gould, A.L.1
  • 17
    • 0029001316 scopus 로고
    • Planning and revising the sample size for a trial
    • Gould AL. Planning and revising the sample size for a trial. Statistics in Medicine 1995; 14:1039-1051.
    • (1995) Statistics in Medicine , vol.14 , pp. 1039-1051
    • Gould, A.L.1
  • 18
    • 0036713341 scopus 로고    scopus 로고
    • Sample size re-determination for repeated measures studies
    • Zucker DM, Denne J. Sample size re-determination for repeated measures studies. Biometri 2002; 48(3): 548-559.
    • (2002) Biometri , vol.48 , Issue.3 , pp. 548-559
    • Zucker, D.M.1    Denne, J.2
  • 20
    • 0000065971 scopus 로고
    • Sample size re-estimation without unblinding for normally distributed data with unknown variance
    • Gould AL, Shih WJ. Sample size re-estimation without unblinding for normally distributed data with unknown variance. Communications in Statistics Theory and Methods 1992; 21:2833-2853.
    • (1992) Communications in Statistics Theory and Methods , vol.21 , pp. 2833-2853
    • Gould, A.L.1    Shih, W.J.2
  • 21
    • 0032518741 scopus 로고    scopus 로고
    • Modifying the design of ongoing trials without unblinding
    • Gould AL, Shih WJ. Modifying the design of ongoing trials without unblinding. Statistics in Medicine 1998; 17:89-100.
    • (1998) Statistics in Medicine , vol.17 , pp. 89-100
    • Gould, A.L.1    Shih, W.J.2
  • 22
    • 0028595559 scopus 로고
    • Internal Pilot studies for estimating sample size
    • Birkett MA, Day SJ. Internal Pilot studies for estimating sample size. Statistics in Medicine 1994; 13: 2455-2403.
    • (1994) Statistics in Medicine , vol.13 , pp. 2403-2455
    • Birkett, M.A.1    Day, S.J.2
  • 23
    • 0034656288 scopus 로고    scopus 로고
    • Re-calculating the sample size in internal pilot study designs with control of the type I error rate
    • Kieser M, Friede T. Re-calculating the sample size in internal pilot study designs with control of the type I error rate. Statistics in Medicine 2000; 19:901-911.
    • (2000) Statistics in Medicine , vol.19 , pp. 901-911
    • Kieser, M.1    Friede, T.2
  • 24
    • 0024994537 scopus 로고
    • The role of internal pilot studies in increasing the efficacy of clinical trials
    • Wittes J, Brittain E. The role of internal pilot studies in increasing the efficacy of clinical trials. Statistics in Medicine 1990; 9:65-72.
    • (1990) Statistics in Medicine , vol.9 , pp. 65-72
    • Wittes, J.1    Brittain, E.2
  • 25
    • 0000992663 scopus 로고
    • On the use and interpretation of test criteria
    • Neyman J, Pearson ES. On the use and interpretation of test criteria. Biometrika 1928: 20(A):175-194.
    • (1928) Biometrika , vol.20 , Issue.A , pp. 175-194
    • Neyman, J.1    Pearson, E.S.2
  • 27
    • 84952965566 scopus 로고
    • The testing of statistical hypotheses in relation to the probabilities a priori
    • Neyman J, Pearson ES. The testing of statistical hypotheses in relation to the probabilities a priori. Proceeds of the Cambridge Philosophical Society 1933; 29:492-510.
    • (1933) Proceeds of the Cambridge Philosophical Society , vol.29 , pp. 492-510
    • Neyman, J.1    Pearson, E.S.2
  • 31
    • 0029071491 scopus 로고
    • Sample size: How many patients are necessary?
    • Fayers P, Machin D. Sample size: how many patients are necessary? British Journal of Cancer 1995; 72:1-9.
    • (1995) British Journal of Cancer , vol.72 , pp. 1-9
    • Fayers, P.1    Machin, D.2
  • 33
    • 0033555868 scopus 로고    scopus 로고
    • Sample size formula with a continuous outcome for unequal group sizes and unequal variances
    • Schouten HJA. Sample size formula with a continuous outcome for unequal group sizes and unequal variances. Statistics in Medicine 1999; 18:87-91.
