Pharmaceutical Statistics

Article	Citations
Frailty model with change points for survival analysis	29
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	20
Pre‐Posterior Distributions in Drug Development and Their Properties	17
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	17
Average Hazard as Harmonic Mean	16
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	15
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	13
Selection bias, investment decisions and treatment effect distributions	12
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	12
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose	12
Estimating and comparing adverse event probabilities in the presence of varying follow‐up times and competing events	12
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	12
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A Model‐Based Trial Design With a Randomization Scheme Considering Pharmacokinetics Exposure for Dose Optimization in Oncology	11
Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery	11
A conservative approach to leveraging external evidence for effective clinical trial design	10
The individual‐level surrogate threshold effect in a causal‐inference setting with normally distributed endpoints	10
CUSUMIN: A cumulative sum interval design for cancer phase I dose finding studies	9
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	9
Applying the Estimand Framework to Non‐Inferiority Trials	9
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	9
Weighted log‐rank test to compare two survival functions in the presence of dependent censoring	9
Issue Information	9
Control of Unconditional Type I Error in Clinical Trials With External Control Borrowing—A Two‐Stage Adaptive Design Perspective	9
A two‐stage drop‐the‐losers design for time‐to‐event outcome using a historical control arm	9

A meta‐analytic framework to adjust for bias in external control studies	8
Current developments of the estimand concept	8
Why “Minimal Clinically Important Difference” for Interpreting the Magnitude of the Treatment Effect Is Not Useful	8
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	8
From innovative thinking to pharmaceutical industry implementation: Some success stories	7
Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components	7
A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	7
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior	7
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	7
Simulation‐based sequential design	6
Mixture Experimentation in Pharmaceutical Formulations: A Tutorial	6
Estimation of a treatment policy estimand for time to event data using data collected post discontinuation of randomised treatment	6
Simultaneous Inference Using Multiple Marginal Models	6
Reflecting on Andy Grieve's influence and innovation: A personal perspective	5
Issue Information	5
An evolutionary algorithm for the direct optimization of covariate balance between nonrandomized populations	5
An illness–death multistate model to implement delta adjustment and reference‐based imputation with time‐to‐event endpoints	5
Bayes shrinkage estimator for consistency assessment of treatment effects in multi‐regional clinical trials	5
Interim decision making in seamless trial designs: An application in an adaptive dose‐finding study in a rare kidney disease	5
Bayesian borrowing from historical control data in a vaccine efficacy trial	4
Should responder analyses be conducted on continuous outcomes?	4
Frequentist and Bayesian tolerance intervals for setting specification limits for left‐censored gamma distributed drug quality attributes	4
Balance diagnostics in propensity score analysis following multiple imputation: A new method	4
Evaluating hybrid controls methodology in early‐phase oncology trials: A simulation study based on the MORPHEUS‐UC trial	4
Regulatory issues and the potential use of Bayesian approaches for early drug approval systems in Japan	4
Using an interim analysis based exclusively on an early outcome in a randomized clinical trial with a long‐term clinical endpoint	4
Bayesian Response Adaptive Randomization for Randomized Clinical Trials With Continuous Outcomes: The Role of Covariate Adjustment	4
Chauhan Weighted Trajectory Analysis of Combined Efficacy and Safety Outcomes for Risk–Benefit Analysis	4
Study‐design in pandemics: From surveillance and performance‐evaluation to licensing and pharmacovigilance	4
Tutorial on Firth's Logistic Regression Models for Biomarkers in Preclinical Space	4
What they forgot to tell you about machine learning with an application to pharmaceutical manufacturing	4
Geometric approaches to assessing the numerical feasibility for conducting matching‐adjusted indirect comparisons	4
Statistical methods for handling missing data to align with treatment policy strategy	4
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Statistical detection of synergy: New methods and a comparative study	4
Estimation of Treatment Policy Estimands for Continuous Outcomes Using Off‐Treatment Sequential Multiple Imputation	4
