Journal of Biopharmaceutical Statistics最新文献_第2页

The power prior for zero-inflated count models: Analysis based on episodes of urinary incontinence. 零膨胀计数模型的功率先验：基于尿失禁发作的分析。

IF 1.3 4区医学

Journal of Biopharmaceutical Statistics Pub Date : 2026-10-11 DOI: 10.1080/10543406.2026.2741426

Akalu Banbeta Tereda, Legesse Kassa Debusho

{"title":"The power prior for zero-inflated count models: Analysis based on episodes of urinary incontinence.","authors":"Akalu Banbeta Tereda, Legesse Kassa Debusho","doi":"10.1080/10543406.2026.2741426","DOIUrl":"https://doi.org/10.1080/10543406.2026.2741426","url":null,"abstract":"We have extended the power prior approach for the zero-inflated count models. This helps to incorporate historical information into the analysis of a current study for the zero-inflated Poisson and negative binomial models. Our motivation is based on clinical trials conducted to evaluate an active treatment versus the control treatment for patients with an overactive bladder. The endpoint of interest is the number of incontinence episodes observed three days prior to week 12 after starting treatment. This is a count outcome that contains an excess of zeros. We used the principle of path sampling to compute the scaling constant of the modified power prior (MPP). Our algorithm efficiently works for two historical data sets. A simulation study was conducted to assess operating characteristics under both homogeneous and heterogeneous settings. When heterogeneity among studies arises from differences in dispersion and/or probabilities of zeros, the MPP approach maintains type I error rates close to the nominal 5%, whereas pooling and meta-analytic-predictive (MAP) approaches lead to inflated type I error rates. In contrast, when heterogeneity is due to the mean parameters, the MAP approach maintains the nominal type I error rate, while the MPP method leads to an inflated type I error rate. Across scenarios in which type I error is adequately controlled, the dependent MPP, i.e. the MPP which assumes similar power parameters, consistently achieves the highest statistical power among the borrowing methods.","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"1-22"},"PeriodicalIF":1.3,"publicationDate":"2026-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149169988","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Comparison of multiple testing procedures for clinical trials with two doses and multiple ranked endpoints. 两个剂量和多个分级终点的临床试验的多个测试程序的比较。

IF 1.3 4区医学

Journal of Biopharmaceutical Statistics Pub Date : 2026-10-11 DOI: 10.1080/10543406.2026.2741424

Pascal Minini, Gautier Paux, Alex Dmitrienko

{"title":"Comparison of multiple testing procedures for clinical trials with two doses and multiple ranked endpoints.","authors":"Pascal Minini, Gautier Paux, Alex Dmitrienko","doi":"10.1080/10543406.2026.2741424","DOIUrl":"https://doi.org/10.1080/10543406.2026.2741424","url":null,"abstract":"Clinical trials comparing two doses of an investigational product versus placebo are becoming more and more frequent in the confirmatory phase of clinical development. These clinical trials generally include a primary endpoint, and several key secondary endpoints ranked hierarchically. Strong control of the family‑wise error rate (FWER) is required in particular if the study is aimed at supporting approval of the investigational product. This paper describes a simulation study comparing different multiple testing procedures (MTP), under a wide range of plausible scenarios. A case study including two doses and three ranked endpoints was introduced to illustrate the MTPs and evaluate their performance. Our findings provide practical guidance for statisticians and clinical researchers designing trials with two doses and multiple endpoints, highlighting which methods offer optimal performance under different anticipated scenarios. The results also challenge some common assumptions about hierarchical testing approaches and demonstrate the robust performance of enhanced mixture methods across diverse dose-response scenarios. This paper also shows that the enhanced mixture method can be simplified in trials that include two doses and hierarchically ranked endpoints.","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"1-20"},"PeriodicalIF":1.3,"publicationDate":"2026-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149170857","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The bivariate defective Marshall-Olkin Weibull distribution based on Clayton Copula and its application to medical survival data. 基于Clayton Copula的二元缺陷Marshall-Olkin Weibull分布及其在医疗生存数据中的应用。

IF 1.3 4区医学

Journal of Biopharmaceutical Statistics Pub Date : 2026-10-05 DOI: 10.1080/10543406.2026.2726747

Dariush Kadkhoda, Ali Akbar Khadem Maboudi, Mohammad Mehdi Dindarloo, Ahmad Reza Baghestani

