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    Statistical Methods Guide

    Concepts, procedure selection, required inputs, and formulae for planning sample size and statistical power

    Core Concepts

    Review the statistical choices and assumptions that shape a defensible sample-size plan.

    Common Planning Pitfalls

    • Using unrealistic effect sizes (too optimistic)
    • Ignoring dropout/attrition rates
    • Not accounting for unequal group sizes
    • Forgetting to adjust for multiple comparisons
    • Using wrong test for study design
    • Inadequate pilot data for parameter estimates
    Already know your variables? Jump to the procedure finder

    Statistical Analysis Framework

    Understanding the fundamentals of statistical analysis and sample size determination

    Sample Size Determinants

    Questions to be answered to know how to calculate the sample size:

    1

    Type of statistical analysis:

    Descriptive
    or
    Analytical
    2

    Type of analytical statistics:

    Estimation
    or
    Hypothesis testing
    3

    Type of the Outcome variable and the risk factor:

    Numerical
    or
    Categorical

    Areas of Statistics

    There are two main areas of Statistics:

    Descriptive statistics

    Provides tabular and graphical techniques and numerical measures for describing data.

    Analytical (Inferential) statistics

    Provides procedures for analyzing data and making decisions. Using the sample to infer about the population (parameter).

    Types of Inferential Statistics

    Estimation

    The answer for the research question is a value or a range of values (Confidence interval)

    Example:

    "What is the prevalence of Diabetes in a certain population?"

    Hypothesis testing

    The data may provide enough evidence to reject the null hypothesis; otherwise, fail to reject it. A non-significant result does not prove the null is true.

    Example:

    "Is there a significant difference in the prevalence of Diabetes between males and females in a certain population?"

    Types of Variables

    Understanding variable types is crucial for selecting the appropriate statistical test

    Variables can be classified into two main categories:

    Qualitative (Categorical)

    Labels or names used to identify attributes of the sample elements. The labels can be numbers with no real numerical meaning.

    Binary
    2 categories

    It has only two categories or levels

    • Smoking status: yes or no
    • Survival status: alive or dead
    • Diabetes status: diabetic or non-diabetic
    Appropriate Tests:
    • • Chi-Square Test
    • • Fisher's Exact Test
    • • McNemar's Test (paired)
    • • One/Two-Proportion Z-Test
    • • Logistic Regression
    • • Odds Ratio (case-control)
    • • Relative Risk (cohort)
    Nominal
    No ranking

    The order of the categories is not essential (no ranking)

    • Blood type: A, B, AB, O
    • Medical specialty: surgery, internal medicine, family medicine, pediatrics, dermatology, or orthopedics
    • Type of diabetes: type 1, type 2, and gestational diabetes (diabetes while pregnant)
    Appropriate Tests:
    • • Chi-Square Test of Independence
    • • Chi-Square Goodness of Fit
    • • Multinomial Logistic Regression
    • • Fisher's Exact Test (small samples)
    Ordinal
    Ordered

    The categories can be ordered

    • Stage of cancer: stage I, II, III, or IV
    • Disease severity: mild, moderate, or severe
    • Likert scale: e.g., level of satisfaction: strongly-agree, agree, neutral, disagree, or strongly-disagree
    Appropriate Tests:
    • • Mann-Whitney U Test
    • • Wilcoxon Signed-Rank Test
    • • Kruskal-Wallis Test
    • • Spearman Correlation
    • • Ordinal Logistic Regression
    • • Cochran-Armitage Trend Test
    • • Jonckheere-Terpstra Test

    Quantitative (Numerical)

    Numbers (with real meaning), representing measurements, obtained from the sample elements.

    Discrete
    Countable

    Is measured on a numeric or a quantitative scale and can take countable values (how many)

    • Number of children
    • Length of stay at the hospital
    • Parity, which is the number of times a woman has given birth to a live neonate
    Appropriate Tests:
    • • Poisson Regression
    • • Negative Binomial Regression
    • • Chi-Square Test (when grouped)
    • • Mann-Whitney U (non-parametric)
    • • Kruskal-Wallis (non-parametric)
    • • Can also use tests for continuous data if assumptions met
    Continuous
    Uncountable

    Is measured on a numeric or a quantitative scale and can take uncountable real values (how much)

    • Age
    • Weight
    • Height
    • LDL cholesterol
    • HbA1C (blood glucose)
    Appropriate Tests:
    • • One-Sample t-Test
    • • Two-Sample t-Test
    • • Paired t-Test
    • • ANOVA (One-Way, Two-Way)
    • • Linear Regression
    • • Pearson Correlation
    • • ANCOVA
    • • Time-to-event outcomes require survival methods that account for censoring
    • • Outcomes counted over time — exacerbations, falls, admissions — are compared as rates over person-time, not as single measurements
    Note: The terms used in the calculator correspond to these variable types:
    Binary = 2 categories
    Nominal = 3+ unordered categories
    Ordinal = Ordered categories
    Numerical = Continuous or Discrete numbers

    Sampling designs: simple, stratified and clustered

    How the people in a survey are chosen changes how many are needed. Stratification and clustering are separate choices, and a survey can use either, both or neither.

    Simple random sample

    Everyone in the population has the same chance of selection, independently.

    n = z² × variance / E²

    The baseline every other design is compared with.

    Stratified sample

    The population is divided into strata, such as regions or urban and rural areas, and every stratum is sampled.

    DE_w = Σ W_h² / a_h

    Sampling in proportion to the population costs nothing (DE_w = 1). Giving small strata more than their share, to report on them, weights respondents unequally and costs the combined estimate DE_w > 1.

    Cluster sample

    Groups such as villages, schools or clinics are sampled first, then people within them.

    DE = 1 + (m − 1) × ICC

    People in the same cluster resemble each other, so each adds less information: the sample grows by the design effect, more so for large clusters.

    • Both together: clusters sampled inside every stratum, as most national surveys do. The two design effects multiply, and each stratum needs at least two clusters.
    • A figure for every stratum, not just the national one, means sizing each stratum as its own survey. It usually costs far more people; the national estimate then comes out more precise than needed.
    • Finite populations: with strata, each stratum carries its own finite-population correction. With clusters, the correction belongs to the clusters (1 − c/C), and matters only when a large share of all clusters is sampled.
    • Stratified randomisation in a trial is a different idea: it balances the arms and barely changes the sample size.

    In the calculator, open “Estimate precisely”, choose one mean, proportion, median, paired difference or category proportions, and tick “Stratified sampling”, “cluster sampling”, or both. The formulas and worked examples are in Sample size for a stratified survey

    Find a Suitable Statistical Procedure

    Tell us what you want to measure and compare. We suggest a starting method and explain what to check.

    Step 1

    1Planning goal

    Estimate a value, such as prevalence or an ICC, or test a hypothesis, such as a difference between groups or that reliability exceeds a benchmark.

    The shortlist will narrow the options. Pairing, repeated measurements, clustering, censoring, sampling, and model assumptions can still change the appropriate method.