OOIR: Observatory of International Research

Papers

(The TQCC of Statistics in Biopharmaceutical Research 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 2021-12-01 to 2025-12-01.)

Article	Citations
Treatment Selection at Interim Analysis Based on a Correlated Early Endpoint in Adaptive Two-Stage Trials with a Primary Endpoint Based on Count Data	35
U-MET: Utility-Based Dose Optimization Approach for Multiple-Dose Randomized Trial Designs	23
Predicting Probability of Success for Phase III Trials via Propensity-Score-Based External Data Borrowing	16
A landscape assessment of key evidence needs in study design and statistical methodologies for HTA submissions	15
DODII: Bayesian Dose Optimization Design for Randomized Phase II Trials	14
Consistency Based Multiplicity Adjustment Approach—Multiple Doses in Phase III Studies	12
The Current Landscape of HTA Framework and Key Challenges	11
Modified Simon’s Two-Stage Design for Phase IIA Clinical Trials in Oncology—Dynamic Monitoring and More Flexibility	11
We Need Subject Matter Expertise to Choose and Identify Causal Estimands: Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	11
Bayesian and Frequentist Approaches to Rescuing Disrupted Trials: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	10
A Basket Trial Design Based on Power Priors	10
Missing Data Imputation for a Multivariate Outcome of Mixed Variable Types	10
Joint Analysis of Longitudinal Ordinal Categorical Item Response Data and Survival Times with Cure Fraction	10
Some Multiplicity Adjustment Procedures for Clinical Trials with Sequential Design and Multiple Endpoints	10
Some Group Sequential Trials from Industry over the Last 30 Years	9
Statistical Considerations and Challenges for Pivotal Clinical Studies of Artificial Intelligence Medical Tests for Widespread Use: Opportunities for Inter-Disciplinary Collaboration	9
Design Strategy and Consideration for Oncology Dose-Optimization: An Industry Perspective	9
A Two-Stage Covariate-Adjusted Response-Adaptive Enrichment Design	9
A New Centralized Statistical Monitoring Method for Detecting Atypical Distribution of Qualitative Variables in Multicenter Randomized Controlled Trials	8
Backward Sequential Significance Testing in Survival Trials	8
Monitoring Ongoing Clinical Trials Under Fractional Brownian Motion With Drift	8
Closing a Chapter: A Farewell from the Editor of Statistics in Biopharmaceutical Research	8
LORDs: Locally Optimal Restricted Designs for Phase I/II Dose-Finding Studies	8
Joint Analysis of Longitudinal Data and Zero-Inflated Recurrent Events	8
MT-Keyboard: A Bayesian Model-Assisted Interval Design to Account for Toxicity Grades and Types for Phase I Trials	8

Augmenting the Control Arm of Randomized Trials by Incorporating Multiple External Data Sources Using Propensity Score Stratification and Data-Driven Mixture Prior	8
Exact Matching as an Alternative to Propensity Score Matching	7
Bayesian Hierarchical Model with Adaptive Similarity Evaluation of Treatment Effects in Oncology Basket Trials	7
Statistics in Biopharmaceutical Research Best Papers Award 2023	7
Pre-Specified Safety Analysis of OS Data for Trials in Indolent or Early-Stage Cancers	6
Statistical Issues and Challenges in Clinical Trials for COVID-19 Treatments, Vaccines, Medical Devices and Diagnostics	6
Novel Statistical Designs and Considerations to Support Diversity and Inclusion in Clinical Trials	6
Which Randomization Methods Are Used Most Frequently in Clinical Trials? Results of a Survey by the Randomization Working Group	6
Effects of Allocation Method and Time Trends on Identification of the Best Arm in Multi-Arm Trials	6
A Comparison of Different Approaches to Bayesian Hierarchical Models in a Basket Trial to Evaluate the Benefits of Increasing Complexity	6
Statistical Thinking and Innovation with Global Impact—Special Issue for the 2023 Regulatory-Industry Statistics Workshop	5
Editor’s Note	5
A Case Study of 2-Stage Seamless Adaptive Sample Size Re-Estimation Design with Efficacy Interim Analysis When Slope Is the Primary Endpoint	5
Statistical Consideration for Fit-for-Use Real-World Data to Support Regulatory Decision Making in Drug Development	5
Treatment-Control Comparisons in Platform Trials Including Non-Concurrent Controls	5
Bayesian Basket Trial Design Accounting for Multiple Cutoffs of an Ambiguous Biomarker	5
Nonconcurrent Controls in Platform Trials: Can We Borrow Their Concurrent Observation Data?	5
Rejoinder to Comments on “Non-Proportional Hazards – An Evaluation of the MaxCombo Test in Cancer Clinical Trials”	5
A Primer of Quasi-Exact Tests for Randomized Clinical Trials with Binary Endpoints	4
Adaptive Endpoints Selection with Application in Rare Disease	4
Bias-Corrected Group Sequential Design in the Presence of Surrogate Endpoints with Application to PALM Trial	4
On Variance Estimation for the One-Sample Log-Rank Test	4
Estimands for Continuous Longitudinal Outcomes in the Presence of Treatment Discontinuation—A Simulation Study in Hyperkalemia Treatments	4
Rejoinder to Commentaries on the “Monitoring OS in Pivotal Trials in Indolent Cancers” Article	4
A Comprehensive Bayesian Approach to Quantify and Compare Benefit-Risk of Medical Products	3
Comment on “Monitoring Overall Survival in Pivotal Trials in Indolent Cancers”	3
A Propensity-Score Integrated Approach to Bayesian Dynamic Power Prior Borrowing	3
A Simulation Study on the Estimation of the Effect in the Hypothetical Scenario of No Use of Symptomatic Treatment in Trials for Disease-Modifying Agents for Alzheimer’s Disease	3
Performance of LTMLE in the Presence of Missing Data in Control-Matched Longitudinal Studies	3
Accuracy and Safety of Novel Designs for Phase I Drug-Combination Oncology Trials	3
Rejoinder to Letter to the Editor—Selecting an Optimal Design for a Nonrandomized Comparative Study: A Commentary on “Some Considerations on Design and Analysis Plan on a Nonrandomized Comparative Stu	3
Correction	3
An Alternative to Traditional Sample Size Determination for Small Patient Populations	3
Use of Common Control Arms Across Substudies in a Master Protocol via Ridge Estimation	3
Evaluating the Impact of Outcome Delay on the Efficiency of Two-Arm Group-Sequential Trials	3
A Comparison of Estimand and Estimation Strategies for Clinical Trials in Early Parkinson’s Disease	3
Assessing the Commonly Used Assumptions in Estimating the Principal Causal Effect in Clinical Trials	3
A Multi-Arm Multi-Stage Group Sequential Phase 2/3 Design with Dose Selection for Oncology Trials	3
The Use of Machine Learning in Regulatory Drug Safety Evaluation	3
Bayesian Optimal Phase II Design for Randomized Clinical Trials	3
A Rényi-Divergence-Based Family of Metrics for the Evaluation of Surrogate Endpoints in a Causal Inference Framework	3
Random Effects Meta-Analysis of Contingency Tables with Complete and Partially Complete Data, with Application to COVID-19 Research	3
The NISS Ingram Olkin Forum on Unplanned Clinical Trial Disruptions	3