Pub Date : 2024-01-05DOI: 10.1080/19466315.2023.2289514
Bharati Kumar, Jonathan W. Bartlett
Nonproportional hazards (NPH) have been observed in confirmatory clinical trials with time to event outcomes. Under NPH, the hazard ratio does not stay constant over time and the log rank test is n...
{"title":"Weighted Hazard Ratio Estimation for Delayed and Diminishing Treatment Effect","authors":"Bharati Kumar, Jonathan W. Bartlett","doi":"10.1080/19466315.2023.2289514","DOIUrl":"https://doi.org/10.1080/19466315.2023.2289514","url":null,"abstract":"Nonproportional hazards (NPH) have been observed in confirmatory clinical trials with time to event outcomes. Under NPH, the hazard ratio does not stay constant over time and the log rank test is n...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139103532","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}
Pub Date : 2024-01-02DOI: 10.1080/19466315.2023.2298961
Lindsay R Berry, Elizabeth Lorenzi, Nicholas S Berry, Amy M Crawford, Peter Jacko, Kert Viele
Many trial designs, such as dose-finding trials, shared-control designs, or adaptive platform trials, investigate multiple therapies simultaneously. Often these trials seek to identify the best arm...
{"title":"Effects of Allocation Method and Time Trends on Identification of the Best Arm in Multi-arm Trials","authors":"Lindsay R Berry, Elizabeth Lorenzi, Nicholas S Berry, Amy M Crawford, Peter Jacko, Kert Viele","doi":"10.1080/19466315.2023.2298961","DOIUrl":"https://doi.org/10.1080/19466315.2023.2298961","url":null,"abstract":"Many trial designs, such as dose-finding trials, shared-control designs, or adaptive platform trials, investigate multiple therapies simultaneously. Often these trials seek to identify the best arm...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139083546","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}
Pub Date : 2023-12-18DOI: 10.1080/19466315.2023.2296648
Francois Mercier, Victoria Homer, Junxian Geng, Hontao Zhang, Stefan Englert, Natalia Kan-Dobrosky, Anja Victor
The R1 addendum to ICH E9 (E9-R1) provides guidance on the definition of estimands in clinical drug development. While the E9-R1 has seen uptake in randomized late-stage clinical trials, its implem...
ICH E9 的 R1 增补（E9-R1）为临床药物开发中估算指标的定义提供了指导。虽然 E9-R1 已在后期随机临床试验中得到采纳，但其执行情况仍不容乐观。
{"title":"Estimands in oncology early clinical development: Assessing the impact of intercurrent events on the dose-toxicity relationship","authors":"Francois Mercier, Victoria Homer, Junxian Geng, Hontao Zhang, Stefan Englert, Natalia Kan-Dobrosky, Anja Victor","doi":"10.1080/19466315.2023.2296648","DOIUrl":"https://doi.org/10.1080/19466315.2023.2296648","url":null,"abstract":"The R1 addendum to ICH E9 (E9-R1) provides guidance on the definition of estimands in clinical drug development. While the E9-R1 has seen uptake in randomized late-stage clinical trials, its implem...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138821316","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}
Pub Date : 2023-12-12DOI: 10.1080/19466315.2023.2292774
Jiawei Wei, Jiajun Xu, Björn Bornkamp, Ray Lin, Hong Tian, Dong Xi, Xin Zhang, Ziqiang Zhao, Satrajit Roychoudhury
ICH E9(R1) specifies the importance of precisely defining the treatment effect for clinical trials – to inform patient choices and facilitate evidence-based decision-making. FDA's guidance on covar...
ICH E9(R1)明确规定了精确定义临床试验治疗效果的重要性--为患者的选择提供信息并促进循证决策。美国食品和药物管理局（FDA）关于临床试验疗效一致性的指导意见...
{"title":"Conditional and Unconditional treatment effects in randomized clinical trials: Estimands, Estimation, and Interpretation","authors":"Jiawei Wei, Jiajun Xu, Björn Bornkamp, Ray Lin, Hong Tian, Dong Xi, Xin Zhang, Ziqiang Zhao, Satrajit Roychoudhury","doi":"10.1080/19466315.2023.2292774","DOIUrl":"https://doi.org/10.1080/19466315.2023.2292774","url":null,"abstract":"ICH E9(R1) specifies the importance of precisely defining the treatment effect for clinical trials – to inform patient choices and facilitate evidence-based decision-making. FDA's guidance on covar...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138686311","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}
Pub Date : 2023-12-12DOI: 10.1080/19466315.2023.2292815
Jennifer L. Proper, Veronica Bunn, Bradley Hupf, Jianchang Lin
Given the rising costs and time length of confirmatory phase III trials, drug developers have become increasingly reliant on quantitative methods to support critical decisions such as whether drug ...
