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This short “snippet” covers three important aspects related to statistics – the concept of variables, the importance, and practical aspects related to descriptive statistics and issues related to sampling – types of sampling and sample size estimation.
Keywords: Biostatistics, descriptive statistics, sample size, variablesWhat is a variable?[1,2] To put it in very simple terms, a variable is an entity whose value varies. A variable is an essential component of any statistical data. It is a feature of a member of a given sample or population, which is unique, and can differ in quantity or quantity from another member of the same sample or population. Variables either are the primary quantities of interest or act as practical substitutes for the same. The importance of variables is that they help in operationalization of concepts for data collection. For example, if you want to do an experiment based on the severity of urticaria, one option would be to measure the severity using a scale to grade severity of itching. This becomes an operational variable. For a variable to be “good,” it needs to have some properties such as good reliability and validity, low bias, feasibility/practicality, low cost, objectivity, clarity, and acceptance. Variables can be classified into various ways as discussed below.
A variable can collect either qualitative or quantitative data. A variable differing in quantity is called a quantitative variable (e.g., weight of a group of patients), whereas a variable differing in quality is called a qualitative variable (e.g., the Fitzpatrick skin type)
A simple test which can be used to differentiate between qualitative and quantitative variables is the subtraction test. If you can subtract the value of one variable from the other to get a meaningful result, then you are dealing with a quantitative variable (this of course will not apply to rating scales/ranks).
Discrete variables are variables in which no values may be assumed between the two given values (e.g., number of lesions in each patient in a sample of patients with urticaria).
Continuous variables, on the other hand, can take any value in between the two given values (e.g., duration for which the weals last in the same sample of patients with urticaria). One way of differentiating between continuous and discrete variables is to use the “mid-way” test. If, for every pair of values of a variable, a value exactly mid-way between them is meaningful, the variable is continuous. For example, two values for the time taken for a weal to subside can be 10 and 13 min. The mid-way value would be 11.5 min which makes sense. However, for a number of weals, suppose you have a pair of values – 5 and 8 – the midway value would be 6.5 weals, which does not make sense.
Nominal/categorical variables are, as the name suggests, variables which can be slotted into different categories (e.g., gender or type of psoriasis).
Ordinal variables or ranked variables are similar to categorical, but can be put into an order (e.g., a scale for severity of itching).
In the context of an experimental study, the dependent variable (also called outcome variable) is directly linked to the primary outcome of the study. For example, in a clinical trial on psoriasis, the PASI (psoriasis area severity index) would possibly be one dependent variable. The independent variable (sometime also called explanatory variable) is something which is not affected by the experiment itself but which can be manipulated to affect the dependent variable. Other terms sometimes used synonymously include blocking variable, covariate, or predictor variable. Confounding variables are extra variables, which can have an effect on the experiment. They are linked with dependent and independent variables and can cause spurious association. For example, in a clinical trial for a topical treatment in psoriasis, the concomitant use of moisturizers might be a confounding variable. A control variable is a variable that must be kept constant during the course of an experiment.
Statistics can be broadly divided into descriptive statistics and inferential statistics.[3,4] Descriptive statistics give a summary about the sample being studied without drawing any inferences based on probability theory. Even if the primary aim of a study involves inferential statistics, descriptive statistics are still used to give a general summary. When we describe the population using tools such as frequency distribution tables, percentages, and other measures of central tendency like the mean, for example, we are talking about descriptive statistics. When we use a specific statistical test (e.g., Mann–Whitney U-test) to compare the mean scores and express it in terms of statistical significance, we are talking about inferential statistics. Descriptive statistics can help in summarizing data in the form of simple quantitative measures such as percentages or means or in the form of visual summaries such as histograms and box plots.
Descriptive statistics can be used to describe a single variable (univariate analysis) or more than one variable (bivariate/multivariate analysis). In the case of more than one variable, descriptive statistics can help summarize relationships between variables using tools such as scatter plots.
Descriptive statistics can be broadly put under two categories:
Sorting/grouping and illustration/visual displays Summary statistics.Sorting and grouping is most commonly done using frequency distribution tables. For continuous variables, it is generally better to use groups in the frequency table. Ideally, group sizes should be equal (except in extreme ends where open groups are used; e.g., age “greater than” or “less than”).
Another form of presenting frequency distributions is the “stem and leaf” diagram, which is considered to be a more accurate form of description.
