Pharmaceutical Statistics

Article	Citations
Bayesian single‐to‐double arm transition design using both short‐term and long‐term endpoints	52
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	23
On Some Modeling Issues in Estimating Vaccine Efficacy	20
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	17
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Issue Information	16
Conditional power and information fraction calculations at an interim analysis for random coefficient models	15
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Inverse probability of censoring weighting for visual predictive checks of time‐to‐event models with time‐varying covariates	12
A semiparametric modeling approach for analyzing clinical biomarkers restricted to limits of detection	12
Informed decision‐making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments	12
Using the Bayesian detection of potential risk using inference on blinded safety data (BDRIBS) method to support the decision to refer an event for unblinded evaluation	11
Issue Information	11
Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation	11
Evaluating response‐adaptive randomization procedures for recurrent events and terminal event data using a composite endpoint	11
A Bayesian optimal interval design for dose optimization with a randomization scheme based on pharmacokinetics outcomes in oncology	10
The Acute Stroke Therapy by Inhibition of Neutrophils study – Key features and impact	9
Sample size re‐estimation for response‐adaptive randomized clinical trials	9
Identifying treatment effects using trimmed means when data are missing not at random	9
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	9
Frailty model with change points for survival analysis	9
A marginalized two‐part joint model for a longitudinal biomarker and a terminal event with application to advanced head and neck cancers	9
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	9
Sample size re‐estimation in Phase 2 dose‐finding: Conditional power versus Bayesian predictive power	9
Evaluation of a flexible piecewise linear mixed‐effects model in the analysis of randomized cross‐over trials	9

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Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	8
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design	8
Sample size calculation for comparing two ROC curves	7
Using marginal structural models to analyze the impact of subsequent therapy on the treatment effect in survival data: Simulations and clinical trial examples	7
Multiple imputation analysis of Miettinen‐Nurminen interval for difference in proportions	7
Estimating and comparing adverse event probabilities in the presence of varying follow‐up times and competing events	7
Sample size calculation in clinical trials with two co‐primary endpoints including overdispersed count and continuous outcomes	7
A comparative study of adaptive trial designs for dose optimization	7
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	6
Flexible Spline Models for Blinded Sample Size Reestimation in Event‐Driven Clinical Trials	5
The utilities and pitfalls of stratified analysis in challenging situations	5
Comparison of Prior Distributions for the Heterogeneity Parameter in a Rare Events Meta‐Analysis of a Few Studies	5
Estimation of treatment effects in short‐term depression studies. An evaluation based on the ICH E9(R1) estimands framework	5
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The Role of CMC Statisticians: Co‐Practitioners of the Scientific Method	5
T3 + 3: 3 + 3 Design With Delayed Outcomes	4
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	4
Left truncation in linked data: A practical guide to understanding left truncation and applying it using SAS and R	4
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	4
Pre‐Posterior Distributions in Drug Development and Their Properties	4
Selection bias, investment decisions and treatment effect distributions	4
Improving sample size recalculation in adaptive clinical trials by resampling	4
Confirmatory efficacy testing for individual dose–placebo comparisons using serial gatekeeping procedure in dose‐finding trials with multiple comparison procedures–modeling	4
Bayesian optimal phase II designs with dual‐criterion decision making	4
A Federated Data Analysis Approach for the Evaluation of Surrogate Endpoints	4
Average Hazard as Harmonic Mean	4
Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study	4
Issue Information	4
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A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	4
Estimation of the Restricted Mean Duration of Response (RMDoR) in Oncology	3
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Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	3
On “Re‐randomization tests as sensitivity analyses to confirm immunological noninferiority of an investigational vaccine: Case study” by Luca Grassano et al. (2023, Pharmaceutical Statistics)	3
The individual‐level surrogate threshold effect in a causal‐inference setting with normally distributed endpoints	3
Weighted log‐rank test to compare two survival functions in the presence of dependent censoring	3
