Journal of Biopharmaceutical Statistics最新文献

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A basket trial design based on constrained hierarchical Bayesian model for latent subgroups. 基于分层贝叶斯约束模型的潜伏亚组篮式试验设计。
IF 1.2 4区 医学
Journal of Biopharmaceutical Statistics Pub Date : 2025-03-01 Epub Date: 2024-02-18 DOI: 10.1080/10543406.2024.2311851
Kentaro Takeda, Atsuki Hashimoto, Shufang Liu, Alan Rong
{"title":"A basket trial design based on constrained hierarchical Bayesian model for latent subgroups.","authors":"Kentaro Takeda, Atsuki Hashimoto, Shufang Liu, Alan Rong","doi":"10.1080/10543406.2024.2311851","DOIUrl":"10.1080/10543406.2024.2311851","url":null,"abstract":"<p><p>It is well known a basket trial consisting of multiple cancer types has the potential of borrowing strength across the baskets defined by the cancer types, leading to an efficient design in terms of sample size and trial duration. The treatment effects in those baskets are often heterogeneous and categorized by the cancer types being sensitive or insensitive to the treatment. Hence, the assumption of exchangeability in many existing basket trials may be violated, and there is a need to design trials without this assumption. In this paper, we simplify the constrained hierarchical Bayesian model for latent subgroups (CHBM-LS) for two classifiers to deal with the potential heterogeneity of treatment effects due to the single classifier of the cancer type. Different baskets are aggregated into subgroups using a latent subgroup modeling approach. The treatment effects are similar and exchangeable to facilitate information borrowing within each latent subgroup. Applying the simplified CHBM-LS approach to the real basket trials where baskets defined by only cancer types shows better performance than other available approaches. Further simulation study also demonstrates this CHBM-LS approach outperforms other approaches with higher statistical power and better-controlled type I error rates under various scenarios.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"271-282"},"PeriodicalIF":1.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139900903","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
Sequential monitoring of cancer immunotherapy trial with random delayed treatment effect. 对随机延迟治疗效果的癌症免疫疗法试验进行序列监测。
IF 1.2 4区 医学
Journal of Biopharmaceutical Statistics Pub Date : 2025-03-01 Epub Date: 2023-12-25 DOI: 10.1080/10543406.2023.2296055
Jianrong Wu, Liang Zhu, Yimei Li
{"title":"Sequential monitoring of cancer immunotherapy trial with random delayed treatment effect.","authors":"Jianrong Wu, Liang Zhu, Yimei Li","doi":"10.1080/10543406.2023.2296055","DOIUrl":"10.1080/10543406.2023.2296055","url":null,"abstract":"<p><p>Cancer immunotherapy trials are frequently characterized by a delayed treatment effect that violates the proportional hazards assumption. The log-rank test (LRT) suffers a substantial loss of statistical power under the nonproportional hazards model. Various group sequential designs using weighted LRTs (WLRTs) have been proposed under the fixed delayed treatment effect model. However, patients enrolled in immunotherapy trials are often heterogeneous, and the duration of the delayed treatment effect is a random variable. Therefore, we propose group sequential designs under the random delayed effect model using the random delayed distribution WLRT. The proposed group sequential designs are developed for monitoring the efficacy of the trial using the method of Lan-DeMets alpha-spending function with O'Brien-Fleming stopping boundaries or a gamma family alpha-spending function. The maximum sample size for the group sequential design is obtained by multiplying an inflation factor with the sample size for the fixed sample design. Simulations are conducted to study the operating characteristics of the proposed group sequential designs. The robustness of the proposed group sequential designs for misspecifying random delay time distribution and domain is studied via simulations.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"227-240"},"PeriodicalIF":1.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11196386/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139038220","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Interval estimation of relative risks for combined unilateral and bilateral correlated data. 合并单侧和双侧相关数据的相对风险区间估计。
IF 1.2 4区 医学
Journal of Biopharmaceutical Statistics Pub Date : 2025-03-01 Epub Date: 2024-01-09 DOI: 10.1080/10543406.2023.2297789