Suppose the weight in kilograms of a group of 10 patients is as follows:
56, 34, 48, 43, 87, 78, 54, 62, 61, 59
The “stem” records the value of the “ten's” place (or higher) and the “leaf” records the value in the “one's” place [ Table 1 ].
Stem and leaf plot
| Stem | Leaf |
|---|---|
| 0 | - |
| 1 | - |
| 2 | - |
| 3 | 4 |
| 4 | 3 8 |
| 5 | 4 6 9 |
| 6 | 1 2 |
| 7 | 8 |
| 8 | 7 |
| 9 | - |
The most common tools used for visual display include frequency diagrams, bar charts (for noncontinuous variables) and histograms (for continuous variables). Composite bar charts can be used to compare variables. For example, the frequency distribution in a sample population of males and females can be illustrated as given in Figure 1 .
Composite bar chart
A pie chart helps show how a total quantity is divided among its constituent variables. Scatter diagrams can be used to illustrate the relationship between two variables. For example, global scores given for improvement in a condition like acne by the patient and the doctor [ Figure 2 ].
The main tools used for summary statistics are broadly grouped into measures of central tendency (such as mean, median, and mode) and measures of dispersion or variation (such as range, standard deviation, and variance).
Imagine that the data below represent the weights of a sample of 15 pediatric patients arranged in ascending order:
30, 35, 37, 38, 38, 38, 42, 42, 44, 46, 47, 48, 51, 53, 86
Just having the raw data does not mean much to us, so we try to express it in terms of some values, which give a summary of the data.
The mean is basically the sum of all the values divided by the total number. In this case, we get a value of 45.
The problem is that some extreme values (outliers), like “'86,” in this case can skew the value of the mean. In this case, we consider other values like the median, which is the point that divides the distribution into two equal halves. It is also referred to as the 50 th percentile (50% of the values are above it and 50% are below it). In our previous example, since we have already arranged the values in ascending order we find that the point which divides it into two equal halves is the 8 th value – 42. In case of a total number of values being even, we choose the two middle points and take an average to reach the median.
The mode is the most common data point. In our example, this would be 38. The mode as in our case may not necessarily be in the center of the distribution.
The median is the best measure of central tendency from among the mean, median, and mode. In a “symmetric” distribution, all three are the same, whereas in skewed data the median and mean are not the same; lie more toward the skew, with the mean lying further to the skew compared with the median. For example, in Figure 3 , a right skewed distribution is seen (direction of skew is based on the tail); data values' distribution is longer on the right-hand (positive) side than on the left-hand side. The mean is typically greater than the median in such cases.
Location of mode, median, and mean
The range gives the spread between the lowest and highest values. In our previous example, this will be 86-30 = 56.
A more valuable measure is the interquartile range. A quartile is one of the values which break the distribution into four equal parts. The 25 th percentile is the data point which divides the group between the first one-fourth and the last three-fourth of the data. The first one-fourth will form the first quartile. The 75 th percentile is the data point which divides the distribution into a first three-fourth and last one-fourth (the last one-fourth being the fourth quartile). The range between the 25 th percentile and 75 th percentile is called the interquartile range.
Variance is also a measure of dispersion. The larger the variance, the further the individual units are from the mean. Let us consider the same example we used for calculating the mean. The mean was 45.
For the first value (30), the deviation from the mean will be 15; for the last value (86), the deviation will be 41. Similarly we can calculate the deviations for all values in a sample. Adding these deviations and averaging will give a clue to the total dispersion, but the problem is that since the deviations are a mix of negative and positive values, the final total becomes zero. To calculate the variance, this problem is overcome by adding squares of the deviations. So variance would be the sum of squares of the variation divided by the total number in the population (for a sample we use “n − 1”). To get a more realistic value of the average dispersion, we take the square root of the variance, which is called the “standard deviation.”
The box plot is a composite representation that portrays the mean, median, range, and the outliers [ Figure 4 ].
Skewness is a measure of the symmetry of distribution. Basically if the distribution curve is symmetric, it looks the same on either side of the central point. When this is not the case, it is said to be skewed. Kurtosis is a representation of outliers. Distributions with high kurtosis tend to have “heavy tails” indicating a larger number of outliers, whereas distributions with low kurtosis have light tails, indicating lesser outliers. There are formulas to calculate both skewness and kurtosis [Figures [Figures5 5 – 8 ].
