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Precision Based Sample Size Calculation

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CTU-Bern/presize

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Bland (2009) recommended tobase study sizes on the width of the confidence interval rather thepower of a statistical test. The goal ofpresize is to providefunctions for such precision based sample size calculations. For a givensample size, the functions will return the precision (width of theconfidence interval), and vice versa.

Installation

presize can be installed from CRAN in the usual manner:

install.packages("presize")

You can install the development version ofpresize with:

install.packages('presize',repos= c('https://ctu-bern.r-universe.dev','https://cloud.r-project.org'))

Overview

presize provides functions for

MeasureFunctionMethods available
Descriptive measures
Meanprec_mean
Proportionprec_propWilson, Agresti-Coull, exact, Wald (see Brown, Cai, and DasGupta 2001)
Rateprec_rateScore, variance stabilizing, exact, Wald (see Barker 2002)
Absolute differences
Mean differenceprec_meandiff
Risk differenceprec_riskdiffNewcombe (Newcombe 1998), Miettinen-Nurminen (Miettinen and Nurminen 1985), Agresti-Caffo (Agresti and Caffo 2000), Wald
Relative differences
Odds ratioprec_orGart, Wolff, independence smoothed logit (see Fagerland, Lydersen, and Laake 2015)
Risk ratioprec_riskratioKoopman (Koopman 1984), Katz (Katz et al. 1978)
Rate ratioprec_rateratioRothman (Rothman and Greenland 2018)
Correlation measures
Correlation coefficientprec_corPearson, Kendall, Spearman (see Bonnett and Wright 2000)
Intraclass correlationprec_iccBonnett (2002)
Limit of agreementprec_lim_agreeBland and Altman (1986)
Cohen’s kappaprec_kappaRotondi and Donner (2012)
Cronbach’s alphaprec_cronbBonett and Wright (2015)
Diagnostic measures
Sensitivity1prec_sensAs perprec_prop
Specificity1prec_specAs perprec_prop
Area under the curveprec_aucHanley and McNeil (1982)
Negative likelihood ratio2prec_neg_lrSimel, Samsa, and Matchar (1991)
Positive likelihood ratio2prec_pos_lrSimel, Samsa, and Matchar (1991)
Generic likelihood ratioprec_lrSimel, Samsa, and Matchar (1991)

1 Simple wrappers forprec_prop.

2 Wrappers forprec_lr with values provided via sens andspec

Example

Suppose we want to estimate the proportion of hospital admissions withdiabetes. Diabetes has a prevalence of approximately 10% (Emerging RiskFactors Collaboration et al. (2010)). We assume a slightly higherproportion of diabetics, 15%, as diabetes is a risk factor for a widerange of conditions. We want to estimate the prevalence of diabetes towithin 5% (plus/minus 2.5%). Withpresize, this is simple. We use theprec_prop (precision of a proportion) function and pass our 15% and 5%as argumentsp andconf.width:

library(presize)# load the packageprec_prop(p=0.15,conf.width=0.05)#> Warning in prec_prop(p = 0.15, conf.width = 0.05): more than one method was#> chosen, 'wilson' will be used#>#>      sample size for a proportion with Wilson confidence interval.#>#>      p      padj        n conf.width conf.level       lwr       upr#> 1 0.15 0.1517077 783.4897       0.05       0.95 0.1267077 0.1767077#>#> NOTE: padj is the adjusted proportion, from which the ci is calculated.

In the n column, we see that we would need to ask 784 (rounding 783.5up) patients to achieve the desired CI width. Disappointingly, we alsoknow that we only have funds to collect the data from 600 patients. Wewonder if 600 patients would yield sufficient precision - we could alsoaccept a CI width of 6% (plus/minus 3%). In such a case, we can pass theargumentsp andn.

prec_prop(p=0.15,n=600)#> Warning in prec_prop(p = 0.15, n = 600): more than one method was chosen,#> 'wilson' will be used#>#>      precision for a proportion with Wilson confidence interval.#>#>      p      padj   n conf.width conf.level       lwr       upr#> 1 0.15 0.1522266 600 0.05713404       0.95 0.1236596 0.1807936#>#> NOTE: padj is the adjusted proportion, from which the ci is calculated.

Now we see that with 600 patients, the CI would have a width of 5.7%. Weare happy with this and continue planning our study with those values.All of the functions listed in Table 1 can be used similarly.

We can also look at a range of scenarios simulatenously by passing avector to one of the arguments, which could be used to create somethinganalogous to a power curve:

prec_prop(p=0.15,n= seq(600,800,50))#> Warning in prec_prop(p = 0.15, n = seq(600, 800, 50)): more than one method was#> chosen, 'wilson' will be used#>#>      precision for a proportion with Wilson confidence interval.#>#>      p      padj   n conf.width conf.level       lwr       upr#> 1 0.15 0.1522266 600 0.05713404       0.95 0.1236596 0.1807936#> 2 0.15 0.1520563 650 0.05489329       0.95 0.1246097 0.1795030#> 3 0.15 0.1519102 700 0.05289705       0.95 0.1254617 0.1783588#> 4 0.15 0.1517835 750 0.05110386       0.95 0.1262316 0.1773355#> 5 0.15 0.1516726 800 0.04948148       0.95 0.1269319 0.1764133#>#> NOTE: padj is the adjusted proportion, from which the ci is calculated.

