Pharmaceutical Statistics

Article	Citations
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	39
Prediction Intervals for Overdispersed Binomial Endpoints and Their Application to Toxicological Historical Control Data	29
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	22
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
Average Hazard as Harmonic Mean	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	14
Frailty model with change points for survival analysis	14
Pre‐Posterior Distributions in Drug Development and Their Properties	13
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	13
Control of Unconditional Type I Error in Clinical Trials With External Control Borrowing—A Two‐Stage Adaptive Design Perspective	12
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	12
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose	12
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	12
A conservative approach to leveraging external evidence for effective clinical trial design	12
Issue Information	12
Extending Multiple Testing With Unknown Test Dependency via the CoCo Test: With Applications to Cancer Studies	10
Current developments of the estimand concept	10
A meta‐analytic framework to adjust for bias in external control studies	10
Multiplicity Adjustment Methods for a Three‐Way Crossover Bioequivalence Study	10
Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery	9
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	9
A Model‐Based Trial Design With a Randomization Scheme Considering Pharmacokinetics Exposure for Dose Optimization in Oncology	9

Why “Minimal Clinically Important Difference” for Interpreting the Magnitude of the Treatment Effect Is Not Useful	9
Applying the Estimand Framework to Non‐Inferiority Trials	8
Confidence Intervals for Validation of Analytical Procedures Under ICH Q2 ( R2 )	8
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	8
Finding the Optimal Number of Splits and Repetitions in Double Cross‐Fitting Targeted Maximum Likelihood Estimators	7
Mixture Experimentation in Pharmaceutical Formulations: A Tutorial	7
Applying the Principal Stratum Strategy in Equivalence Trials: A Case Study	7
Issue Information	7
Simultaneous Inference Using Multiple Marginal Models	7
An illness–death multistate model to implement delta adjustment and reference‐based imputation with time‐to‐event endpoints	7
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	7
A Unified Approach to Covariate Adjustment for Survival Endpoints in Randomized Clinical Trials	7
Chauhan Weighted Trajectory Analysis of Combined Efficacy and Safety Outcomes for Risk–Benefit Analysis	7
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An evolutionary algorithm for the direct optimization of covariate balance between nonrandomized populations	6
Should responder analyses be conducted on continuous outcomes?	6
Frequentist and Bayesian tolerance intervals for setting specification limits for left‐censored gamma distributed drug quality attributes	6
Simulation‐Based Bayesian Predictive Probability of Success for Interim Monitoring of Clinical Trials With Competing Event Data: Two Case Studies	6
Application of Causal Inference to Establish Assay Effect in the Absence of a Bridging Study: A Case Study of MenACWY ‐ CRM<	6
BOIN ‐ MEM : A Two‐Stage Design for Dose Optimization With Information Borrowing Across Dose Levels and Stages in Onco	6
Bayesian Response Adaptive Randomization for Randomized Clinical Trials With Continuous Outcomes: The Role of Covariate Adjustment	6
Interim decision making in seamless trial designs: An application in an adaptive dose‐finding study in a rare kidney disease	6
Statistical methods for handling missing data to align with treatment policy strategy	6
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Informing the Borrowing Process for Dose‐Finding Trials by Estimating the Similarity Between Population‐Specific Dose‐Toxicity Curves	6
An Ensemble Classifier for Ordinal Outcomes in High‐Dimensional Genomics Data	5
A Tree‐Based Scan Statistic for Detecting Signals of Drug–Drug Interactions in Spontaneous Reporting Databases	5
Bayesian borrowing from historical control data in a vaccine efficacy trial	5
Issue Information	5
Improving precision and power in randomized trials with a two‐stage study design: Stratification using clustering method	5
Balance diagnostics in propensity score analysis following multiple imputation: A new method	5
Tutorial on Firth's Logistic Regression Models for Biomarkers in Preclinical Space	5
A Bayesian Hybrid Design With Borrowing From Historical Study	5
A Personalized Dose‐Finding Algorithm Based on Adaptive Gaussian Process Regression	5
Beyond the Fragility Index	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
CUSUMIN Combination: A Cumulative Sum Interval Design for Phase I Cancer Drug‐Combination Trials	5
Evaluating hybrid controls methodology in early‐phase oncology trials: A simulation study based on the MORPHEUS‐UC trial	5
Comparative Analyses of Bioequivalence Assessment Methods for In Vitro Permeation Test Data	4
Designing and Evaluating Bayesian Advanced Adaptive Randomised Clinical Trials: A Practical Guide	4
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Issue Information	4
What they forgot to tell you about machine learning with an application to pharmaceutical manufacturing	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
Bayesian optimal phase II designs with dual‐criterion decision making	4
