Pharmaceutical Statistics

Article	Citations
Frailty model with change points for survival analysis	52
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	33
Prediction Intervals for Overdispersed Binomial Endpoints and Their Application to Toxicological Historical Control Data	32
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	25
Average Hazard as Harmonic Mean	21
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	20
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	18
Pre‐Posterior Distributions in Drug Development and Their Properties	17
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	16
Adaptive Constrained Weighted Estimation for Incorporating Multiple External Information Sources	15
Prospectively Specified Adaptive Bayesian Borrowing: Considerations, Methodologies, and Implementations	14
A Weighted Geometric Average Hazard Ratio Based Sample Size Formula for Non‐Proportional Hazards and Its Application to a Smoothed Piecewise Model in Cancer Immunotherapy Trial Design	14
Using Off‐Treatment Sequential Multiple Imputation for Binary Outcomes to Address Intercurrent Events Handled by a Treatment Policy Strategy	13
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	13
Issue Information	12
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CUSUMIN: A cumulative sum interval design for cancer phase I dose finding studies	12
Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery	10
Control of Unconditional Type I Error in Clinical Trials With External Control Borrowing—A Two‐Stage Adaptive Design Perspective	10
A Model‐Based Trial Design With a Randomization Scheme Considering Pharmacokinetics Exposure for Dose Optimization in Oncology	10
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	10
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose	10
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	10
Why “Minimal Clinically Important Difference” for Interpreting the Magnitude of the Treatment Effect Is Not Useful	10
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	10

Extending Multiple Testing With Unknown Test Dependency via the CoCo Test: With Applications to Cancer Studies	9
Weighted log‐rank test to compare two survival functions in the presence of dependent censoring	9
A meta‐analytic framework to adjust for bias in external control studies	9
Multiplicity Adjustment Methods for a Three‐Way Crossover Bioequivalence Study	8
Applying the Estimand Framework to Non‐Inferiority Trials	8
Simulation‐based sequential design	7
From innovative thinking to pharmaceutical industry implementation: Some success stories	7
Confidence Intervals for Validation of Analytical Procedures Under ICH Q2 ( R2 )	7
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	7
A conservative approach to leveraging external evidence for effective clinical trial design	7
Finding the Optimal Number of Splits and Repetitions in Double Cross‐Fitting Targeted Maximum Likelihood Estimators	7
A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	7
Current developments of the estimand concept	7
Issue Information	6
BOIN ‐ MEM : A Two‐Stage Design for Dose Optimization With Information Borrowing Across Dose Levels and Stages in Onco	6
Mixture Experimentation in Pharmaceutical Formulations: A Tutorial	6
An illness–death multistate model to implement delta adjustment and reference‐based imputation with time‐to‐event endpoints	6
A Unified Approach to Covariate Adjustment for Survival Endpoints in Randomized Clinical Trials	6
Simultaneous Inference Using Multiple Marginal Models	6
Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components	6
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Balance diagnostics in propensity score analysis following multiple imputation: A new method	5
Interim decision making in seamless trial designs: An application in an adaptive dose‐finding study in a rare kidney disease	5
Should responder analyses be conducted on continuous outcomes?	5
Application of Causal Inference to Establish Assay Effect in the Absence of a Bridging Study: A Case Study of MenACWY ‐ CRM<	5
Bayesian Response Adaptive Randomization for Randomized Clinical Trials With Continuous Outcomes: The Role of Covariate Adjustment	5
An evolutionary algorithm for the direct optimization of covariate balance between nonrandomized populations	5
Frequentist and Bayesian tolerance intervals for setting specification limits for left‐censored gamma distributed drug quality attributes	5
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Applying the Principal Stratum Strategy in Equivalence Trials: A Case Study	5
Chauhan Weighted Trajectory Analysis of Combined Efficacy and Safety Outcomes for Risk–Benefit Analysis	5
Simulation‐Based Bayesian Predictive Probability of Success for Interim Monitoring of Clinical Trials With Competing Event Data: Two Case Studies	5
Bayesian borrowing from historical control data in a vaccine efficacy trial	5
Informing the Borrowing Process for Dose‐Finding Trials by Estimating the Similarity Between Population‐Specific Dose‐Toxicity Curves	5
Statistical methods for handling missing data to align with treatment policy strategy	5
Reflecting on Andy Grieve's influence and innovation: A personal perspective	5
CUSUMIN Combination: A Cumulative Sum Interval Design for Phase I Cancer Drug‐Combination Trials	4
A Tree‐Based Scan Statistic for Detecting Signals of Drug–Drug Interactions in Spontaneous Reporting Databases	4
The Wilcoxon–Mann–Whitney Estimand Versus Differences in Medians or Means	4
Evaluating hybrid controls methodology in early‐phase oncology trials: A simulation study based on the MORPHEUS‐UC trial	4
What they forgot to tell you about machine learning with an application to pharmaceutical manufacturing	4
“Super‐covariates”: Using predicted control group outcome as a covariate in randomized clinical trials	4
The Acute Stroke Therapy by Inhibition of Neutrophils study – Key features and impact	4
A Bayesian Hybrid Design With Borrowing From Historical Study	4