Kejia Wang, Chang-Xing Ma
{"title":"Interval estimation of relative risks for combined unilateral and bilateral correlated data.","authors":"Kejia Wang, Chang-Xing Ma","doi":"10.1080/10543406.2023.2297789","DOIUrl":"10.1080/10543406.2023.2297789","url":null,"abstract":"<p><p>Measurements are generally collected as unilateral or bilateral data in clinical trials, epidemiology, or observational studies. For example, in ophthalmology studies, the primary outcome is often obtained from one eye or both eyes of an individual. In medical studies, the relative risk is usually the parameter of interest and is commonly used. In this article, we develop three confidence intervals for the relative risk for combined unilateral and bilateral correlated data under the equal dependence assumption. The proposed confidence intervals are based on maximum likelihood estimates of parameters derived using the Fisher scoring method. Simulation studies are conducted to evaluate the performance of proposed confidence intervals with respect to the empirical coverage probability, the mean interval width, and the ratio of mesial non-coverage probability to the distal non-coverage probability. We also compare the proposed methods with the confidence interval based on the method of variance estimates recovery and the confidence interval obtained from the modified Poisson regression model with correlated binary data. We recommend the score confidence interval for general applications because it best controls converge probabilities at the 95% level with reasonable mean interval width. We illustrate the methods with a real-world example.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"163-186"},"PeriodicalIF":1.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139405311","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
Miettinen and Nurminen score statistics revisited. 米特宁和努尔米宁分数统计再探。
IF 1.2 4区 医学
Journal of Biopharmaceutical Statistics Pub Date : 2025-03-01 Epub Date: 2024-02-09 DOI: 10.1080/10543406.2024.2311242
Antonio Martín Andrés, Francisco Gayá Moreno, María Álvarez Hernández, Inmaculada Herranz Tejedor
{"title":"Miettinen and Nurminen score statistics revisited.","authors":"Antonio Martín Andrés, Francisco Gayá Moreno, María Álvarez Hernández, Inmaculada Herranz Tejedor","doi":"10.1080/10543406.2024.2311242","DOIUrl":"10.1080/10543406.2024.2311242","url":null,"abstract":"<p><p>It is commonly necessary to perform inferences on the difference, ratio, and odds ratio of two proportions <i>p</i><sub><i>1</i></sub> and <i>p</i><sub><i>2</i></sub> based on two independent samples. For this purpose, the most common asymptotic statistics are based on the score statistics (<i>S</i>-type statistics). As these do not correct the bias of the estimator of the product <i>p</i><sub><i>i</i></sub> (1-<i>p</i><sub><i>i</i></sub>), Miettinen and Nurminen proposed the <i>MN</i>-type statistics, which consist of multiplying the statistics <i>S</i> by (<i>N</i>-1)/<i>N</i>, where <i>N</i> is the sum of the two sample sizes. This paper demonstrates that the factor (<i>N</i>-1)/<i>N</i> is only correct in the case of the test of equality of two proportions, providing the estimation of the correct factor (<i>AU</i>-type statistics) and the minimum value of the same (<i>AUM-</i>type statistics). Moreover, this paper assesses the performance of the four-type statistics mentioned (<i>S</i>, <i>MN</i>, <i>AU</i> and <i>AUM</i>) in one and two-tailed tests, and for each of the three parameters cited (<i>d</i>, <i>R</i> and <i>OR</i>). We found that the <i>AUM-</i>type statistics are the best, followed by the <i>MN</i> type (whose performance was most similar to that of <i>AU-</i>type). Finally, this paper also provides the correct factors when the data are from a multinomial distribution, with the novelty that the <i>MN</i> and AU statistics are similar in the case of the test for the odds ratio.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"283-296"},"PeriodicalIF":1.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139713444","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
Combination MCP-Mod for two-drug combination dose-ranging studies. 用于双药联合剂量范围研究的 MCP-Mod 组合。
IF 1.2 4区 医学
Journal of Biopharmaceutical Statistics Pub Date : 2025-03-01 Epub Date: 2024-02-09 DOI: 10.1080/10543406.2024.2311254
Yifan Zhou, Abigail Sloan, Sandeep Menon, Ling Wang