Applying Bias Correction Methods to Build Hybrid Controls Using Real‐World Patients for a Phase IIb Randomized Controlled Trial of Baricitinib for Rheumatoid Arthritis	4
“Super‐covariates”: Using predicted control group outcome as a covariate in randomized clinical trials	4
Issue Information	4
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	4

Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials	4
Comparing various Bayesian random‐effects models for pooling randomized controlled trials with rare events	3
Rejoinder to the letter: “Standard and reference‐based conditional mean imputation: Regulators and trial statisticians be aware!”	3
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Adaptively leveraging external data with robust meta‐analytical‐predictive prior using empirical Bayes	3
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	3
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Multiple Comparisons Procedures for Analyses of Joint Primary Endpoints and Secondary Endpoints	3
Penalized Variable Selection for Joint AFT Random‐Effect Model With Clustered Competing‐Risks Data	3
Multiple Comparisons With Overdispersed Multinomial Data: Methods, Properties and Application	3
The Estimand Framework and Causal Inference: Complementary Not Competing Paradigms	3
Principled leveraging of external data in the evaluation of diagnostic devices via the propensity score‐integrated composite likelihood approach	3
Application of hypothetical strategies in acute pain	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
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	3
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Using Propensity Score Weighting to Enhance the Operating Characteristics of Power Prior in Leveraging External Data to Augment a Traditional Clinical Study	3
On the relative conservativeness of Bayesian logistic regression method in oncology dose‐finding studies	3
On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	3
A Tipping Point Method to Evaluate Sensitivity to Potential Violations in Missing Data Assumptions	3
Quality by Design for Preclinical In Vitro Assay Development	3
Variable Duration Trial as an Alternative Design for Continuous Endpoints	3
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	3
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Web based resource for Statistical Consultants in the Pharmaceutical Industry	3
Principles for Defining Estimands in Clinical Trials—A Proposal	3
Issue Information	3
Predictive Ppk calculations for biologics and vaccines using a Bayesian approach – a tutorial	3
Group sequential design with maximin efficiency robust test for immunotherapy with generalized delayed treatment effect	3
A Commensurate Prior Model With Random Effects for Survival and Competing Risk Outcomes to Accommodate Historical Controls	3
Issue Information	2
Statistical considerations for design and analysis of stability, comparability and formulation tests	2
Do You Want to Stay Single? Considerations on Single‐Arm Trials in Drug Development and the Postregulatory Space	2
Handling Missing Data in Participants with Baseline but No Post‐Baseline Data	2
AIDE: Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials	2
Predictive Biomarker Graphical Approach ( PRIME ) for Precision Medicine	2
Visualizing hypothesis tests in survival analysis under anticipated delayed effects	2
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design	2
Probability of success and group sequential designs	2
Alone, together: On the benefits of Bayesian borrowing in a meta‐analytic setting	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
Sample Size Estimation Using a Partially Clustered Frailty Model for Biomarker‐Strategy Designs With Multiple Treatments	2
Bayesian Power‐Based Sample Size Determination for Single‐Arm Clinical Trials With Time‐to‐Event Endpoints	2
Issue Information	2
Correction to “Mixture Experimentation in Pharmaceutical Formulations: A Tutorial”	2
Issue Information	2
Leveraging Two‐Stage δ Global Sensibility Analysis Method to Inform Parameter Estimation in PBPK Models	2
PubPredict: Prediction of progression and survival in oncology leveraging publications and early efficacy data	2
A Federated Data Analysis Approach for the Evaluation of Surrogate Endpoints	2
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Statistical analysis of actigraphy data with generalised additive models	2
Sample Size Estimation for Correlated Count Data With Changes in Dispersion	2
Transporting randomized trial results to estimate counterfactual survival functions in target populations	2
Investigating Stability in Subgroup Identification for Stratified Medicine	2
Setting the control limit at release for stability assurance	2
“Within‐Trial” Prognostic Score Adjustment Is Targeted Maximum Likelihood Estimation	2
Subgroup Identification Based on Quantitative Objectives	2
Personalized Treatment Selection for Multivariate Ordinal Scale Outcomes and Multiple Treatments	2
Should the two‐trial paradigm still be the gold standard in drug assessment?	2
Sample size calculation for comparing two ROC curves	2
WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors	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
Tree‐temporal scan statistics for safety signal detection in vaccine clinical trials	2
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	2
Introduction to Nonclinical Biopharmaceutical Statistics Tutorials—A Special Issue	2
Just say no to data listings!	2
Potential Bias Models With Bayesian Shrinkage Priors for Dynamic Borrowing of Multiple Historical Control Data	2
A comparison of statistical methods for animal oncology studies	1
