Pharmaceutical Statistics

Article	Citations
Frailty model with change points for survival analysis	32
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	20
Pre‐Posterior Distributions in Drug Development and Their Properties	18
Average Hazard as Harmonic Mean	16
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	16
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	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
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	13
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	13
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior	12
Weighted log‐rank test to compare two survival functions in the presence of dependent censoring	12
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Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery	12
A meta‐analytic framework to adjust for bias in external control studies	11
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	11
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose	11
A two‐stage drop‐the‐losers design for time‐to‐event outcome using a historical control arm	10
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	10
Issue Information	10
A conservative approach to leveraging external evidence for effective clinical trial design	10
A Model‐Based Trial Design With a Randomization Scheme Considering Pharmacokinetics Exposure for Dose Optimization in Oncology	10
Applying the Estimand Framework to Non‐Inferiority Trials	9
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	9
CUSUMIN: A cumulative sum interval design for cancer phase I dose finding studies	9
Why “Minimal Clinically Important Difference” for Interpreting the Magnitude of the Treatment Effect Is Not Useful	8

Control of Unconditional Type I Error in Clinical Trials With External Control Borrowing—A Two‐Stage Adaptive Design Perspective	8
Mixture Experimentation in Pharmaceutical Formulations: A Tutorial	7
A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	7
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	7
Simultaneous Inference Using Multiple Marginal Models	7
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
Current developments of the estimand concept	7
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
Issue Information	6
Reflecting on Andy Grieve's influence and innovation: A personal perspective	6
An illness–death multistate model to implement delta adjustment and reference‐based imputation with time‐to‐event endpoints	6
Bayesian Response Adaptive Randomization for Randomized Clinical Trials With Continuous Outcomes: The Role of Covariate Adjustment	6
Estimation of Treatment Policy Estimands for Continuous Outcomes Using Off‐Treatment Sequential Multiple Imputation	5
Tutorial on Firth's Logistic Regression Models for Biomarkers in Preclinical Space	5
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Chauhan Weighted Trajectory Analysis of Combined Efficacy and Safety Outcomes for Risk–Benefit Analysis	5
Using an interim analysis based exclusively on an early outcome in a randomized clinical trial with a long‐term clinical endpoint	5
Statistical detection of synergy: New methods and a comparative study	5
Bayesian borrowing from historical control data in a vaccine efficacy trial	5
Should responder analyses be conducted on continuous outcomes?	5
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
Balance diagnostics in propensity score analysis following multiple imputation: A new method	5
Frequentist and Bayesian tolerance intervals for setting specification limits for left‐censored gamma distributed drug quality attributes	5
Applying the Principal Stratum Strategy in Equivalence Trials: A Case Study	5
Statistical methods for handling missing data to align with treatment policy strategy	5
Regulatory issues and the potential use of Bayesian approaches for early drug approval systems in Japan	4
A Personalized Dose‐Finding Algorithm Based on Adaptive Gaussian Process Regression	4
Beyond the Fragility Index	4
Propensity score matching and stratification using multiparty data without pooling	4
Methodological challenges in the analysis of recurrent events for randomised controlled trials with application to cardiovascular events in LEADER	4
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	4
Study‐design in pandemics: From surveillance and performance‐evaluation to licensing and pharmacovigilance	4
A Bayesian Hybrid Design With Borrowing From Historical Study	4
Comparative Analyses of Bioequivalence Assessment Methods for In Vitro Permeation Test Data	4
“Super‐covariates”: Using predicted control group outcome as a covariate in randomized clinical trials	4
Evaluating hybrid controls methodology in early‐phase oncology trials: A simulation study based on the MORPHEUS‐UC trial	4
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What they forgot to tell you about machine learning with an application to pharmaceutical manufacturing	4
