Pharmaceutical Statistics

Article	Citations
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	57
Prediction Intervals for Overdispersed Binomial Endpoints and Their Application to Toxicological Historical Control Data	34
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	25
Average Hazard as Harmonic Mean	22
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	21
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	19
Using Off‐Treatment Sequential Multiple Imputation for Binary Outcomes to Address Intercurrent Events Handled by a Treatment Policy Strategy	18
Prospectively Specified Adaptive Bayesian Borrowing: Considerations, Methodologies, and Implementations	17
Adaptive Constrained Weighted Estimation for Incorporating Multiple External Information Sources	16
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	15
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	14
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	13
Frailty model with change points for survival analysis	13
Pre‐Posterior Distributions in Drug Development and Their Properties	13
CUSUMIN: A cumulative sum interval design for cancer phase I dose finding studies	12
Issue Information	12
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	11
Why “Minimal Clinically Important Difference” for Interpreting the Magnitude of the Treatment Effect Is Not Useful	11
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	11
Control of Unconditional Type I Error in Clinical Trials With External Control Borrowing—A Two‐Stage Adaptive Design Perspective	11
Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery	11
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose	10
A Model‐Based Trial Design With a Randomization Scheme Considering Pharmacokinetics Exposure for Dose Optimization in Oncology	10
Weighted log‐rank test to compare two survival functions in the presence of dependent censoring	10
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	10

A meta‐analytic framework to adjust for bias in external control studies	9
A conservative approach to leveraging external evidence for effective clinical trial design	9
Extending Multiple Testing With Unknown Test Dependency via the CoCo Test: With Applications to Cancer Studies	8
Applying the Estimand Framework to Non‐Inferiority Trials	8
Issue Information	7
From innovative thinking to pharmaceutical industry implementation: Some success stories	7
Finding the Optimal Number of Splits and Repetitions in Double Cross‐Fitting Targeted Maximum Likelihood Estimators	7
A Unified Approach to Covariate Adjustment for Survival Endpoints in Randomized Clinical Trials	7
Confidence Intervals for Validation of Analytical Procedures Under ICH Q2 ( R2 )	7
A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	7
Simulation‐based sequential design	7
Current developments of the estimand concept	7
Multiplicity Adjustment Methods for a Three‐Way Crossover Bioequivalence Study	7
Mixture Experimentation in Pharmaceutical Formulations: A Tutorial	6
Chauhan Weighted Trajectory Analysis of Combined Efficacy and Safety Outcomes for Risk–Benefit Analysis	6
BOIN ‐ MEM : A Two‐Stage Design for Dose Optimization With Information Borrowing Across Dose Levels and Stages in Onco	6
Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components	6
Reflecting on Andy Grieve's influence and innovation: A personal perspective	6
Simultaneous Inference Using Multiple Marginal Models	6
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	6
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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
Bayesian Response Adaptive Randomization for Randomized Clinical Trials With Continuous Outcomes: The Role of Covariate Adjustment	5
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Bayesian borrowing from historical control data in a vaccine efficacy trial	5
Study‐design in pandemics: From surveillance and performance‐evaluation to licensing and pharmacovigilance	5
Simulation‐Based Bayesian Predictive Probability of Success for Interim Monitoring of Clinical Trials With Competing Event Data: Two Case Studies	5
Applying the Principal Stratum Strategy in Equivalence Trials: A Case Study	5
Should responder analyses be conducted on continuous outcomes?	5
Frequentist and Bayesian tolerance intervals for setting specification limits for left‐censored gamma distributed drug quality attributes	5
Estimation of Treatment Policy Estimands for Continuous Outcomes Using Off‐Treatment Sequential Multiple Imputation	5
An illness–death multistate model to implement delta adjustment and reference‐based imputation with time‐to‐event endpoints	5
Application of Causal Inference to Establish Assay Effect in the Absence of a Bridging Study: A Case Study of MenACWY ‐ CRM<	5
An evolutionary algorithm for the direct optimization of covariate balance between nonrandomized populations	5
Interim decision making in seamless trial designs: An application in an adaptive dose‐finding study in a rare kidney disease	5
Balance diagnostics in propensity score analysis following multiple imputation: A new method	5
The Wilcoxon–Mann–Whitney Estimand Versus Differences in Medians or Means	5
Evaluating hybrid controls methodology in early‐phase oncology trials: A simulation study based on the MORPHEUS‐UC trial	4
Tutorial on Firth's Logistic Regression Models for Biomarkers in Preclinical Space	4
Issue Information	4
What they forgot to tell you about machine learning with an application to pharmaceutical manufacturing	4
