Pharmaceutical Statistics

Article	Citations
Frailty model with change points for survival analysis	41
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	26
Prediction Intervals for Overdispersed Binomial Endpoints and Their Application to Toxicological Historical Control Data	23
Pre‐Posterior Distributions in Drug Development and Their Properties	21
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	19
Average Hazard as Harmonic Mean	18
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	17
Adaptive Constrained Weighted Estimation for Incorporating Multiple External Information Sources	16
Prospectively Specified Adaptive Bayesian Borrowing: Considerations, Methodologies, and Implementations	15
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	15
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	15
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	14
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	13
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Issue Information	12
CUSUMIN: A cumulative sum interval design for cancer phase I dose finding studies	11
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
Why “Minimal Clinically Important Difference” for Interpreting the Magnitude of the Treatment Effect Is Not Useful	10
Control of Unconditional Type I Error in Clinical Trials With External Control Borrowing—A Two‐Stage Adaptive Design Perspective	10
A conservative approach to leveraging external evidence for effective clinical trial design	9
Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery	9
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior	9
Current developments of the estimand concept	8

Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	8
From innovative thinking to pharmaceutical industry implementation: Some success stories	7
Confidence Intervals for Validation of Analytical Procedures Under ICH Q2 ( R2 )	7
A meta‐analytic framework to adjust for bias in external control studies	7
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	7
Applying the Estimand Framework to Non‐Inferiority Trials	7
A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	7
Extending Multiple Testing With Unknown Test Dependency via the CoCo Test: With Applications to Cancer Studies	7
Weighted log‐rank test to compare two survival functions in the presence of dependent censoring	7
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	6
Finding the Optimal Number of Splits and Repetitions in Double Cross‐Fitting Targeted Maximum Likelihood Estimators	6
Mixture Experimentation in Pharmaceutical Formulations: A Tutorial	6
Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components	6
Issue Information	6
Simultaneous Inference Using Multiple Marginal Models	6
Estimation of a treatment policy estimand for time to event data using data collected post discontinuation of randomised treatment	6
Simulation‐based sequential design	6
A Unified Approach to Covariate Adjustment for Survival Endpoints in Randomized Clinical Trials	6
Bayesian Response Adaptive Randomization for Randomized Clinical Trials With Continuous Outcomes: The Role of Covariate Adjustment	5
Reflecting on Andy Grieve's influence and innovation: A personal perspective	5
An evolutionary algorithm for the direct optimization of covariate balance between nonrandomized populations	5
Chauhan Weighted Trajectory Analysis of Combined Efficacy and Safety Outcomes for Risk–Benefit Analysis	5
Issue Information	5
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Interim decision making in seamless trial designs: An application in an adaptive dose‐finding study in a rare kidney disease	5
An illness–death multistate model to implement delta adjustment and reference‐based imputation with time‐to‐event endpoints	5
Applying the Principal Stratum Strategy in Equivalence Trials: A Case Study	5
Simulation‐Based Bayesian Predictive Probability of Success for Interim Monitoring of Clinical Trials With Competing Event Data: Two Case Studies	4
Statistical methods for handling missing data to align with treatment policy strategy	4
The Wilcoxon–Mann–Whitney Estimand Versus Differences in Medians or Means	4
Tutorial on Firth's Logistic Regression Models for Biomarkers in Preclinical Space	4
Propensity score matching and stratification using multiparty data without pooling	4
CUSUMIN Combination: A Cumulative Sum Interval Design for Phase I Cancer Drug‐Combination Trials	4
Bayesian borrowing from historical control data in a vaccine efficacy trial	4
Should responder analyses be conducted on continuous outcomes?	4
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Frequentist and Bayesian tolerance intervals for setting specification limits for left‐censored gamma distributed drug quality attributes	4
A Tree‐Based Scan Statistic for Detecting Signals of Drug–Drug Interactions in Spontaneous Reporting Databases	4
Beyond the Fragility Index	4
BOIN ‐ MEM : A Two‐Stage Design for Dose Optimization With Information Borrowing Across Dose Levels and Stages in Onco	4
Informing the Borrowing Process for Dose‐Finding Trials by Estimating the Similarity Between Population‐Specific Dose‐Toxicity Curves	4
Balance diagnostics in propensity score analysis following multiple imputation: A new method	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