{"title":"The bivariate defective Marshall-Olkin Weibull distribution based on Clayton Copula and its application to medical survival data.","authors":"Dariush Kadkhoda, Ali Akbar Khadem Maboudi, Mohammad Mehdi Dindarloo, Ahmad Reza Baghestani","doi":"10.1080/10543406.2026.2726747","DOIUrl":"https://doi.org/10.1080/10543406.2026.2726747","url":null,"abstract":"Traditional survival analysis models assume that all individuals will eventually experience the event, while cure models account for subsets that remain \"cured\" or immune. These models estimate cure rates and have been applied extensively, particularly in cancer research. Recent advancements have introduced defective distributions as a novel approach in cure rate modeling. In defective models, the total probability mass is less than one, and the survival function approaches a constant value, referred to as time progresses toward infinity. In this context, p represents the cure rate. This innovative method is particularly beneficial in real-world applications involving correlated time-to-event outcomes. A significant gap exists in the study of defective models, which fundamentally contradict probability theory. We proposed a new bivariate cure model using a copula-based approach to address dependencies between survival outcomes, introducing the use of defective distributions in multivariate survival analysis as a novel topic. We investigated the behavior of the defective Marshall-Olkin Weibull distribution in bivariate survival analysis using Clayton copula through a simulation study and on real-world datasets. The simulation study confirmed the model's adequacy, with estimations improving as the sample size increased. Our model demonstrated exceptional flexibility compared to previous models in the literature; its correlation estimates (Kendall and Spearman) closely matched empirical values, achieved lower AIC and BIC values, and accurately identified significant covariates. The model performed well even without a cure fraction, highlighting its robustness and potential for various scenarios.","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"1-30"},"PeriodicalIF":1.3,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149118727","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Post-test medical diagnostic accuracy measures: an innovative approach based on the area under F-scores curves. 测试后医学诊断准确性测量：一种基于f分数曲线下面积的创新方法。

IF 1.3 4区医学

Journal of Biopharmaceutical Statistics Pub Date : 2026-10-01 Epub Date: 2025-06-17 DOI: 10.1080/10543406.2025.2512989

Hani Samawi, Jing Kersey, Marwan Alsharman

引用次数: 0

Confidence intervals for the covariate-specific overlap coefficient (OVL). 协变量特异性重叠系数（OVL）的置信区间。

IF 1.3 4区医学

Journal of Biopharmaceutical Statistics Pub Date : 2026-10-01 Epub Date: 2025-08-25 DOI: 10.1080/10543406.2025.2547587

M Carmen Pardo, Alba M Franco-Pereira, Benjamin Reiser, Christos T Nakas

引用次数: 0

Curtailed procedures for binomial random-sized subset selection. 二项随机大小子集选择的简化程序。

IF 1.3 4区医学

Journal of Biopharmaceutical Statistics Pub Date : 2026-10-01 Epub Date: 2025-11-12 DOI: 10.1080/10543406.2025.2575945

Yifang Zhang, Pinyuen Chen

{"title":"Curtailed procedures for binomial random-sized subset selection.","authors":"Yifang Zhang, Pinyuen Chen","doi":"10.1080/10543406.2025.2575945","DOIUrl":"10.1080/10543406.2025.2575945","url":null,"abstract":"Randomized subset selection procedures are important statistical tools in clinical trials involving multiple treatments. However, traditional methods lack built-in early stopping criteria, leading to potential inefficiencies and unnecessary patient exposure. Inspired by Gupta and Sobel's (1960) foundational subset selection approach and Bechhofer and Kulkarni's (1982) idea of curtailment, this paper introduces a curtailed subset selection procedure for binomial populations under a frequentist framework. Specifically, our method includes a mathematically driven stopping rule that terminates sampling as soon as non-leading treatments can no longer statistically surpass the current leader. We derive explicit formulas for calculating the probability of correct selection and the expected sample size, and we also introduce an optional randomization extension to precisely achieve pre-specified accuracy targets. Simulation studies confirm that the proposed curtailed procedure maintains comparable accuracy levels while substantially reducing expected sample sizes compared to existing procedures. Illustrative examples from clinical trial scenarios demonstrate the practical benefits and ease of implementation. This approach provides researchers and practitioners with an efficient, statistically rigorous tool for optimizing subset selection in biopharmaceutical research.","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"1587-1614"},"PeriodicalIF":1.3,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145508132","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Tables, listings and figures in a clinical study report - quality or quantity? 临床研究报告中的表格、清单和数据——质量还是数量？

IF 1.3 4区医学

Journal of Biopharmaceutical Statistics Pub Date : 2026-10-01 Epub Date: 2025-11-10 DOI: 10.1080/10543406.2025.2575942

Ning Li, Yaohua Zhang, Naitee Ting

引用次数: 0

Change-point detection in Weibull-accelerated failure time models via narrowest significance pursuit. 基于最窄显著性追求的威布尔加速故障时间模型的变化点检测。

IF 1.3 4区医学

Journal of Biopharmaceutical Statistics Pub Date : 2026-10-01 Epub Date: 2025-12-21 DOI: 10.1080/10543406.2025.2602464