Designing and Evaluating Bayesian Advanced Adaptive Randomised Clinical Trials: A Practical Guide	4
“Super‐covariates”: Using predicted control group outcome as a covariate in randomized clinical trials	4
A Tree‐Based Scan Statistic for Detecting Signals of Drug–Drug Interactions in Spontaneous Reporting Databases	4
A Bayesian Hybrid Design With Borrowing From Historical Study	4
Improving precision and power in randomized trials with a two‐stage study design: Stratification using clustering method	4
Propensity score matching and stratification using multiparty data without pooling	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
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An Ensemble Classifier for Ordinal Outcomes in High‐Dimensional Genomics Data	4
CUSUMIN Combination: A Cumulative Sum Interval Design for Phase I Cancer Drug‐Combination Trials	4
Beyond the Fragility Index	4
A Personalized Dose‐Finding Algorithm Based on Adaptive Gaussian Process Regression	4
Comparative Analyses of Bioequivalence Assessment Methods for In Vitro Permeation Test Data	4
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Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	3
The Acute Stroke Therapy by Inhibition of Neutrophils study – Key features and impact	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
Bayesian optimal phase II designs with dual‐criterion decision making	3
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	3
Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance	3
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Web based resource for Statistical Consultants in the Pharmaceutical Industry	3
An adaptive biomarker basket design in phase II oncology trials	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
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	3
Multiple Comparisons Procedures for Analyses of Joint Primary Endpoints and Secondary Endpoints	3
Quality by Design for Preclinical In Vitro Assay Development	3
Multiple Comparisons With Overdispersed Multinomial Data: Methods, Properties and Application	3
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Issue Information	3
Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials	3
Issue Information	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
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	3
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Bayesian Power‐Based Sample Size Determination for Single‐Arm Clinical Trials With Time‐to‐Event Endpoints	2
A Commensurate Prior Model With Random Effects for Survival and Competing Risk Outcomes to Accommodate Historical Controls	2
Probability of success and group sequential designs	2
Variable Duration Trial as an Alternative Design for Continuous Endpoints	2
WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors	2
Just say no to data listings!	2
Statistical analysis of actigraphy data with generalised additive models	2
On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	2
Alone, together: On the benefits of Bayesian borrowing in a meta‐analytic setting	2
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Principles for Defining Estimands in Clinical Trials—A Proposal	2
Transporting randomized trial results to estimate counterfactual survival functions in target populations	2
Do statisticians count? A prior and posterior viewpoint	2
Personalized Treatment Selection for Multivariate Ordinal Scale Outcomes and Multiple Treatments	2
Issue Information	2
On the relative conservativeness of Bayesian logistic regression method in oncology dose‐finding studies	2
Investigating Stability in Subgroup Identification for Stratified Medicine	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
Tree‐temporal scan statistics for safety signal detection in vaccine clinical trials	2
Visualizing hypothesis tests in survival analysis under anticipated delayed effects	2
Sample Size Estimation Using a Partially Clustered Frailty Model for Biomarker‐Strategy Designs With Multiple Treatments	2
Potential Bias Models With Bayesian Shrinkage Priors for Dynamic Borrowing of Multiple Historical Control Data	2
Predictive Ppk calculations for biologics and vaccines using a Bayesian approach – a tutorial	2
AIDE: Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials	2
The Estimand Framework and Causal Inference: Complementary Not Competing Paradigms	2
Group sequential design with maximin efficiency robust test for immunotherapy with generalized delayed treatment effect	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
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design	2
Introduction to Nonclinical Biopharmaceutical Statistics Tutorials—A Special Issue	2
Application of hypothetical strategies in acute pain	2
The design and analysis of vaccine trials for COVID‐19 for the purpose of estimating efficacy	2
Rejoinder to the letter: “Standard and reference‐based conditional mean imputation: Regulators and trial statisticians be aware!”	2
PubPredict: Prediction of progression and survival in oncology leveraging publications and early efficacy data	2
A Tipping Point Method to Evaluate Sensitivity to Potential Violations in Missing Data Assumptions	2
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Penalized Variable Selection for Joint AFT Random‐Effect Model With Clustered Competing‐Risks Data	2
Should the two‐trial paradigm still be the gold standard in drug assessment?	2
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Adaptively leveraging external data with robust meta‐analytical‐predictive prior using empirical Bayes	2
