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bootf2 (version 0.4.1)

bootf2: Estimate 90% Confidence Intervals of \(f_2\) with Bootstrap Methodology

Description

Main function to estimate 90% confidence intervals of \(f_2\) using bootstrap methodology.

Usage

bootf2(test, ref, path.in, file.in, path.out, file.out,
       n.boots = 10000L, seed = 306L, digits = 2L, alpha = 0.05,
       regulation = c("EMA", "FDA", "WHO","Canada", "ANVISA"),
       min.points = 1L, both.TR.85 = FALSE, print.report = TRUE,
       report.style = c("concise", "intermediate", "detailed"),
       f2.type = c("all", "est.f2", "exp.f2", "bc.f2",
                   "vc.exp.f2", "vc.bc.f2"),
       ci.type = c("all", "normal", "basic", "percentile",
                   "bca.jackknife", "bca.boot"),
       quantile.type = c("all", as.character(1:9), "boot"),
       jackknife.type = c("all", "nt+nr", "nt*nr", "nt=nr"),
       time.unit = c("min", "h"), output.to.screen = FALSE,
       sim.data.out = FALSE)

Arguments

test, ref

Data frames of dissolution profiles of test and reference product if path.in and file.in are not specified; otherwise, they should be character strings indicating the worksheet names of the Excel file where the dissolution data is saved. See Input/Output in Details.

path.in, file.in, path.out, file.out

Character strings of input and output directories and file names. See Input/Output in Details.

n.boots

An integer indicating the number of bootstrap samples.

seed

Integer seed value for reproducibility. If missing, a random seed will be generated for reproducibility purpose.

digits

An integer indicating the decimal points for the output.

alpha

A numeric value between 0 and 1 to estimate \((1-2\times \alpha)\times 100\) confidence interval.

regulation

Character strings indicating regulatory guidelines. @seealso calcf2() for details on regulation rules.

min.points

An integer indicating the minimum time points to be used to calculate \(f_2\). For conventional \(f_2\) calculation, the default is 3, however, for bootstrap \(f_2\), the value should be lower as there might be less time points available in certain bootstrap samples. The default is 1. @seealso calcf2().

both.TR.85

Logical. If TRUE, and if regulation = "FDA", all measurements up to the time points at which both test and reference products dissolve more than 85% will be used to calculate \(f_2\). This is the conventional, but incorrect, interpretation of the US FDA rule. Therefore, the argument should only be set to TRUE for validation purpose such as comparing the results from old literature that use the wrong interpretation to calculate \(f_2\). @seealso calcf2() for details on regulation rules.

print.report

Logical. If TRUE, a plain text report will be produced. See Input/Output in Details.

report.style

"concise" style produces the estimators and their confidence intervals; "intermediate" style adds a list of individual \(f_2\)s for all bootstrap samples in the end of "concise" report; "detailed" style further adds individual bootstrap samples along with their \(f_2\)s in the end of "intermediate" report. See Input/Output in Details.

f2.type

Character strings indicating which type of \(f_2\) estimator should be calculated. See Types of estimators in Details.

ci.type

Character strings indicating which type of confidence interval should be estimated. See Types of confidence intervals in Details.

quantile.type

Character strings indicating the type of percentile.

jackknife.type

Character strings indicating the type of jackknife method. See Details.

time.unit

Character strings indicating the unit of time. It should be either "min" for minute or "h" for hour. It is mainly used for checking CV rules and making plot. @seealso calcf2().

output.to.screen

Logical. If TRUE, a "concise" style summary report will be printed on screen. See Input/Output in Details.

sim.data.out

Logical. If TRUE, all individual bootstrap data sets will be included in the output.

Value

A list of 3 or 5 components.

  • boot.ci: A data frame of bootstrap \(f_2\) confidence intervals.

  • boot.f2: A data frame of all individual \(f_2\) values for all bootstrap data set.

