OOIR: Observatory of International Research

Papers

(The TQCC of Pharmaceutical Statistics is 3. 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 2022-08-01 to 2026-08-01.)

Article	Citations
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	39
Prediction Intervals for Overdispersed Binomial Endpoints and Their Application to Toxicological Historical Control Data	29
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	22
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	19
Average Hazard as Harmonic Mean	19
Using Off‐Treatment Sequential Multiple Imputation for Binary Outcomes to Address Intercurrent Events Handled by a Treatment Policy Strategy	18
Prospectively Specified Adaptive Bayesian Borrowing: Considerations, Methodologies, and Implementations	17
Adaptive Constrained Weighted Estimation for Incorporating Multiple External Information Sources	16
A Weighted Geometric Average Hazard Ratio Based Sample Size Formula for Non‐Proportional Hazards and Its Application to a Smoothed Piecewise Model in Cancer Immunotherapy Trial Design	14
Frailty model with change points for survival analysis	14
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	13
Pre‐Posterior Distributions in Drug Development and Their Properties	13
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	12
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose	12
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	12
A conservative approach to leveraging external evidence for effective clinical trial design	12
Issue Information	12
Control of Unconditional Type I Error in Clinical Trials With External Control Borrowing—A Two‐Stage Adaptive Design Perspective	12
Extending Multiple Testing With Unknown Test Dependency via the CoCo Test: With Applications to Cancer Studies	10
Current developments of the estimand concept	10
A meta‐analytic framework to adjust for bias in external control studies	10
Multiplicity Adjustment Methods for a Three‐Way Crossover Bioequivalence Study	10
Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery	9
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	9
A Model‐Based Trial Design With a Randomization Scheme Considering Pharmacokinetics Exposure for Dose Optimization in Oncology	9

Why “Minimal Clinically Important Difference” for Interpreting the Magnitude of the Treatment Effect Is Not Useful	9
Applying the Estimand Framework to Non‐Inferiority Trials	8
Confidence Intervals for Validation of Analytical Procedures Under ICH Q2 ( R2 )	8
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	8
Finding the Optimal Number of Splits and Repetitions in Double Cross‐Fitting Targeted Maximum Likelihood Estimators	7
Mixture Experimentation in Pharmaceutical Formulations: A Tutorial	7
Applying the Principal Stratum Strategy in Equivalence Trials: A Case Study	7
Issue Information	7
Simultaneous Inference Using Multiple Marginal Models	7
An illness–death multistate model to implement delta adjustment and reference‐based imputation with time‐to‐event endpoints	7
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	7
A Unified Approach to Covariate Adjustment for Survival Endpoints in Randomized Clinical Trials	7
Chauhan Weighted Trajectory Analysis of Combined Efficacy and Safety Outcomes for Risk–Benefit Analysis	7
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Frequentist and Bayesian tolerance intervals for setting specification limits for left‐censored gamma distributed drug quality attributes	6
Simulation‐Based Bayesian Predictive Probability of Success for Interim Monitoring of Clinical Trials With Competing Event Data: Two Case Studies	6
Application of Causal Inference to Establish Assay Effect in the Absence of a Bridging Study: A Case Study of MenACWY ‐ CRM<	6
BOIN ‐ MEM : A Two‐Stage Design for Dose Optimization With Information Borrowing Across Dose Levels and Stages in Onco	6
Bayesian Response Adaptive Randomization for Randomized Clinical Trials With Continuous Outcomes: The Role of Covariate Adjustment	6
Interim decision making in seamless trial designs: An application in an adaptive dose‐finding study in a rare kidney disease	6
Statistical methods for handling missing data to align with treatment policy strategy	6
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Informing the Borrowing Process for Dose‐Finding Trials by Estimating the Similarity Between Population‐Specific Dose‐Toxicity Curves	6
An evolutionary algorithm for the direct optimization of covariate balance between nonrandomized populations	6
Should responder analyses be conducted on continuous outcomes?	6
An Ensemble Classifier for Ordinal Outcomes in High‐Dimensional Genomics Data	5
A Tree‐Based Scan Statistic for Detecting Signals of Drug–Drug Interactions in Spontaneous Reporting Databases	5
Bayesian borrowing from historical control data in a vaccine efficacy trial	5
Issue Information	5
Improving precision and power in randomized trials with a two‐stage study design: Stratification using clustering method	5
Balance diagnostics in propensity score analysis following multiple imputation: A new method	5
Tutorial on Firth's Logistic Regression Models for Biomarkers in Preclinical Space	5
A Bayesian Hybrid Design With Borrowing From Historical Study	5
A Personalized Dose‐Finding Algorithm Based on Adaptive Gaussian Process Regression	5
Beyond the Fragility Index	5
Estimation of Treatment Policy Estimands for Continuous Outcomes Using Off‐Treatment Sequential Multiple Imputation	5
The Wilcoxon–Mann–Whitney Estimand Versus Differences in Medians or Means	5
CUSUMIN Combination: A Cumulative Sum Interval Design for Phase I Cancer Drug‐Combination Trials	5
Evaluating hybrid controls methodology in early‐phase oncology trials: A simulation study based on the MORPHEUS‐UC trial	5
Designing and Evaluating Bayesian Advanced Adaptive Randomised Clinical Trials: A Practical Guide	4
	4
Issue Information	4
What they forgot to tell you about machine learning with an application to pharmaceutical manufacturing	4
A Tobit Partly Linear Mixed and Mixture Cure Model for the Joint Analysis of Interval‐Bounded Longitudinal Measurements and Survival Times With Cure Proportion	4
Bayesian optimal phase II designs with dual‐criterion decision making	4
Applying Bias Correction Methods to Build Hybrid Controls Using Real‐World Patients for a Phase IIb Randomized Controlled Trial of Baricitinib for Rheumatoid Arthritis	4
“Super‐covariates”: Using predicted control group outcome as a covariate in randomized clinical trials	4
Issue Information	4
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	4
Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials	4

