Pharmaceutical Statistics

Article	Citations
Frailty model with change points for survival analysis	39
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	25
Prediction Intervals for Overdispersed Binomial Endpoints and Their Application to Toxicological Historical Control Data	22
Pre‐Posterior Distributions in Drug Development and Their Properties	19
Average Hazard as Harmonic Mean	18
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	18
Using an early outcome as the sole source of information of interim decisions regarding treatment effect on a long‐term endpoint: The non‐Gaussian case	17
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	16
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	14
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	14
	14
Issue Information	13
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose	13
CUSUMIN: A cumulative sum interval design for cancer phase I dose finding studies	13
A Model‐Based Trial Design With a Randomization Scheme Considering Pharmacokinetics Exposure for Dose Optimization in Oncology	12
Control of Unconditional Type I Error in Clinical Trials With External Control Borrowing—A Two‐Stage Adaptive Design Perspective	12
Why “Minimal Clinically Important Difference” for Interpreting the Magnitude of the Treatment Effect Is Not Useful	11
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	10
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior	10
Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery	10
Weighted log‐rank test to compare two survival functions in the presence of dependent censoring	10
A conservative approach to leveraging external evidence for effective clinical trial design	9
A meta‐analytic framework to adjust for bias in external control studies	9
Applying the Estimand Framework to Non‐Inferiority Trials	8
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	8

Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	8
Extending Multiple Testing With Unknown Test Dependency via the CoCo Test: With Applications to Cancer Studies	8
From innovative thinking to pharmaceutical industry implementation: Some success stories	7
Simultaneous Inference Using Multiple Marginal Models	7
Estimation of a treatment policy estimand for time to event data using data collected post discontinuation of randomised treatment	7
A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	7
Mixture Experimentation in Pharmaceutical Formulations: A Tutorial	7
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	7
Current developments of the estimand concept	7
Finding the Optimal Number of Splits and Repetitions in Double Cross‐Fitting Targeted Maximum Likelihood Estimators	7
Simulation‐based sequential design	7
Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components	7
An evolutionary algorithm for the direct optimization of covariate balance between nonrandomized populations	6
Issue Information	6
	6
Interim decision making in seamless trial designs: An application in an adaptive dose‐finding study in a rare kidney disease	6
BOIN‐MEM: A Two‐Stage Design for Dose Optimization With Information Borrowing Across Dose Levels and Stages in Oncology Phase I/II Trials	6
An illness–death multistate model to implement delta adjustment and reference‐based imputation with time‐to‐event endpoints	6
Chauhan Weighted Trajectory Analysis of Combined Efficacy and Safety Outcomes for Risk–Benefit Analysis	6
Bayesian Response Adaptive Randomization for Randomized Clinical Trials With Continuous Outcomes: The Role of Covariate Adjustment	6
Reflecting on Andy Grieve's influence and innovation: A personal perspective	6
Applying the Principal Stratum Strategy in Equivalence Trials: A Case Study	6
Statistical methods for handling missing data to align with treatment policy strategy	5
Estimation of Treatment Policy Estimands for Continuous Outcomes Using Off‐Treatment Sequential Multiple Imputation	5
The Wilcoxon–Mann–Whitney Estimand Versus Differences in Medians or Means	5
	5
Bayesian borrowing from historical control data in a vaccine efficacy trial	5
Tutorial on Firth's Logistic Regression Models for Biomarkers in Preclinical Space	5
Should responder analyses be conducted on continuous outcomes?	5
Study‐design in pandemics: From surveillance and performance‐evaluation to licensing and pharmacovigilance	5
Frequentist and Bayesian tolerance intervals for setting specification limits for left‐censored gamma distributed drug quality attributes	5
Balance diagnostics in propensity score analysis following multiple imputation: A new method	5
A Bayesian Hybrid Design With Borrowing From Historical Study	4
Comparative Analyses of Bioequivalence Assessment Methods for In Vitro Permeation Test Data	4
Issue Information	4
Designing and Evaluating Bayesian Advanced Adaptive Randomised Clinical Trials: A Practical Guide	4
	4
What they forgot to tell you about machine learning with an application to pharmaceutical manufacturing	4
Geometric approaches to assessing the numerical feasibility for conducting matching‐adjusted indirect comparisons	4
Beyond the Fragility Index	4
Evaluating hybrid controls methodology in early‐phase oncology trials: A simulation study based on the MORPHEUS‐UC trial	4
“Super‐covariates”: Using predicted control group outcome as a covariate in randomized clinical trials	4
A Personalized Dose‐Finding Algorithm Based on Adaptive Gaussian Process Regression	4
CUSUMIN Combination: A Cumulative Sum Interval Design for Phase I Cancer Drug‐Combination Trials	4
Propensity score matching and stratification using multiparty data without pooling	4
Regulatory issues and the potential use of Bayesian approaches for early drug approval systems in Japan	4