  • boot.info: A data frame with detailed information of bootstrap for reproducibility purpose, such as all arguments used in the function, time points used for calculation of \(f_2\), and the number of NAs.

  • boot.summary: A data frame with descriptive statistics of the bootstrap \(f_2\).

  • boot.t and boot.r: Lists of individual bootstrap samples for test and reference product if sim.data.out = TRUE.

Details

Minimum required arguments that must be provided by the user

Arguments test and ref must be provided by the user. They should be R data frames, with time as the first column, and all individual profiles profiles as the rest columns. The actual names of the columns do not matter since they will be renamed internally.

Input/Output

The dissolution data of test and reference product can either be provided as data frames for test and ref, as explained above, or be read from an Excel file with data of test and reference stored in separate worksheets. In the latter case, the argument path.in, the directory where the Excel file is, and file.in, the name of the Excel file including the file extension .xls or .xlsx, must be provided. In such case, the argument test and ref must be the names of the worksheets in quotation marks. The first column of each Excel worksheet must be time, and the rest columns are individual dissolution profiles. The first row should be column names, such as time, unit01, unit02, ... The actual names of the columns do not matter as they will be renamed internally.

Arguments path.out and file.out are the names of the output directory and file. If they are not provided, but argument print.report is TRUE, then a plain text report will be generated automatically in the current working directory with file name test_vs_ref_TZ_YYYY-MM-DD_HHMMSS.txt, where test and ref are data set names of test and reference, TZ is the time zone such as CEST, YYYY-MM-DD is the numeric date format and HHMMSS is the numeric time format for hour, minute, and second.

For a quick check, set argument output.to.screen = TRUE, a summary report very similar to concise style report will be printed on screen.

Types of Estimators

According to Shah et al, the population \(f_2\) for the inference is $$f_2 = 100-25\log\left(1 + \frac{1}{P}\sum_{i=1}^P% \left(\mu_{\mathrm{T},i} - \mu_{\mathrm{R},i}\right)^2 \right)\,,$$ where \(P\) is the number of time points; \(\mu_{\mathrm{T},i}\) and \(\mu_{\mathrm{R},i}\) are population mean of test and reference product at time point \(i\), respectively; \(\sum_{i=1}^P\) is the summation from \(i = 1\) to \(P\).

Five estimators for \(f_2\) are included in the function:

  1. The estimated \(f_2\), denoted by \(\hat{f}_2\), is the one written in various regulatory guidelines. It is expressed differently, but mathematically equivalently, as $$\hat{f}_2 = 100-25\log\left(1 + \frac{1}{P}\sum_{i=1}^P\left(% \bar{X}_{\mathrm{T},i} - \bar{X}_{\mathrm{R},i}\right)^2 \right)\:,$$ where \(P\) is the number of time points; \(\bar{X}_{\mathrm{T},i}\) and \(\bar{X}_{\mathrm{R},i}\) are mean dissolution data at the \(i\)th time point of random samples chosen from the test and the reference population, respectively. Compared to the equation of population \(f_2\) above, the only difference is that in the equation of \(\hat{f}_2\) the sample means of dissolution profiles replace the population means for the approximation. In other words, a point estimate is used for the statistical inference in practice.

  2. The Bias-corrected \(f_2\), denoted by \(\hat{f}_{2,\mathrm{bc}}\), was described in the article of Shah et al, as $$\hat{f}_{2,\mathrm{bc}} = 100-25\log\left(1 + \frac{1}{P}% \left(\sum_{i=1}^P\left(\bar{X}_{\mathrm{T},i} - % \bar{X}_{\mathrm{R},i}\right)^2 - \frac{1}{n}\sum_{i=1}^P% \left(S_{\mathrm{T},i}^2 + S_{\mathrm{R},i}^2\right)\right)\right)\,,$$ where \(S_{\mathrm{T},i}^2\) and \(S_{\mathrm{R},i}^2\) are unbiased estimates of variance at the \(i\)th time points of random samples chosen from test and reference population, respectively; and \(n\) is the sample size.

