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 2020-11-01 to 2024-11-01.)

Article	Citations
The use of external controls: To what extent can it currently be recommended?	43
Principal stratum strategy: Potential role in drug development	40
Win statistics (win ratio, win odds, and net benefit) can complement one another to show the strength of the treatment effect on time‐to‐event outcomes	23
Joint hypothesis testing of the area under the receiver operating characteristic curve and the Youden index	21
Adjusting win statistics for dependent censoring	20
Assessing efficacy in important subgroups in confirmatory trials: An example using Bayesian dynamic borrowing	17
How to choose a time zero for patients in external control arms	17
Standard and reference‐based conditional mean imputation	16
Estimating and comparing adverse event probabilities in the presence of varying follow‐up times and competing events	16
Non‐proportional hazards in immuno‐oncology: Is an old perspective needed?	15
Estimands in observational studies: Some considerations beyondICH E9(R1)	13
TITE‐gBOIN: Time‐to‐event Bayesian optimal interval design to accelerate dose‐finding accounting for toxicity grades	13
Sample size calculations for single‐arm survival studies using transformations of the Kaplan–Meier estimator	12
A novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: A comparison to standard methods	12
Incorporating historical information to improve phase I clinical trials	12
Decision rules for identifying combination therapies in open‐entry, randomized controlled platform trials	12
Estimands for overall survival in clinical trials with treatment switching in oncology	11
Methodological challenges in the analysis of recurrent events for randomised controlled trials with application to cardiovascular events inLEADER	10
Estimand framework: Are we asking the right questions? A case study in the solid tumor setting	10
Estimands in hematologic oncology trials	9
The detailed clinical objectives approach to designing clinical trials and choosing estimands	9
Ensuring exchangeability indata‐basedpriors for a Bayesian analysis of clinical trials	9
Informed decision‐making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments	9
Utilizing restricted mean duration of response for efficacy evaluation of cancer treatments	9
Improving the assessment of the probability of success in late stage drug development	9

Estimation of treatment effects in short‐term depression studies. An evaluation based on the ICH E9(R1) estimands framework	9
Left truncation in linked data: A practical guide to understanding left truncation and applying it using SAS and R	8
Flexible parametric copula modeling approaches for clustered survival data	8
Optimising the trade‐off between type I andIIerror rates in the Bayesian context	8
A simulation‐free group sequential design with max‐combo tests in the presence of non‐proportional hazards	8
Counterfactual mediation analysis in the multistate model framework for surrogate and clinical time‐to‐event outcomes in randomized controlled trials	7
Covariate handling approaches in combination with dynamic borrowing for hybrid control studies	7
A robust permutation test for the concordance correlation coefficient	6
Assessing and communicating heterogeneity of treatment effects for patient subpopulations: The hardest problem there is	6
The stratified win statistics (win ratio, win odds, and net benefit)	6
The power prior with multiple historical controls for the linear regression model	6
Bayesian optimal phase II designs with dual‐criterion decision making	6
A weighted log‐rank test and associated effect estimator for cancer trials with delayed treatment effect	6
Conditional assurance: the answer to the questions that should be asked within drug development	6
Incorporating covariates information in adaptive clinical trials for precision medicine	6
Return‐to‐baseline multiple imputation for missing values in clinical trials	5
Statistical methods for handling missing data to align with treatment policy strategy	5
Parametric and nonparametric methods for confidence intervals and sample size planning for win probability in parallel‐group randomized trials with Likert item and Likert scale data	5
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	5
Bayesian MCPMod	5
Propensity score‐based methods for causal inference and external data leveraging in regulatory settings: From basic ideas to implementation	5
Estimating survival parameters under conditionally independent left truncation	5
Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study	5
Natural cubic splines for the analysis of Alzheimer's clinical trials	5
Some design considerations incorporating early futility for single‐arm clinical trials with time‐to‐event primary endpoints using Weibull distribution	5
Progression‐free survival in oncological clinical studies: Assessment time bias and methods for its correction	5
An adaptive design for early clinical development including interim decision for single‐arm trial with external controls or randomized trial	5
Treatment effect measures under nonproportional hazards	4
Generalized phase I‐II designs to increase long term therapeutic success rate	4
Estimation of a treatment policy estimand for time to event data using data collected post discontinuation of randomised treatment	4
Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials	4
Adjusting for bias in the mean for primary and secondary outcomes when trials are in sequence	4
Operational characteristics of generalized pairwise comparisons for hierarchically ordered endpoints	4
Time‐to‐event calibration‐free odds design: A robust efficient design for phase I trials with late‐onset outcomes	4
SCI: A Bayesian adaptive phase I/II dose‐finding design accounting for semi‐competing risks outcomes for immunotherapy trials	4
Selection bias, investment decisions and treatment effect distributions	4
Assessing safety at the end of clinical trials using system organ classes: A case and comparative study	4
Potential impact of COVID‐19 on ongoing clinical trials: a simulation study with the neurological Yale Global Tic Severity Scale based on the CANNA‐TICS study	4
Bayesian optimization design for dose‐finding based on toxicity and efficacy outcomes in phase I/II clinical trials	4
	4
Empirical likelihood confidence interval for sensitivity to the early disease stage	3
Identifying treatment effects using trimmed means when data are missing not at random	3
Heterogeneity in treatment effects across diverse populations	3
Bayesian adaptive randomization design incorporating propensity score‐matched historical controls	3
Statistical methodology for highly variable compounds: A novel design approach for the ofatumumab Phase 2 bioequivalence study	3
Should responder analyses be conducted on continuous outcomes?	3
A dose‐finding design for dual‐agent trials with patient‐specific doses for one agent with application to an opiate detoxification trial	3
Adaptive designs for IVPT data with mixed scaled average bioequivalence	3
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	3
Propensity score matching and stratification using multiparty data without pooling	3

