Výsledky vyhledávání - "Margaret Gamalo"

Extent of safety database in pediatric drug development: types of assessment, analytical precision, and pathway for extrapolation through on-target effects

Autor: Margaret Gamalo, Yihua Zhao, Aijun Gao, Jingjing Ye, Junjing Lin, Eiji Eshida, YounJeong Choi, Robert Nelson

Publikováno v: Journal of Biopharmaceutical Statistics. :1-16

Pediatric patients should have access to medicines that have been appropriately evaluated for safety and efficacy. Given this goal of revised labelling, the adequacy of the pediatric clinical development plan and resulting safety database must inform

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e7ce8da3a3c5df424d057f6225e7c572
https://doi.org/10.1080/10543406.2023.2172425

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Design of paediatric trials with benefit-risk endpoints using a composite score of adverse events of interest (AEI) and win-statistics

Autor: Yodit Seifu, Shahrul Mt-Isa, Kyle Duke, Margaret Gamalo-Siebers, William Wang, Gaohong Dong, John Kolassa

Publikováno v: Journal of Biopharmaceutical Statistics. :1-12

A fundamental problem in the regulatory evaluation of a therapy is assessing whether the benefit outweighs the associated risks. This work proposes designing a trial that assesses a composite endpoint consisting of benefit and risk, hence, making the

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4faf399c10b9d36eaf936b92bc22a3c3
https://doi.org/10.1080/10543406.2022.2153202

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Strategies to facilitate adolescent access to medicines: Improving regulatory guidance

Autor: Christina Bucci-Rechtweg, Angeliki Siapkara, Kristina An Haack Bonnet, Solange Corriol Rohou, Elin Haf Davies, Martine Dehlinger Kremer, Margaret Gamalo, Carmen Moreno, Robert M Nelson, Rhian Thomas Turner

Publikováno v: Clinical Trials. 20:13-21

Background: Historically, pediatric medicines are developed after adult trials are completed, even when identical drug targets and disease similarities exist across the populations. This has resulted in significant delays in the authorization of medi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::269c4fe54475265e57b18d1fec0eb398
https://doi.org/10.1177/17407745221132302

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The win odds: statistical inference and regression

Autor: James Song, Johan Verbeeck, Bo Huang, David C. Hoaglin, Margaret Gamalo-Siebers, Yodit Seifu, Duolao Wang, Freda Cooner, Gaohong Dong

Publikováno v: Journal of Biopharmaceutical Statistics. 33:140-150

Generalized pairwise comparisons and win statistics (i.e., win ratio, win odds and net benefit) are advantageous in analyzing and interpreting a composite of multiple outcomes in clinical trials. An important limitation of these statistics is their i

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a9b959881e966e7679f0b2e5bb9e6641
https://doi.org/10.1080/10543406.2022.2089156

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Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components

Autor: Ran Liao, Sujatro Chakladar, Margaret Gamalo

Publikováno v: Pharmaceutical Statistics. 21:1342-1356

There is an increasing interest in the use of win ratio with composite time-to-event due to its flexibility in combining component endpoints. Exploring this flexibility further, one interesting question is in assessing the impact when there is a diff

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::552f26a5d9f447cf7aa21018a4d2c704
https://doi.org/10.1002/pst.2248

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Real-World Data as External Controls: Practical Experience from Notable Marketing Applications of New Therapies

Autor: Rima Izem, Joan Buenconsejo, Ruthanna Davi, Jingyu Julia Luan, LaRee Tracy, Margaret Gamalo

Publikováno v: Therapeutic Innovation & Regulatory Science. 56:704-716

Real-world data (RWD) can contextualize findings from single-arm trials when randomized comparative trials are unethical or unfeasible. Findings from single-arm trials alone are difficult to interpret and a comparison, when feasible and meaningful, t

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::df24aa0758fd7bb1588ea74060982083
https://doi.org/10.1007/s43441-022-00413-0

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Power priors with entropy balancing weights in data augmentation of partially controlled randomized trials

Autor: Guanglei Yu, Yuanyuan Bian, Margaret Gamalo

Publikováno v: Journal of Biopharmaceutical Statistics. 32:4-20

In pediatric or orphan diseases, there are many instances where it is unfeasible to conduct randomized and controlled clinical trials. This is due in part to the difficulty of enrolling a sufficient number of patients over a reasonable time period to

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0504f114bd917ec3e68c52937bb60ccf
https://doi.org/10.1080/10543406.2021.2021226

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