Improving precision and power in randomized trials with a two‐stage study design: Stratification using clustering method	4
	4
The Acute Stroke Therapy by Inhibition of Neutrophils study – Key features and impact	3
Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study	3

Issue Information	3
Issue Information	3
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	3
Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials	3
	3
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	3
Response to the letter to the editor regarding our article ‘statistical methodology for highly variable compounds: A novel design approach for the ofatumumab phase 2 bioequivalence study’ https://doi.	3
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	3
An adaptive design for early clinical development including interim decision for single‐arm trial with external controls or randomized trial	3
Multiple Comparisons Procedures for Analyses of Joint Primary Endpoints and Secondary Endpoints	3
Improving the assessment of the probability of success in late stage drug development	3
	3
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	3
An adaptive biomarker basket design in phase II oncology trials	3
Bayesian optimal phase II designs with dual‐criterion decision making	3
Using Propensity Score Weighting to Enhance the Operating Characteristics of Power Prior in Leveraging External Data to Augment a Traditional Clinical Study	3
Issue Information	3
Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance	3
Applying Bias Correction Methods to Build Hybrid Controls Using Real‐World Patients for a Phase IIb Randomized Controlled Trial of Baricitinib for Rheumatoid Arthritis	3
Introduction to Nonclinical Biopharmaceutical Statistics Tutorials—A Special Issue	2
A note on point estimation and interval estimation of the relative treatment effect under a simple crossover design	2
Visualizing hypothesis tests in survival analysis under anticipated delayed effects	2
Estimation of multivariate treatment effects in contaminated clinical trials	2
Transporting randomized trial results to estimate counterfactual survival functions in target populations	2
Predictive Ppk calculations for biologics and vaccines using a Bayesian approach – a tutorial	2
Sample Size Estimation Using a Partially Clustered Frailty Model for Biomarker‐Strategy Designs With Multiple Treatments	2
	2
Just say no to data listings!	2
	2
Adaptively leveraging external data with robust meta‐analytical‐predictive prior using empirical Bayes	2
A Tipping Point Method to Evaluate Sensitivity to Potential Violations in Missing Data Assumptions	2
Inclusion of binary proxy variables in logistic regression improves treatment effect estimation in observational studies in the presence of binary unmeasured confounding variables	2
The design and analysis of vaccine trials for COVID‐19 for the purpose of estimating efficacy	2
Application of hypothetical strategies in acute pain	2
Confidence intervals for point‐of‐stabilization of content uniformity	2
Two‐stage subgroup‐specific time‐to‐event (2S‐Sub‐TITE): An adaptive two‐stage time‐to‐toxicity design for subgroup‐specific dose finding in phase I oncology trials	2
Alone, together: On the benefits of Bayesian borrowing in a meta‐analytic setting	2
Comments on “Statistical methodology for highly variable compounds: A novel design approach for the ofatumumab phase 2 bioequivalence study”	2
Sample Size Estimation for Correlated Count Data With Changes in Dispersion	2
A Commensurate Prior Model With Random Effects for Survival and Competing Risk Outcomes to Accommodate Historical Controls	2
Variable Duration Trial as an Alternative Design for Continuous Endpoints	2
Quality by Design for Preclinical In Vitro Assay Development	2
Group sequential design with maximin efficiency robust test for immunotherapy with generalized delayed treatment effect	2
The Estimand Framework and Causal Inference: Complementary Not Competing Paradigms	2
Comparing various Bayesian random‐effects models for pooling randomized controlled trials with rare events	2
Issue Information	2
	2
Should the two‐trial paradigm still be the gold standard in drug assessment?	2
Statistical analysis of actigraphy data with generalised additive models	2
Principled leveraging of external data in the evaluation of diagnostic devices via the propensity score‐integrated composite likelihood approach	2
Potential Bias Models With Bayesian Shrinkage Priors for Dynamic Borrowing of Multiple Historical Control Data	2
Rejoinder to the letter: “Standard and reference‐based conditional mean imputation: Regulators and trial statisticians be aware!”	2
Tree‐temporal scan statistics for safety signal detection in vaccine clinical trials	2
	2
	2
Web based resource for Statistical Consultants in the Pharmaceutical Industry	2
On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	2
Principles for Defining Estimands in Clinical Trials—A Proposal	2
On the relative conservativeness of Bayesian logistic regression method in oncology dose‐finding studies	2
WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors	2
Investigating Stability in Subgroup Identification for Stratified Medicine	2
Real Effect or Bias? Good Practices for Evaluating the Robustness of Evidence From Comparative Observational Studies Through Quantitative Sensitivity Analysis for Unmeasured Confounding	1
A propensity score‐integrated approach for leveraging external data in a randomized controlled trial with time‐to‐event endpoints	1
Standard and reference‐based conditional mean imputation	1
Comparison of nonparametric estimators of the expected number of recurrent events	1
How to avoid concerns with the interpretation of two primary endpoints if significant superiority in one is sufficient for formal proof of efficacy	1