Tutorial on Firth's Logistic Regression Models for Biomarkers in Preclinical Space	4
Comparative Analyses of Bioequivalence Assessment Methods for In Vitro Permeation Test Data	4
A Tobit Partly Linear Mixed and Mixture Cure Model for the Joint Analysis of Interval‐Bounded Longitudinal Measurements and Survival Times With Cure Proportion	4
Propensity score matching and stratification using multiparty data without pooling	4
Designing and Evaluating Bayesian Advanced Adaptive Randomised Clinical Trials: A Practical Guide	4
Improving precision and power in randomized trials with a two‐stage study design: Stratification using clustering method	4

Issue Information	4
Beyond the Fragility Index	4
Study‐design in pandemics: From surveillance and performance‐evaluation to licensing and pharmacovigilance	4
Estimation of Treatment Policy Estimands for Continuous Outcomes Using Off‐Treatment Sequential Multiple Imputation	4
Issue Information	4
A Personalized Dose‐Finding Algorithm Based on Adaptive Gaussian Process Regression	4
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Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	4
On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	3
Bayesian optimal phase II designs with dual‐criterion decision making	3
Issue Information	3
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	3
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	3
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Web based resource for Statistical Consultants in the Pharmaceutical Industry	3
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	3
Multiple Comparisons With Overdispersed Multinomial Data: Methods, Properties and Application	3
Using Propensity Score Weighting to Enhance the Operating Characteristics of Power Prior in Leveraging External Data to Augment a Traditional Clinical Study	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
An adaptive biomarker basket design in phase II oncology trials	3
Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance	3
Quality by Design for Preclinical In Vitro Assay Development	3
Comparing various Bayesian random‐effects models for pooling randomized controlled trials with rare events	3
Issue Information	3
Effects of duration of follow‐up and lag in data collection on the performance of adaptive 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
Multiple Comparisons Procedures for Analyses of Joint Primary Endpoints and Secondary Endpoints	3
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Bayesian Power‐Based Sample Size Determination for Single‐Arm Clinical Trials With Time‐to‐Event Endpoints	2
The design and analysis of vaccine trials for COVID‐19 for the purpose of estimating efficacy	2
Penalized Variable Selection for Joint AFT Random‐Effect Model With Clustered Competing‐Risks Data	2
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Tree‐temporal scan statistics for safety signal detection in vaccine clinical trials	2
Should the two‐trial paradigm still be the gold standard in drug assessment?	2
Rejoinder to the letter: “Standard and reference‐based conditional mean imputation: Regulators and trial statisticians be aware!”	2
Do statisticians count? A prior and posterior viewpoint	2
Comments on “Statistical methodology for highly variable compounds: A novel design approach for the ofatumumab phase 2 bioequivalence study”	2
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Adaptively leveraging external data with robust meta‐analytical‐predictive prior using empirical Bayes	2
Transporting randomized trial results to estimate counterfactual survival functions in target populations	2
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Probability of success and group sequential designs	2
WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors	2
Principled leveraging of external data in the evaluation of diagnostic devices via the propensity score‐integrated composite likelihood approach	2
Do You Want to Stay Single? Considerations on Single‐Arm Trials in Drug Development and the Postregulatory Space	2
A Tipping Point Method to Evaluate Sensitivity to Potential Violations in Missing Data Assumptions	2
Statistical analysis of actigraphy data with generalised additive models	2
Sample Size Estimation Using a Partially Clustered Frailty Model for Biomarker‐Strategy Designs With Multiple Treatments	2
Alone, together: On the benefits of Bayesian borrowing in a meta‐analytic setting	2
Application of hypothetical strategies in acute pain	2
Predictive Ppk calculations for biologics and vaccines using a Bayesian approach – a tutorial	2
The Estimand Framework and Causal Inference: Complementary Not Competing Paradigms	2
Just say no to data listings!	2
AIDE: Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials	2
Issue Information	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
Visualizing hypothesis tests in survival analysis under anticipated delayed effects	2
A Commensurate Prior Model With Random Effects for Survival and Competing Risk Outcomes to Accommodate Historical Controls	2
PubPredict: Prediction of progression and survival in oncology leveraging publications and early efficacy data	2
Variable Duration Trial as an Alternative Design for Continuous Endpoints	2
Potential Bias Models With Bayesian Shrinkage Priors for Dynamic Borrowing of Multiple Historical Control Data	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
Sample Size Estimation for Correlated Count Data With Changes in Dispersion	2
Principles for Defining Estimands in Clinical Trials—A Proposal	2
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Group sequential design with maximin efficiency robust test for immunotherapy with generalized delayed treatment effect	2
On the relative conservativeness of Bayesian logistic regression method in oncology dose‐finding studies	2
Introduction to Nonclinical Biopharmaceutical Statistics Tutorials—A Special Issue	2