Shiny app

An online interactive version of the package is availablehere. The app can also be launchedlocally vialaunch_presize_app() in RStudio.

Getting help

The package website, including more details on the functions, can befoundhere.

If you have a question, feel free to make a thread on thediscussion page.

If you encounter a bug, please create anissue.

Contributing

Contributions topresize are welcome. If you have ideas, open anissue or adiscussionthread on GitHub.

If you want to contribute code, please feel free to fork the repository,make your changes and make a pull request to have them integrated intothe package. New functionality should have accompanying tests and passcontinuous integration. See also thecontributingguidelines.

Funding

presize was largely developed at CTU Bern, with collaboration from CTUBasel. Funding was provided by the Swiss Clinical Trial Organisation.

CitationDOI

If you usepresize, please cite it in your publication as:
Haynes et al., (2021). presize: An R-package for precision-based samplesize calculation in clinical research. Journal of Open Source Software,6(60), 3118,https://doi.org/10.21105/joss.03118

Acknowledgements

The package logo was created withggplot2 andhexSticker with iconsfromFont Awesome (via theemojifontpackage).

References

Agresti, A, and B Caffo. 2000. “Simple and Effective ConfidenceIntervals for Proportions and Differences of Proportions Result fromAdding Two Successes and Two Failures.”The Americal Statistician 54(4): 280–88.https://doi.org/10.2307/2685779.

Barker, L. 2002. “A Comparison of Nine Confidence Intervals for aPoisson Parameter When the Expected Number of Events Is ≤ 5.”TheAmerical Statistician 56 (2): 85–89.https://doi.org/10.1198/000313002317572736.

Bland, J M, and D G Altman. 1986. “Statistical Methods for AssessingAgreement Between Two Methods of Clinical Measurement.”Lanceti(8476): 307–10.https://doi.org/10.1016/S0140-6736(86)90837-8.

Bonett, Douglas G., and Thomas A. Wright. 2015. “Cronbach’s AlphaReliability: Interval Estimation, Hypothesis Testing, and Sample SizePlanning.”Journal of Organizational Behavior 36 (1): 3–15.https://doi.org/https://doi.org/10.1002/job.1960.

Bonnett, D G. 2002. “Sample Size Requirements for Estimating IntraclassCorrelations with Desired Precision.”Statistics in Medicine 21:1331–5.https://doi.org/10.1002/sim.1108.

Bonnett, D G, and T A Wright. 2000. “Sample Size Requirements forEstimating Pearson, Kendall and Spearman Correlations.”Psychometrika65: 23–28.https://doi.org/10.1007/BF02294183.

Brown, L D, T T Cai, and A DasGupta. 2001. “Interval Estimation for aBinomial Proportion.”Statistical Science 16 (2): 101–17.https://doi.org/10.1214/ss/1009213286.

Emerging Risk Factors Collaboration, N Sarwar, P Gao, S R Seshasai, RGobin, S Kaptoge, E Di Angelantonio, et al. 2010. “Diabetes Mellitus,Fasting Blood Glucose Concentration, and Risk of Vascular Disease: ACollaborative Meta-Analysis of 102 Prospective Studies.”Lancet 375(9733): 2215–22.https://doi.org/10.1016/S0140-6736(10)60484-9.

Fagerland, M W, S Lydersen, and P Laake. 2015. “Recommended ConfidenceIntervals for Two Independent Binomial Proportions.”StatisticalMethods in Medical Research 24 (2): 224–54.https://doi.org/10.1177/0962280211415469.

Hanley, J A, and B J McNeil. 1982. “The Meaning and Use of the AreaUnder a Receiver Operating Characteristic (Roc) Curve.”Radiology 148:29–36.https://doi.org/10.1148/radiology.143.1.7063747.

Katz, D, J Baptista, S P Azen, and M C Pike. 1978. “Obtaining ConfidenceIntervals for the Risk Ratio in Cohort Studies.”Biometrics 34:469–74.https://doi.org/10.2307/2530610.

Koopman, P A R. 1984. “Confidence Intervals for the Ratio of TwoBinomial Proportions.”Biometrics 40: 513–17.https://doi.org/10.2307/2531551.

Miettinen, O, and M Nurminen. 1985. “Comparative Analysis of Two Rates.”Statistics in Medicine 4: 213–26.https://doi.org/10.1002/sim.4780040211.

Newcombe, R G. 1998. “Interval Estimation for the Difference BetweenIndependent Proportions: Comparison of Eleven Methods.”Statistics inMedicine 17: 873–90.https://doi.org/10.1002/(sici)1097-0258(19980430)17:8<873::aid-sim779>3.0.co;2-i.

Rothman, K J, and S Greenland. 2018. “Planning Study Size Based onPrecision Rather Than Power.”Epidemiology 29: 599–603.https://doi.org/10.1097/EDE.0000000000000876.

Rotondi, M A, and A Donner. 2012. “A Confidence Interval Approach toSample Size Estimation for Interobserver Agreement Studies with MultipleRaters and Outcomes.”Journal of Clinical Epidemiology 65: 778–84.https://doi.org/10.1016/j.jclinepi.2011.10.019.

Simel, D L, G P Samsa, and D B Matchar. 1991. “Likelihood Ratios withConfidence: Sample Size Estimation for Diagnostic Test Studies.”Journal of Clinical Epidemiology 44 (8): 763–70.https://doi.org/10.1016/0895-4356(91)90128-v.


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