Cited by 426Statistical Methods in Clinical TrialsStatistical Methods and Bayesian InferenceStatistical Methods in Epidemiology

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The statistical interpretation of pilot trials: should significance thresholds be reconsidered?

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Lee, Ellen C, Whitehead, Amy L, Jacques, Richard M, Julious, Steven A·2014·BMC Medical Research Methodology

DOI 10.1186/1471-2288-14-41PMC PMC3994566

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Abstract

BACKGROUND: In an evaluation of a new health technology, a pilot trial may be undertaken prior to a trial that makes a definitive assessment of benefit. The objective of pilot studies is to provide sufficient evidence that a larger definitive trial can be undertaken and, at times, to provide a preliminary assessment of benefit. METHODS: We describe significance thresholds, confidence intervals and surrogate markers in the context of pilot studies and how Bayesian methods can be used in pilot trials. We use a worked example to illustrate the issues raised. RESULTS: We show how significance levels other than the traditional 5% should be considered to provide preliminary evidence for efficacy and how estimation and confidence intervals should be the focus to provide an estimated range of possible treatment effects. We also illustrate how Bayesian methods could also assist in the early assessment of a health technology. CONCLUSIONS: We recommend that in pilot trials the focus should be on descriptive statistics and estimation, using confidence intervals, rather than formal hypothesis testing and that confidence intervals other than 95% confidence intervals, such as 85% or 75%, be used for the estimation. The confidence interval should then be interpreted with regards to the minimum clinically important difference. We also recommend that Bayesian methods be used to assist in the interpretation of pilot trials. Surrogate endpoints can also be used in pilot trials but they must reliably predict the overall effect on the clinical outcome.

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Figure 1 Mean difference in SF-36 GH dimension scores between treatment and control with confidence intervals (based on n = 31 patients).CC BY · PubMed Central

Figure 2 Prior, observed and posterior distributions for non-informative, pessimistic and optimistic priors.CC BY · PubMed Central

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Biomarkers and surrogate endpoints: Preferred definitions and conceptual framework
Biomarkers Definitions Working Group (2001) · Clinical Pharmacology & Therapeutics · doi:10.1067/mcp.2001.113989 · Cited by 6,771
A tutorial on pilot studies: the what, why and how
Thabane, L., Ma, J., Chu, R., Cheng, J., Ismaila, A., Rios, L. P., Robson, R., Thabane, M., Giangregorio, L., & Goldsmith, C. H. (2010) · BMC Medical Research Methodology · doi:10.1186/1471-2288-10-1 · Cited by 3,384
Design and analysis of pilot studies: recommendations for good practice
Lancaster, G. A., Dodd, S., & Williamson, P. R. (2004) · Journal of Evaluation in Clinical Practice · doi:10.1111/j..2002.384.doc.x · Cited by 2,572
Surrogate endpoints in clinical trials: Definition and operational criteria
Prentice, R. L. (1989) · Statistics in Medicine · doi:10.1002/sim.4780080407 · Cited by 2,057
Bayesian Approaches to Clinical Trials and Health‐Care Evaluation
Spiegelhalter, D. J., Abrams, K. R., & Myles, J. P. (2003) · Wiley eBooks · doi:10.1002/0470092602 · Cited by 1,687
Surrogate End Points in Clinical Trials: Are We Being Misled?
Fleming, T. R., & DeMets, D. L. (1996) · Annals of Internal Medicine · doi:10.7326/0003-4819-125-7-199610010-00011 · Cited by 1,651
What is a pilot or feasibility study? A review of current practice and editorial policy
Arain, M., Campbell, M. J., Cooper, C. L., & Lancaster, G. A. (2010) · BMC Medical Research Methodology · doi:10.1186/1471-2288-10-67 · Cited by 1,483
Bayesian Statistics: An Introduction
Ziegel, E. R., & Lee, P. M. (1998) · Technometrics · doi:10.2307/1270551 · Cited by 1,191
Multiple Imputation and its Application
Carpenter, J. R., & Kenward, M. G. (2013) · doi:10.1002/9781119942283 · Cited by 913
Considerations in the Evaluation of Surrogate Endpoints in Clinical Trials
Gruttola, V. D., Clax, P. A., DeMets, D. L., Downing, G. J., Ellenberg, S. S., Friedman, L., Gail, M. H., Prentice, R. L., Wittes, J., & Zeger, S. L. (2001) · Controlled Clinical Trials · doi:10.1016/s0197-2456(01)00153-2 · Cited by 428

Show all 31 references

Research works printed but not linked: 1

International Conference on Harmonisation: ICH E9 statistical principals for clinical trials. 1998, http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/Step4/E9_Guideline.pdf,

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Authors: Lee, E. C., Whitehead, A. L., Jacques, R. M., & Julious, S. A.
Affiliations: University of Sheffield (GB)
Corresponding author: Julious, Steven A
ORCID: Lee, Ellen C
Whitehead, Amy L
Jacques, Richard M
Julious, Steven A
Venue: BMC Medical Research Methodology
Publisher: Springer Science and Business Media LLC
Published: 2014-03-20
Volume: 14
Issue: 1
Pages: 41-41
ISSN: 1471-2288 (ISSN-L)
DOI: 10.1186/1471-2288-14-41
PMC: PMC3994566
OpenAlex: W2108052506
Cited by: 423
References: 31
Citations by year: 2026: 26 · 2025: 60 · 2024: 50 · 2023: 59 · 2022: 52 · 2021: 40 · 2020: 34 · 2019: 30 · 2018: 31 · 2017: 16 · 2016: 14 · 2015: 9 · 2014: 2
FWCI: 14.45
Type: journal article
Language: en
Open access: gold
doi.org
Licence: cc-by
www.springer.com/tdm (unspecified)
Locations: BMC Medical Research Methodology (publishedVersion, cc-by)
Europe PMC (PubMed Central) (publishedVersion, cc-by)
PubMed Central (publishedVersion, cc-by)
PubMed (publishedVersion)
White Rose Research Online (University of Leeds, The University of Sheffield, University of York) (acceptedVersion)
ePrints Soton (University of Southampton) (acceptedVersion)
Topics: Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods and Bayesian Inference
Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Epidemiology
Keywords: pilot trial, significance threshold, confidence intervals, Bayesian analysis, surrogate endpoints, minimal clinically important difference, health technology assessment, treatment effect estimation, hypothesis testing
MeSH terms: Bayes Theorem, Data Interpretation, Statistical, Humans, Leg Ulcer, Pilot Projects, Research Design, Biomarkers, Confidence Intervals, Treatment Outcome

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The statistical interpretation of pilot trials: should significance thresholds be reconsidered?

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