Skip to main content
Top
Published in: BMC Medicine 1/2010

Open Access 01-12-2010 | Debate

Current sample size conventions: Flaws, harms, and alternatives

Author: Peter Bacchetti

Published in: BMC Medicine | Issue 1/2010

Login to get access

Abstract

Background

The belief remains widespread that medical research studies must have statistical power of at least 80% in order to be scientifically sound, and peer reviewers often question whether power is high enough.

Discussion

This requirement and the methods for meeting it have severe flaws. Notably, the true nature of how sample size influences a study's projected scientific or practical value precludes any meaningful blanket designation of <80% power as "inadequate". In addition, standard calculations are inherently unreliable, and focusing only on power neglects a completed study's most important results: estimates and confidence intervals. Current conventions harm the research process in many ways: promoting misinterpretation of completed studies, eroding scientific integrity, giving reviewers arbitrary power, inhibiting innovation, perverting ethical standards, wasting effort, and wasting money. Medical research would benefit from alternative approaches, including established value of information methods, simple choices based on cost or feasibility that have recently been justified, sensitivity analyses that examine a meaningful array of possible findings, and following previous analogous studies. To promote more rational approaches, research training should cover the issues presented here, peer reviewers should be extremely careful before raising issues of "inadequate" sample size, and reports of completed studies should not discuss power.