A Bayesian Hybrid Design With Borrowing From Historical Study	4
Using Propensity Score Weighting to Enhance the Operating Characteristics of Power Prior in Leveraging External Data to Augment a Traditional Clinical Study	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
“Super‐covariates”: Using predicted control group outcome as a covariate in randomized clinical trials	3
Improving precision and power in randomized trials with a two‐stage study design: Stratification using clustering method	3

Regulatory issues and the potential use of Bayesian approaches for early drug approval systems in Japan	3
Evaluating hybrid controls methodology in early‐phase oncology trials: A simulation study based on the MORPHEUS‐UC trial	3
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	3
Issue Information	3
Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance	3
An adaptive biomarker basket design in phase II oncology trials	3
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Geometric approaches to assessing the numerical feasibility for conducting matching‐adjusted indirect comparisons	3
Comparative Analyses of Bioequivalence Assessment Methods for In Vitro Permeation Test Data	3
What they forgot to tell you about machine learning with an application to pharmaceutical manufacturing	3
Issue Information	3
Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study	3
Multiple Comparisons Procedures for Analyses of Joint Primary Endpoints and Secondary Endpoints	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
Issue Information	3
Designing and Evaluating Bayesian Advanced Adaptive Randomised Clinical Trials: A Practical Guide	3
A Personalized Dose‐Finding Algorithm Based on Adaptive Gaussian Process Regression	3
The Acute Stroke Therapy by Inhibition of Neutrophils study – Key features and impact	3
Issue Information	3
An adaptive design for early clinical development including interim decision for single‐arm trial with external controls or randomized trial	3
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
Just say no to data listings!	2
Comments on “Statistical methodology for highly variable compounds: A novel design approach for the ofatumumab phase 2 bioequivalence study”	2
Sample Size Estimation Using a Partially Clustered Frailty Model for Biomarker‐Strategy Designs With Multiple Treatments	2
Adaptively leveraging external data with robust meta‐analytical‐predictive prior using empirical Bayes	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
Quality by Design for Preclinical In Vitro Assay Development	2
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	2
On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	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
Investigating Stability in Subgroup Identification for Stratified Medicine	2
Principles for Defining Estimands in Clinical Trials—A Proposal	2
Introduction to Nonclinical Biopharmaceutical Statistics Tutorials—A Special Issue	2
Predictive Ppk calculations for biologics and vaccines using a Bayesian approach – a tutorial	2
Statistical analysis of actigraphy data with generalised additive models	2
Web based resource for Statistical Consultants in the Pharmaceutical Industry	2
Sample Size Estimation for Correlated Count Data With Changes in Dispersion	2
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Variable Duration Trial as an Alternative Design for Continuous Endpoints	2
Group sequential design with maximin efficiency robust test for immunotherapy with generalized delayed treatment effect	2
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Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials	2
Application of hypothetical strategies in acute pain	2
Do statisticians count? A prior and posterior viewpoint	2
The design and analysis of vaccine trials for COVID‐19 for the purpose of estimating efficacy	2
Potential Bias Models With Bayesian Shrinkage Priors for Dynamic Borrowing of Multiple Historical Control Data	2
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Confidence intervals for point‐of‐stabilization of content uniformity	2
Rejoinder to the letter: “Standard and reference‐based conditional mean imputation: Regulators and trial statisticians be aware!”	2
Transporting randomized trial results to estimate counterfactual survival functions in target populations	2
A Tipping Point Method to Evaluate Sensitivity to Potential Violations in Missing Data Assumptions	2
Tree‐temporal scan statistics for safety signal detection in vaccine clinical trials	2
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The Estimand Framework and Causal Inference: Complementary Not Competing Paradigms	2
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	2
On the relative conservativeness of Bayesian logistic regression method in oncology dose‐finding studies	2
Alone, together: On the benefits of Bayesian borrowing in a meta‐analytic setting	2
Probability of success and group sequential designs	2
Principled leveraging of external data in the evaluation of diagnostic devices via the propensity score‐integrated composite likelihood approach	2
Comparing various Bayesian random‐effects models for pooling randomized controlled trials with rare events	2
Should the two‐trial paradigm still be the gold standard in drug assessment?	2
