Statistics in Medicine

Tutorials in Biostatistics Papers

The primary objective of Tutorials in Biostatistics is to present introductory tutorials on current biostatistical methods.  Each tutorial presents the topic, defines vocabulary, reviews its uses, illustrates the uses with numerical examples relevant to biostatistical applications, includes demonstrations and references to available computer software for performing the method, and supplies references to articles and books for further reading. The tutorial should act as an introduction to those not already familiar with the method and as a review and update to others. The mathematical level of each depends upon the topic. In all cases, however, the tutorial will strive for the broadest possible audience of researchers and clinicians who comprise the readership of Statistics in Medicine.

Tutorials Published in 2025:

Continuous-Time Causal Inference With Marked Point Process Weights: An Example on Sodium-Glucose Co-Transporters 2 Inhibitor Medications and Urinary Tract Infection (Open Access)

The Mathematics of Serocatalytic Models With Applications to Public Health Data (Open Access)

Futility Monitoring in Clinical Trials (Open Access)

Design and Analysis of Group Sequential Trials for Repeated Measurements When Pipeline Data Occurs: A Tutorial

Surrogate Marker Evaluation: A Tutorial Using R (Open Access)

So Many Choices: A Guide to Selecting Among Methods to Adjust for Observed Confounders (Open Access)

Joint Latent Class Models: A Tutorial on Practical Applications in Clinical Research (Open Access)

Incorporating Additional Evidence as Prior Information to Resolve Non-Identifiability in Bayesian Disease Model Calibration: A Tutorial (Open Access)

Guidelines and Best Practices for the Use of Targeted Maximum Likelihood and Machine Learning When Estimating Causal Effects of Exposures on Time-To-Event Outcomes (Open Access)

Multiple Imputation for Longitudinal Data: A Tutorial (Open Access)

Estimation of Diagnostic Test Accuracy Without Gold Standards

Tutorials Published in 2024:

Confidence distributions for treatment effects in clinical trials: Posteriors without priors (Open Access)

A Brief Introduction on Latent Variable Based Ordinal Regression Models With an Application to Survey Data (Open Access)

Statistical Inference for Box–Cox based Receiver Operating Characteristic Curves

Dynamic path analysis for exploring treatment effect mediation processes in clinical trials with time-to-event endpoints

Modern approaches for evaluating treatment effect heterogeneity from clinical trials and observational data

Novel non-linear models for clinical trial analysis with longitudinal data: A tutorial using SAS for both frequentist and Bayesian methods

Standardization and other approaches to meta-analyze differences in means (Open Access)

Bayesian survival analysis with INLA (Open Access)

Bayesian transition models for ordinal longitudinal outcome (Open Access)

Design of randomized clinical trials with a binary endpoint: Conditional versus unconditional analyses of a two-by-two table (Open Access)

On variance estimation of the inverse probability-of-treatment weighting estimator: A tutorial for different types of propensity score weights (Open Access)

Parameter estimation and forecasting with quantified uncertainty for ordinary differential equation models using QuantDiffForecast: A MATLAB toolbox and tutorial (Open Access)

Statistical plasmode simulations–Potentials, challenges and recommendations (Open Access)

Tutorials Published in 2023:

Modeling multiple correlated end-organ disease trajectories: A tutorial for multistate and joint models with applications in diabetes complications

Minimization in randomized clinical trials (Open Access)

Single-world intervention graphs for defining, identifying, and communicating estimands in clinical trials

A guide to regression discontinuity designs in medical applications

Joint clustering multiple longitudinal features: A comparison of methods and software packages with practical guidance (Open Access)

Tutorials Published in 2022 Issues:

Review and evaluation of imputation methods for multivariate longitudinal data with mixed-type incomplete variable

Gaussian graphical models with applications to omics analyses

DL 101: Basic Introduction to deep learning with its application in biomedical related fields

Generalized additive models to analyze nonlinear trends in biomedical longitudinal data using R: Beyond repeated measures ANOVA and linear mixed models 

Using principal stratification in analysis of clinical trials

Translating questions to estimands in randomized clinical trials with intercurrent events (Open Access)

Correcting for partial verification bias in diagnostic accuracy studies: A tutorial using R

Tutorials Published in 2021 Issues:

Using fractional polynomials and restricted cubic splines to model non-proportional hazards or time-varying covariate effects in the Cox regression model (Open Access)

Introduction to computational causal inference using reproducible Stata, R, and Python code: A tutorial (Open Access)

Bayesian workflow for disease transmission modeling in Stan

A tutorial on individualized treatment effect prediction from randomized trials with a binary endpoint (Open Access)

Optimal planning of adaptive two‐stage designs (Open Access)

Bayesian survival analysis with BUGS

A practical introduction to Bayesian estimation of causal effects: Parametric and nonparametric approaches

Analysis of time‐to‐event for observational studies: Guidance to the use of intensity models

Tutorials Published in 2020 Issues:

Randomization tests for multiarmed randomized clinical trials

A primer on strong vs weak control of familywise error rate

STRATOS guidance document on measurement error and misclassification of variables in observational epidemiology: Part 1—Basic theory and simple methods of adjustment

STRATOS guidance document on measurement error and misclassification of variables in observational epidemiology: Part 2—More complex methods of adjustment and advanced topics

Matching with time‐dependent treatments: A review and look forward

Extending inferences from a randomized trial to a new target population

Individual participant data meta‐analysis to examine interactions between treatment effect and participant‐level covariates: Statistical recommendations for conduct and planning

Relative rate of change in cognitive score network dynamics via Bayesian hierarchical models reveal spatial patterns of neurodegeneration

Sensitivity analysis for clinical trials with missing continuous outcome data using controlled multiple imputation: A practical guide

Randomization‐based interval estimation in randomized clinical trials

A general presentation on how to carry out a CHARMS analysis for prognostic multivariate models

Empirical use of causal inference methods to evaluate survival differences in a real‐world registry vs those found in randomized clinical trials

A tutorial on dealing with time‐varying eligibility for treatment: Comparing the risk of major bleeding with direct‐acting oral anticoagulants vs warfarin

To tolerate or to agree: A tutorial on tolerance intervals in method comparison studies with BivRegBLS R Package

Formulating causal questions and principled statistical answers

Tutorials Published in 2019 Issues:

What makes a biostatistician?

Using simulation studies to evaluate statistical methods

Re‐randomization tests in clinical trials

Evaluating classification accuracy for modern learning approaches

How to analyze and interpret recurrent events data in the presence of a terminal event: An application on readmission after colorectal cancer surgery

Sequential trials in the context of competing risks: Concepts and case study, with R and SAS code

Cloud‐based simulation studies in R ‐ A tutorial on using doRedis with Amazon spot fleets

P value functions: An underused method to present research results and to promote quantitative reasoning

Maximum likelihood estimation with missing outcomes: From simplicity to complexity

The relations among three popular indices of risks

Bayesian additive regression trees and the General BART model

Tutorials Published in 2018 Issues:

Tutorial on kernel estimation of continuous spatial and spatiotemporal relative risk

Targeted maximum likelihood estimation for a binary treatment: A tutorial

A tutorial in assessing disclosure risk in microdata

Tutorials Published in 2017 Issues:

Data-driven subgroup identification and analysis in clinical trials

Meta-analysis using individual participant data: one-stage and two-stage approaches, and why they may differ

Tutorial on statistical considerations on subgroup analysis in confirmatory clinical trials

Intermediate and advanced topics in multilevel logistic regression analysis

Linear combinations come alive in crossover designs

Understanding MCP‐MOD dose finding as a method based on linear regression

Tutorials Published in 2016 Issues:

Latent class instrumental variables: a clinical and biostatistical perspective

Statistical methods for studying disease subtype heterogeneity

A tutorial on Bayesian bivariate meta‐analysis of mixed binary‐continuous outcomes with missing treatment effects

Low-event-rate meta-analyses of clinical trials: implementing good practices

Simple generalized estimating equations (GEEs) and weighted generalized estimating equations (WGEEs) in longitudinal studies with dropouts: guidelines and implementation in R

Developing points‐based risk‐scoring systems in the presence of competing risks

Modeling zero-modified count and semicontinuous data in health services research part 1: background and overview

Modeling zero-modified count and semicontinuous data in health services research part 2: case studies

Prevalence odds ratio versus prevalence ratio: choice comes with consequences

Tutorials Published in 2015 Issues:

Statistical methods for studying disease subtype heterogeneity

Random forest classification of etiologies for an orphan disease

A tutorial on structural equation modeling for analysis of overlapping symptoms in co-occurring conditions using MPlus

A guide to genome-wide association analysis and post-analytic interrogation

Tutorials Published in 2014 Issues:

Graphical assessment of internal and external calibration of logistic regression models by using loess smoothers

Advanced multiplicity adjustment methods in clinical trials

Funnel plots for population-based cancer survival: principles, methods and applications

The use of propensity score methods with survival or time-to-event outcomes: reporting measures of effect similar to those used in randomized experiments

Multiple hypothesis testing in genomics

A toolkit for measurement error correction, with a focus on nutritional epidemiology

Instrumental variable methods for causal inference

Recommended tests and confidence intervals for paired binomial proportions

How to interpret a small increase in AUC with an additional risk prediction marker: decision analysis comes through

Tutorials Published in 2013 Issues:

Methods for dealing with time-dependent confounding

Discovering, comparing, and combining moderators of treatment on outcome after randomized clinical trials: a parametric approach

A tutorial on propensity score estimation for multiple treatments using generalized boosted models

Multilevel modeling versus cross‐sectional analysis for assessing the longitudinal tracking of cardiovascular risk factors over time

Traditional multiplicity adjustment methods in clinical trials

Tutorials Published in 2012 Issues:

Designing a pilot sequential multiple assignment randomized trial for developing an adaptive treatment strategy

Using R and WinBUGS to fit a generalized partial credit model for developing and evaluating patient-reported outcomes assessments

Tutorial in biostatistics: sample sizes for parallel group clinical trials with binary data

Tutorials Published in 2011 Issues:

Multiple imputation using chained equations: Issues and guidance for practice

Frailty models: Applications to biomedical and genetic studies

Tutorials Published in 2010 Issues:

Efficiency robust statistics for genetic linkage and association studies under genetic model uncertainty

Logistic quantile regression for bounded outcomes

Real longitudinal data analysis for real people: Building a good enough mixed model

Dose-response analyses using restricted cubic spline functions in public health research

The analysis of treatment effects for recurring episodic conditions

Random effects meta-analysis of event outcome in the framework of the generalized linear mixed model with applications in sparse data

Tutorials Published in 2009 Issues:

Events per person‐time (incidence rate): A misleading statistic?

Recommended tests for association in 2×2 tables

Adaptive designs for confirmatory clinical trials

Practical application of the vanishing tetrad test for causal indicator measurement models: An example from health‐related quality of life

Student t‐tests for potentially abnormal data

Hip psychometrics

Tutorials Published in 2008 Issues:

Evaluation of diagnostic scores with adjustment for covariates

Analysis of longitudinal laboratory data in the presence of common selection mechanisms: A view toward greater emphasis on pre‐marketing pharmaceutical safety

Checking hazard regression models using pseudo‐observations

Empirical estimation of life expectancy from large clinical trials: Use of left‐truncated, right‐censored survival analysis methodology

Analysis of cross‐over designs with serial correlation within periods using semi‐parametric mixed models

Tutorials Published in 2007 Issues:

Interval estimation for individual categories in cumulative logit models

Parametric survival analysis and taxonomy of hazard functions for the generalized gamma distribution

Frequentist evaluation of group sequential clinical trial designs

Tutorials Published in 2006 Issues:

On the application of the von Mises distribution and angular regression methods to investigate the seasonality of disease onset

Tutorials Published in 2005 Issues:

The use of quantile regression in health care research: a case study examining gender differences in the timeliness of thrombolytic therapy

Tutorials Published in 2004 Issues:

Handling drop‐out in longitudinal studies

Presentation of multivariate data for clinical use: The Framingham Study risk score functions

Sample sizes for clinical trials with Normal data

Regression analysis of multiple source and multiple informant data from complex survey samples

Tutorials Published in 2003 Issues:

Hierarchical linear models for the development of growth curves: an example with body mass index in overweight/obese adults

Comparison of multiple regression to two latent variable techniques for estimation and prediction

Tutorials Published in 2002 Issues:

Covariance models for nested repeated measures data: analysis of ovarian steroid secretion data

Advanced methods in meta‐analysis: multivariate approach and meta‐regression

Kappa coefficients in medical research

Likelihood methods for measuring statistical evidence

Multilevel modelling of medical data

Tutorials Published in 2001 Issues:

Disease map reconstruction

Using observational data to estimate prognosis: an example using a coronary artery disease registry

The applications of capture‐recapture models to epidemiological data

Tutorials Published in 2000 Issues:

Categorizing a prognostic variable: review of methods, code for easy implementation and applications to decision‐making about cancer treatments

Repeated measures in clinical trials: simple strategies for analysis using summary measures

Strategies for comparing treatments on a binary response with multi‐centre data

Modelling covariance structure in the analysis of repeated measures data

Tutorials Published in 1999 Issues:

Meta‐analysis: formulating, evaluating, combining, and reporting

An introduction to hierarchical linear modelling

Longitudinal data analysis (repeated measures) in clinical trials

Analysis of binary outcomes in longitudinal studies using weighted estimating equations and discrete‐time survival methods: prevalence and incidence of smoking in an adolescent cohort

Genetic mapping of complex traits

Tutorials Published in 1998 Issues:

Methods for interval‐censored data

Development of a clinical prediction model for an ordinal outcome: the World Health Organization Multicentre Study of Clinical Signs and Etiological Agents of Pneumonia, Sepsis and Meningitis in Young Infants

Extending the simple linear regression model to account for correlated responses: An introduction to generalized estimating equations and multi‐level mixed modelling

Propensity score methods for bias reduction in the comparison of a treatment to a non‐randomized control group

Tutorials Published in 1997 Issues:

Using the general linear mixed model to analyse unbalanced repeated measures and longitudinal data

Tutorials Published in 1996 Issues:

Editorial on Tutorials in Biostatistics

Designing studies for dose response

Multivariate prognostic models: Issues in developing models, evaluating assumptions and adequacy, and measuring and reducing errors

Statistical approaches to human brain mapping by functional magnetic resonance imaging

Tutorials Published in 1992 Issues:

An overview of methods for the analysis of longitudinal data