A model‐assisted design for partially or completely ordered groups	1
Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation	1
	1
Using the Bayesian detection of potential risk using inference on blinded safety data (BDRIBS) method to support the decision to refer an event for unblinded evaluation	1
Return‐to‐baseline multiple imputation for missing values in clinical trials	1
Sample size re‐estimation in Phase 2 dose‐finding: Conditional power versus Bayesian predictive power	1
PubPredict: Prediction of progression and survival in oncology leveraging publications and early efficacy data	1
A Bayesian optimal interval design for dose optimization with a randomization scheme based on pharmacokinetics outcomes in oncology	1
Issue Information	1
A Flexible Seamless Phase 2/3 Design With Biomarker‐Driven Subgroup Enrichment and Sample Size Re‐Estimation	1
Confirmatory efficacy testing for individual dose–placebo comparisons using serial gatekeeping procedure in dose‐finding trials with multiple comparison procedures–modeling	1
Empirical likelihood inference for area under the receiver operating characteristic curve using ranked set samples	1
Getting (More Out of) Graphics—Practices and Principles of Data Visualisation. Data Science SeriesBy AntonyUnwin, Chapman & Hall/CRC, 2024. 446 pp. $ 74.99. ISBN: 978‐0‐36‐767399‐4	1
Information fraction estimation: Strategies for a phase 3 non‐inferiority maximum duration design with time to event outcome	1

Correction to “Mixture Experimentation in Pharmaceutical Formulations: A Tutorial”	1
A Federated Data Analysis Approach for the Evaluation of Surrogate Endpoints	1
An improved score‐type confidence interval for stratified risk differences involving rare events	1
A Phase I Dose‐Finding Design Incorporating Intra‐Patient Dose Escalation	1
Comment on “Average Hazard as Harmonic Mean” by Chiba (2025)	1
Issue Information	1
A Bayesian Dynamic Model‐Based Adaptive Design for Oncology Dose Optimization in Phase I/II Clinical Trials	1
A comparison of statistical methods for animal oncology studies	1
A treatment‐specific marginal structural Cox model for the effect of treatment discontinuation	1
Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	1
Survival Analysis Without Sharing of Individual Patient Data by Using a Gaussian Copula	1
Estimating the treatment effect for adherers using multiple imputation	1
Long‐Term Safety Evaluations in the Presence of Switching: Evaluation of Two Approaches	1
Issue Information	1
Simulating and reporting frequentist operating characteristics of clinical trials that borrow external information: Towards a fair comparison in case of one‐arm and hybrid control two‐arm trials	1
Mediation Analysis of Path‐Specific Effects in Randomised Clinical Trials With Repeatedly Measured Mediators and Outcomes	1
Do You Want to Stay Single? Considerations on Single‐Arm Trials in Drug Development and the Postregulatory Space	1
Informed decision‐making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments	1
Probability of success and group sequential designs	1
Optimal unplanned design modification in adaptive two‐stage trials	1
AIDE: Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials	1
Replenishing the pipeline: A quantitative approach to optimising the sourcing of new projects	1
Sample size calculation for comparing two ROC curves	1
A practical guide to sample size calculations: Installation of the app SampSize	1
Detection of Outlying Correlation Coefficients in Multicenter Clinical Trials	1
Subgroup Identification Based on Quantitative Objectives	1
Issue Information	1
Defining estimands for efficacy assessment in single arm phase 1b or phase 2 clinical trials in oncology early development	1
Estimating survival parameters under conditionally independent left truncation	1
Correction to “Sample Size Estimation for Correlated Count Data With Changes in Dispersion”	1
Issue Information	1
A Tutorial on Improving RCT Power Using Prognostic Score Adjustment for Linear Models	1
Adjusting for bias in the mean for primary and secondary outcomes when trials are in sequence	1
Decision rules for identifying combination therapies in open‐entry, randomized controlled platform trials	1
PKBOIN‐12: A Bayesian Optimal Interval Phase I/II Design Incorporating Pharmacokinetics Outcomes to Find the Optimal Biological Dose	1
Setting the control limit at release for stability assurance	1
Natural cubic splines for the analysis of Alzheimer's clinical trials	1
Statistical modeling approaches for the comparison of dissolution profiles	1
The utilities and pitfalls of stratified analysis in challenging situations	1
	1
Issue Information	1
A simulation‐based comparison of estimation methods for adaptive and classical group sequential clinical trials	1
	1
Do statisticians count? A prior and posterior viewpoint	1
Evaluation of a flexible piecewise linear mixed‐effects model in the analysis of randomized cross‐over trials	1
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design	1
Introduction to qualification and validation of an immunoassay	1
Personalized Treatment Selection for Multivariate Ordinal Scale Outcomes and Multiple Treatments	1
Time‐to‐event calibration‐free odds design: A robust efficient design for phase I trials with late‐onset outcomes	1
Statistical considerations for design and analysis of stability, comparability and formulation tests	1
Taylor Series Approximation for Accurate Generalized Confidence Intervals of Ratios of Log‐Normal Standard Deviations for Meta‐Analysis Using Means and Standard Deviations in Time Scale	1
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data	1
Issue Information	1
Issue Information	1
Assessment of pharmacokinetic linearity after repeated drug administration	1