Issue Information	4
Improving precision and power in randomized trials with a two‐stage study design: Stratification using clustering method	4
CUSUMIN Combination: A Cumulative Sum Interval Design for Phase I Cancer Drug‐Combination Trials	4
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Geometric approaches to assessing the numerical feasibility for conducting matching‐adjusted indirect comparisons	4
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The Acute Stroke Therapy by Inhibition of Neutrophils study – Key features and impact	3
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	3
Issue Information	3

Bayesian optimal phase II designs with dual‐criterion decision making	3
Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study	3
An adaptive biomarker basket design in phase II oncology trials	3
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Improving the assessment of the probability of success in late stage drug development	3
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	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
Issue Information	3
Using Propensity Score Weighting to Enhance the Operating Characteristics of Power Prior in Leveraging External Data to Augment a Traditional Clinical Study	3
Semi‐parametric accelerated failure‐time model: A useful alternative to the proportional‐hazards model in cancer clinical trials	3
Issue Information	3
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	3
Multiple Comparisons Procedures for Analyses of Joint Primary Endpoints and Secondary Endpoints	3
Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance	3
Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials	3
An adaptive design for early clinical development including interim decision for single‐arm trial with external controls or randomized trial	3
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Principled leveraging of external data in the evaluation of diagnostic devices via the propensity score‐integrated composite likelihood approach	2
Variable Duration Trial as an Alternative Design for Continuous Endpoints	2
Comments on “Statistical methodology for highly variable compounds: A novel design approach for the ofatumumab phase 2 bioequivalence study”	2
Comparing various Bayesian random‐effects models for pooling randomized controlled trials with rare events	2
On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	2
Principles for Defining Estimands in Clinical Trials—A Proposal	2
A Commensurate Prior Model With Random Effects for Survival and Competing Risk Outcomes to Accommodate Historical Controls	2
Do You Want to Stay Single? Considerations on Single‐Arm Trials in Drug Development and the Postregulatory Space	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
Inclusion of binary proxy variables in logistic regression improves treatment effect estimation in observational studies in the presence of binary unmeasured confounding variables	2
Rejoinder to the letter: “Standard and reference‐based conditional mean imputation: Regulators and trial statisticians be aware!”	2
Tree‐temporal scan statistics for safety signal detection in vaccine 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
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On the relative conservativeness of Bayesian logistic regression method in oncology dose‐finding studies	2
Adaptively leveraging external data with robust meta‐analytical‐predictive prior using empirical Bayes	2
Confidence intervals for exposure‐adjusted rate differences in randomized trials	2
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Transporting randomized trial results to estimate counterfactual survival functions in target populations	2
Robust estimates of regional treatment effects in multiregional randomized clinical trials with semiparametric logistic model	2
A Tipping Point Method to Evaluate Sensitivity to Potential Violations in Missing Data Assumptions	2
Just say no to data listings!	2
Sample Size Estimation for Correlated Count Data With Changes in Dispersion	2
A note on point estimation and interval estimation of the relative treatment effect under a simple crossover design	2
Sample Size Estimation Using a Partially Clustered Frailty Model for Biomarker‐Strategy Designs With Multiple Treatments	2
Estimation of multivariate treatment effects in contaminated clinical trials	2
Issue Information	2
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Group sequential design with maximin efficiency robust test for immunotherapy with generalized delayed treatment effect	2
Quality by Design for Preclinical In Vitro Assay Development	2
Web based resource for Statistical Consultants in the Pharmaceutical Industry	2
Application of hypothetical strategies in acute pain	2
Potential Bias Models With Bayesian Shrinkage Priors for Dynamic Borrowing of Multiple Historical Control Data	2
