Use of the bootstrap in analysing cost data from cluster randomised trials: some simulation results
Open accessAbstract
Background: This work has investigated under what conditions confidence intervals around the differences in mean costs from a cluster RCT are suitable for estimation using a commonly used cluster-adjusted bootstrap in preference to methods that utilise the Huber-White robust estimator of variance. The bootstrap's main advantage is in dealing with skewed data, which often characterise patient costs. However, it is insufficiently well recognised that one method of adjusting the bootstrap to deal with clustered data is only valid in large samples. In particular, the requirement that the number of clusters randomised should be large would not be satisfied in many cluster RCTs performed to date. Methods: The performances of confidence intervals for simple differences in mean costs utilising a robust (cluster-adjusted) standard error and from two cluster-adjusted bootstrap procedures were compared in terms of confidence interval coverage in a large number of simulations. Parameters varied included the intracluster correlation coefficient, the sample size and the distributions used to generate the data. Results: The bootstrap's advantage in dealing with skewed data was found to be outweighed by its poor confidence interval coverage when the number of clusters was at the level frequently found in cluster RCTs in practice. Simulations showed that confidence intervals based on robust methods of standard error estimation achieved coverage rates between 93.5% and 94.8% for a 95% nominal level whereas those for the bootstrap ranged between 86.4% and 93.8%. Conclusion: In general, 24 clusters per treatment arm is probably the minimum number for which one would even begin to consider the bootstrap in preference to traditional robust methods, for the parameter combinations investigated here. At least this number of clusters and extremely skewed data would be necessary for the bootstrap to be considered in favour of the robust method. There is a need for further investigation of more complex bootstrap procedures if economic data from cluster RCTs are to be analysed appropriately.
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This study supports Flynn et al.'s reasoning that we need a large number of clusters, more than 20 per group, to start considering bootstrap methods in place of more traditional robust methods like mixed models.
“Moreover, this study supports Flynn et al.’s reasoning [23] that we need a large number of clusters, more than 20 per group, to start considering bootstrap methods in place of more traditional robust methods like mixed models.”
From Evaluation of the type I error rate when using parametric bootstrap analysis of a cluster randomized controlled trial with binary outcomes and a small number of clusters (2022) · cites this paper as [23]
The Conclusions (page unavailable) say that 24 clusters per treatment arm is probably the minimum at which to begin considering bootstrap methods over traditional robust methods, for the simulated conditions. But the Methods section, “Comparison of methods that allow for clustering” (page unavailable), identifies the comparator as a Huber–White cluster-adjusted method, not a mixed model. The conclusion also requires extremely skewed data for the bootstrap to be considered in its favour. (LLM, conf=0.87)
Experimental — accuracy not yet measuredFlynn et al. concluded that in general, 24 clusters per treatment arm is probably the minimum number for which one would even begin to consider the bootstrap in preference to traditional robust methods, for the parameter combinations investigated.
“Flynn et al [23] concluded that “In general, 24 clusters per treatment arm is probably the minimum number for which one would even begin to consider the bootstrap in preference to traditional robust methods, for the parameter combinations investigated here.”
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- Authors
- Flynn, T. N., & Peters, T. J.
- Affiliations
- University of Bristol (GB)
- Corresponding author
- Flynn, Terry N
- Venue
- BMC Health Services Research
- Published
- 2004-11-18
- Volume
- 4
- Issue
- 1
- Pages
- 33-33
- ISSN
- 1472-6963 (ISSN-L)
- PMC
- PMC535558
- OpenAlex
- W1569909498
- Cited by
- 32
- References
- 17
- Citations by year
- 2024: 1 · 2023: 2 · 2022: 2 · 2021: 1 · 2020: 1 · 2018: 1 · 2017: 2 · 2016: 1 · 2014: 1 · 2013: 2 · 2012: 3
- FWCI
- 2.03
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- BMC Health Services Research (publishedVersion, cc-by)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- Medical Research Council
- Topics
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods and Bayesian Inference
- Social Sciences › Decision Sciences › Statistics, Probability and Uncertainty › Meta-analysis and systematic reviews
- Keywords
- bootstrapping, cost data, confidence interval coverage, intra-cluster correlation, number of clusters, skewed data, economic evaluation
- MeSH terms
- Analysis of Variance, Data Interpretation, Statistical, Humans, Models, Statistical, Cluster Analysis, Confidence Intervals, Randomized Controlled Trials as Topic, Treatment Outcome, Health Care Costs, Sample Size, Statistics, Nonparametric
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