A simulation‐based comparison of estimation methods for adaptive and classical group sequential clinical trials	3
Encouragement of subgroup assessment by the FDA	3
The use of local and nonlocal priors in Bayesian test‐based monitoring for single‐arm phase II clinical trials	3
Defining estimands for efficacy assessment in single arm phase 1b or phase 2 clinical trials in oncology early development	3
Bayesiansingle‐armphaseIItrial designs withtime‐to‐eventendpoints	3
A two‐stage adaptive clinical trial design with data‐driven subgroup identification at interim analysis	3
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	3
The design and analysis of vaccine trials for COVID‐19 for the purpose of estimating efficacy	3
Regulatory issues and the potential use of Bayesian approaches for early drug approval systems in Japan	3
Statistical detection of synergy: New methods and a comparative study	3
Response‐adaptive trial designs with accelerated Thompson sampling	3
Novel concentration‐QTc models for early clinical studies with parallel placebo controls: A simulation study	3
Flexible diagnostic measures and new cut‐point selection methods under multiple ordered classes	3
Reflecting on Andy Grieve's influence and innovation: A personal perspective	2
Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components	2
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	2
Statistical modeling approaches for the comparison of dissolution profiles	2
Kernel hazard estimation for visualisation of the effect of a continuous covariate on time‐to‐event endpoints	2
An adaptive biomarker basket design in phase II oncology trials	2
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	2
A comparison of reweighting estimators of average treatment effects in real world populations	2
A note on confidence intervals for the restricted mean survival time based on transformations in small sample size	2
Should the two‐trial paradigm still be the gold standard in drug assessment?	2
Using recurrent time‐to‐event models with multinomial outcomes to generate toxicity profiles	2
On kernel machine learning for propensity score estimation under complex confounding structures	2
A practical guide to sample size calculations: Installation of the app SampSize	2
Effects of sceptical priors on the performance of adaptive clinical trials with binary outcomes.	2
Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	2
Dose intra‐subject escalation to an event (DIETE): A new method for phase 1 dose‐finding utilizing systematic intra‐subject dose escalation with application to T‐cell engagers	2
Adjusting for covariates in analysis based on restricted mean survival times	2
Estimating the treatment effect for adherers using multiple imputation	2
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior	2
A natural lead‐in approach to response‐adaptive allocation for continuous outcomes	2
A semi‐mechanistic dose‐finding design in oncology using pharmacokinetic/pharmacodynamic modeling	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
Joint confidence region estimation on predictive values	2
Improving early phase oncology clinical trial design: A case study	2
Using an interim analysis based exclusively on an early outcome in a randomized clinical trial with a long‐term clinical endpoint	2
Empirical likelihood inference for area under the receiver operating characteristic curve using ranked set samples	2
A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	2
Doubly‐robust methods for differences in restricted mean lifetimes using pseudo‐observations	2
A meta‐analytic framework to adjust for bias in external control studies	2
Semi‐parametric accelerated failure‐time model: A useful alternative to the proportional‐hazards model in cancer clinical trials	2
Geometric approaches to assessing the numerical feasibility for conducting matching‐adjusted indirect comparisons	2
Sample size re‐estimation for response‐adaptive randomized clinical trials	2
	2
Joint analysis of longitudinal measurements and survival times with a cure fraction based on partly linear mixed and semiparametric cure models	2
Estimation of Treatment Policy Estimands for Continuous Outcomes Using Off‐Treatment Sequential Multiple Imputation	2
A fiducial approach for testing the non‐inferiority of proportion difference in matched‐pairs design	2
A Bayesian phase I/II design to determine subgroup‐specific optimal dose for immunotherapy sequentially combined with radiotherapy	2