  3. The variance- and bias-corrected \(f_2\), denoted by \(\hat{f}_{2,\mathrm{vcbc}}\), does not assume equal weight of variance as \(\hat{f}_{2,\mathrm{bc}}\) does. $$\hat{f}_{2, \mathrm{vcbc}} = 100-25\log\left(1 +% \frac{1}{P}\left(\sum_{i=1}^P \left(\bar{X}_{\mathrm{T},i} -% \bar{X}_{\mathrm{R},i}\right)^2 - \frac{1}{n}\sum_{i=1}^P% \left(w_{\mathrm{T},i}\cdot S_{\mathrm{T},i}^2 +% w_{\mathrm{R},i}\cdot S_{\mathrm{R},i}^2\right)\right)\right)\,,$$ where \(w_{\mathrm{T},i}\) and \(w_{\mathrm{R},i}\) are weighting factors for variance of test and reference products, respectively, which can be calculated as follows: $$w_{\mathrm{T},i} = 0.5 + \frac{S_{\mathrm{T},i}^2}% {S_{\mathrm{T},i}^2 + S_{\mathrm{R},i}^2}\,,$$ and $$w_{\mathrm{R},i} = 0.5 + \frac{S_{\mathrm{R},i}^2}% {S_{\mathrm{T},i}^2 + S_{\mathrm{R},i}^2}\,.$$

  4. The expected \(f_2\), denoted by \(\hat{f}_{2, \mathrm{exp}}\), is calculated based on the mathematical expectation of estimated \(f_2\), $$\hat{f}_{2, \mathrm{exp}} = 100-25\log\left(1 + \frac{1}{P}% \left(\sum_{i=1}^P\left(\bar{X}_{\mathrm{T},i} - % \bar{X}_{\mathrm{R},i}\right)^2 + \frac{1}{n}\sum_{i=1}^P% \left(S_{\mathrm{T},i}^2 + S_{\mathrm{R},i}^2\right)\right)\right)\,,$$ using mean dissolution profiles and variance from samples for the approximation of population values.

  5. The variance-corrected expected \(f_2\), denoted by \(\hat{f}_{2, \mathrm{vcexp}}\), is calculated as $$\hat{f}_{2, \mathrm{vcexp}} = 100-25\log\left(1 +% \frac{1}{P}\left(\sum_{i=1}^P \left(\bar{X}_{\mathrm{T},i} -% \bar{X}_{\mathrm{R},i}\right)^2 + \frac{1}{n}\sum_{i=1}^P% \left(w_{\mathrm{T},i}\cdot S_{\mathrm{T},i}^2 +% w_{\mathrm{R},i}\cdot S_{\mathrm{R},i}^2\right)\right)\right)\,.$$

Types of Confidence Interval

The following confidence intervals are included:

  1. The Normal interval with bias correction, denoted by normal in the function, is estimated according to Davison and Hinkley, $$\hat{f}_{2, \mathrm{L,U}} = \hat{f}_{2, \mathrm{S}} - E_B \mp % \sqrt{V_B}\cdot Z_{1-\alpha}\,,$$ where \(\hat{f}_{2, \mathrm{L,U}}\) are the lower and upper limit of the confidence interval estimated from bootstrap samples; \(\hat{f}_{2, \mathrm{S}}\) denotes the estimators described above; \(Z_{1-\alpha}\) represents the inverse of standard normal cumulative distribution function with type I error \(\alpha\); \(E_B\) and \(V_B\) are the resampling estimates of bias and variance calculated as $$E_B = \frac{1}{B}\sum_{b=1}^{B}\hat{f}_{2,b}^\star - % \hat{f}_{2, \mathrm{S}} = \bar{f}_2^\star - \hat{f}_{2,\mathrm{S}}\,,$$ and $$V_B = \frac{1}{B-1}\sum_{b=1}^{B} \left(\hat{f}_{2,b}^\star -\bar{f}_2^\star\right)^2\,,$$ where \(B\) is the number of bootstrap samples; \(\hat{f}_{2,b}^\star\) is the \(f_2\) estimate with \(b\)th bootstrap sample, and \(\bar{f}_2^\star\) is the mean value.