{"title":"Combination MCP-Mod for two-drug combination dose-ranging studies.","authors":"Yifan Zhou, Abigail Sloan, Sandeep Menon, Ling Wang","doi":"10.1080/10543406.2024.2311254","DOIUrl":"10.1080/10543406.2024.2311254","url":null,"abstract":"<p><p>Combination therapies with multiple mechanisms of action can offer improved efficacy and/or safety profiles when compared to a single therapy with one mechanism of action. Consequently, the number of combination therapy studies have increased multi-fold, both in oncology and non-oncology indications. However, identifying the optimal doses of each drug in a combination therapy can require a large sample size and prolong study timelines, especially when full factorial designs are used. In this paper, we extend the MCP-Mod design of Bretz, Pinheiro, and Branson to a three-dimensional space to model the dose-response surface of a two-drug combination under the framework of Combination (Comb) MCP-Mod. The resulting model yields a set of dosages for each drug in the combination that elicits the target response so that an optimal dose for the combination can be selected for pivotal studies. We construct three-dimensional dose-response models for the combination and formulate the contrast test statistic to select the best model, which can then be used to select the optimal dose. Guidance to calculate power and sample size calculations are provided to assist study design. Simulation studies show that Comb MCP-Mod performs as well as the conventional multiple comparisons approach in controlling the family-wise error rate at the desired alpha level. However, Comb MCP-Mod is more powerful than the classical multiple comparisons approach in detecting dose-response relationships when treatment is non-null. The probability of correctly identifying the underlying dose-response relationship is generally higher when using Comb MCP-Mod than when using the multiple comparisons approach.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"257-270"},"PeriodicalIF":1.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139713443","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
Directed Acyclic Graph Assisted Method For Estimating Average Treatment Effect. 估算平均治疗效果的有向无环图辅助方法
IF 1.2 4区 医学
Journal of Biopharmaceutical Statistics Pub Date : 2025-03-01 Epub Date: 2023-12-27 DOI: 10.1080/10543406.2023.2296047
Jingchao Sun, Scott Duncan, Subhadip Pal, Maiying Kong
{"title":"Directed Acyclic Graph Assisted Method For Estimating Average Treatment Effect.","authors":"Jingchao Sun, Scott Duncan, Subhadip Pal, Maiying Kong","doi":"10.1080/10543406.2023.2296047","DOIUrl":"10.1080/10543406.2023.2296047","url":null,"abstract":"<p><p>Observational data, such as electronic clinical records and claims data, can prove invaluable for evaluating the Average Treatment Effect (ATE) and supporting decision-making, provided they are employed correctly. The Inverse Probability of Treatment Weighting (IPTW) method, based on propensity scores, has demonstrated remarkable efficacy in estimating ATE, assuming that the assumptions of exchangeability, consistency, and positivity are met. Directed Acyclic Graphs (DAGs) offer a practical approach to assess the exchangeability assumption, which asserts that treatment assignment and potential outcomes are independent given a set of confounding variables that block all backdoor paths from treatment assignment to potential outcomes. To ensure a consistent ATE estimator, one can adjust for a minimally sufficient adjustment set of confounding variables that block all backdoor paths from treatment assignment to the outcome. To enhance the efficiency of ATE estimators, our proposal involves incorporating both the minimally sufficient adjustment set of confounding variables and predictors into the propensity score model. Extensive simulations were conducted to evaluate the performance of propensity score-based IPTW methods in estimating ATE when different sets of covariates were included in the propensity score models. The simulation results underscored the significance of including the minimally sufficient adjustment set of confounding variables along with predictors in the propensity score models to obtain a consistent and efficient ATE estimator. We applied this proposed method to investigate whether tracheostomy was causally associated with in-hospital infant mortality, utilizing the 2016 Healthcare Cost and Utilization Project Kids' Inpatient Database. The estimated ATE was found to be approximately 2.30%-2.46% with p-value >0.05.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"187-206"},"PeriodicalIF":1.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11209833/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139049806","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Bayesian phase II adaptive randomization by jointly modeling efficacy and toxicity as time-to-event outcomes. 通过将疗效和毒性联合建模为时间到事件结果,进行贝叶斯 II 期适应性随机化。
