Pub Date : 2024-06-21DOI: 10.1080/19466315.2024.2370405
Yu-Chieh Cheng, Hsiao-Hui Tsou, H.M.James Hung, Byron Fan, Brandon Fan
Clinical trials are an essential aspect of the drug development process. Clinical endpoints and surrogate endpoints are two terms used in clinical trials to measure the effectiveness of a treatment...
{"title":"Establishing and using threshold of surrogate endpoint in relation to clinical endpoint#","authors":"Yu-Chieh Cheng, Hsiao-Hui Tsou, H.M.James Hung, Byron Fan, Brandon Fan","doi":"10.1080/19466315.2024.2370405","DOIUrl":"https://doi.org/10.1080/19466315.2024.2370405","url":null,"abstract":"Clinical trials are an essential aspect of the drug development process. Clinical endpoints and surrogate endpoints are two terms used in clinical trials to measure the effectiveness of a treatment...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":"247 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2024-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141613836","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 : 2024-06-17DOI: 10.1080/19466315.2024.2368789
Se Yoon Lee
In Bayesian clinical trial design, the power prior methodology offers a flexible framework for incorporating historical data into the analysis. One critical aspect of this approach is determining t...
{"title":"Eliciting the discount parameter in a power prior method on the basis of the type I error consideration","authors":"Se Yoon Lee","doi":"10.1080/19466315.2024.2368789","DOIUrl":"https://doi.org/10.1080/19466315.2024.2368789","url":null,"abstract":"In Bayesian clinical trial design, the power prior methodology offers a flexible framework for incorporating historical data into the analysis. One critical aspect of this approach is determining t...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":"90 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2024-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141613969","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 : 2024-06-17DOI: 10.1080/19466315.2024.2368787
David K. Lim, Naim U. Rashid, Junier B. Oliva, Joseph G. Ibrahim
Deep Learning (DL) methods have dramatically increased in popularity in recent years. While its initial success was demonstrated in the classification and manipulation of image data, there has been...
{"title":"Unsupervised Imputation of Non-ignorably Missing Data Using Importance-Weighted Autoencoders","authors":"David K. Lim, Naim U. Rashid, Junier B. Oliva, Joseph G. Ibrahim","doi":"10.1080/19466315.2024.2368787","DOIUrl":"https://doi.org/10.1080/19466315.2024.2368787","url":null,"abstract":"Deep Learning (DL) methods have dramatically increased in popularity in recent years. While its initial success was demonstrated in the classification and manipulation of image data, there has been...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":"63 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2024-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141530915","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 : 2024-06-10DOI: 10.1080/19466315.2024.2365648
Thomas R Fleming, Lisa V Hampson, Bharani-Dharan Bharani, Frank Bretz, Arunava Chakravartty, Thibaud Coroller, Evanthia Koukouli, Janet Wittes, Nigel Yateman, Emmanuel Zuber
Indolent cancers are characterized by long overall survival (OS) times. Therefore, powering a clinical trial to provide definitive assessment of the effects of an experimental intervention on OS in...
{"title":"Monitoring overall survival in pivotal trials in indolent cancers","authors":"Thomas R Fleming, Lisa V Hampson, Bharani-Dharan Bharani, Frank Bretz, Arunava Chakravartty, Thibaud Coroller, Evanthia Koukouli, Janet Wittes, Nigel Yateman, Emmanuel Zuber","doi":"10.1080/19466315.2024.2365648","DOIUrl":"https://doi.org/10.1080/19466315.2024.2365648","url":null,"abstract":"Indolent cancers are characterized by long overall survival (OS) times. Therefore, powering a clinical trial to provide definitive assessment of the effects of an experimental intervention on OS in...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":"26 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2024-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141507322","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 : 2024-04-30DOI: 10.1080/19466315.2024.2342817
Nicky Best, Maxine Ajimi, Beat Neuenschwander, Gaëlle Saint-Hilary, Simon Wandel
There is growing interest in Bayesian clinical trial designs with informative prior distributions, e.g. for extrapolation of adult data to pediatrics, or use of external controls. While the classic...
{"title":"Beyond the classical type I error: Bayesian metrics for Bayesian designs using informative priors","authors":"Nicky Best, Maxine Ajimi, Beat Neuenschwander, Gaëlle Saint-Hilary, Simon Wandel","doi":"10.1080/19466315.2024.2342817","DOIUrl":"https://doi.org/10.1080/19466315.2024.2342817","url":null,"abstract":"There is growing interest in Bayesian clinical trial designs with informative prior distributions, e.g. for extrapolation of adult data to pediatrics, or use of external controls. While the classic...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":"15 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2024-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140886861","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 : 2024-03-26DOI: 10.1080/19466315.2024.2334354
Keaven M. Anderson, Yujie Zhao, Nan Xiao, Joy Ge, Harlan F. Weisman
We consider several industry group sequential trials and associated issues over the last 30 years. Generally, group sequential design has provided a great deal of flexibility to overcome many chall...