Comparing various Bayesian random‐effects models for pooling randomized controlled trials with rare events	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
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	2
Handling Missing Data in Participants with Baseline but No Post‐Baseline Data	1
A Flexible Seamless Phase 2/3 Design With Biomarker‐Driven Subgroup Enrichment and Sample Size Re‐Estimation	1
Correction to “Mixture Experimentation in Pharmaceutical Formulations: A Tutorial”	1
A Dose‐Finding Design for Drug Combinations Using a Bayesian 4 Parameter Logistic Model With Penalised D‐Optimality	1
“Within‐Trial” Prognostic Score Adjustment Is Targeted Maximum Likelihood Estimation	1
Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation	1
Robust Modestly Weighted Log‐Rank Tests	1
Issue Information	1

A Bayesian optimal interval design for dose optimization with a randomization scheme based on pharmacokinetics outcomes in oncology	1
A Bayesian Dynamic Model‐Based Adaptive Design for Oncology Dose Optimization in Phase I/II Clinical Trials	1
Combining evidence from clinical trials in conditional or accelerated approval	1
Natural cubic splines for the analysis of Alzheimer's clinical trials	1
Issue Information	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
An improved score‐type confidence interval for stratified risk differences involving rare events	1
Issue Information	1
Survival Analysis Without Sharing of Individual Patient Data by Using a Gaussian Copula	1
Issue Information	1
Correction to “Sample Size Estimation for Correlated Count Data With Changes in Dispersion”	1
Predictive Biomarker Graphical Approach ( PRIME ) for Precision Medicine	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
Issue Information	1
Setting the control limit at release for stability assurance	1
A Statistical Concept for Conditional Marketing Authorisation Based on the Intermediate and Final Outcomes of a Single Confirmatory Randomised Clinical Trial	1
Statistical considerations for design and analysis of stability, comparability and formulation tests	1
Issue Information	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
Mediation Analysis of Path‐Specific Effects in Randomised Clinical Trials With Repeatedly Measured Mediators and Outcomes	1
Comparison of nonparametric estimators of the expected number of recurrent events	1
Great Wall: A Generalized Dose Optimization Design for Drug Combination Trials Maximizing Survival Benefit	1
Issue Information	1
A Tutorial on Improving RCT Power Using Prognostic Score Adjustment for Linear Models	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
Time‐to‐event calibration‐free odds design: A robust efficient design for phase I trials with late‐onset outcomes	1
A comparison of statistical methods for animal oncology studies	1
Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	1
Statistical modeling approaches for the comparison of dissolution profiles	1
Statistical Consideration for Event‐Free Survival With Cure Rate in Acute Myeloid Leukemia Studies	1
Detection of Outlying Correlation Coefficients in Multicenter Clinical Trials	1
Methodological Approaches for the Estimation of Confidence Intervals on Partial Youden Index Under Verification Bias	1
Confirmatory efficacy testing for individual dose–placebo comparisons using serial gatekeeping procedure in dose‐finding trials with multiple comparison procedures–modeling	1
Introduction to qualification and validation of an immunoassay	1
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Comment on “Average Hazard as Harmonic Mean” by Chiba (2025)	1
Subgroup Identification Based on Quantitative Objectives	1
Replenishing the pipeline: A quantitative approach to optimising the sourcing of new projects	1
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data	1
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PKBOIN‐12: A Bayesian Optimal Interval Phase I/II Design Incorporating Pharmacokinetics Outcomes to Find the Optimal Biological Dose	1
Defining estimands for efficacy assessment in single arm phase 1b or phase 2 clinical trials in oncology early development	1
Informed decision‐making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments	1
A Phase I Dose‐Finding Design Incorporating Intra‐Patient Dose Escalation	1
Shrinkage priors for isotonic probability vectors and binary data modeling, with applications to dose–response modeling	1
Evaluation of a flexible piecewise linear mixed‐effects model in the analysis of randomized cross‐over trials	1
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Assessment of Responder Analyses for Patient‐Reported Outcomes With Varying Variability Between Intervention Groups: Maximum Responder Threshold and Effect	1
A propensity score‐integrated approach for leveraging external data in a randomized controlled trial with time‐to‐event endpoints	1
Sample size re‐estimation in Phase 2 dose‐finding: Conditional power versus Bayesian predictive power	1
Information fraction estimation: Strategies for a phase 3 non‐inferiority maximum duration design with time to event outcome	1
Issue Information	1
Order of Addition in Mixture‐Amount Experiments	1
A Federated Data Analysis Approach for the Evaluation of Surrogate Endpoints	1
A model‐assisted design for partially or completely ordered groups	1
Sample size calculation for comparing two ROC curves	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