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相关论文: Multi-indication evidence synthesis in oncology he…

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A growing number of oncology treatments, such as bevacizumab, are used across multiple indications. However, in health technology assessment (HTA), their clinical and cost-effectiveness are typically appraised within a single target…

Background: Evidence maps have been used in healthcare to understand existing evidence and to support decision-making. In oncology they have been used to summarise evidence within a disease area but have not been used to compare evidence…

Progression free survival (PFS) and tumour response (TR) have been investigated as surrogate endpoints for overall survival (OS) in advanced colorectal cancer (aCRC), however their validity has been shown to be suboptimal. In recent years,…

应用统计 · 统计学 2018-09-11 Eleni G. Elia , Nicolas Städler , Oriana Ciani , Rod S. Taylor , Sylwia Bujkiewicz

We introduce a statistical procedure that integrates survival data from multiple biomedical studies, to improve the accuracy of predictions of survival or other events, based on individual clinical and genomic profiles, compared to models…

应用统计 · 统计学 2020-07-20 Steffen Ventz , Rahul Mazumder , Lorenzo Trippa

In health technology assessment, decisions are based on complex cost-effectiveness models which, to be implemented, require numerous input parameters. When some of relevant estimates are not available the model may have to be simplified.…

应用统计 · 统计学 2019-01-23 Sze Huey Tan , Keith R Abrams , Sylwia Bujkiewicz

In meta-analytic modeling, the functional relationship between a primary and surrogate endpoint is estimated using summary data from a set of completed clinical trials. Parameters in the meta-analytic model are used to assess the quality of…

统计方法学 · 统计学 2025-08-07 James P. Long , Abhishikta Roy , Ehsan Irajizad , Kim-Anh Do , Yu Shen

Surrogate endpoints play an important role in drug development when they can be used to measure treatment effect early compared to the final clinical outcome and to predict clinical benefit or harm. Such endpoints are assessed for their…

Purpose: PFS is often used as a surrogate endpoint for OS in metastatic breast cancer studies. We have evaluated the association of treatment effect on PFS with significant HR$_{OS}$ (and how this association is affected by other factors)…

应用统计 · 统计学 2020-06-30 Madan G. Kundu , Suddhasatta Acharyya

Surrogate endpoint (SE) for overall survival in cancer patients is essential to improving the efficiency of oncology drug development. In practice, we may discover a new patient level association with survival, based on one or more clinical…

应用统计 · 统计学 2022-11-08 Wei Zou

Data from observational studies (OSs) is widely available and readily obtainable yet frequently contains confounding biases. On the other hand, data derived from randomized controlled trials (RCTs) helps to reduce these biases; however, it…

统计方法学 · 统计学 2024-10-30 Dong Yao , Caizhi Tang , Qing Cui , Longfei Li

Heterogeneous treatment effects (HTEs) are commonly identified during randomized controlled trials (RCTs). Identifying subgroups of patients with similar treatment effects is of high interest in clinical research to advance precision…

机器学习 · 计算机科学 2022-12-06 Peniel N. Argaw , Elizabeth Healey , Isaac S. Kohane

The development of oncology drugs progresses through multiple phases, where after each phase a decision is made about whether to move a molecule forward. Early phase efficacy decisions are often made on the basis of single arm studies based…

应用统计 · 统计学 2023-04-17 Ulrich Beyer , David Dejardin , Matthias Meller , Kaspar Rufibach , Hans Ulrich Burger

Objective: Traditionally validation of surrogate endpoints has been carried out using RCT data. However, RCT data may be too limited to validate surrogate endpoints. In this paper, we sought to improve validation of surrogate endpoints with…

应用统计 · 统计学 2021-12-17 Lorna Wheaton , Anastasios Papanikos , Anne Thomas , Sylwia Bujkiewicz

The primary benefit of identifying a valid surrogate marker is the ability to use it in a future trial to test for a treatment effect with shorter follow-up time or less cost. However, previous work has demonstrated potential heterogeneity…

统计方法学 · 统计学 2022-09-20 Layla Parast , Tianxi Cai , Lu Tian

Surrogate endpoints are very important in regulatory decision-making in healthcare, in particular if they can be measured early compared to the long-term final clinical outcome and act as good predictors of clinical benefit. Bivariate…

统计方法学 · 统计学 2023-07-06 Sylwia Bujkiewicz , Dan Jackson , John R Thompson , Rebecca Turner , Keith R Abrams , Ian R White

Evaluating treatment effects is critical in clinical trials but sometimes involves lengthy, invasive, or costly follow-up procedures. In these cases, surrogate markers, which provide intermediate measures of the long-term treatment effect,…

统计方法学 · 统计学 2026-03-24 Sarah C. Lotspeich , P. D. Anh. Nguyen , Layla Parast

Subgroup analyses are common in epidemiologic and clinical research. Unfortunately, restriction to subgroup members to test for heterogeneity can yield imprecise effect estimates. If the true effect differs between members and non-members…

The Galleri (R) (GRAIL) multi-cancer early detection test measures circulating tumour DNA (ctDNA) to predict the presence of more than 50 different cancers, from a blood test. If sensitivity of the test to detect early-stage cancers is…

统计方法学 · 统计学 2025-05-01 Sofia Dias , Yiwen Liu , Stephen Palmer , Marta O Soares

Understanding whether and how treatment effects vary across subgroups is crucial to inform clinical practice and recommendations. Accordingly, the assessment of heterogeneous treatment effects (HTE) based on pre-specified potential effect…

统计方法学 · 统计学 2023-12-04 Bryan S. Blette , Scott D. Halpern , Fan Li , Michael O. Harhay

While randomized controlled trials (RCTs) are critical for establishing the efficacy of new therapies, there are limitations regarding what comparisons can be made directly from trial data. RCTs are limited to a small number of comparator…

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