Issue Information	1
Natural cubic splines for the analysis of Alzheimer's clinical trials	1
Great Wall: A Generalized Dose Optimization Design for Drug Combination Trials Maximizing Survival Benefit	1
Evaluation of a flexible piecewise linear mixed‐effects model in the analysis of randomized cross‐over trials	1
A Tutorial on Improving RCT Power Using Prognostic Score Adjustment for Linear Models	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
Statistical modeling approaches for the comparison of dissolution profiles	1
Replenishing the pipeline: A quantitative approach to optimising the sourcing of new projects	1
Comment on “Average Hazard as Harmonic Mean” by Chiba (2025)	1

Propensity score‐incorporated adaptive design approaches when incorporating real‐world data	1
A Dose‐Finding Design for Drug Combinations Using a Bayesian 4 Parameter Logistic Model With Penalised D‐Optimality	1
Bayesian Solutions for Assessing Differential Effects in Biomarker Positive and Negative Subgroups	1
Order of Addition in Mixture‐Amount Experiments	1
The Choice Between Pearson's χ2 Test and Fisher's Exact Test for 2 × 2 Tables	1
Detection of Outlying Correlation Coefficients in Multicenter Clinical Trials	1
Shrinkage priors for isotonic probability vectors and binary data modeling, with applications to dose–response modeling	1
A model‐assisted design for partially or completely ordered groups	1
Potency Assay Variability Estimation in Practice	1
Issue Information	1
Issue Information	1
Issue Information	1
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A Bayesian Dynamic Model‐Based Adaptive Design for Oncology Dose Optimization in Phase I/II Clinical Trials	1
Assessment of Responder Analyses for Patient‐Reported Outcomes With Varying Variability Between Intervention Groups: Maximum Responder Threshold and Effect	1
Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	1
Issue Information	1
A Phase I Dose‐Finding Design Incorporating Intra‐Patient Dose Escalation	1
Sample size re‐estimation in Phase 2 dose‐finding: Conditional power versus Bayesian predictive power	1
Long‐Term Safety Evaluations in the Presence of Switching: Evaluation of Two Approaches	1
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data	1
Key considerations for choosing a statistical method to deal with incomplete treatment adherence in pragmatic trials	1
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Parametric and nonparametric methods for confidence intervals and sample size planning for win probability in parallel‐group randomized trials with Likert item and Likert scale data	1
Going beyond probability of success: Opportunities for statisticians to influence quantitative decision‐making at the portfolio level	1
An improved score‐type confidence interval for stratified risk differences involving rare events	1
Combining evidence from clinical trials in conditional or accelerated approval	1
Introduction to qualification and validation of an immunoassay	1
Issue Information	1
Time‐to‐event calibration‐free odds design: A robust efficient design for phase I trials with late‐onset outcomes	1
A Bayesian optimal interval design for dose optimization with a randomization scheme based on pharmacokinetics outcomes in oncology	1
A Statistical Concept for Conditional Marketing Authorisation Based on the Intermediate and Final Outcomes of a Single Confirmatory Randomised Clinical Trial	1
Statistical Consideration for Event‐Free Survival With Cure Rate in Acute Myeloid Leukemia Studies	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
How to avoid concerns with the interpretation of two primary endpoints if significant superiority in one is sufficient for formal proof of efficacy	1
Survival Analysis Without Sharing of Individual Patient Data by Using a Gaussian Copula	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
PKBOIN‐12: A Bayesian Optimal Interval Phase I/II Design Incorporating Pharmacokinetics Outcomes to Find the Optimal Biological Dose	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
CUI ‐ MET : A Clinical Utility Index Based Analysis and Decision Framework for Dose Optimization in Multiple‐Dose, Mul	1
On sample size calculation in drug interaction trials	1
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Adaptive designs for best treatment identification with top‐two Thompson sampling and acceleration	1
A Flexible Seamless Phase 2/3 Design With Biomarker‐Driven Subgroup Enrichment and Sample Size Re‐Estimation	1
Will the Pharmaceutical Industry Need Statisticians in an AI World?	1
Methodological Approaches for the Estimation of Confidence Intervals on Partial Youden Index Under Verification Bias	1
A case study: Assessing the efficacy of the revised dosage regimen via prediction model for recurrent event rate using biomarker data	1
Information fraction estimation: Strategies for a phase 3 non‐inferiority maximum duration design with time to event outcome	1
Experimental design considerations and statistical analyses in preclinical tumor growth inhibition studies	1
Correction to “Sample Size Estimation for Correlated Count Data With Changes in Dispersion”	1
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Defining estimands for efficacy assessment in single arm phase 1b or phase 2 clinical trials in oncology early development	1
A Bayesian Optimal Interval Design Considering Efficacy and Toxicity in Early Phase Basket Trials	1
A propensity score‐integrated approach for leveraging external data in a randomized controlled trial with time‐to‐event endpoints	1