    • (1999) Statistics in Medicine , vol.18 , pp. 87-91
    • Schouten, H.J.A.1
  • 34
    • 0035044593 scopus 로고    scopus 로고
    • A simple procedure to compute sample size needed to compared two independent groups when the population variances are unknown
    • Singer J. A simple procedure to compute sample size needed to compared two independent groups when the population variances are unknown. Statistics in Medicine 2001; 20:1995.
    • (2001) Statistics in Medicine , vol.20 , pp. 1995
    • Singer, J.1
  • 37
    • 0036452029 scopus 로고    scopus 로고
    • A note on sample size calculations for mean comparisons based on noncentral t-statistics
    • Chow SC, Shao J, Wang H. A note on sample size calculations for mean comparisons based on noncentral t-statistics. Journal of Pharmaceutical Statistics 2002; 12:441-456.
    • (2002) Journal of Pharmaceutical Statistics , vol.12 , pp. 441-456
    • Chow, S.C.1    Shao, J.2    Wang, H.3
  • 38
    • 0000985097 scopus 로고
    • Sample size formulas for normal theory t-tests
    • Guenther WC. Sample size formulas for normal theory t-tests. The American Statistician 1981; 35:243-244.
    • (1981) The American Statistician , vol.35 , pp. 243-244
    • Guenther, W.C.1
  • 39
    • 0043005476 scopus 로고    scopus 로고
    • Bioequivalence trials, intersection-union tests and equivalence confidence sets
    • Berger RL, Hsu, JC. Bioequivalence trials, intersection-union tests and equivalence confidence sets. Statistical Science 1996; 11:283-319.
    • (1996) Statistical Science , vol.11 , pp. 283-319
    • Berger, R.L.1    Hsu, J.C.2
  • 41
    • 25544432142 scopus 로고    scopus 로고
    • Choice of control group in clinical trials
    • ICH E10 2000. May 2001 (last accessed 10 November)
    • ICH E10 Choice of control group in clinical trials, 2000. May 2001 http://www.fda.gov/cder/guidance/4155fnl.pdf (last accessed 10 November 2003).
    • (2003)
  • 42
    • 25544452266 scopus 로고    scopus 로고
    • Points to consider on switching between superiority and non-inferiority
    • CPMP. (CPMP/EWP/482/99) 17 July 2000. (last accessed 21 October)
    • CPMP. Points to consider on switching between superiority and non-inferiority. (CPMP/EWP/482/99) 17 July 2000. http://www.emea.eu.int/pdfs/human/ewp/048299en.pdf (last accessed 21 October 2003).
    • (2003)
  • 43
    • 2942701201 scopus 로고    scopus 로고
    • Concept paper on the development of a committee for proprietary medicinal products (CPMP) points to consider on biostatistical methodological issues arising from recent CPMP discussions on licensing applications: Choice of delta
    • CPMP. (CPMP/EWP/2158/99) 23 September 1999. (last accessed 21 October)
    • CPMP. Concept paper on the development of a committee for proprietary medicinal products (CPMP) points to consider on biostatistical methodological issues arising from recent CPMP discussions on licensing applications: choice of delta. (CPMP/EWP/2158/99) 23 September 1999. http://www.emea.eu.int/pdfs/human/ewp/215899en.pdf (last accessed 21 October 2003).
    • (2003)
  • 44
    • 12244264188 scopus 로고    scopus 로고
    • Some fundamental issues with non-inferiority testing in active controlled trials
    • Hung HMJ, Wang SJ, Lawrence J, O'Neil RT. Some fundamental issues with non-inferiority testing in active controlled trials. Statistics in Medicine 2003; 22:213-225.
    • (2003) Statistics in Medicine , vol.22 , pp. 213-225
    • Hung, H.M.J.1    Wang, S.J.2    Lawrence, J.3    O'Neil, R.T.4
  • 45
    • 0037472837 scopus 로고    scopus 로고
    • Non-inferiority trials: Design concepts and issues - The encounters of academic consultants in statistics
    • D'Agostino RB, Massaro J, Sullivan LM. Non-inferiority trials: design concepts and issues - the encounters of academic consultants in statistics. Statistics in Medicine 2003; 22:169-186.