Statistical analysis of actigraphy data with generalised additive models	2
Alone, together: On the benefits of Bayesian borrowing in a meta‐analytic setting	2
Predictive Ppk calculations for biologics and vaccines using a Bayesian approach – a tutorial	2
A unified approach for evaluating the prediction of treatment effect across different types of endpoints	2
Return‐to‐baseline multiple imputation for missing values in clinical trials	1
Issue Information	1
A propensity score‐integrated approach for leveraging external data in a randomized controlled trial with time‐to‐event endpoints	1
Defining estimands for efficacy assessment in single arm phase 1b or phase 2 clinical trials in oncology early development	1
A practical guide to sample size calculations: Installation of the app SampSize	1
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Issue Information	1
Issue Information	1
Estimating survival parameters under conditionally independent left truncation	1
Heterogeneity in treatment effects across diverse populations	1
Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	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
Confirmatory efficacy testing for individual dose–placebo comparisons using serial gatekeeping procedure in dose‐finding trials with multiple comparison procedures–modeling	1
Probability of success and group sequential designs	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
Subgroup Identification Based on Quantitative Objectives	1
Investigating Stability in Subgroup Identification for Stratified Medicine	1
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A Federated Data Analysis Approach for the Evaluation of Surrogate Endpoints	1
Ensuring exchangeability indata‐basedpriors for a Bayesian analysis of clinical trials	1
Issue Information	1
Detection of Outlying Correlation Coefficients in Multicenter Clinical Trials	1
Time‐to‐event calibration‐free odds design: A robust efficient design for phase I trials with late‐onset outcomes	1
Empirical likelihood inference for area under the receiver operating characteristic curve using ranked set samples	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
AIDE: Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials	1
PKBOIN‐12: A Bayesian Optimal Interval Phase I/II Design Incorporating Pharmacokinetics Outcomes to Find the Optimal Biological Dose	1
Statistical considerations for design and analysis of stability, comparability and formulation tests	1
The design and analysis of vaccine trials for COVID‐19 for the purpose of estimating efficacy	1
Comparison of nonparametric estimators of the expected number of recurrent events	1
Identifying treatment effects using trimmed means when data are missing not at random	1
A model‐assisted design for partially or completely ordered groups	1
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data	1
WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors	1
A Bayesian Dynamic Model‐Based Adaptive Design for Oncology Dose Optimization in Phase I/II Clinical Trials	1
A mixed effects model for analyzing area under the curve of longitudinally measured biomarkers with missing data	1
Visualizing hypothesis tests in survival analysis under anticipated delayed effects	1
A Phase I Dose‐Finding Design Incorporating Intra‐Patient Dose Escalation	1
Flexible parametric copula modeling approaches for clustered survival data	1
Separation and the information theory surrogate evaluation approach: A penalised likelihood solution	1
Information fraction estimation: Strategies for a phase 3 non‐inferiority maximum duration design with time to event outcome	1
An improved score‐type confidence interval for stratified risk differences involving rare events	1
Decision rules for identifying combination therapies in open‐entry, randomized controlled platform trials	1
PubPredict: Prediction of progression and survival in oncology leveraging publications and early efficacy data	1
Bayesiansingle‐armphaseIItrial designs withtime‐to‐eventendpoints	1
Do statisticians count? A prior and posterior viewpoint	1
Standard and reference‐based conditional mean imputation	1
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design	1
A simulation‐based comparison of estimation methods for adaptive and classical group sequential clinical trials	1
Assessment of pharmacokinetic linearity after repeated drug administration	1
Sample size calculation for comparing two ROC curves	1
Issue Information	1
Issue Information	1
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Setting the control limit at release for stability assurance	1
Statistical modeling approaches for the comparison of dissolution profiles	1
Bivariate Bayesian hypothesis testing with missing data in components	0
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Estimation Methods for Estimands Using the Treatment Policy Strategy; a Simulation Study Based on the PIONEER 1 Trial	0
