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 2021-09-01 to 2025-09-01.)

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
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
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	18
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	17
Average Hazard as Harmonic Mean	16
	15
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
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
Estimation of a treatment policy estimand for time to event data using data collected post discontinuation of randomised treatment	7
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
Reflecting on Andy Grieve's influence and innovation: A personal perspective	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
	5
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
	5
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
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
The Wilcoxon–Mann–Whitney Estimand Versus Differences in Medians or Means	4
A Personalized Dose‐Finding Algorithm Based on Adaptive Gaussian Process Regression	4
	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

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
Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study	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
	3
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	3
Issue Information	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
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
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	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
	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
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
	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
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
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
	2
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
	2
On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	2
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	2