High kurtosis (positive kurtosis – also called leptokurtic)
Low kurtosis (negative kurtosis – also called “Platykurtic”)
In an ideal study, we should be able to include all units of a particular population under study, something that is referred to as a census.[5,6] This would remove the chances of sampling error (difference between the outcome characteristics in a random sample when compared with the true population values – something that is virtually unavoidable when you take a random sample). However, it is obvious that this would not be feasible in most situations. Hence, we have to study a subset of the population to reach to our conclusions. This representative subset is a sample and we need to have sufficient numbers in this sample to make meaningful and accurate conclusions and reduce the effect of sampling error.
We also need to know that broadly sampling can be divided into two types – probability sampling and nonprobability sampling. Examples of probability sampling include methods such as simple random sampling (each member in a population has an equal chance of being selected), stratified random sampling (in nonhomogeneous populations, the population is divided into subgroups – followed be random sampling in each subgroup), systematic (sampling is based on a systematic technique – e.g., every third person is selected for a survey), and cluster sampling (similar to stratified sampling except that the clusters here are preexisting clusters unlike stratified sampling where the researcher decides on the stratification criteria), whereas nonprobability sampling, where every unit in the population does not have an equal chance of inclusion into the sample, includes methods such as convenience sampling (e.g., sample selected based on ease of access) and purposive sampling (where only people who meet specific criteria are included in the sample).
An accurate calculation of sample size is an essential aspect of good study design. It is important to calculate the sample size much in advance, rather than have to go for post hoc analysis. A sample size that is too less may make the study underpowered, whereas a sample size which is more than necessary might lead to a wastage of resources.
We will first go through the sample size calculation for a hypothesis-based design (like a randomized control trial).
The important factors to consider for sample size calculation include study design, type of statistical test, level of significance, power and effect size, variance (standard deviation for quantitative data), and expected proportions in the case of qualitative data. This is based on previous data, either based on previous studies or based on the clinicians' experience. In case the study is something being conducted for the first time, a pilot study might be conducted which helps generate these data for further studies based on a larger sample size). It is also important to know whether the data follow a normal distribution or not.
Two essential aspects we must understand are the concept of Type I and Type II errors. In a study that compares two groups, a null hypothesis assumes that there is no significant difference between the two groups, and any observed difference being due to sampling or experimental error. When we reject a null hypothesis, when it is true, we label it as a Type I error (also denoted as “alpha,” correlating with significance levels). In a Type II error (also denoted as “beta”), we fail to reject a null hypothesis, when the alternate hypothesis is actually true. Type II errors are usually expressed as “1- β,” correlating with the power of the test. While there are no absolute rules, the minimal levels accepted are 0.05 for α (corresponding to a significance level of 5%) and 0.20 for β (corresponding to a minimum recommended power of “1 − 0.20,” or 80%).
For a clinical trial, the investigator will have to decide in advance what clinically detectable change is significant (for numerical data, this is could be the anticipated outcome means in the two groups, whereas for categorical data, it could correlate with the proportions of successful outcomes in two groups.). While we will not go into details of the formula for sample size calculation, some important points are as follows:
In the context where effect size is involved, the sample size is inversely proportional to the square of the effect size. What this means in effect is that reducing the effect size will lead to an increase in the required sample size.
Reducing the level of significance (alpha) or increasing power (1-β) will lead to an increase in the calculated sample size.
An increase in variance of the outcome leads to an increase in the calculated sample size.
A note is that for estimation type of studies/surveys, sample size calculation needs to consider some other factors too. This includes an idea about total population size (this generally does not make a major difference when population size is above 20,000, so in situations where population size is not known we can assume a population of 20,000 or more). The other factor is the “margin of error” – the amount of deviation which the investigators find acceptable in terms of percentages. Regarding confidence levels, ideally, a 95% confidence level is the minimum recommended for surveys too. Finally, we need an idea of the expected/crude prevalence – either based on previous studies or based on estimates.
Sample size calculation also needs to add corrections for patient drop-outs/lost-to-follow-up patients and missing records. An important point is that in some studies dealing with rare diseases, it may be difficult to achieve desired sample size. In these cases, the investigators might have to rework outcomes or maybe pool data from multiple centers. Although post hoc power can be analyzed, a better approach suggested is to calculate 95% confidence intervals for the outcome and interpret the study results based on this.
