使用贝叶斯动态借用来最大限度地利用现有数据:一个案例研究
Dawn Edwards1, N Best2, J Crawford2
1GSK, 980 Great West Road, Brentford, TW8 9GS, Middlesex, UK. dawn.m.webber@gsk.com.
Therapeutic innovation & regulatory science
|November 1, 2023
概括
贝叶斯动态借用 (BDB) 通过整合先前的试验数据来增强临床药物开发. 这种强有力的方法加强了证据,提高了试验效率,帮助监管提交.
科学领域:
- 临床药物开发 临床药物开发
- 生物统计学 生物统计学
- 监管科学 监管科学
背景情况:
- 贝叶斯动态借款 (BDB) 在临床试验中越来越多地被采用.
- BDB提供了一种数学上严格的方法,用于将现有数据集成到新的试验中.
- 对BDB的监管接受正在不断发展,并且在各机构之间存在差异.
研究的目的:
- 用BDB与外部数据描述一个新的随机临床试验的设计.
- 讨论药物开发计划中数据再利用和BDB的关键考虑因素.
- 介绍一篇关于BDB在中国药物注册的案例研究.
主要方法:
- 设计一个随机临床试验,通过BDB. 结合外部数据.
- 评估外部数据的适当性,选择和权重,以获得贷款.
- 评估新研究中治疗效益的成功证明.
主要成果:
- 这篇论文详细介绍了BDB在设计临床试验中的应用.
- 它解决了有效借用和集成数据的关键因素.
- 一个案例研究说明了BDB在支持药物注册方面的作用.
结论:
- BDB为高效和基于证据的临床药物开发提供了一个强大的框架.
- 仔细考虑数据选择,权重和监管准则对于BDB实施至关重要.
- 本案例研究展示了BDB在监管环境中的实际应用和评估.
相关概念视频
Bootstrapping
612
The term "bootstrap" originated in the 19th century as a metaphor for self-improvement or achieving something independently, without external assistance. This concept extends to statistical bootstrapping, a self-contained method for estimating population parameters through resampling, even though it can be computationally intensive. Developed by the American statistician Dr. Bradley Efron in 1979, bootstrapping provides a robust way to perform inference when the original sample size is...
612
Model Approaches for Pharmacokinetic Data: Distributed Parameter Models
74
Pharmacokinetic models are mathematical constructs that represent and predict the time course of drug concentrations in the body, providing meaningful pharmacokinetic parameters. These models are categorized into compartment, physiological, and distributed parameter models.
The distributed parameter models are specifically designed to account for variations and differences in some drug classes. This model is particularly useful for assessing regional concentrations of anticancer or...
The distributed parameter models are specifically designed to account for variations and differences in some drug classes. This model is particularly useful for assessing regional concentrations of anticancer or...
74
Distributions to Estimate Population Parameter
4.1K
The accurate values of population parameters such as population proportion, population mean, and population standard deviation (or variance) are usually unknown. These are fixed values that can only be estimated from the data collected from the samples. The estimates of each of these parameters are sample proportion, the sample mean, and sample standard deviation (or variance). To obtain the values of these sample statistics, data are required that have particular distribution and central...
4.1K
Mechanistic Models: Compartment Models in Individual and Population Analysis
44
Mechanistic models are utilized in individual analysis using single-source data, but imperfections arise due to data collection errors, preventing perfect prediction of observed data. The mathematical equation involves known values (Xi), observed concentrations (Ci), measurement errors (εi), model parameters (ϕj), and the related function (ƒi) for i number of values. Different least-squares metrics quantify differences between predicted and observed values. The ordinary least...
44
Cluster Sampling Method
11.9K
Appropriate sampling methods ensure that samples are drawn without bias and accurately represent the population. Because measuring the entire population in a study is not practical, researchers use samples to represent the population of interest.
To choose a cluster sample, divide the population into clusters (groups) and then randomly select some of the clusters. All the members from these clusters are in the cluster sample. For example, if you randomly sample four departments from your...
To choose a cluster sample, divide the population into clusters (groups) and then randomly select some of the clusters. All the members from these clusters are in the cluster sample. For example, if you randomly sample four departments from your...
11.9K
Biostatistics: Overview
254
Biostatistics plays a crucial role in understanding and analyzing data in healthcare and biology. Biostatisticians conduct experiments, gather evidence, and draw meaningful conclusions using statistical methods and techniques. Different variables form the foundation of biostatistical analysis, allowing researchers to understand and interpret data effectively. These variables are classified into different types, each serving a specific purpose in statistical analysis.
Discrete variables are...
Discrete variables are...
254


