Pharmaceutical Statistics

Article	Citations
Frailty model with change points for survival analysis	37
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	24
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	18
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	18
Pre‐Posterior Distributions in Drug Development and Their Properties	18
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	18
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	17
Average Hazard as Harmonic Mean	16
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Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	15
Weighted log‐rank test to compare two survival functions in the presence of dependent censoring	14
A conservative approach to leveraging external evidence for effective clinical trial design	13
Applying the Estimand Framework to Non‐Inferiority Trials	13
A meta‐analytic framework to adjust for bias in external control studies	13
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	13
Issue Information	12
A two‐stage drop‐the‐losers design for time‐to‐event outcome using a historical control arm	12
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	11
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
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose	10
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
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior	10
Why “Minimal Clinically Important Difference” for Interpreting the Magnitude of the Treatment Effect Is Not Useful	8

Current developments of the estimand concept	8
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	8
Estimation of a treatment policy estimand for time to event data using data collected post discontinuation of randomised treatment	7
Simulation‐based sequential design	7
From innovative thinking to pharmaceutical industry implementation: Some success stories	7
Simultaneous Inference Using Multiple Marginal Models	7
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	7
Mixture Experimentation in Pharmaceutical Formulations: A Tutorial	7
A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	7
Reflecting on Andy Grieve's influence and innovation: A personal perspective	6
Issue Information	6
An illness–death multistate model to implement delta adjustment and reference‐based imputation with time‐to‐event endpoints	6
Should responder analyses be conducted on continuous outcomes?	6
Bayesian Response Adaptive Randomization for Randomized Clinical Trials With Continuous Outcomes: The Role of Covariate Adjustment	6
Chauhan Weighted Trajectory Analysis of Combined Efficacy and Safety Outcomes for Risk–Benefit Analysis	6
Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components	6
Interim decision making in seamless trial designs: An application in an adaptive dose‐finding study in a rare kidney disease	5
An evolutionary algorithm for the direct optimization of covariate balance between nonrandomized populations	5
Using an interim analysis based exclusively on an early outcome in a randomized clinical trial with a long‐term clinical endpoint	5
Balance diagnostics in propensity score analysis following multiple imputation: A new method	5
Study‐design in pandemics: From surveillance and performance‐evaluation to licensing and pharmacovigilance	5
Applying the Principal Stratum Strategy in Equivalence Trials: A Case Study	5
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Bayesian borrowing from historical control data in a vaccine efficacy trial	5
Statistical detection of synergy: New methods and a comparative study	5
Statistical methods for handling missing data to align with treatment policy strategy	5
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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
The Wilcoxon–Mann–Whitney Estimand Versus Differences in Medians or Means	4
A Personalized Dose‐Finding Algorithm Based on Adaptive Gaussian Process Regression	4
Methodological challenges in the analysis of recurrent events for randomised controlled trials with application to cardiovascular events in LEADER	4
Geometric approaches to assessing the numerical feasibility for conducting matching‐adjusted indirect comparisons	4
“Super‐covariates”: Using predicted control group outcome as a covariate in randomized clinical trials	4
A Bayesian Hybrid Design With Borrowing From Historical Study	4
Regulatory issues and the potential use of Bayesian approaches for early drug approval systems in Japan	4
Beyond the Fragility Index	4
What they forgot to tell you about machine learning with an application to pharmaceutical manufacturing	4
Improving precision and power in randomized trials with a two‐stage study design: Stratification using clustering method	4
Issue Information	4
Tutorial on Firth's Logistic Regression Models for Biomarkers in Preclinical Space	4
Evaluating hybrid controls methodology in early‐phase oncology trials: A simulation study based on the MORPHEUS‐UC trial	4
Comparative Analyses of Bioequivalence Assessment Methods for In Vitro Permeation Test Data	4
Issue Information	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
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Issue Information	3
Bayesian optimal phase II designs with dual‐criterion decision making	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
Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study	3
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	3
Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance	3
An adaptive design for early clinical development including interim decision for single‐arm trial with external controls or randomized trial	3
Using Propensity Score Weighting to Enhance the Operating Characteristics of Power Prior in Leveraging External Data to Augment a Traditional Clinical Study	3
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	3
Semi‐parametric accelerated failure‐time model: A useful alternative to the proportional‐hazards model in cancer clinical trials	3
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	3
Improving the assessment of the probability of success in late stage drug development	3
The Acute Stroke Therapy by Inhibition of Neutrophils study – Key features and impact	3
An adaptive biomarker basket design in phase II oncology trials	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
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	3
Introduction to Nonclinical Biopharmaceutical Statistics Tutorials—A Special Issue	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
Principles for Defining Estimands in Clinical Trials—A Proposal	2
Transporting randomized trial results to estimate counterfactual survival functions in target populations	2
Estimation of multivariate treatment effects in contaminated clinical trials	2
Sample Size Estimation Using a Partially Clustered Frailty Model for Biomarker‐Strategy Designs With Multiple Treatments	2
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Issue Information	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
Comparing various Bayesian random‐effects models for pooling randomized controlled trials with rare events	2
Predictive Ppk calculations for biologics and vaccines using a Bayesian approach – a tutorial	2
Just say no to data listings!	2
Sample Size Estimation for Correlated Count Data With Changes in Dispersion	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
A note on point estimation and interval estimation of the relative treatment effect under a simple crossover design	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
Variable Duration Trial as an Alternative Design for Continuous Endpoints	2
Adaptively leveraging external data with robust meta‐analytical‐predictive prior using empirical Bayes	2
On the relative conservativeness of Bayesian logistic regression method in oncology dose‐finding studies	2
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On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	2
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Potential Bias Models With Bayesian Shrinkage Priors for Dynamic Borrowing of Multiple Historical Control Data	2
Confidence intervals for point‐of‐stabilization of content uniformity	2
Should the two‐trial paradigm still be the gold standard in drug assessment?	2
Principled leveraging of external data in the evaluation of diagnostic devices via the propensity score‐integrated composite likelihood approach	2
Tree‐temporal scan statistics for safety signal detection in vaccine clinical trials	2
Rejoinder to the letter: “Standard and reference‐based conditional mean imputation: Regulators and trial statisticians be aware!”	2
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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
Application of hypothetical strategies in acute pain	2
Web based resource for Statistical Consultants in the Pharmaceutical Industry	2
Quality by Design for Preclinical In Vitro Assay Development	2
Issue Information	2
A Commensurate Prior Model With Random Effects for Survival and Competing Risk Outcomes to Accommodate Historical Controls	2
Visualizing hypothesis tests in survival analysis under anticipated delayed effects	2
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Empirical likelihood inference for area under the receiver operating characteristic curve using ranked set samples	1
Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	1
Time‐to‐event calibration‐free odds design: A robust efficient design for phase I trials with late‐onset outcomes	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
Correction to “Mixture Experimentation in Pharmaceutical Formulations: A Tutorial”	1
Optimal unplanned design modification in adaptive two‐stage trials	1
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data	1
Issue Information	1
A Phase I Dose‐Finding Design Incorporating Intra‐Patient Dose Escalation	1
Adjusting for bias in the mean for primary and secondary outcomes when trials are in sequence	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
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
Introduction to qualification and validation of an immunoassay	1
Detection of Outlying Correlation Coefficients in Multicenter Clinical Trials	1
Assessment of pharmacokinetic linearity after repeated drug administration	1
Do statisticians count? A prior and posterior viewpoint	1
Defining estimands for efficacy assessment in single arm phase 1b or phase 2 clinical trials in oncology early development	1
PubPredict: Prediction of progression and survival in oncology leveraging publications and early efficacy data	1
Do You Want to Stay Single? Considerations on Single‐Arm Trials in Drug Development and the Postregulatory Space	1