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Estimation and expected sample size in Simon's two‐stage designs that stop as early as possible	3
A note from the editors	3
Improving early phase oncology clinical trial design: An opportunity for statisticians	3
A conservative approach to leveraging external evidence for effective clinical trial design	3
Issue Information	3
Re‐randomization tests as sensitivity analyses to confirm immunological noninferiority of an investigational vaccine: Case study	3
Combining evidence from clinical trials in conditional or accelerated approval	3
CUSUMIN: A cumulative sum interval design for cancer phase I dose finding studies	3
A targeted simulation‐extrapolation method for evaluating biomarkers based on new technologies in precision medicine	3
A mixed effects model for analyzing area under the curve of longitudinally measured biomarkers with missing data	3
Bayesian Sample Size Calculation in Small n, Sequential Multiple Assignment Randomized Trials (snSMART)	3
An adaptive biomarker basket design in phase II oncology trials	3

Setting the control limit at release for stability assurance	3
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior	3
Shrinkage priors for isotonic probability vectors and binary data modeling, with applications to dose–response modeling	3
Issue Information	3
Statistical considerations for design and analysis of stability, comparability and formulation tests	3
Rationale for the update algorithm of the graphical approach to sequentially rejective multiple test procedures	3
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The partnership between statisticians and the Institutional Animal Care and Use Committee (IACUC)	2
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	2
Identifying treatment effect heterogeneity in dose‐finding trials using Bayesian hierarchical models	2
Current developments of the estimand concept	2
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	2
Assessing the performance of group‐based trajectory modeling method to discover different patterns of medication adherence	2
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A meta‐analytic framework to adjust for bias in external control studies	2
Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery	2
A model selection criterion for clustered survival analysis with informative cluster size	2
Optimizing Sample Size Determinations for Phase 3 Clinical Trials in Type 2 Diabetes	2
Estimating the Strength of Binding Affinity via Delta–Delta‐G for Hit Screening After a Deming Regression Calibration	2
Improving the assessment of the probability of success in late stage drug development	2
Prediction Intervals for Overdispersed Poisson Data and Their Application in Medical and Pre‐Clinical Quality Control	2
Issue Information	2
Adjusting for covariates in analysis based on restricted mean survival times	2
Applying the Estimand Framework to Non‐Inferiority Trials	2
Optimal sample size allocation for two‐arm superiority and non‐inferiority trials with binary endpoints	2
Semi‐parametric accelerated failure‐time model: A useful alternative to the proportional‐hazards model in cancer clinical trials	2
Practical and robust test for comparing binomial proportions in the randomized phase II setting	2
Potency Assay Variability Estimation in Practice	2
Balance Index to Determine the Follow‐Up Duration of Oncology Trials	2
Using Propensity Score Weighting to Enhance the Operating Characteristics of Power Prior in Leveraging External Data to Augment a Traditional Clinical Study	2
An adaptive design for early clinical development including interim decision for single‐arm trial with external controls or randomized trial	2
A Model‐Based Trial Design With a Randomization Scheme Considering Pharmacokinetics Exposure for Dose Optimization in Oncology	2
Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance	2
Statistical modeling approaches for the comparison of dissolution profiles	2
Sample Size Reestimation in Stochastic Curtailment Tests With Time‐to‐Events Outcome in the Case of Nonproportional Hazards Utilizing Two Weibull Distributions With Unknown Shape Parameters	2
A two‐stage drop‐the‐losers design for time‐to‐event outcome using a historical control arm	2
Counterfactual mediation analysis in the multistate model framework for surrogate and clinical time‐to‐event outcomes in randomized controlled trials	2
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose	2
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	2
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.	2
Efficient Study Design and Analysis of Longitudinal Dose–Response Data Using Fractional Polynomials	2
Experimental design considerations and statistical analyses in preclinical tumor growth inhibition studies	2
The detailed clinical objectives approach to designing clinical trials and choosing estimands	2
Issue Information	2
Empirical likelihood confidence interval for sensitivity to the early disease stage	2
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	2
A dynamic power prior approach to non‐inferiority trials for normal means	1
Comparison of nonparametric estimators of the expected number of recurrent events	1
Estimating survival parameters under conditionally independent left truncation	1