A targeted simulation‐extrapolation method for evaluating biomarkers based on new technologies in precision medicine	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
Kernel hazard estimation for visualisation of the effect of a continuous covariate on time‐to‐event endpoints	0
Practical and robust test for comparing binomial proportions in the randomized phase II setting	0
Leading beyond regulatory approval: Opportunities for statisticians to optimize evidence generation and impact clinical practice	0
Sample size calculation in clinical trials with two co‐primary endpoints including overdispersed count and continuous outcomes	0
Bayesian MCPMod	0
Bayesian single‐to‐double arm transition design using both short‐term and long‐term endpoints	0
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Assessing the performance of group‐based trajectory modeling method to discover different patterns of medication adherence	0
T3 + 3: 3 + 3 Design With Delayed Outcomes	0
Optimal sample size division in two‐stage seamless designs	0
Foreword	0
Cautionary note on regional consistency evaluation in multiregional clinical trials with binary outcomes	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
Mathematical programming tools for randomization purposes in small two‐arm clinical trials: A case study with real data	0
Statistical methodology for highly variable compounds: A novel design approach for the ofatumumab Phase 2 bioequivalence study	0
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A Bayesian latent class model for predicting gestational age in health administrative data	0
Improving early phase oncology clinical trial design: An opportunity for statisticians	0
Issue Information	0
Prediction Intervals for Overdispersed Poisson Data and Their Application in Medical and Pre‐Clinical Quality Control	0
Counterfactual mediation analysis in the multistate model framework for surrogate and clinical time‐to‐event outcomes in randomized controlled trials	0
Utilizing restricted mean duration of response for efficacy evaluation of cancer treatments	0
Issue Information	0
Duration of and time to response in oncology clinical trials from the perspective of the estimand framework	0
A comparative study of adaptive trial designs for dose optimization	0
A fiducial approach for testing the non‐inferiority of proportion difference in matched‐pairs design	0
Evaluating response‐adaptive randomization procedures for recurrent events and terminal event data using a composite endpoint	0
A note on confidence intervals for the restricted mean survival time based on transformations in small sample size	0
Trial Probability of Success for Testing 3‐Way PK/PD Similarity With Multiple Endpoints	0
Covariate adjustment and estimation of difference in proportions in randomized clinical trials	0
Stability analysis using mixed models: A critique of tolerance interval methods and a probabilistic solution	0
The flaw of averages: Bayes factors as posterior means of the likelihood ratio	0
TITE‐gBOIN: Time‐to‐event Bayesian optimal interval design to accelerate dose‐finding accounting for toxicity grades	0
A Bayesian phase I/II design to determine subgroup‐specific optimal dose for immunotherapy sequentially combined with radiotherapy	0
The Role of CMC Statisticians: Co‐Practitioners of the Scientific Method	0
Bayesian adaptive randomization design incorporating propensity score‐matched historical controls	0
Balance Index to Determine the Follow‐Up Duration of Oncology Trials	0
Operational characteristics of generalized pairwise comparisons for hierarchically ordered endpoints	0
Rationale for the update algorithm of the graphical approach to sequentially rejective multiple test procedures	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
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
A marginalized two‐part joint model for a longitudinal biomarker and a terminal event with application to advanced head and neck cancers	0
Statistical approaches to evaluate in vitro dissolution data against proposed dissolution specifications	0
Multiple imputation analysis of Miettinen‐Nurminen interval for difference in proportions	0
Treatment effect measures under nonproportional hazards	0
A note from the editors	0
Correction to “The Flaw of Averages: Bayes Factors as Posterior Means of the Likelihood Ratio”	0
BF‐BOIN‐ET: A Backfill Bayesian Optimal Interval Design Using Efficacy and Toxicity Outcomes for Dose Optimization	0
New Methods for Two‐Stage Treatment Switching Estimation	0
On implementing Jeffreys' substitution likelihood for Bayesian inference concerning the medians of unknown distributions	0
Modeling immunogenecity data to establish screening bioassays cut point	0
Performance of phase‐I dose finding designs with and without a run‐in intra‐patient dose escalation stage	0
Improving early phase oncology clinical trial design: A case study	0
Sample size calculation for recurrent event data with additive rates models	0