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Issue Information	1
A comparison of statistical methods for animal oncology studies	1
Personalized Treatment Selection for Multivariate Ordinal Scale Outcomes and Multiple Treatments	1
Optimal unplanned design modification in adaptive two‐stage trials	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
Comment on “Average Hazard as Harmonic Mean” by Chiba (2025)	1
Issue Information	1
Statistical modeling approaches for the comparison of dissolution profiles	1
A Bayesian Dynamic Model‐Based Adaptive Design for Oncology Dose Optimization in Phase I/II Clinical Trials	1
A treatment‐specific marginal structural Cox model for the effect of treatment discontinuation	1
Estimating survival parameters under conditionally independent left truncation	1
A practical guide to sample size calculations: Installation of the app SampSize	1
Comparison of nonparametric estimators of the expected number of recurrent events	1
Information fraction estimation: Strategies for a phase 3 non‐inferiority maximum duration design with time to event outcome	1

Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	1
Defining estimands for efficacy assessment in single arm phase 1b or phase 2 clinical trials in oncology early development	1
Issue Information	1
A model‐assisted design for partially or completely ordered groups	1
Detection of Outlying Correlation Coefficients in Multicenter Clinical Trials	1
Visualizing hypothesis tests in survival analysis under anticipated delayed effects	1
Mediation Analysis of Path‐Specific Effects in Randomised Clinical Trials With Repeatedly Measured Mediators and Outcomes	1
Return‐to‐baseline multiple imputation for missing values in clinical trials	1
Natural cubic splines for the analysis of Alzheimer's clinical trials	1
Confirmatory efficacy testing for individual dose–placebo comparisons using serial gatekeeping procedure in dose‐finding trials with multiple comparison procedures–modeling	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
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
Replenishing the pipeline: A quantitative approach to optimising the sourcing of new projects	1
Issue Information	1
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data	1
Statistical Consideration for Event‐Free Survival With Cure Rate in Acute Myeloid Leukemia Studies	1
A Phase I Dose‐Finding Design Incorporating Intra‐Patient Dose Escalation	1
A Tutorial on Improving RCT Power Using Prognostic Score Adjustment for Linear Models	1
A propensity score‐integrated approach for leveraging external data in a randomized controlled trial with time‐to‐event endpoints	1
PKBOIN‐12: A Bayesian Optimal Interval Phase I/II Design Incorporating Pharmacokinetics Outcomes to Find the Optimal Biological Dose	1
An improved score‐type confidence interval for stratified risk differences involving rare events	1
Subgroup Identification Based on Quantitative Objectives	1
Introduction to qualification and validation of an immunoassay	1
Correction to “Mixture Experimentation in Pharmaceutical Formulations: A Tutorial”	1
Great Wall: A Generalized Dose Optimization Design for Drug Combination Trials Maximizing Survival Benefit	1
Survival Analysis Without Sharing of Individual Patient Data by Using a Gaussian Copula	1
Decision rules for identifying combination therapies in open‐entry, randomized controlled platform trials	1
Empirical likelihood inference for area under the receiver operating characteristic curve using ranked set samples	1
Do You Want to Stay Single? Considerations on Single‐Arm Trials in Drug Development and the Postregulatory Space	1
A Bayesian optimal interval design for dose optimization with a randomization scheme based on pharmacokinetics outcomes in oncology	1
Setting the control limit at release for stability assurance	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
Sample size calculation for comparing two ROC curves	1
Issue Information	1
A Federated Data Analysis Approach for the Evaluation of Surrogate Endpoints	1
A Flexible Seamless Phase 2/3 Design With Biomarker‐Driven Subgroup Enrichment and Sample Size Re‐Estimation	1
Issue Information	1
Statistical considerations for design and analysis of stability, comparability and formulation tests	1
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Assessment of pharmacokinetic linearity after repeated drug administration	1
Long‐Term Safety Evaluations in the Presence of Switching: Evaluation of Two Approaches	1
Correction to “Sample Size Estimation for Correlated Count Data With Changes in Dispersion”	1
Robust Modestly Weighted Log‐Rank Tests	1
Time‐to‐event calibration‐free odds design: A robust efficient design for phase I trials with late‐onset outcomes	1
Standard and reference‐based conditional mean imputation	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
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design	1
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Order of Addition in Mixture‐Amount Experiments	1
AIDE: Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials	1
Issue Information	1
WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors	1
Evaluation of a flexible piecewise linear mixed‐effects model in the analysis of randomized cross‐over trials	1