Estimation of a treatment policy estimand for time to event data using data collected post discontinuation of randomised treatment	1
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A generalized Bayesian optimal interval design for dose optimization in immunotherapy	1
Issue Information	1
Adaptive designs for IVPT data with mixed scaled average bioequivalence	1
Assessment of pharmacokinetic linearity after repeated drug administration	1
Principles for Defining Estimands in Clinical Trials—A Proposal	1
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A case study: Assessing the efficacy of the revised dosage regimen via prediction model for recurrent event rate using biomarker data	1
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Key considerations for choosing a statistical method to deal with incomplete treatment adherence in pragmatic trials	1
Issue Information	1
Doubly‐robust methods for differences in restricted mean lifetimes using pseudo‐observations	1
Multiple Comparisons Procedures for Analyses of Joint Primary Endpoints and Secondary Endpoints	1
SCI: A Bayesian adaptive phase I/II dose‐finding design accounting for semi‐competing risks outcomes for immunotherapy trials	1
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data	1
The importance of using ordinal scores for patient classification based on health‐related quality of life trajectories	1
Subgroup Identification Based on Quantitative Objectives	1
The cross‐over of statistical thinking and practices: A pandemic catalyst	1
Flexible diagnostic measures and new cut‐point selection methods under multiple ordered classes	1
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	1
On sample size calculation in drug interaction trials	1
Addressing and communicating heterogeneous treatment effects for patient subpopulations: Challenges and opportunities	1
Adaptively leveraging external data with robust meta‐analytical‐predictive prior using empirical Bayes	1
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Mixture Experimentation in Pharmaceutical Formulations: A Tutorial	1
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A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	1
Randomization in Pre‐Clinical Studies: When Evolution Theory Meets Statistics	1
From innovative thinking to pharmaceutical industry implementation: Some success stories	1
Web based resource for Statistical Consultants in the Pharmaceutical Industry	1

A two‐stage adaptive clinical trial design with data‐driven subgroup identification at interim analysis	1
The Choice Between Pearson's χ2 Test and Fisher's Exact Test for 2 × 2 Tables	1
Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components	1
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	1
Ensuring exchangeability indata‐basedpriors for a Bayesian analysis of clinical trials	1
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Analysis of an incomplete binary outcome dichotomized from an underlying continuous variable in clinical trials	1
Issue Information	1
Group sequential design with maximin efficiency robust test for immunotherapy with generalized delayed treatment effect	1
Adaptive designs for best treatment identification with top‐two Thompson sampling and acceleration	1
Strategy for Designing In Vivo Dose–Response Comparison Studies	1
Issue Information	1
Synergy detection: A practical guide to statistical assessment of potential drug combinations	1
Simulation‐based sequential design	1
Simultaneous Inference Using Multiple Marginal Models	1
Determining the minimum duration of treatment in tuberculosis: An order restricted non‐inferiority trial design	1
Propensity score‐incorporated adaptive design approaches when incorporating real‐world data	1
Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials	1
On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	1
A fiducial approach for testing the non‐inferiority of proportion difference in matched‐pairs design	0
Incorporating historical information to improve phase I clinical trials	0
Covariate adjustment and estimation of difference in proportions in randomized clinical trials	0
Potential Bias Models With Bayesian Shrinkage Priors for Dynamic Borrowing of Multiple Historical Control Data	0
Operational characteristics of generalized pairwise comparisons for hierarchically ordered endpoints	0
Issue Information	0
Variable Duration Trial as an Alternative Design for Continuous Endpoints	0
Incorporating covariates information in adaptive clinical trials for precision medicine	0
Robust estimates of regional treatment effects in multiregional randomized clinical trials with semiparametric logistic model	0
Issue Information	0
AIDE: Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials	0
Sample Size Estimation for Correlated Count Data With Changes in Dispersion	0
The flaw of averages: Bayes factors as posterior means of the likelihood ratio	0
Evaluating hybrid controls methodology in early‐phase oncology trials: A simulation study based on the MORPHEUS‐UC trial	0
Bayesian adaptive randomization design incorporating propensity score‐matched historical controls	0
Alone, together: On the benefits of Bayesian borrowing in a meta‐analytic setting	0
