Obtaining evidence by a single well-powered trial or several modestly powered trials

Statistical Methods in Medical Research

Volumn 25, Issue 2, 2016, Pages 538-552

  Inthout, Joanna ^a   Ioannidis, John P A ^b,c   Borm, George F ^a  

^a RADBOUD UNIVERSITY MEDICAL CENTER   (Netherlands)

^b STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c STANFORD UNIVERSITY   (United States)

Author keywords

Clinical trial; heterogeneity; meta analysis; publication bias; type I error

Indexed keywords

CONTROLLED STUDY; HUMAN; META ANALYSIS; PUBLICATION; QUANTITATIVE STUDY; CLINICAL TRIAL (TOPIC); META ANALYSIS (TOPIC); METHODOLOGY; PROCEDURES; PUBLISHING;

CLINICAL TRIALS AS TOPIC; HUMANS; META-ANALYSIS AS TOPIC; PUBLICATION BIAS; RESEARCH DESIGN;

EID: 84964300648     PISSN: 09622802     EISSN: 14770334     Source Type: Journal    
DOI: 10.1177/0962280212461098     Document Type: Article

References (39)

1. Schulz KF, Grimes DA,. Sample size calculations in randomised trials: mandatory and mystical. Lancet 2005; 365 (9467): 1348-1353.
2. Edwards SJL, Lilford RJ, Braunholz D, et al. Why "underpowered" trials are not necessarily unethical. Lancet 1997; 350: 804-807.
3. Halpern SD, Karlawish JH, Berlin JA,. The continuing unethical conduct of underpowered clinical trials. JAMA 2002; 288 (3): 358-362.
4. Borm GF, Den Heijer M, Zielhuis GA,. Publication bias was not a good reason to discourage trials with low power. J Clin Epidemiol 2009; 62: 47e53-47e53.
5. Rothstein HR, Sutton AJ, Borenstein M, Rothstein HR, Sutton AJ, Borenstein M,. Publication bias in meta-analysis. Publication bias in meta-analysis: prevention, assessment and adjustments, Chichester, UK: John Wiley & Sons, Ltd, 2006., doi:10.1002/0470870168.ch1.
6. Song F, Eastwood AJ, Gilbody S, et al. Publication and related biases. Health Technol Assess 2000; 4 (10): 1-115.
7. Ioannidis JP, Patsopoulos NA, Rothstein HR,. Reasons or excuses for avoiding meta-analysis in forest plots. BMJ 2008; 336 (7658): 1413-1415.
8. Shrier I, Platt RW, Steele RJ,. Mega-trials vs. meta-analysis: precision vs. heterogeneity?. Contemp Clin Trials 2007; 28: 324-328.
9. Ioannidis JP,. Contradicted and initially stronger effects in highly cited clinical research. JAMA 2005; 294 (2): 218-228.
10. Borm GF, Lemmers O, Fransen J, et al. The evidence provided by a single trial is less reliable than its statistical analysis suggests. J Clin Epidemiol 2009; 62: 711-715.
11. Borm GF, Donders R,. A treatment should be evaluated by small trials. Response to letter. J Clin Epidemiol 2009; 62: 886-889.
12. Higgins JP, Thompson SG, Deeks JJ, et al. Measuring inconsistency in meta-analyses. BMJ 2003; 327 (7414): 557-560.
13. Ioannidis JPA, Patsopoulos NA, Evangelou E,. Uncertainty in heterogeneity estimates in meta-analyses. BMJ 2007; 335 (7626): 914-916.
14. Ross JS, Mulvey GK, Hines EM, et al. Trial publication after registration in ClinicalTrials.Gov: a cross-sectional analysis. PLoS Med 2009; 6 (9): e1000144-e1000144.
15. Lee K, Bacchetti P, Sim I,. Publication of clinical trials supporting successful new drug applications: a literature analysis. PLoS Med 2008; 5 (9): e191-e191.
16. Hopewell S, Loudon K, Clarke MJ, et al. Publication bias in clinical trials due to statistical significance or direction of trial results. Cochrane Database Syst Rev 2009 Jan; 21 (1): MR000006-MR000006.