  2. The basic interval, denoted by basic in the function, is estimated according to Davison and Hinkley, $$\hat{f}_{2, \mathrm{L}} = 2\hat{f}_{2, \mathrm{S}} -% \hat{f}_{2,(B+1)(1-\alpha)}^\star\,,$$ and $$\hat{f}_{2, \mathrm{U}} = 2\hat{f}_{2, \mathrm{S}} -% \hat{f}_{2,(B+1)\alpha}^\star\,,$$ where \(\hat{f}_{2,(B+1)\alpha}^\star\) and \(\hat{f}_{2,(B+1)(1-\alpha)}^\star\) are the \((B+1)\alpha\)th and \((B+1)(1-\alpha)\)th ordered resampling estimates of \(f_2\), respectively. When \((B+1)\alpha\) is not an integer, the following equation is used for interpolation, $$\hat{f}_{2,(B+1)\alpha}^\star = \hat{f}_{2,k}^\star + % \frac{\Phi^{-1}\left(\alpha\right)-\Phi^{-1}\left(\frac{k}{B+1}\right)}% {\Phi^{-1}\left(\frac{k+1}{B+1}\right)-\Phi^{-1}% \left(\frac{k}{B+1}\right)}\left(\hat{f}_{2,k+1}^\star-% \hat{f}_{2,k}^\star\right),$$ where \(k\) is the integer part of \((B+1)\alpha\), \(\hat{f}_{2,k+1}^\star\) and \(\hat{f}_{2,k}^\star\) are the \((k+1)\)th and the \(k\)th ordered resampling estimates of \(f_2\), respectively.

  3. The percentile intervals, denoted by percentile in the function, are estimated using nine different types of quantiles, Type 1 to Type 9, as summarized in Hyndman and Fan's article and implemented in R's quantile function. Using R's boot package, program bootf2BCA outputs a percentile interval using the equation above for interpolation. To be able to compare the results among different programs, the same interval, denoted by Percentile (boot) in the function, is also included in the function.

  4. The bias-corrected and accelerated (BCa) intervals are estimated according to Efron and Tibshirani, $$\hat{f}_{2, \mathrm{L}} = \hat{f}_{2, \alpha_1}^\star\,,$$ $$\hat{f}_{2, \mathrm{U}} = \hat{f}_{2, \alpha_2}^\star\,,$$ where \(\hat{f}_{2, \alpha_1}^\star\) and \(\hat{f}_{2, \alpha_2}^\star\) are the \(100\alpha_1\)th and the \(100\alpha_2\)th percentile of the resampling estimates of \(f_2\), respectively. Type I errors \(\alpha_1\) and \(\alpha_2\) are obtained as $$\alpha_1 = \Phi\left(\hat{z}_0 + \frac{\hat{z}_0 + \hat{z}_\alpha}% {1-\hat{a}\left(\hat{z}_0 + \hat{z}_\alpha\right)}\right),$$ and $$\alpha_2 = \Phi\left(\hat{z}_0 + \frac{\hat{z}_0 + % \hat{z}_{1-\alpha}}{1-\hat{a}\left(\hat{z}_0 + % \hat{z}_{1-\alpha}\right)}\right),$$ where \(\hat{z}_0\) and \(\hat{a}\) are called bias-correction and acceleration, respectively.