Summary

Common conventions and expectations concerning sample size are deeply flawed, cause serious harm to the research process, and should be replaced by more rational alternatives.
Appendix
Available only for authorised users
Literature
1.
go back to reference Halpern SD, Karlawish JHT, Berlin JA: The continuing unethical conduct of underpowered clinical trials. JAMA-Journal of the American Medical Association. 2002, 288: 358-362. 10.1001/jama.288.3.358.CrossRef Halpern SD, Karlawish JHT, Berlin JA: The continuing unethical conduct of underpowered clinical trials. JAMA-Journal of the American Medical Association. 2002, 288: 358-362. 10.1001/jama.288.3.358.CrossRef
2.
go back to reference Edwards SJL, Lilford RJ, Braunholtz D, Jackson J: Why "underpowered" trials are not necessarily unethical. Lancet. 1997, 350: 804-807. 10.1016/S0140-6736(97)02290-3.CrossRefPubMed Edwards SJL, Lilford RJ, Braunholtz D, Jackson J: Why "underpowered" trials are not necessarily unethical. Lancet. 1997, 350: 804-807. 10.1016/S0140-6736(97)02290-3.CrossRefPubMed
3.
go back to reference Guyatt GH, Mills EJ, Elbourne D: In the era of systematic reviews, does the size of an individual trial still matter?. PLoS Medicine. 2008, 5: 3-5. 10.1371/journal.pmed.0050004.CrossRef Guyatt GH, Mills EJ, Elbourne D: In the era of systematic reviews, does the size of an individual trial still matter?. PLoS Medicine. 2008, 5: 3-5. 10.1371/journal.pmed.0050004.CrossRef
4.
go back to reference Vail A: Experiences of a biostatistician on a UK research ethics committee. Statistics in Medicine. 1998, 17: 2811-2814. 10.1002/(SICI)1097-0258(19981230)17:24<2811::AID-SIM22>3.0.CO;2-1.CrossRefPubMed Vail A: Experiences of a biostatistician on a UK research ethics committee. Statistics in Medicine. 1998, 17: 2811-2814. 10.1002/(SICI)1097-0258(19981230)17:24<2811::AID-SIM22>3.0.CO;2-1.CrossRefPubMed
5.
go back to reference Bacchetti P, McCulloch CE, Segal MR: Simple, defensible sample sizes based on cost efficiency. Biometrics. 2008, 64: 577-585. 10.1111/j.1541-0420.2008.01004_1.x.CrossRefPubMedPubMedCentral Bacchetti P, McCulloch CE, Segal MR: Simple, defensible sample sizes based on cost efficiency. Biometrics. 2008, 64: 577-585. 10.1111/j.1541-0420.2008.01004_1.x.CrossRefPubMedPubMedCentral
6.
go back to reference Kraemer HC, Mintz J, Noda A, Tinklenberg J, Yesavage JA: Caution regarding the use of pilot studies to guide power calculations for study proposals. Archives of General Psychiatry. 2006, 63: 484-489. 10.1001/archpsyc.63.5.484.CrossRefPubMed Kraemer HC, Mintz J, Noda A, Tinklenberg J, Yesavage JA: Caution regarding the use of pilot studies to guide power calculations for study proposals. Archives of General Psychiatry. 2006, 63: 484-489. 10.1001/archpsyc.63.5.484.CrossRefPubMed
7.
go back to reference Horrobin DF: Are large clinical trials in rapidly lethal diseases usually unethical?. Lancet. 2003, 361: 695-697. 10.1016/S0140-6736(03)12571-8.CrossRefPubMed Horrobin DF: Are large clinical trials in rapidly lethal diseases usually unethical?. Lancet. 2003, 361: 695-697. 10.1016/S0140-6736(03)12571-8.CrossRefPubMed
8.
go back to reference Matthews JNS: Small clinical-trials - are they all bad?. Statistics in Medicine. 1995, 14: 115-126. 10.1002/sim.4780140204.CrossRefPubMed Matthews JNS: Small clinical-trials - are they all bad?. Statistics in Medicine. 1995, 14: 115-126. 10.1002/sim.4780140204.CrossRefPubMed
9.
go back to reference Vickers AJ: Underpowering in randomized trials reporting a sample size calculation. Journal of Clinical Epidemiology. 2003, 56: 717-720. 10.1016/S0895-4356(03)00141-0.CrossRefPubMed Vickers AJ: Underpowering in randomized trials reporting a sample size calculation. Journal of Clinical Epidemiology. 2003, 56: 717-720. 10.1016/S0895-4356(03)00141-0.CrossRefPubMed
10.
go back to reference Charles P, Giraudeau B, Dechartres A, Baron G, Ravaud P: Reporting of sample size calculation in randomised controlled trials: review. British Medical Journal. 2009, 338: b1732-10.1136/bmj.b1732.CrossRefPubMedPubMedCentral Charles P, Giraudeau B, Dechartres A, Baron G, Ravaud P: Reporting of sample size calculation in randomised controlled trials: review. British Medical Journal. 2009, 338: b1732-10.1136/bmj.b1732.CrossRefPubMedPubMedCentral
11.
