Sample sizes for clinical trials with Normal data

Statistics in Medicine - Tập 23 Số 12 - Trang 1921-1986 - 2004
Steven A. Julious1
1Institute of General Practice and Community Care, Community Science Centre, Northern General Hospital, Herries Road, Sheffield, S5 7AU, U.K.

Tóm tắt

AbstractThis article gives an overview of sample size calculations for parallel group and cross‐over studies with Normal data. Sample size derivation is given for trials where the objective is to demonstrate: superiority, equivalence, non‐inferiority, bioequivalence and estimation to a given precision, for different types I and II errors. It is demonstrated how the different trial objectives influence the null and alternative hypotheses of the trials and how these hypotheses influence the calculations. Sample size tables for the different types of trials and worked examples are given. Copyright © 2004 John Wiley & Sons, Ltd.

Từ khóa


Tài liệu tham khảo

10.1136/bmj.2.4582.769

10.1002/pst.6

ICH E9. Statistical principals for clinical trials. September1998.http://www.fda.gov/cder/guidance/ICH_E9‐fnl.PDF[last accessed 23 October 2003].

Altman DG, 1999, Treatment allocation in controlled trials: why randomize?, British Medical Journal, 318, 1209, 10.1136/bmj.318.7192.1209

Day S, 1998, Encyclopedia of Biostatistics, 410

10.1136/bmj.281.6251.1336

ICH E3. Structure and content of clinical study reports. July1996.http://www.fda.gov/cder/guidance/iche3.pdf[last accessed 23 October 2003].

HintzJL. PASS 2000 User's Guide. Kaysville 2000.

ElashoffJD. nQuery advisor version 4 user's guide. Los Angeles 2000.

10.1081/BIP-100101174

10.1136/bmj.311.7013.1145

10.1002/sim.4780030302

Machin D, 1997, Statistical Tables for the Design of Clinical Studies

10.1177/009286159603000228

CPMP. Points to consider on multiplicity issues in clinical trials. (CPMP/EWP/908/99) 19 September 2002http://www.emea.eu.int/pdfs/human/ewp/090899en.pdf(last accessed 21 October 2003 ).

10.1002/sim.4780110107

10.1002/sim.4780140922

10.1111/j.0006-341X.2002.00548.x

10.1002/(SICI)1097-0258(19991230)18:24<3493::AID-SIM302>3.0.CO;2-2

10.1080/03610929208830947

10.1002/(SICI)1097-0258(19980115)17:1<89::AID-SIM730>3.0.CO;2-D

10.1002/sim.4780132309

10.1002/(SICI)1097-0258(20000415)19:7<901::AID-SIM405>3.0.CO;2-L

10.1002/sim.4780090113

Neyman J, 1928, On the use and interpretation of test criteria, Biometrika, 20, 175

Neyman J, 1933, On the problem of the most efficient tests of statistical hypotheses, Philosophical Transitions Royal Society (London), 23, 289

10.1017/S030500410001152X

Neyman J, 1936, Contributions to the theory of testing hypotheses, Journal Statistical Research Memoirs (University of London), 1, 1

Neyman J, 1936, Sufficient statistics and uniformly most powerful test of statistical hypothesis, Journal Statistical Research Memoirs (University of London), 1, 113

Neyman J, 1938, Contributions to the theory of testing statistical hypotheses, Journal Statistical Research Memoirs (University of London), 2, 25

10.1038/bjc.1995.268

Armitage P, Statistical Methods in Medical Research, 10.1002/9780470773666

10.1002/(SICI)1097-0258(19990115)18:1<87::AID-SIM958>3.0.CO;2-K

10.1002/sim.722

BrushGG. How to choose the proper sample size vol. 12 The ASQC Basic References in Quality Control Statistical Techniques American Society for Quality Control: Milwaukee WI 1988.

Senn S, 1993, Cross‐over Trials in Clinical Research

Chow SC, 2002, A note on sample size calculations for mean comparisons based on noncentral t‐statistics, Journal of Pharmaceutical Statistics, 12, 441

10.2307/2683297

10.1214/ss/1032280304

10.1136/bmj.313.7048.36

ICH E10 Choice of control group in clinical trials 2000. May2001http://www.fda.gov/cder/guidance/4155fnl.pdf(last accessed 10 November 2003).

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).

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 September1999.http://www.emea.eu.int/ pdfs/human/ewp/215899en.pdf(last accessed 21 October 2003).

10.1002/sim.1315

10.1002/sim.1425

10.1016/S0197-2456(01)00196-9

10.1093/biomet/52.3-4.437

Diletti E, 1991, Sample size determination form bioequivalence assessment by means of confidence intervals, International Journal of Clinical Pharmacology, Therapy and Toxicology, 29, 1

10.1080/10543409508835115

10.1016/S0140-6736(98)85007-1

FDA. Guidance for industry. Bioavailability and bioequivalence studies for orally administered drug products—general considerations. October2000.http://www.fda.gov/cder/guidance/3615fnl.pdf(last accessed 23 October 2003).

FDA. Statistical approaches to establishing bioequivalence. January2001.http://www.fda.gov/cder/guidance/3616fnl.pdf(last accessed 23 October 2003).

CPMP. Note for guidance on the investigation of bioavailability and bioequivalence. (CPMP/EWP/QWP1401/98) 26 July2001.http://www.emea.eu.int/pdfs/human/ewp/140198en.pdf(last accessed 21 October 2003).

FDA. Guidance for industry. Food‐effect bioavailability and fed bioequivalence studies. December2000.http://www.fda.gov/cder/guidance/5194fnl.pdf(last accessed 23 October 03).

CPMP. Note for guidance on the investigation of drug interactions. (CPMP/EWP/560/95) 17 December1997.http://www.emea.eu.int/pdfs/human/ewp/056095en.pdf(last accessed 21 October 2003).

FDA. Guidance for industry. In vivo drug metabolism/drug interaction studies—study design data analysis and recommendations for dosing and labelling. November1999.http://www.fda.gov/cder/guidance/2635fnl.pdf(last accessed 23 October 2003).

FDA. Guidance for industry. Pharmacokinetics in patients with impaired renal function—study design data analysis and impact on dosing and labelling. May1998.http://www.fda.gov/cder/guidance/1449fnl.pdfpdf (last accessed 23 October 2003).

FDA. Guidance for industry. Pharmacokinetics in patients with impaired hepatic function: study design data analysis and impact on dosing and labelling. May2003.http://www.fda.gov/cder/guidance/3625fnl.pdf(last accessed 23 October 2003).

10.1081/BIP-100101013

10.1080/10543409708835177

10.1002/sim.4780140926

10.1002/sim.4780101105

10.1007/BF02353513

Hauck WW, 1992, Types of bioequivalence and related statistical considerations, International Journal of Clinical Pharmacology, Therapy and Toxicology, 30, 181

10.1016/S0140-6736(88)90620-4

Wood J, 1999, Sample‐size calculations for trials in health services research, Journal of Health Services and Research and Policy, 4, 226, 10.1177/135581969900400408

Desu MM, 1990, Sample Size Methodology

CPMP. Points to consider on adjustment for baseline covariates. (CPMP/EWP/2863/99) 23 May2003.http://www.emea.eu.int/pdfs/human/ewp/286399en.pdf(last accessed 21 October 2003).

10.1002/sim.4780111304

Diggle PJ, 1996, Analysis of Longitudinal Data

10.1002/1097-0258(20001130)19:22<3133::AID-SIM744>3.0.CO;2-0