    • (2003) Statistics in Medicine , vol.22 , pp. 169-186
    • D'Agostino, R.B.1    Massaro, J.2    Sullivan, L.M.3
  • 46
    • 0036169283 scopus 로고    scopus 로고
    • Choosing an equivalence limit for noninferiority and or equivalence studies
    • Wiens BL. Choosing an equivalence limit for noninferiority and or equivalence studies. Controlled Clinical Trials 2002; 23:2-14.
    • (2002) Controlled Clinical Trials , vol.23 , pp. 2-14
    • Wiens, B.L.1
  • 47
    • 0006075504 scopus 로고
    • A special case of a bivariate non-central t-distribution
    • Owen DB. A special case of a bivariate non-central t-distribution. Biometrika 1965; 52:437-446.
    • (1965) Biometrika , vol.52 , pp. 437-446
    • Owen, D.B.1
  • 49
    • 0028807804 scopus 로고
    • A useful testing strategy in phase III trials: Combined test of superiority and test of equivalence
    • Morikawa T, Yoshida M. A useful testing strategy in phase III trials: combined test of superiority and test of equivalence. Journal of Biopharmaceutical Statistics 1995; 5(3):297-306.
    • (1995) Journal of Biopharmaceutical Statistics , vol.5 , Issue.3 , pp. 297-306
    • Morikawa, T.1    Yoshida, M.2
  • 50
    • 0032508256 scopus 로고    scopus 로고
    • In the blood: Proposed new requirements for the registering of generic drugs
    • Senn S. In the blood: proposed new requirements for the registering of generic drugs. The Lancet 1998; 352:85-86.
    • (1998) The Lancet , vol.352 , pp. 85-86
    • Senn, S.1
  • 51
    • 0003455042 scopus 로고    scopus 로고
    • Guidance for industry. Bioavailability and bioequivalence studies for orally administered drug products - General considerations
    • FDA. October 2000. (last accessed 23 October)
    • FDA. Guidance for industry. Bioavailability and bioequivalence studies for orally administered drug products - general considerations. October 2000. http://www.fda.gov/eder/guidance/3615fnl.pdf (last accessed 23 October 2003).
    • (2003)
  • 52
    • 25544449552 scopus 로고    scopus 로고
    • Statistical approaches to establishing bioequivalence
    • FDA. January 2001. (last accessed 23 October)
    • FDA. Statistical approaches to establishing bioequivalence. January 2001. http://www.fda.gov/cder/guidance/3616fnl.pdf (last accessed 23 October 2003).
    • (2003)
  • 53
    • 33646248680 scopus 로고    scopus 로고
    • Note for guidance on the investigation of bioavailability and bioequivalence
    • CPMP. (CPMP/EWP/QWP1401/98) 26 July 2001. (last accessed 21 October)
    • CPMP. Note for guidance on the investigation of bioavailability and bioequivalence. (CPMP/EWP/QWP1401/98) 26 July 2001. http://www.emea.cu.int/pdf/human/ewp/140198en.pdf (last accessed 21 October 2003).
    • (2003)
  • 54
    • 0242350785 scopus 로고    scopus 로고
    • Guidance for industry. Food-effect bioavailability and fed bioequivalence studies
    • FDA. December 2000 (last accessed 23 October 03)
    • FDA. Guidance for industry. Food-effect bioavailability and fed bioequivalence studies. December 2000. http://www.fda.gov/cder/guidance/5194fnl.pdf (last accessed 23 October 03).
  • 55
    • 25544438880 scopus 로고    scopus 로고
    • Note for guidance on the investigation of drug interactions
    • CPMP. (CPMP/EWP/560/95) 17 December 1997. (last accessed 21 October)
    • CPMP. Note for guidance on the investigation of drug interactions. (CPMP/EWP/560/95) 17 December 1997. http://www.emea.cu.int/pdfs/human/ewp/056095en.pdf (last accessed 21 October 2003).
    • (2003)
  • 56
    • 2942747527 scopus 로고    scopus 로고
    • Guidance for industry. In vivo drug metabolism/drug interaction studies - Study design, data analysis, and recommendations for dosing and labelling
    • FDA. November 1999. (last accessed 23 October)
    • FDA. Guidance for industry. In vivo drug metabolism/drug interaction studies - study design, data analysis, and recommendations for dosing and labelling. November 1999. http://www.fda.gov/cder/guidance/2635fnl.pdf (last accessed 23 October 2003).