IF 1.2 4区 医学
Journal of Biopharmaceutical Statistics Pub Date : 2025-03-01 Epub Date: 2024-01-01 DOI: 10.1080/10543406.2023.2297782
Yu-Mei Chang, Pao-Sheng Shen, Chun-Ying Ho
{"title":"Bayesian phase II adaptive randomization by jointly modeling efficacy and toxicity as time-to-event outcomes.","authors":"Yu-Mei Chang, Pao-Sheng Shen, Chun-Ying Ho","doi":"10.1080/10543406.2023.2297782","DOIUrl":"10.1080/10543406.2023.2297782","url":null,"abstract":"<p><p>The main goals of Phase II trials are to identify the therapeutic efficacy of new treatments and continue monitoring all the possible adverse effects. In Phase II trials, it is important to develop an adaptive randomization (AR) procedure that takes into account both the efficacy and toxicity. In most existing articles, toxicity is modeled as a binary endpoint through an unobservable random effect (frailty) to link the efficacy and toxicity. However, this approach does not capture toxicity profiles that evolve over time. In this article, we propose a new Bayesian adaptive randomization (BAR) procedure using the covariate-adjusted efficacy-toxicity ratio (ETR) index, where efficacy and toxicity are jointly modelled as time-to-event (TTE) outcomes. Furthermore, we also propose early stopping rules for toxicity and futility such that inferior treatments can be dropped at earlier time of trial. Simulation results show that compared to the BAR procedures based solely on the efficacy and that based on TTE efficacy and binary toxicity outcomes, the proposed BAR procedure can better identify the difference in treatment toxicity such that it can assign more patients to the superior treatment arm under some scenarios.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"207-226"},"PeriodicalIF":1.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139075923","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
Sample size estimation for recurrent event data using multifrailty and multilevel survival models. 使用多变量和多层次生存模型估算复发事件数据的样本量。
IF 1.2 4区 医学
Journal of Biopharmaceutical Statistics Pub Date : 2025-03-01 Epub Date: 2024-02-09 DOI: 10.1080/10543406.2024.2310306
Derek Dinart, Carine Bellera, Virginie Rondeau
{"title":"Sample size estimation for recurrent event data using multifrailty and multilevel survival models.","authors":"Derek Dinart, Carine Bellera, Virginie Rondeau","doi":"10.1080/10543406.2024.2310306","DOIUrl":"10.1080/10543406.2024.2310306","url":null,"abstract":"<p><p>In epidemiology and clinical research, recurrent events refer to individuals who are likely to experience transient clinical events repeatedly over an observation period. Examples include hospitalizations in patients with heart failure, fractures in osteoporosis studies and the occurrence of new lesions in oncology. We provided an in-depth analysis of the sample size required for the analysis of recurrent time-to-event data using multifrailty or multilevel survival models. We covered the topic from the simple shared frailty model to models with hierarchical or joint frailties. We relied on a Wald-type test statistic to estimate the sample size assuming either a single or multiple endpoints. Simulations revealed that the sample size increased as heterogeneity increased. We also observed that it was more attractive to include more patients and reduce the duration of follow-up than to include fewer patients and increase the duration of follow-up to obtain the number of events required. Each model investigated can address the question of the number of subjects for recurrent events. However, depending on the research question, one model will be more suitable than another. We illustrated our methodology with the AFFIRM-AHF trial investigating the effect of intravenous ferric carboxymaltose in patients hospitalised for acute heart failure.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"241-256"},"PeriodicalIF":1.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139708580","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
Considerations for master protocols using external controls. 使用外部控制的主协议的注意事项。
IF 1.2 4区 医学
Journal of Biopharmaceutical Statistics Pub Date : 2025-03-01 Epub Date: 2024-02-16 DOI: 10.1080/10543406.2024.2311248