{"title":"Some group sequential trials from industry over the last 30 years","authors":"Keaven M. Anderson, Yujie Zhao, Nan Xiao, Joy Ge, Harlan F. Weisman","doi":"10.1080/19466315.2024.2334354","DOIUrl":"https://doi.org/10.1080/19466315.2024.2334354","url":null,"abstract":"We consider several industry group sequential trials and associated issues over the last 30 years. Generally, group sequential design has provided a great deal of flexibility to overcome many chall...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":"73 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2024-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140311791","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 : 2024-03-25DOI: 10.1080/19466315.2024.2332650
Rong Liu, Ying Yuan, Suman Sen, Olga Marchenko, Qi Jiang, Hong Tian, Xiaoyun Li, Ruitao Lin, Cindy Lu, Heng Zhou
The conventional more-is-better dose selection paradigm is often inappropriate for novel molecularly targeted agents. In response, the US Food and Drug Administration (FDA) initiated Project Optimu...
{"title":"Design Strategy and Consideration for Oncology Dose-Optimization: An Industry Perspective","authors":"Rong Liu, Ying Yuan, Suman Sen, Olga Marchenko, Qi Jiang, Hong Tian, Xiaoyun Li, Ruitao Lin, Cindy Lu, Heng Zhou","doi":"10.1080/19466315.2024.2332650","DOIUrl":"https://doi.org/10.1080/19466315.2024.2332650","url":null,"abstract":"The conventional more-is-better dose selection paradigm is often inappropriate for novel molecularly targeted agents. In response, the US Food and Drug Administration (FDA) initiated Project Optimu...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":"143 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2024-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140311921","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 : 2024-03-20DOI: 10.1080/19466315.2024.2332675
Tuo Wang, Ying Li, Yongming Qu
The win ratio is a novel approach to analyzing prioritized composite endpoints in clinical trials. The win ratio has grown increasingly popular and has been applied in many ongoing cardiovascular c...
{"title":"Restricted time win ratio: from estimands to estimation","authors":"Tuo Wang, Ying Li, Yongming Qu","doi":"10.1080/19466315.2024.2332675","DOIUrl":"https://doi.org/10.1080/19466315.2024.2332675","url":null,"abstract":"The win ratio is a novel approach to analyzing prioritized composite endpoints in clinical trials. The win ratio has grown increasingly popular and has been applied in many ongoing cardiovascular c...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":"68 3 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2024-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140205584","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 : 2024-03-08DOI: 10.1080/19466315.2024.2327290
Peng Zhang, Weichung Joe Shih, Yong Lin, K.K. Gordon Lan, Tai Xie
The standard Brownian motion (Bm) with a linear drift is a convenient statistical structure for monitoring ongoing clinical trials in practice for more than four decades (Lan and DeMets, 1983). Und...
{"title":"Monitoring Ongoing Clinical Trials under Fractional Brownian Motion with Drift","authors":"Peng Zhang, Weichung Joe Shih, Yong Lin, K.K. Gordon Lan, Tai Xie","doi":"10.1080/19466315.2024.2327290","DOIUrl":"https://doi.org/10.1080/19466315.2024.2327290","url":null,"abstract":"The standard Brownian motion (Bm) with a linear drift is a convenient statistical structure for monitoring ongoing clinical trials in practice for more than four decades (Lan and DeMets, 1983). Und...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":"19 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2024-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140070410","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 : 2024-03-06DOI: 10.1080/19466315.2024.2327303
Zhiwei Zhang, Jixian Wang, Dong Xi
We consider the problem of estimating the causal effect of drug exposure on clinical response in a randomized dose comparison trial where each group receives a different dose. Because exposure is n...
{"title":"An Instrumental Variable Approach to Learning the Causal Exposure-Response Relationship in a Randomized Dose Comparison Trial","authors":"Zhiwei Zhang, Jixian Wang, Dong Xi","doi":"10.1080/19466315.2024.2327303","DOIUrl":"https://doi.org/10.1080/19466315.2024.2327303","url":null,"abstract":"We consider the problem of estimating the causal effect of drug exposure on clinical response in a randomized dose comparison trial where each group receives a different dose. Because exposure is n...","PeriodicalId":51280,"journal":{"name":"Statistics in Biopharmaceutical Research","volume":"5 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2024-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140044210","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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