go back to reference Altman DG, Schulz KF, Moher D, Egger M, Davidoff F, Elbourne D, Gotzsche PC, Lang T, Grp C: The revised CONSORT statement for reporting randomized trials: Explanation and elaboration. Annals of Internal Medicine. 2001, 134: 663-694.CrossRefPubMed Altman DG, Schulz KF, Moher D, Egger M, Davidoff F, Elbourne D, Gotzsche PC, Lang T, Grp C: The revised CONSORT statement for reporting randomized trials: Explanation and elaboration. Annals of Internal Medicine. 2001, 134: 663-694.CrossRefPubMed
12.
go back to reference Gardner MJ, Altman DG: Confidence-intervals rather than P-values - estimation rather than hypothesis-testing. British Medical Journal. 1986, 292: 746-750. 10.1136/bmj.292.6522.746.CrossRefPubMedPubMedCentral Gardner MJ, Altman DG: Confidence-intervals rather than P-values - estimation rather than hypothesis-testing. British Medical Journal. 1986, 292: 746-750. 10.1136/bmj.292.6522.746.CrossRefPubMedPubMedCentral
13.
go back to reference Goodman SN: P-values, hypothesis tests, and likelihood - implications for epidemiology of a neglected historical debate. American Journal of Epidemiology. 1993, 137: 485-496.PubMed Goodman SN: P-values, hypothesis tests, and likelihood - implications for epidemiology of a neglected historical debate. American Journal of Epidemiology. 1993, 137: 485-496.PubMed
14.
go back to reference Prentice RL, Caan B, Chlebowski RT, Patterson R, Kuller LH, Ockene JK, Margolis KL, Limacher MC, Manson JE, Parker LM, Paskett E, Phillips L, Robbins J, Rossouw JE, Sarto GE, Shikany JM, Stefanick ML, Thomson CA, Van Horn L, Vitolins MZ, Wactawski-Wende J, Wallace RB, Wassertheil-Smoller S, Whitlock E, Yano K, Adams-Campbell L, Anderson GL, Assaf AR, Beresford SA, Black HR, et al: Low-fat dietary pattern and risk of invasive breast cancer - The women's health initiative randomized controlled dietary modification trial. JAMA-Journal of the American Medical Association. 2006, 295: 629-642. 10.1001/jama.295.6.629.CrossRef Prentice RL, Caan B, Chlebowski RT, Patterson R, Kuller LH, Ockene JK, Margolis KL, Limacher MC, Manson JE, Parker LM, Paskett E, Phillips L, Robbins J, Rossouw JE, Sarto GE, Shikany JM, Stefanick ML, Thomson CA, Van Horn L, Vitolins MZ, Wactawski-Wende J, Wallace RB, Wassertheil-Smoller S, Whitlock E, Yano K, Adams-Campbell L, Anderson GL, Assaf AR, Beresford SA, Black HR, et al: Low-fat dietary pattern and risk of invasive breast cancer - The women's health initiative randomized controlled dietary modification trial. JAMA-Journal of the American Medical Association. 2006, 295: 629-642. 10.1001/jama.295.6.629.CrossRef
15.
go back to reference Hoenig JM, Heisey DM: The abuse of power: The pervasive fallacy of power calculations for data analysis. American Statistician. 2001, 55: 19-24. 10.1198/000313001300339897.CrossRef Hoenig JM, Heisey DM: The abuse of power: The pervasive fallacy of power calculations for data analysis. American Statistician. 2001, 55: 19-24. 10.1198/000313001300339897.CrossRef
16.
go back to reference Rumbold AR, Crowther CA, Haslam RR, Dekker GA, Robinson JS: Vitamins C and E and the risks of preeclampsia and perinatal complications. New England Journal of Medicine. 2006, 354: 1796-1806. 10.1056/NEJMoa054186.CrossRefPubMed Rumbold AR, Crowther CA, Haslam RR, Dekker GA, Robinson JS: Vitamins C and E and the risks of preeclampsia and perinatal complications. New England Journal of Medicine. 2006, 354: 1796-1806. 10.1056/NEJMoa054186.CrossRefPubMed
18.
go back to reference Detsky AS: Using cost-effectiveness analysis to improve the efficiency of allocating funds to clinical-trials. Statistics in Medicine. 1990, 9: 173-184. 10.1002/sim.4780090124.CrossRefPubMed Detsky AS: Using cost-effectiveness analysis to improve the efficiency of allocating funds to clinical-trials. Statistics in Medicine. 1990, 9: 173-184. 10.1002/sim.4780090124.CrossRefPubMed
19.
go back to reference Senn S: Statistical Issues in Drug Development. 2007, Chichester, England; Hoboken, NJ: John Wiley & Sons, 2CrossRef Senn S: Statistical Issues in Drug Development. 2007, Chichester, England; Hoboken, NJ: John Wiley & Sons, 2CrossRef
20.
go back to reference Goodman SN, Berlin JA: The use of predicted confidence-intervals when planning experiments and the misuse of power when interpreting results. Annals of Internal Medicine. 1994, 121: 200-206.CrossRefPubMed Goodman SN, Berlin JA: The use of predicted confidence-intervals when planning experiments and the misuse of power when interpreting results. Annals of Internal Medicine. 1994, 121: 200-206.CrossRefPubMed
21.