{"title":"Predicting Probability of Success for Phase III Trials via Propensity-Score-Based External Data Borrowing","authors":"Jennifer L. Proper, Veronica Bunn, Bradley Hupf, Jianchang Lin","doi":"10.1080/19466315.2023.2292815","DOIUrl":"https://doi.org/10.1080/19466315.2023.2292815","url":null,"abstract":"Given the rising costs and time length of confirmatory phase III trials, drug developers have become increasingly reliant on quantitative methods to support critical decisions such as whether drug ...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138631875","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}
Pub Date : 2023-12-11DOI: 10.1080/19466315.2023.2292238
Beibei Guo, Li Wang, Ying Yuan
An adaptive platform trial (APT) is a multi-arm trial in the context of a single disease where treatment arms are allowed to enter or leave the trial based on some decision rule. If a treatment ent...
{"title":"Treatment Comparisons in Adaptive Platform Trials Adjusting for Temporal Drift","authors":"Beibei Guo, Li Wang, Ying Yuan","doi":"10.1080/19466315.2023.2292238","DOIUrl":"https://doi.org/10.1080/19466315.2023.2292238","url":null,"abstract":"An adaptive platform trial (APT) is a multi-arm trial in the context of a single disease where treatment arms are allowed to enter or leave the trial based on some decision rule. If a treatment ent...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138567738","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}
Pub Date : 2023-12-07DOI: 10.1080/19466315.2023.2292816
Ziji Yu, Yanzhao Wang, Jianchang Lin
The traditional MTD-based dose selection paradigm commonly used for cytotoxic chemotherapies might not be optimal for targeted therapies because a higher dose does not necessarily result in improve...
{"title":"DODII: Bayesian Dose Optimization Design for Randomized Phase II Trials","authors":"Ziji Yu, Yanzhao Wang, Jianchang Lin","doi":"10.1080/19466315.2023.2292816","DOIUrl":"https://doi.org/10.1080/19466315.2023.2292816","url":null,"abstract":"The traditional MTD-based dose selection paradigm commonly used for cytotoxic chemotherapies might not be optimal for targeted therapies because a higher dose does not necessarily result in improve...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-12-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138575947","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}
Pub Date : 2023-11-30DOI: 10.1080/19466315.2023.2290642
Ming Chi, Xiaogang Wang, Hui Song, Yingwei Peng, Dongsheng Tu
For longitudinal ordinal categorical item response data which may not be observable after a subject develops a terminal event, some statistical models have been proposed for the joint analysis of t...
{"title":"Joint Analysis of Longitudinal Ordinal Categorical Item Response Data and Survival Times with Cure Fraction","authors":"Ming Chi, Xiaogang Wang, Hui Song, Yingwei Peng, Dongsheng Tu","doi":"10.1080/19466315.2023.2290642","DOIUrl":"https://doi.org/10.1080/19466315.2023.2290642","url":null,"abstract":"For longitudinal ordinal categorical item response data which may not be observable after a subject develops a terminal event, some statistical models have been proposed for the joint analysis of t...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138516362","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}
Pub Date : 2023-11-29DOI: 10.1080/19466315.2023.2289523
Hege Michiels, An Vandebosch, Stijn Vansteelandt
When choosing estimands and estimators in randomized clinical trials, caution is warranted, as intercurrent events, such as, due to patients who switch treatment after disease progression, are ofte...
{"title":"Adjusting for time-varying treatment switches in randomized clinical trials: the danger of extrapolation and how to address it","authors":"Hege Michiels, An Vandebosch, Stijn Vansteelandt","doi":"10.1080/19466315.2023.2289523","DOIUrl":"https://doi.org/10.1080/19466315.2023.2289523","url":null,"abstract":"When choosing estimands and estimators in randomized clinical trials, caution is warranted, as intercurrent events, such as, due to patients who switch treatment after disease progression, are ofte...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138516352","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}
Pub Date : 2023-11-28DOI: 10.1080/19466315.2023.2288013
Nolan A. Wages, Ruitao Lin
This article considers the concept of designing Phase I clinical trials using both clinician- and patient-reported outcomes to adaptively allocate study participants to tolerable doses and determin...
{"title":"Isotonic Phase I cancer clinical trial design utilizing patient-reported outcomes","authors":"Nolan A. Wages, Ruitao Lin","doi":"10.1080/19466315.2023.2288013","DOIUrl":"https://doi.org/10.1080/19466315.2023.2288013","url":null,"abstract":"This article considers the concept of designing Phase I clinical trials using both clinician- and patient-reported outcomes to adaptively allocate study participants to tolerable doses and determin...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138516325","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}
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