Improving precision and power in randomized trials with a two‐stage study design: Stratification using clustering method	4
A Personalized Dose‐Finding Algorithm Based on Adaptive Gaussian Process Regression	4
Improving the assessment of the probability of success in late stage drug development	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
CUSUMIN Combination: A Cumulative Sum Interval Design for Phase I Cancer Drug‐Combination Trials	3
Propensity score matching and stratification using multiparty data without pooling	3
Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study	3
Comparative Analyses of Bioequivalence Assessment Methods for In Vitro Permeation Test Data	3
Semi‐parametric accelerated failure‐time model: A useful alternative to the proportional‐hazards model in cancer clinical trials	3
Multiple Comparisons Procedures for Analyses of Joint Primary Endpoints and Secondary Endpoints	3
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	3
An adaptive biomarker basket design in phase II oncology trials	3
Issue Information	3
A Bayesian Hybrid Design With Borrowing From Historical Study	3

“Super‐covariates”: Using predicted control group outcome as a covariate in randomized clinical trials	3
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Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	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
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	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
Beyond the Fragility Index	3
Methodological challenges in the analysis of recurrent events for randomised controlled trials with application to cardiovascular events in LEADER	3
The Acute Stroke Therapy by Inhibition of Neutrophils study – Key features and impact	3
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Issue Information	3
The detailed clinical objectives approach to designing clinical trials and choosing estimands	3
Rejoinder to the letter: “Standard and reference‐based conditional mean imputation: Regulators and trial statisticians be aware!”	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
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Application of hypothetical strategies in acute pain	2
Principled leveraging of external data in the evaluation of diagnostic devices via the propensity score‐integrated composite likelihood approach	2
Group sequential design with maximin efficiency robust test for immunotherapy with generalized delayed treatment effect	2
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Adaptively leveraging external data with robust meta‐analytical‐predictive prior using empirical Bayes	2
An adaptive design for early clinical development including interim decision for single‐arm trial with external controls or randomized trial	2
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Predictive Ppk calculations for biologics and vaccines using a Bayesian approach – a tutorial	2
Should the two‐trial paradigm still be the gold standard in drug assessment?	2
Variable Duration Trial as an Alternative Design for Continuous Endpoints	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
A Tipping Point Method to Evaluate Sensitivity to Potential Violations in Missing Data Assumptions	2
Principles for Defining Estimands in Clinical Trials—A Proposal	2
Estimation of multivariate treatment effects in contaminated clinical trials	2
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
Comparing various Bayesian random‐effects models for pooling randomized controlled trials with rare events	2
Issue Information	2
On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	2
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	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
Tree‐temporal scan statistics for safety signal detection in vaccine clinical trials	2
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
On the relative conservativeness of Bayesian logistic regression method in oncology dose‐finding studies	2
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A Commensurate Prior Model With Random Effects for Survival and Competing Risk Outcomes to Accommodate Historical Controls	2
Confidence intervals for exposure‐adjusted rate differences in randomized trials	2
Quality by Design for Preclinical In Vitro Assay Development	2
Web based resource for Statistical Consultants in the Pharmaceutical Industry	2
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Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance	2
Comments on “Statistical methodology for highly variable compounds: A novel design approach for the ofatumumab phase 2 bioequivalence study”	2
Statistical modeling approaches for the comparison of dissolution profiles	1
Sample Size Estimation for Correlated Count Data With Changes in Dispersion	1
Heterogeneity in treatment effects across diverse populations	1
Statistical considerations for design and analysis of stability, comparability and formulation tests	1
Robust estimates of regional treatment effects in multiregional randomized clinical trials with semiparametric logistic model	1