Beyond the Fragility Index	0
Bivariate Bayesian hypothesis testing with missing data in components	0
The stratified win statistics (win ratio, win odds, and net benefit)	0
Issue Information	0
Return‐to‐baseline multiple imputation for missing values in clinical trials	0
Using an interim analysis based exclusively on an early outcome in a randomized clinical trial with a long‐term clinical endpoint	0
Kernel hazard estimation for visualisation of the effect of a continuous covariate on time‐to‐event endpoints	0
Tutorial on Firth's Logistic Regression Models for Biomarkers in Preclinical Space	0
Heterogeneity in treatment effects across diverse populations	0
Estimands for overall survival in clinical trials with treatment switching in oncology	0
Approximate Bayesian Analysis for Borrowing External Controls for Randomized Controlled Trials With Dynamic Borrowing and Covariate Balancing Adjustment	0
A Bayesian phase I/II design to determine subgroup‐specific optimal dose for immunotherapy sequentially combined with radiotherapy	0
Separation and the information theory surrogate evaluation approach: A penalised likelihood solution	0
Trial Probability of Success for Testing 3‐Way PK/PD Similarity With Multiple Endpoints	0
Sample Size Estimation Using a Partially Clustered Frailty Model for Biomarker‐Strategy Designs With Multiple Treatments	0
The use of external controls: To what extent can it currently be recommended?	0
Statistical analysis of actigraphy data with generalised additive models	0
Optimal Cut‐Point Selection Methods Under Binary Classification When Subclasses Are Involved	0
A comparison of statistical methods for animal oncology studies	0
Correction to “The Flaw of Averages: Bayes Factors as Posterior Means of the Likelihood Ratio”	0
Inclusion of binary proxy variables in logistic regression improves treatment effect estimation in observational studies in the presence of binary unmeasured confounding variables	0
Handling Partially Observed Trial Data After Treatment Withdrawal: Introducing Retrieved Dropout Reference‐Base Centred Multiple Imputation	0
Issue Information	0
Visualizing hypothesis tests in survival analysis under anticipated delayed effects	0
Bayesian borrowing from historical control data in a vaccine efficacy trial	0
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Issue Information	0
Just say no to data listings!	0
What they forgot to tell you about machine learning with an application to pharmaceutical manufacturing	0
Foreword	0
Utilizing restricted mean duration of response for efficacy evaluation of cancer treatments	0
A treatment‐specific marginal structural Cox model for the effect of treatment discontinuation	0
WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors	0
A note on confidence intervals for the restricted mean survival time based on transformations in small sample size	0
Methodological challenges in the analysis of recurrent events for randomised controlled trials with application to cardiovascular events in LEADER	0
Estimation of Treatment Policy Estimands for Continuous Outcomes Using Off‐Treatment Sequential Multiple Imputation	0
Transporting randomized trial results to estimate counterfactual survival functions in target populations	0
BF‐BOIN‐ET: A Backfill Bayesian Optimal Interval Design Using Efficacy and Toxicity Outcomes for Dose Optimization	0
A unified approach for evaluating the prediction of treatment effect across different types of endpoints	0
Balance diagnostics in propensity score analysis following multiple imputation: A new method	0
Bayesian Methods for Quality Tolerance Limit (QTL) Monitoring	0
Natural cubic splines for the analysis of Alzheimer's clinical trials	0
Assessing the Operational Characteristics of the Individual Causal Association as a Metric of Surrogacy in the Binary Continuous Setting	0
Mathematical programming tools for randomization purposes in small two‐arm clinical trials: A case study with real data	0
Estimands in observational studies: Some considerations beyondICH E9(R1)	0
Tree‐temporal scan statistics for safety signal detection in vaccine clinical trials	0
Bayesian MCPMod	0
Win statistics (win ratio, win odds, and net benefit) can complement one another to show the strength of the treatment effect on time‐to‐event outcomes	0
Probability of success and group sequential designs	0
Sample size calculation for recurrent event data with additive rates models	0
A Bayesian Hybrid Design With Borrowing From Historical Study	0
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Real Effect or Bias? Good Practices for Evaluating the Robustness of Evidence From Comparative Observational Studies Through Quantitative Sensitivity Analysis for Unmeasured Confounding	0
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Number of Repetitions in Re‐Randomization Tests	0
Study‐design in pandemics: From surveillance and performance‐evaluation to licensing and pharmacovigilance	0
Assessing safety at the end of clinical trials using system organ classes: A case and comparative study	0
Joint confidence region estimation on predictive values	0
Estimation of the odds ratio from multi‐stage randomized trials	0
New Methods for Two‐Stage Treatment Switching Estimation	0