Issue Information	1
Sample size calculation for comparing two ROC curves	1
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design	1
A practical guide to sample size calculations: Installation of the app SampSize	1
Issue Information	1
Information fraction estimation: Strategies for a phase 3 non‐inferiority maximum duration design with time to event outcome	1
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Comparison of nonparametric estimators of the expected number of recurrent events	1
Setting the control limit at release for stability assurance	1
A treatment‐specific marginal structural Cox model for the effect of treatment discontinuation	1
Statistical modeling approaches for the comparison of dissolution profiles	1
A Flexible Seamless Phase 2/3 Design With Biomarker‐Driven Subgroup Enrichment and Sample Size Re‐Estimation	1
Ensuring exchangeability indata‐basedpriors for a Bayesian analysis of clinical trials	1
A comparison of statistical methods for animal oncology studies	1
Estimating survival parameters under conditionally independent left truncation	1
Estimating the treatment effect for adherers using multiple imputation	1
Issue Information	1
WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors	1
Standard and reference‐based conditional mean imputation	1
Investigating Stability in Subgroup Identification for Stratified Medicine	1
Return‐to‐baseline multiple imputation for missing values in clinical trials	1
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Probability of success and group sequential designs	1
Issue Information	1
A model‐assisted design for partially or completely ordered groups	1
AIDE: Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials	1
An improved score‐type confidence interval for stratified risk differences involving rare events	1
A propensity score‐integrated approach for leveraging external data in a randomized controlled trial with time‐to‐event endpoints	1
Personalized Treatment Selection for Multivariate Ordinal Scale Outcomes and Multiple Treatments	1
A Bayesian Dynamic Model‐Based Adaptive Design for Oncology Dose Optimization in Phase I/II Clinical Trials	1
Issue Information	1
Replenishing the pipeline: A quantitative approach to optimising the sourcing of new projects	1
Statistical considerations for design and analysis of stability, comparability and formulation tests	1
Comment on “Average Hazard as Harmonic Mean” by Chiba (2025)	1
PKBOIN‐12: A Bayesian Optimal Interval Phase I/II Design Incorporating Pharmacokinetics Outcomes to Find the Optimal Biological Dose	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
A Tutorial on Improving RCT Power Using Prognostic Score Adjustment for Linear Models	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
Subgroup Identification Based on Quantitative Objectives	1
Estimands for overall survival in clinical trials with treatment switching in oncology	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
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
Survival Analysis Without Sharing of Individual Patient Data by Using a Gaussian Copula	1
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A simulation‐based comparison of estimation methods for adaptive and classical group sequential clinical trials	1
Decision rules for identifying combination therapies in open‐entry, randomized controlled platform trials	1
The design and analysis of vaccine trials for COVID‐19 for the purpose of estimating efficacy	1