Comparative Analyses of Bioequivalence Assessment Methods for In Vitro Permeation Test Data	4
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	3
	3
Web based resource for Statistical Consultants in the Pharmaceutical Industry	3
Issue Information	3
	3
Group sequential design with maximin efficiency robust test for immunotherapy with generalized delayed treatment effect	3
Penalized Variable Selection for Joint AFT Random‐Effect Model With Clustered Competing‐Risks Data	3
The Estimand Framework and Causal Inference: Complementary Not Competing Paradigms	3
Comparing various Bayesian random‐effects models for pooling randomized controlled trials with rare events	3
Application of hypothetical strategies in acute pain	3
	3
Adaptively leveraging external data with robust meta‐analytical‐predictive prior using empirical Bayes	3
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	3
Multiple Comparisons Procedures for Analyses of Joint Primary Endpoints and Secondary Endpoints	3
	3
On the relative conservativeness of Bayesian logistic regression method in oncology dose‐finding studies	3
Multiple Comparisons With Overdispersed Multinomial Data: Methods, Properties and Application	3
A Tipping Point Method to Evaluate Sensitivity to Potential Violations in Missing Data Assumptions	3
Principled leveraging of external data in the evaluation of diagnostic devices via the propensity score‐integrated composite likelihood approach	3
Variable Duration Trial as an Alternative Design for Continuous Endpoints	3
An adaptive biomarker basket design in phase II oncology trials	3
Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance	3
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	3
Principles for Defining Estimands in Clinical Trials—A Proposal	3
Using Propensity Score Weighting to Enhance the Operating Characteristics of Power Prior in Leveraging External Data to Augment a Traditional Clinical Study	3
Predictive Ppk calculations for biologics and vaccines using a Bayesian approach – a tutorial	3
A Commensurate Prior Model With Random Effects for Survival and Competing Risk Outcomes to Accommodate Historical Controls	3
On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes	3
Rejoinder to the letter: “Standard and reference‐based conditional mean imputation: Regulators and trial statisticians be aware!”	3
Quality by Design for Preclinical In Vitro Assay Development	3