OOIR: Observatory of International Research

Papers

(The TQCC of Pharmaceutical Statistics is 2. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2021-04-01 to 2025-04-01.)

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
Issue Information	16
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Conditional power and information fraction calculations at an interim analysis for random coefficient models	15
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Informed decision‐making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments	12
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
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
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
A Bayesian optimal interval design for dose optimization with a randomization scheme based on pharmacokinetics outcomes in oncology	10
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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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
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 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
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
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	6
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The Role of CMC Statisticians: Co‐Practitioners of the Scientific Method	5
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
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
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
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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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
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
Optimizing Sample Size Determinations for Phase 3 Clinical Trials in Type 2 Diabetes	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
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
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
Using Propensity Score Weighting to Enhance the Operating Characteristics of Power Prior in Leveraging External Data to Augment a Traditional Clinical Study	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 model selection criterion for clustered survival analysis with informative cluster size	2
A two‐stage drop‐the‐losers design for time‐to‐event outcome using a historical control arm	2
Estimating the Strength of Binding Affinity via Delta–Delta‐G for Hit Screening After a Deming Regression Calibration	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
Issue Information	2
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	2
The partnership between statisticians and the Institutional Animal Care and Use Committee (IACUC)	2
Balance Index to Determine the Follow‐Up Duration of Oncology Trials	2
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	2
An adaptive design for early clinical development including interim decision for single‐arm trial with external controls or randomized trial	2
Identifying treatment effect heterogeneity in dose‐finding trials using Bayesian hierarchical models	2
Current developments of the estimand concept	2