Investigating Stability in Subgroup Identification for Stratified Medicine	2
Statistical modeling approaches for the comparison of dissolution profiles	1
Issue Information	1
Issue Information	1
Personalized Treatment Selection for Multivariate Ordinal Scale Outcomes and Multiple Treatments	1
Real Effect or Bias? Good Practices for Evaluating the Robustness of Evidence From Comparative Observational Studies Through Quantitative Sensitivity Analysis for Unmeasured Confounding	1
Sample size calculation for comparing two ROC curves	1
A Statistical Concept for Conditional Marketing Authorisation Based on the Intermediate and Final Outcomes of a Single Confirmatory Randomised Clinical Trial	1
Handling Missing Data in Participants with Baseline but No Post‐Baseline Data	1
The utilities and pitfalls of stratified analysis in challenging situations	1
Setting the control limit at release for stability assurance	1

Informed decision‐making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments	1
Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	1
Statistical considerations for design and analysis of stability, comparability and formulation tests	1
Comparison of nonparametric estimators of the expected number of recurrent events	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
A Dose‐Finding Design for Drug Combinations Using a Bayesian 4 Parameter Logistic Model With Penalised D‐Optimality	1
Robust Modestly Weighted Log‐Rank Tests	1
Statistical Consideration for Event‐Free Survival With Cure Rate in Acute Myeloid Leukemia Studies	1
A Flexible Seamless Phase 2/3 Design With Biomarker‐Driven Subgroup Enrichment and Sample Size Re‐Estimation	1
A model‐assisted design for partially or completely ordered groups	1
Correction to “Sample Size Estimation for Correlated Count Data With Changes in Dispersion”	1
Issue Information	1
Methodological Approaches for the Estimation of Confidence Intervals on Partial Youden Index Under Verification Bias	1
“Within‐Trial” Prognostic Score Adjustment Is Targeted Maximum Likelihood Estimation	1
Defining estimands for efficacy assessment in single arm phase 1b or phase 2 clinical trials in oncology early development	1
An improved score‐type confidence interval for stratified risk differences involving rare events	1
Replenishing the pipeline: A quantitative approach to optimising the sourcing of new projects	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
Assessment of Responder Analyses for Patient‐Reported Outcomes With Varying Variability Between Intervention Groups: Maximum Responder Threshold and Effect	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
Evaluation of a flexible piecewise linear mixed‐effects model in the analysis of randomized cross‐over trials	1
Predictive Biomarker Graphical Approach ( PRIME ) for Precision Medicine	1
Sample size re‐estimation in Phase 2 dose‐finding: Conditional power versus Bayesian predictive power	1
Subgroup Identification Based on Quantitative Objectives	1
Long‐Term Safety Evaluations in the Presence of Switching: Evaluation of Two Approaches	1
Leveraging Two‐Stage δ Global Sensibility Analysis Method to Inform Parameter Estimation in PBPK Models	1
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A Phase I Dose‐Finding Design Incorporating Intra‐Patient Dose Escalation	1
Order of Addition in Mixture‐Amount Experiments	1
Issue Information	1
A Tutorial on Improving RCT Power Using Prognostic Score Adjustment for Linear Models	1
A comparison of statistical methods for animal oncology studies	1
Great Wall: A Generalized Dose Optimization Design for Drug Combination Trials Maximizing Survival Benefit	1
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Time‐to‐event calibration‐free odds design: A robust efficient design for phase I trials with late‐onset outcomes	1
A practical guide to sample size calculations: Installation of the app SampSize	1
A propensity score‐integrated approach for leveraging external data in a randomized controlled trial with time‐to‐event endpoints	1
Confirmatory efficacy testing for individual dose–placebo comparisons using serial gatekeeping procedure in dose‐finding trials with multiple comparison procedures–modeling	1
Issue Information	1
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design	1
Optimal unplanned design modification in adaptive two‐stage trials	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 Federated Data Analysis Approach for the Evaluation of Surrogate Endpoints	1
Natural cubic splines for the analysis of Alzheimer's clinical trials	1
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data	1
Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation	1
Issue Information	1
A Bayesian optimal interval design for dose optimization with a randomization scheme based on pharmacokinetics outcomes in oncology	1
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Correction to “Mixture Experimentation in Pharmaceutical Formulations: A Tutorial”	1
PKBOIN‐12: A Bayesian Optimal Interval Phase I/II Design Incorporating Pharmacokinetics Outcomes to Find the Optimal Biological Dose	1
Information fraction estimation: Strategies for a phase 3 non‐inferiority maximum duration design with time to event outcome	1
Mediation Analysis of Path‐Specific Effects in Randomised Clinical Trials With Repeatedly Measured Mediators and Outcomes	1
A Bayesian Dynamic Model‐Based Adaptive Design for Oncology Dose Optimization in Phase I/II Clinical Trials	1
Issue Information	1
Comment on “Average Hazard as Harmonic Mean” by Chiba (2025)	1
Survival Analysis Without Sharing of Individual Patient Data by Using a Gaussian Copula	1
Introduction to qualification and validation of an immunoassay	1
Issue Information	1
Empirical likelihood inference for area under the receiver operating characteristic curve using ranked set samples	1
Standard and reference‐based conditional mean imputation	1
Detection of Outlying Correlation Coefficients in Multicenter Clinical Trials	1