Assessment of pharmacokinetic linearity after repeated drug administration	1
AIDE: Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials	1
Transporting randomized trial results to estimate counterfactual survival functions in target populations	1
Subgroup Identification Based on Quantitative Objectives	1
Statistical analysis of actigraphy data with generalised additive models	1
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Empirical likelihood inference for area under the receiver operating characteristic curve using ranked set samples	1
Bayesiansingle‐armphaseIItrial designs withtime‐to‐eventendpoints	1
Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	1
Flexible parametric copula modeling approaches for clustered survival data	1
A Federated Data Analysis Approach for the Evaluation of Surrogate Endpoints	1
A Bayesian Dynamic Model‐Based Adaptive Design for Oncology Dose Optimization in Phase I/II Clinical Trials	1
Issue Information	1
Return‐to‐baseline multiple imputation for missing values in clinical trials	1
Setting the control limit at release for stability assurance	1
Visualizing hypothesis tests in survival analysis under anticipated delayed effects	1
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Do statisticians count? A prior and posterior viewpoint	1
Just say no to data listings!	1
PKBOIN‐12: A Bayesian Optimal Interval Phase I/II Design Incorporating Pharmacokinetics Outcomes to Find the Optimal Biological Dose	1
A unified approach for evaluating the prediction of treatment effect across different types of endpoints	1
Investigating Stability in Subgroup Identification for Stratified Medicine	1
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data	1

A model‐assisted design for partially or completely ordered groups	1
Information fraction estimation: Strategies for a phase 3 non‐inferiority maximum duration design with time to event outcome	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
Ensuring exchangeability indata‐basedpriors for a Bayesian analysis of clinical trials	1
WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors	1
Estimating survival parameters under conditionally independent left truncation	1
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Comparison of nonparametric estimators of the expected number of recurrent events	1
A simulation‐based comparison of estimation methods for adaptive and classical group sequential clinical trials	1
The design and analysis of vaccine trials for COVID‐19 for the purpose of estimating efficacy	1
A mixed effects model for analyzing area under the curve of longitudinally measured biomarkers with missing data	1
Do You Want to Stay Single? Considerations on Single‐Arm Trials in Drug Development and the Postregulatory Space	1
Issue Information	1
Probability of success and group sequential designs	1
The use of local and nonlocal priors in Bayesian test‐based monitoring for single‐arm phase II clinical trials	1
A Phase I Dose‐Finding Design Incorporating Intra‐Patient Dose Escalation	1
Sample size calculation for comparing two ROC curves	1
Identifying treatment effects using trimmed means when data are missing not at random	1
A practical guide to sample size calculations: Installation of the app SampSize	1
Survival Analysis Without Sharing of Individual Patient Data by Using a Gaussian Copula	1
An improved score‐type confidence interval for stratified risk differences involving rare events	1
Standard and reference‐based conditional mean imputation	1
Confirmatory efficacy testing for individual dose–placebo comparisons using serial gatekeeping procedure in dose‐finding trials with multiple comparison procedures–modeling	1
PubPredict: Prediction of progression and survival in oncology leveraging publications and early efficacy data	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
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design	1
Statistical methodology for highly variable compounds: A novel design approach for the ofatumumab Phase 2 bioequivalence study	0
Comments on ‘standard and reference‐based conditional mean imputation’: Regulators and trial statisticians be aware!	0
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A note on confidence intervals for the restricted mean survival time based on transformations in small sample size	0
Encouragement of subgroup assessment by the FDA	0
Kernel hazard estimation for visualisation of the effect of a continuous covariate on time‐to‐event endpoints	0
Synergy detection: A practical guide to statistical assessment of potential drug combinations	0
A targeted simulation‐extrapolation method for evaluating biomarkers based on new technologies in precision medicine	0
A fiducial approach for testing the non‐inferiority of proportion difference in matched‐pairs design	0
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	0
Correction to “The Flaw of Averages: Bayes Factors as Posterior Means of the Likelihood Ratio”	0
T3 + 3: 3 + 3 Design With Delayed Outcomes	0
A comparative study of adaptive trial designs for dose optimization	0
