Doing clinical trials large enough to achieve adequate reductions in uncertainties about treatment effects

Journal of the Royal Society of Medicine, Supplement

Volumn 106, Issue 2, 2013, Pages 68-71

  Campbell, Michael J ^a  

^a UNIVERSITY OF SHEFFIELD   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; CLINICAL OBSERVATION; CLINICAL STUDY; ERROR; FUNDING; PROBABILITY; SAMPLE SIZE; STATISTICAL ANALYSIS; TREATMENT OUTCOME; BAYES THEOREM; CLINICAL TRIAL (TOPIC); DECISION MAKING; EFFECT SIZE; INFORMED CONSENT; MEDICAL LITERATURE; MEDICAL RESEARCH; OBSERVATION; RECOGNITION; SAMPLING ERROR; THERAPY EFFECT; UNCERTAINTY;

CLINICAL TRIALS AS TOPIC; HUMANS; RESEARCH DESIGN; TREATMENT OUTCOME; UNCERTAINTY;

EID: 84873654131     PISSN: 02675331     EISSN: None     Source Type: Journal    
DOI: 10.1177/0141076813477570     Document Type: Article

References (34)

1. Trohler U. The introduction of numerical methods to assess the effects of medical interventions during the 18th century: a brief history. JLL Bulletin: Commentaries on the History of Treatment Evaluation 2010. See www. jameslindlibrary.org
2. Gavarret LDJ. Principes généraux de statistique médicale: ou développement des règles qui doivent présider à son emploi. [General Principles of Medical Statistics: or the Development of Rules that must Govern their Use] Paris: Bechet jeune & Labé 1840
3. Balfour TG. Quoted in West C. Lectures on the Diseases of Infancy and Childhood. London: Longman, Brown, Green and Longmans, 1854:600
4. Chalmers I, Toth B. 19th century controlled trials to test whether belladonna prevents scarlet fever. JLL Bulletin: Commentaries on the History of Treatment Evaluation 2009. See www.jameslindlibrary.org
5. Heiberg P. Studier over den statistiske undersøgelsesmetode som hjælpemiddel ved terapeutiske undersøgelser (Studies on the statistical study design as an aid in therapeutic trials). Bibliotek for Læger 1897;89:1-40
6. Gluud C, Hilden J. Povl Heiberg's 1897 methodological study on the statistical method as an aid in therapeutic trials. JLL Bulletin: Commentaries on the History of Treatment Evaluation 2008. See www. jameslindlibrary.org
7. Neyman J, Pearson ES. On the use and interpretation of certain test criteria for purposes of statistical inference: Part I. Biometrika 1928;20A:175-240
8. Hill AB. Principles of Medical Statistics. London: Lancet, 1937
9. Bell JA. Pertussis prophylaxis with two doses of alum-precipitated vaccine. Public Health Rep 1941;56:1535-46
10. Medical Research Council. Clinical trial of patulin in the common cold. Lancet 1944;2:373-5
11. Medical Research Council. Streptomycin treatment of pulmonary tuberculosis: a Medical Research Council Investigation. Br Med J 1948;2:769-82
12. Paulson E, Wallis WA. Planning and analysing experiments for comparing two percentages. In: Eisenhart C, Hastay MW, Wallis WA, eds. Selected Techniques of Statistical Analysis for Scientific and Industrial Research and Production and Management Engineering. New York: McGraw-Hill, Chapter 7, 1947:247-66
13. Sillitto GP. Note on approximations to the power function of the 2 × 2 comparative trial. Biometrika 1949;4:347-52
14. Cochran WG, Cox GM. Experimental Designs. 2nd edn. New York: Wiley, 1957
15. Zubrod CG, Schneiderman M, Frei E, et al. Appraisal of methods for the study of chemotherapy in man: comparative therapeutic trial of nitrogen mustard and thiophosphoramide. J Chronic Dis 1960;11:7-33
16. Armitage P. Sequential Medical Trials. Springfield, Illinois: Thomas, 1960
17. Kilpatrick GS, Oldham PD. Calcium chloride and adrenaline as bronchial dilators compared by sequential analysis. Br Med J 1954;2:1388-91
18. Snell ES, Armitage P. Clinical comparison of diamorphine and pholcodine as cough suppressants by a new method of sequential analysis. Lancet 1957;1:860-2
19. Truelove SC, Watkinson G, Draper G. Comparison of corticosteroid and sulphasalazine therapy in ulcerative colitis. Br Med J 1962;2:1708-11
20. Anscombe FJ. Review of 'Sequential Medical Trials'. J Am Stat Assoc 1963;58:365-83
21. Freiman JA, Chalmers TC, Smith H, Kuebler RR. The importance of beta, the type II error and sample size in the design and interpretation of the randomized control trial: survey of 71 'negative' trials. N Engl J Med 1978;299:690-4
22. Machin D, Campbell MJ, Tan SB, Tan SH. Sample Size Tables for Clinical Studies. 3rd edn. Chichester: Wiley-Blackwell, 2008
23. Detsky AS, Sackett DL. When was a 'negative' clinical trial big enough? How many patients you needed depends on what you found. Arch Intern Med 1985;145:709-12
24. Schulz KF, Grimes DA. Sample size calculations in randomised trials: mandatory and mystical. Lancet 2005;365:1348-53
25. Bacchetti P, Wolf LE, Segal MR, McCulloch CE. Ethics and sample size. Am J Epidemiol 2005;161:105-10
26. Bland JM. The tyranny of power: is there a better way to calculate sample size? Br Med J 2009;339:1133-5 10.1136/ bmj.b3985
27. Williamson P, Hutton JL, Bliss J, Blunt J, Campbell MJ, Nicholson R. Statistical review by research ethics committees. J R Stat Soc A 2000;163:5-13
28. Spiegelhalter DJ, Abrams KR, Myles JR. Bayesian Approaches to Clinical Trials and Health-Care Evaluation. Chichester: Wiley, 2004
29. Cooper NJ, Jones DR, Sutton AJ. The use of systematic reviews when designing new studies. Clin Trials 2005;2:260-4
30. Robinson KA, Goodman SN. A systematic examination of the citation of prior research in reports of randomized, controlled trials. Ann Intern Med 2011;154:50-5
31. Chalmers I, Matthews R. What are the implications of optimism bias in clinical research? Lancet 2006;367:449-50
32. Sutton AJ, Cooper NJ, Jones DR, Lambert PC, Thompson JR, Abrams KR. Evidence-based sample size calculations based upon updated meta-analysis. Stat Med 2007;26:2479-500
33. Briggs A, Claxton K, Sculpher M. Decision modelling for health economic evaluation. Oxford: Oxford University Press, 2006
34. Phillips AJ, Keene ON. Adaptive designs for pivotal trials: discussion points from the PSI adaptive design expert group. Pharm Stat 2006;5:61-6