Md Hasinur Rahaman Khan, Samia Ashrafi

{"title":"Change-point detection in Weibull-accelerated failure time models via narrowest significance pursuit.","authors":"Md Hasinur Rahaman Khan, Samia Ashrafi","doi":"10.1080/10543406.2025.2602464","DOIUrl":"10.1080/10543406.2025.2602464","url":null,"abstract":"Survival data are often subject to abrupt structural changes due to unforeseen natural or anthropogenic events, which may significantly alter the hazard function and introduce shifts in model parameters. Traditional survival models typically assume time-invariant hazard structures and are therefore inadequate in capturing such distributional shifts. To address this limitation, we adapt the Narrowest Significance Pursuit (NSP) methodology proposed by Fryzlewicz (2024) for change-point detection within the framework of accelerated failure time (AFT) models. The NSP algorithm employs a multiscale sup-norm loss to fit Weibull AFT models across varying subintervals, recursively identifying the narrowest intervals where linearity is statistically violated at a user-specified global significance level. Change-points are detected as abrupt changes in the underlying parameters of the survival model, and the method does not require prior specification of the number or location of these points. We conduct an extensive simulation study to evaluate the performance of the proposed approach under various change-point scenarios, censoring levels, and compare it with the existing methods in the literature. Additionally, a real-world dataset is analyzed to demonstrate the practical applicability of the method. The empirical results highlight the accuracy and robustness of NSP in detecting structural changes in survival distributions, making it a valuable tool for analyzing heterogeneous survival data in dynamic risk environments.","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"1713-1730"},"PeriodicalIF":1.3,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145806392","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Assessing predictive probability of success for future clinical trials. 评估未来临床试验成功的预测概率。

IF 1.3 4区医学

Journal of Biopharmaceutical Statistics Pub Date : 2026-10-01 Epub Date: 2025-06-16 DOI: 10.1080/10543406.2025.2510262

Archie Sachdeva, Ram Tiwari, Ming Zhou

{"title":"Assessing predictive probability of success for future clinical trials.","authors":"Archie Sachdeva, Ram Tiwari, Ming Zhou","doi":"10.1080/10543406.2025.2510262","DOIUrl":"10.1080/10543406.2025.2510262","url":null,"abstract":"Data-driven decision-making is crucial in drug development, with the predictive probability of success (PoS) being a key quantitative tool. PoS estimates the likelihood of success of a future trial based on the same or surrogate endpoint(s) of interest, utilizing information from interim analyses, or completed historical studies. While it has been extensively studied and broadly applied in clinical practice, there is a growing need of a unified approach for PoS that can effectively incorporate information from surrogate endpoints and multiple historical studies. This paper investigates and assesses a unified Bayesian approach for PoS. We first review PoS based on historical data on the same endpoint and then extend it to include information from a surrogate endpoint with a closed-form solution. Additionally, we utilize a Bayesian meta-analytic approach to incorporate data from multiple historical studies. We illustrate the unified approach with examples from oncology and immunology trials and provide an R package \"PPoS\" for practical implementation. By integrating the assessment of PoS with information from surrogate endpoints and historical studies, we aim to enhance the decision-making process in drug development.","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"1257-1279"},"PeriodicalIF":1.3,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144303656","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

A Bayesian approach for studying COVID-19 contagion dynamics in Algeria using a Poisson autoregressive (PAR) model. 使用泊松自回归（PAR）模型研究阿尔及利亚COVID-19传染动力学的贝叶斯方法

IF 1.3 4区医学

Journal of Biopharmaceutical Statistics Pub Date : 2026-10-01 Epub Date: 2025-04-17 DOI: 10.1080/10543406.2025.2489361

Ahmed Hamimes, Hani Amir Aouissi, Feriel Kheira Kebaili, Zeinab A Kasemy

{"title":"A Bayesian approach for studying COVID-19 contagion dynamics in Algeria using a Poisson autoregressive (PAR) model.","authors":"Ahmed Hamimes, Hani Amir Aouissi, Feriel Kheira Kebaili, Zeinab A Kasemy","doi":"10.1080/10543406.2025.2489361","DOIUrl":"10.1080/10543406.2025.2489361","url":null,"abstract":"Global emphasis has been focused on tracking the trends of the COVID-19 pandemic. Numerous techniques have been developed or utilized for this purpose. In this study, we seek to present and evaluate a model that, in our opinion, has not received adequate attention, using Algeria as a case study. We developed two distinct Poisson autoregressive (PAR) models using the Monte Carlo Markov Chain (MCMC) simulation method and the Bayesian method: one based solely on short-term dependence and the other incorporating both short- and long-term dependence. The study aimed to apply these models to enhance the prediction of new infections and determine whether the disease is spreading or declining. This information can guide decisions on implementing or relaxing containment measures. Our findings suggest that Algeria's epidemiological state was relatively stable at the end of the study period, with the combined long-term and short-term dependence factors being less than 1 (<math><mi>α</mi><mo>+</mo><mi>β</mi><mo>=</mo><mn>0.994</mn><mo>)</mo></math>. This indicates that while the epidemic is in decline, the infection rates are not expected to drop significantly in the near future. Furthermore, the short-term dependence parameter <math><mi>α</mi><mo>=</mo><mn>0.987</mn></math>constitutes a significant portion (99%) of the total dependence. This high value of <math><mi>α</mi></math> suggest that the COVID-19 epidemic in Algeria is experiencing a strong decline, though the rate of new infections is expected to persist at a lower level for the foreseeable future. Given these findings, it is recommended that authorities remain vigilant and continue public health measures, including educational campaigns and awareness efforts, to promote COVID-19 vaccination and adherence to health guidelines.","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"1227-1243"},"PeriodicalIF":1.3,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144033680","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0