Bayesian MCPMod	0
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Balance Index to Determine the Follow‐Up Duration of Oncology Trials	0
Bayesian single‐to‐double arm transition design using both short‐term and long‐term endpoints	0
Number of Repetitions in Re‐Randomization Tests	0
Stability analysis using mixed models: A critique of tolerance interval methods and a probabilistic solution	0
Leading beyond regulatory approval: Opportunities for statisticians to optimize evidence generation and impact clinical practice	0
Modeling immunogenecity data to establish screening bioassays cut point	0
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The flaw of averages: Bayes factors as posterior means of the likelihood ratio	0
Conditional assurance: the answer to the questions that should be asked within drug development	0
A Bayesian latent class model for predicting gestational age in health administrative data	0
A two‐stage adaptive clinical trial design with data‐driven subgroup identification at interim analysis	0
Cautionary note on regional consistency evaluation in multiregional clinical trials with binary outcomes	0
Foreword	0
Issue Information	0
Counterfactual mediation analysis in the multistate model framework for surrogate and clinical time‐to‐event outcomes in randomized controlled trials	0
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)	0
Practical and robust test for comparing binomial proportions in the randomized phase II setting	0
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A note from the editors	0
Mathematical programming tools for randomization purposes in small two‐arm clinical trials: A case study with real data	0
Multiple imputation analysis of Miettinen‐Nurminen interval for difference in proportions	0
Sample size calculation in clinical trials with two co‐primary endpoints including overdispersed count and continuous outcomes	0
Trial Probability of Success for Testing 3‐Way PK/PD Similarity With Multiple Endpoints	0
The Role of CMC Statisticians: Co‐Practitioners of the Scientific Method	0
Utilizing restricted mean duration of response for efficacy evaluation of cancer treatments	0
A marginalized two‐part joint model for a longitudinal biomarker and a terminal event with application to advanced head and neck cancers	0
Covariate adjustment and estimation of difference in proportions in randomized clinical trials	0
Performance of phase‐I dose finding designs with and without a run‐in intra‐patient dose escalation stage	0
Operational characteristics of generalized pairwise comparisons for hierarchically ordered endpoints	0
Comments on “Equal‐tailed confidence intervals for comparison of rates”	0
Generalized phase I‐II designs to increase long term therapeutic success rate	0
Strategy for Designing In Vivo Dose–Response Comparison Studies	0
Improving early phase oncology clinical trial design: A case study	0
TITE‐gBOIN: Time‐to‐event Bayesian optimal interval design to accelerate dose‐finding accounting for toxicity grades	0
Bayesian adaptive randomization design incorporating propensity score‐matched historical controls	0
Statistical approaches to evaluate in vitro dissolution data against proposed dissolution specifications	0
The partnership between statisticians and the Institutional Animal Care and Use Committee (IACUC)	0
Identifying treatment effect heterogeneity in dose‐finding trials using Bayesian hierarchical models	0
Author's reply to the letter to the editor by Yongqiang Tang: Comments on “Equal‐tailed confidence intervals for comparison of rates”	0
Improving early phase oncology clinical trial design: An opportunity for statisticians	0
Treatment effect measures under nonproportional hazards	0
Rationale for the update algorithm of the graphical approach to sequentially rejective multiple test procedures	0
Prediction Intervals for Overdispersed Poisson Data and Their Application in Medical and Pre‐Clinical Quality Control	0
Sample size calculation for recurrent event data with additive rates models	0
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BF‐BOIN‐ET: A Backfill Bayesian Optimal Interval Design Using Efficacy and Toxicity Outcomes for Dose Optimization	0
Evaluating response‐adaptive randomization procedures for recurrent events and terminal event data using a composite endpoint	0
Handling Partially Observed Trial Data After Treatment Withdrawal: Introducing Retrieved Dropout Reference‐Base Centred Multiple Imputation	0
Sensitivity analyses for the principal ignorability assumption using multiple imputation	0
Duration of and time to response in oncology clinical trials from the perspective of the estimand framework	0
A Bayesian phase I/II design to determine subgroup‐specific optimal dose for immunotherapy sequentially combined with radiotherapy	0
On implementing Jeffreys' substitution likelihood for Bayesian inference concerning the medians of unknown distributions	0
New Methods for Two‐Stage Treatment Switching Estimation	0
Effects of sceptical priors on the performance of adaptive clinical trials with binary outcomes.	0