Jie Chen, Xiaoyun Nicole Li, Chengxing Cindy Lu, Sammy Yuan, Godwin Yung, Jingjing Ye, Hong Tian, Jianchang Lin
{"title":"Considerations for master protocols using external controls.","authors":"Jie Chen, Xiaoyun Nicole Li, Chengxing Cindy Lu, Sammy Yuan, Godwin Yung, Jingjing Ye, Hong Tian, Jianchang Lin","doi":"10.1080/10543406.2024.2311248","DOIUrl":"10.1080/10543406.2024.2311248","url":null,"abstract":"<p><p>There has been an increasing use of master protocols in oncology clinical trials because of its efficiency to accelerate cancer drug development and flexibility to accommodate multiple substudies. Depending on the study objective and design, a master protocol trial can be a basket trial, an umbrella trial, a platform trial, or any other form of trials in which multiple investigational products and/or subpopulations are studied under a single protocol. Master protocols can use external data and evidence (e.g. external controls) for treatment effect estimation, which can further improve efficiency of master protocol trials. This paper provides an overview of different types of external controls and their unique features when used in master protocols. Some key considerations in master protocols with external controls are discussed including construction of estimands, assessment of fit-for-use real-world data, and considerations for different types of master protocols. Similarities and differences between regular randomized controlled trials and master protocols when using external controls are discussed. A targeted learning-based causal roadmap is presented which constitutes three key steps: (1) define a target statistical estimand that aligns with the causal estimand for the study objective, (2) use an efficient estimator to estimate the target statistical estimand and its uncertainty, and (3) evaluate the impact of causal assumptions on the study conclusion by performing sensitivity analyses. Two illustrative examples for master protocols using external controls are discussed for their merits and possible improvement in causal effect estimation.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"297-319"},"PeriodicalIF":1.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139747780","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
On Fβ -score for medical diagnostics tests of binary diseases: proposing new measures of accuracy.
IF 1.2 4区 医学
Journal of Biopharmaceutical Statistics Pub Date : 2025-02-27 DOI: 10.1080/10543406.2025.2469866
Marwan Alsharman, Hani Samawi, Jing Kersey, Divine Wanduku
{"title":"On F<sub>β</sub> -score for medical diagnostics tests of binary diseases: proposing new measures of accuracy.","authors":"Marwan Alsharman, Hani Samawi, Jing Kersey, Divine Wanduku","doi":"10.1080/10543406.2025.2469866","DOIUrl":"https://doi.org/10.1080/10543406.2025.2469866","url":null,"abstract":"<p><p>Accurate differentiation between health states - diseased or non-diseased - is essential in clinical diagnostics. Optimal cut-off points, or thresholds used to classify test results, are crucial for precise diagnoses. This work introduces the Harmonic Mean of F-score and inverse F-score (<i>HF</i>), a novel metric for a balanced assessment of diagnostic accuracy. <i>HF</i> integrates Specificity (<i>Sp</i>) and Negative Predictive Value (NPV) into the Negative F-score (<i>NF</i><sub><i>γ</i></sub>), ensuring a comprehensive evaluation of true negatives and negative test reliability. Prioritizing both true positives and true negatives, <i>HF</i> was used in optimal cut-off point estimation under binary disease classification. Simulation results revealed that the <i>HF</i> measure performed well, often surpassing established methods in specific settings. The <i>HF</i> measure and cut-off point selection criterion were applied to real-life data, showcasing its ability to provide a balanced evaluation of diagnostic accuracy. The <i>HF</i> measure frequently outperformed traditional metrics. The <i>HF</i> metric's flexibility, allowing parameter adjustments to accommodate diverse scenarios, enables researchers and clinicians to tailor its emphasis on specific aspects of diagnostic performance depending on the context.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"1-27"},"PeriodicalIF":1.2,"publicationDate":"2025-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143525256","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
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