    • (2003)
  • 57
    • 0003556717 scopus 로고    scopus 로고
    • Guidance for industry. Pharmacokinetics in patients with impaired renal function - Study design, data analysis and impact on dosing and labelling
    • FDA. May 1998. (last accessed 23 October)
    • FDA. Guidance for industry. Pharmacokinetics in patients with impaired renal function - study design, data analysis and impact on dosing and labelling. May 1998. http://www.fda.gov/cder/guidance/1449fnl.pdf pdf (last accessed 23 October 2003).
    • (2003)
  • 58
    • 0003556717 scopus 로고    scopus 로고
    • Guidance for industry. Pharmacokinetics in patients with impaired hepatic function: Study design, data analysis and impact on dosing and labelling
    • FDA. May 2003. (last accessed 23 October)
    • FDA. Guidance for industry. Pharmacokinetics in patients with impaired hepatic function: study design, data analysis and impact on dosing and labelling. May 2003. http://www.fda.gov/cder/guidance/3625fnl.pdf (last accessed 23 October 2003).
    • (2003)
  • 60
    • 0030949860 scopus 로고    scopus 로고
    • Common non-compartmental pharmacokinetic variables: Are they normally or log-normally distributed?
    • Lacey JM, Keene ON, Pritchard JF, Bye A. Common non-compartmental pharmacokinetic variables: are they normally or log-normally distributed? Journal of Biopharmaceutical Statistics 1997; 7(1):171-178.
    • (1997) Journal of Biopharmaceutical Statistics , vol.7 , Issue.1 , pp. 171-178
    • Lacey, J.M.1    Keene, O.N.2    Pritchard, J.F.3    Bye, A.4
  • 61
    • 0029028391 scopus 로고
    • Use of the repeated cross-over designs in assessing bioequivalence
    • Liu JP. Use of the repeated cross-over designs in assessing bioequivalence. Statistics in Medicine 1995; 14:1067-1078.
    • (1995) Statistics in Medicine , vol.14 , pp. 1067-1078
    • Liu, J.P.1
  • 62
    • 0026009636 scopus 로고
    • A general parametric approach to the meta-analysis of randomized trials
    • Whitehead A, Whitehead J. A general parametric approach to the meta-analysis of randomized trials. Statistics in Medicine 1991; 10:1665-1677.
    • (1991) Statistics in Medicine , vol.10 , pp. 1665-1677
    • Whitehead, A.1    Whitehead, J.2
  • 65
    • 2942749383 scopus 로고
    • Clinical trial numbers and confidence intervals of pre-specified size
    • Day S. Clinical trial numbers and confidence intervals of pre-specified size. The Lancet 1988; 17:1427.
    • (1988) The Lancet , vol.17 , pp. 1427
    • Day, S.1
  • 68
    • 1442271816 scopus 로고    scopus 로고
    • Points to consider on adjustment for baseline covariates
    • CPMP. (CPMP/EWP/2863/99) 23 May 2003. (last accessed 21 October)
    • CPMP. Points to consider on adjustment for baseline covariates. (CPMP/EWP/2863/99) 23 May 2003. http://www.emea.eu.int/pdfs/human/ewp/286399en.pdf (last accessed 21 October 2003).
    • (2003)
  • 69
    • 0026445783 scopus 로고
    • Repeated measures in clinical trials: Analysis using mean summary statistics and its implication for design
    • Frison LJ, Pocock SJ. Repeated measures in clinical trials: analysis using mean summary statistics and its implication for design. Statistics in Medicine 1992; 11:1685-1704.
    • (1992) Statistics in Medicine , vol.11 , pp. 1685-1704
    • Frison, L.J.1    Pocock, S.J.2
  • 71
    • 0034736416 scopus 로고    scopus 로고
    • Repeated measures in clinical trials: Analysis using means summary statistics and its implications for design
    • Julious SA. Repeated measures in clinical trials: analysis using means summary statistics and its implications for design. Statistics in Medicine 2000; 19:3133-3135.
    • (2000) Statistics in Medicine , vol.19 , pp. 3133-3135
    • Julious, S.A.1


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