17. Dwan K, Altman DG, Arnaiz JA, et al. Systematic review of the empirical evidence of study publication bias and outcome reporting bias. PLoS ONE 2008; 3 (8): e3081-e3081.
18. Song F, Parekh-Bhurke S, Hooper L, et al. Extent of publication bias in different categories of research cohorts: a meta-analysis of empirical studies. BMC Med Res Methodol 2009; 9: 79-79.
19. Rising K, Bacchetti P, Bero L,. Reporting bias in drug trials submitted to the food and drug administration: review of publication and presentation. PLoS Med 2008; 5 (11): e217-e217.
20. DerSimonian R, Laird N,. Meta-analysis in clinical trials. Control Clin Trials 1986; 7: 177-188.
21. Sidik K, Jonkman JN,. A simple confidence interval for meta-analysis. Stat Med 2002; 21: 3153-3159.
22. Brockwell SE, Gordon IR,. A comparison of statistical methods for meta-analysis. Stat Med 2001; 20: 825-840.
23. Henmi M, Copas JB,. Confidence intervals for random effects meta-analysis and robustness to publication bias. Stat Med 2010; 29: 2969-2983.
24. Guidance for industry. Providing evidence of effectiveness for human drug and biological products, Washington, DC: FDA, 1998
25. EMEA. Points to consider on application with 1. meta-analyses; 2. one pivotal study. EMEA: London, 2001.
26. Egger M, Ebrahim S, Smith GD,. Where now for meta-analysis?. Int J Epidemiol 2002; 31: 1-5.
27. LeLorier J, Grégoire G, Benhaddad A, et al. Discrepancies between meta-analyses and subsequent large randomized, controlled trials. N Engl J Med 1997; 337 (8): 536-542.
28. LeLorier J, Gregoire G,. Comparing results from meta-analyses vs large trials. JAMA 1998; 280 (6): 518-519.
29. Flather M, Delahunty N, Collinson J,. Generalizing results of randomized trials to clinical practice: reliability and cautions. Clin Trials 2006; 3: 508-512.
30. Zwarenstein M, Oxman A,. Why are so few randomized trials useful, and what can we do about it?. J Clin Epidemiol 2006; 59: 1125-1126.
31. Ioannidis JP, Cappelleri JC, Lau J,. Meta-analyses and large randomized, controlled trials. N Engl J Med 1998; 338 (1): 59-62.
32. Matthews JNS,. Small trials: are they all bad?. Stat Med 1995; 14: 115-126.
33. Jüni P, Altman DG, Egger M,. Assessing the quality of controlled clinical trials. BMJ 2001; 323: 42-46.
34. Province MA, Hadley EC, Hornbrook MC, et al. The effects of exercise on falls in elderly patients. A preplanned meta-analysis of the FICSIT trials. JAMA 1995; 273: 1341-1347.
35. Stenting Trialists' Collaboration Carotid,. Short-term outcome after stenting versus endarterectomy for symptomatic carotid stenosis: a preplanned meta-analysis of individual patient data. Lancet 2010; 376 (9746): 1062-1073.
36. Giffin RB, Woodcock J,. Comparative effectiveness research: who will do the studies?. Health Aff 2010; 29 (11): 2075-2081.
37. Ioannids JPA, Karassa FB,. The need to consider the wider agenda in systematic reviews and meta-analyses: breadth, timing, and depth of the evidence. BMJ 2010; 341: 762-765.
38. Schulz KF, Altman DG, Moher D,. for the CONSORT Group. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. BMJ 2010; 340: c332-c332.
39. Borm GF, Donders R,. Updating meta-analyses leads to larger type I errors than publication bias. J Clin Epidemiol 2009; 62: 825-830.