Power and sample size when multiple endpoints are considered

Pharmaceutical Statistics

Volumn 6, Issue 3, 2007, Pages 161-170

  Senn, Stephen ^a   Bretz, Frank ^b  

^a UNIVERSITY OF GLASGOW   (United Kingdom)

^b NOVARTIS PHARMA AG   (Switzerland)

Author keywords

Multiple endpoints; Multiple testing; Power

Indexed keywords

ANALYTICAL ERROR; ARTICLE; CONTROLLED STUDY; CORRELATION ANALYSIS; CORRELATION COEFFICIENT; DRUG DOSE REGIMEN; DRUG RESEARCH; DRUG SYNTHESIS; ECONOMIC EVALUATION; INTERMETHOD COMPARISON; MATHEMATICAL COMPUTING; MULTIVARIATE ANALYSIS; NORMAL DISTRIBUTION; NULL HYPOTHESIS; OUTCOMES RESEARCH; PROBABILITY; REGRESSION ANALYSIS; SAMPLE SIZE; STATISTICAL ANALYSIS;

BIAS (EPIDEMIOLOGY); CLINICAL TRIALS; DATA INTERPRETATION, STATISTICAL; ENDPOINT DETERMINATION; HUMANS; MODELS, STATISTICAL; PROBABILITY; RESEARCH DESIGN; SAMPLE SIZE; TREATMENT OUTCOME;

EID: 34648823148     PISSN: 15391604     EISSN: 15391612     Source Type: Journal    
DOI: 10.1002/pst.301     Document Type: Article

References (22)

1. Lewis JA. Discussion of the paper by Senn. The Statistician 2000; 49:156-157.
2. Senn SJ. Statistical issues in drug development. Wiley: Chichester, 1997.
3. Darken PF, Ho S-Y. A note on sample size savings with the use of a single well-controlled clinical trial to support the efficacy of a new drug. Pharmaceutical Statistics 2004; 3:61-63.
4. Fisher LD. One large, well-designed, multicenter study as an alternative to the usual FDA paradigm. Drug Information Journal 1999; 33:265-271.
5. Maca J, Gallo P, Branson M, Maurer W. Reconsidering some aspects of the two-trials paradigm. Journal of Biopharmaceutical Statistics 2002; 12: 107-109.
6. Rosenkranz G. Is it possible to claim efficacy if one of two trials is significant while the other just shows a trend? Drug Information Journal 2002; 36:875-879.
7. Senn SJ. Consensus and controversy in pharmaceutical statistics (with discussion). The Statistician 2000; 49:135-176.
8. Senn SJ. Dicing with death. Cambridge University Press: Cambridge, 2003.
9. Rothman KJ. No adjustments are needed for multiple comparisons. Epidemiology 1990; 1:43-46.
10. Nelder JA. From statistics to statistical science - reply. Journal of the Royal Statistical Society Series D - The Statistician 1999; 48:269.
11. Lindsey JK. Some statistical heresies. Journal of the Royal Statistical Society Series D - The Statistician 1999; 48:1-26.
12. Bauer P. Multiple testing in clinical-trials. Statistics in Medicine 1991; 10:871-890.
13. O'Neill RT. Secondary endpoints cannot be validly analyzed if the primary endpoint does not demonstrate clear statistical significance. Controlled Clinical Trials 1997; 18:550-556.
14. Cook RJ, Farewell VT. Multiplicity considerations in the design and analysis of clinical trials. Journal of the Royal Statistical Society Series A - Statistics in Society 1996; 159:93-110.
15. Chuang-Stein C, Stryszak P, Dmitrienko A, Offen W. Challenge of multiple co-primary end-points: a new approach. Statistics in Medicine 2007; 26:1181-1192.
16. Offen W, Chuang-Stein C, Dmitrienko A, Littman G, Maca J, Meyerson L, Muirhead R, Stryszak P, Boddy A, Chen K, Copley-Merriman K, Dere W, Givens S, Hall D, Henry D, Jackson JD, Krishen A, Liu T, Ryder S, Sankoh AJ, Wang J, Yeh CH. Multiple co-primary endpoints: medical and statistical solutions - a report from the Multiple End-points Expert Team of the Pharmaceutical Research and Manufacturers of America. Drug Information Journal 2007; 41:31-46.
17. Westfall PH, Tobias RD, Rom D, Wolfinger RD, Hochberg Y. Multiple comparisons and multiple tests using the SAS system. SAS: Cary, NC, 1999.
18. Marcus R, Peritz E, Gabriel KR. On closed testing procedures with special reference to ordered analysis of variance. Biometrika 1976; 63:655-660.
19. Dunnett CW. A multiple comparison procedure for comparing several treatments with a control. Journal of the American Statistical Association 1955; 50:1096-1121.
20. Simes RJ. An improved Bonferroni procedure for multiple tests of significance. Biometrika 1986; 73:751-754.
21. Hsu JC. Multiple comparisons: theory and methods. Chapman & Hall: London, 1996.
22. Robertson T, Wright FT, Dykstra RL. Order restricted statistical inference. Wiley: New York, 1988.