    There are different methods to estimate \(\hat{z}_0\) and \(\hat{a}\). Shah et al. used jackknife technique, denoted by bca.jackknife in the function, $$\hat{z}_0 = \Phi^{-1}\left(\frac{N\left\{\hat{f}_{2,b}^\star <% \hat{f}_{2,\mathrm{S}} \right\}}{B}\right),$$ and $$\hat{a} = \frac{\sum_{i=1}^{n}\left(\hat{f}_{2,\mathrm{m}} -% \hat{f}_{2, i}\right)^3}{6\left(\sum_{i=1}^{n}\left(% \hat{f}_{2,\mathrm{m}} - \hat{f}_{2, i}\right)^2\right)^{3/2}}\,,$$ where \(N\left\{\cdot\right\}\) denotes the number of element in the set, \(\hat{f}_{2, i}\) is the \(i\)th jackknife statistic, \(\hat{f}_{2,\mathrm{m}}\) is the mean of the jackknife statistics, and \(\sum\) is the summation from 1 to sample size \(n\).

    Program bootf2BCA gives a slightly different BCa interval with R's boot package. This approach, denoted by bca.boot in the function, is also implemented in the function for estimating the interval.

Notes on the argument jackknife.type

For any sample with size \(n\), the jackknife estimator is obtained by estimating the parameter for each subsample omitting the \(i\)th observation. However, when two samples (e.g., test and reference) are involved, there are several possible ways to do it. Assuming sample size of test and reference are \(n_\mathrm{T}\) and \(n_\mathrm{R}\), the following three possibility are considered:

  • Estimated by removing one observation from both test and reference samples. In this case, the prerequisite is \(n_\mathrm{T} = n_\mathrm{R}\), denoted by nt=nr in the function. So if there are 12 units in test and reference data sets, there will be 12 jackknife estimators.

  • Estimate the jackknife for test sample while keeping the reference data unchanged; and then estimate jackknife for reference sample while keeping the test sample unchanged. This is denoted by nt+nr in the function. This is the default method. So if there are 12 units in test and reference data sets, there will be \(12 + 12 = 24\) jackknife estimators.

  • For each observation deleted from test sample, estimate jackknife for reference sample. This is denoted by nt*nr in the function. So if there are 12 units in test and reference data sets, there will be \(12 \times 12 = 144\) jackknife estimators.

References

Shah, V. P.; Tsong, Y.; Sathe, P.; Liu, J.-P. In Vitro Dissolution Profile Comparison---Statistics and Analysis of the Similarity Factor, \(f_2\). Pharmaceutical Research 1998, 15 (6), 889--896. DOI: 10.1023/A:1011976615750.

Davison, A. C.; Hinkley, D. V. Bootstrap Methods and Their Application. Cambridge University Press, 1997.

Hyndman, R. J.; Fan, Y. Sample Quantiles in Statistical Packages. The American Statistician 1996, 50 (4), 361--365. DOI: /10.1080/00031305.1996.10473566.

Efron, B.; Tibshirani, R. An Introduction to the Bootstrap. Chapman & Hall, 1993.

Examples

Run this code
# NOT RUN {
# time points
tp <- c(5, 10, 15, 20, 30, 45, 60)
# model.par for reference with low variability
par.r <- list(fmax = 100, fmax.cv = 3, mdt = 15, mdt.cv = 14,
              tlag = 0, tlag.cv = 0, beta = 1.5, beta.cv = 8)
# simulate reference data
dr <- sim.dp(tp, model.par = par.r, seed = 100, plot = FALSE)
# model.par for test
par.t <- list(fmax = 100, fmax.cv = 3, mdt = 12.29, mdt.cv = 12,
              tlag = 0, tlag.cv = 0, beta = 1.727, beta.cv = 9)
# simulate test data with low variability
dt <- sim.dp(tp, model.par = par.t, seed = 100, plot = FALSE)

# bootstrap. to reduce test run time, n.boots of 100 was used in the example.
# In practice, it is recommended to use n.boots of 5000--10000.
# Set `output.to.screen = TRUE` to view the result on screen
d <- bootf2(dt$sim.disso, dr$sim.disso, n.boots = 100, print.report = FALSE)


# }

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