go back to reference Schulz KF, Grimes DA: Epidemiology 1 - Sample size calculations in randomised trials: mandatory and mystical. Lancet. 2005, 365: 1348-1353. 10.1016/S0140-6736(05)61034-3.CrossRefPubMed Schulz KF, Grimes DA: Epidemiology 1 - Sample size calculations in randomised trials: mandatory and mystical. Lancet. 2005, 365: 1348-1353. 10.1016/S0140-6736(05)61034-3.CrossRefPubMed
22.
go back to reference Norman GR, Streiner DL: PDQ Statistics. 2003, Hamilton, Ont.: B.C. Decker, 3 Norman GR, Streiner DL: PDQ Statistics. 2003, Hamilton, Ont.: B.C. Decker, 3
23.
go back to reference Bacchetti P: Peer review of statistics in medical research: the other problem. British Medical Journal. 2002, 324: 1271-1273. 10.1136/bmj.324.7348.1271.CrossRefPubMedPubMedCentral Bacchetti P: Peer review of statistics in medical research: the other problem. British Medical Journal. 2002, 324: 1271-1273. 10.1136/bmj.324.7348.1271.CrossRefPubMedPubMedCentral
25.
go back to reference Bacchetti P, Wolf LE, Segal MR, McCulloch CE: Bacchetti et al. Respond to "Ethics and sample size - Another view". American Journal of Epidemiology. 2005, 161: 113-113. 10.1093/aje/kwi016.CrossRef Bacchetti P, Wolf LE, Segal MR, McCulloch CE: Bacchetti et al. Respond to "Ethics and sample size - Another view". American Journal of Epidemiology. 2005, 161: 113-113. 10.1093/aje/kwi016.CrossRef
26.
go back to reference Bacchetti P, Wolf LE, Segal MR, McCulloch CE: Ethics and sample size. American Journal of Epidemiology. 2005, 161: 105-110. 10.1093/aje/kwi014.CrossRefPubMed Bacchetti P, Wolf LE, Segal MR, McCulloch CE: Ethics and sample size. American Journal of Epidemiology. 2005, 161: 105-110. 10.1093/aje/kwi014.CrossRefPubMed
27.
go back to reference Bacchetti P, Wolf LE, Segal MR, McCulloch CE: Re: "Ethics and sample size" - Reply. American Journal of Epidemiology. 2005, 162: 196-196. 10.1093/aje/kwi177.CrossRef Bacchetti P, Wolf LE, Segal MR, McCulloch CE: Re: "Ethics and sample size" - Reply. American Journal of Epidemiology. 2005, 162: 196-196. 10.1093/aje/kwi177.CrossRef
29.
go back to reference Bacchetti P, McCulloch CE, Segal MR: Simple, defensible sample sizes based on cost efficiency - Rejoinder. Biometrics. 2008, 64: 592-594. 10.1111/j.1541-0420.2008.01004_5.x.CrossRef Bacchetti P, McCulloch CE, Segal MR: Simple, defensible sample sizes based on cost efficiency - Rejoinder. Biometrics. 2008, 64: 592-594. 10.1111/j.1541-0420.2008.01004_5.x.CrossRef
30.
go back to reference Willan AR: Optimal sample size determinations from an industry perspective based on the expected value of information. Clinical Trials. 2008, 5: 587-594. 10.1177/1740774508098413.CrossRefPubMed Willan AR: Optimal sample size determinations from an industry perspective based on the expected value of information. Clinical Trials. 2008, 5: 587-594. 10.1177/1740774508098413.CrossRefPubMed
31.
go back to reference Willan AR, Pinto EM: The value of information and optimal clinical trial design. Statistics in Medicine. 2005, 24: 1791-1806. 10.1002/sim.2069.CrossRefPubMed Willan AR, Pinto EM: The value of information and optimal clinical trial design. Statistics in Medicine. 2005, 24: 1791-1806. 10.1002/sim.2069.CrossRefPubMed
32.
go back to reference Altman DG, Moher D, Schulz KF: Peer review of statistics in medical research - Reporting power calculations is important. British Medical Journal. 2002, 325: 492-492. 10.1136/bmj.325.7376.1364/a.CrossRef Altman DG, Moher D, Schulz KF: Peer review of statistics in medical research - Reporting power calculations is important. British Medical Journal. 2002, 325: 492-492. 10.1136/bmj.325.7376.1364/a.CrossRef
33.
go back to reference Bacchetti P: Peer review of statistics in medical research - Author's thoughts on power calculations. British Medical Journal. 2002, 325: 492-493. Bacchetti P: Peer review of statistics in medical research - Author's thoughts on power calculations. British Medical Journal. 2002, 325: 492-493.
34.
35.
go back to reference Tukey JW: Tightening the clinical-trial. Controlled Clinical Trials. 1993, 14: 266-285. 10.1016/0197-2456(93)90225-3.CrossRefPubMed Tukey JW: Tightening the clinical-trial. Controlled Clinical Trials. 1993, 14: 266-285. 10.1016/0197-2456(93)90225-3.CrossRefPubMed
Metadata
Title
Current sample size conventions: Flaws, harms, and alternatives
Author
Peter Bacchetti
Publication date
01-12-2010
Publisher
BioMed Central
Published in
BMC Medicine / Issue 1/2010
Electronic ISSN: 1741-7015
DOI
https://doi.org/10.1186/1741-7015-8-17

Other articles of this Issue 1/2010

BMC Medicine 1/2010 Go to the issue