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Statistical modelling of skewed and semi-continuous data in randomised controlled trials (RCTs): An application of two-part models in RCTs of chronic

随机对照试验（RCT）中偏斜和半连续数据的统计模型：两部分模型在慢性病随机对照试验中的应用

基本信息

批准号：
1939414
负责人：
金额：
--
依托单位：
University of Warwick
依托单位国家：
英国
项目类别：
Studentship
财政年份：
2017
资助国家：
英国
起止时间：
2017 至无数据
项目状态：
已结题

来源：
https://gtr.ukri.org/projects?ref=studentship-1939414
关键词：
Statistical modelling skewed semi continuous

项目摘要

Chronic pain is pain that persists beyond the normal time of healing, and can occur even when no obvious cause can be found with estimates suggesting 14 million people live with chronic pain in the UK and the global burden of pain related conditions increasing with an increasing elderly population. The use of inappropriate analytical techniques seriously affects the findings of RCTs and will at best the precision of effect estimates will be reduced; at worst results might be grossly misleading. Two-part models are a flexible modelling framework often used in econometrics for data with a large number of zeroes and non-zero values that are highly skewed or non-normal. The goal of the research will be to develop and assess the application of two-part models in the context of outcomes that measure pain, and pain related disability, in musculoskeletal RCTs.Performance of two-part models will be explored by fitting these models to simulated data to fully assess the bias and accuracy of estimates of treatment effects and their performance will be assessed by generating simulated datasets. The increased flexibility of two-part models is expected to lead to better fitting models for pain data and thereby, most importantly, more reliable inference.

慢性疼痛是持续超过正常愈合时间的疼痛，即使没有明显的原因也可能发生，估计英国有1400万人患有慢性疼痛，全球疼痛相关疾病的负担随着老年人口的增加而增加。使用不适当的分析技术会严重影响随机对照试验的结果，最好的情况下会降低效应估计的精度;最坏的情况下，结果可能会严重误导。两部分模型是一种灵活的建模框架，经常用于计量经济学中，用于具有大量零和非零值的数据，这些数据高度偏斜或非正态。本研究的目标是开发和评估两部分模型在肌肉骨骼RCT中测量疼痛和疼痛相关残疾的结局背景下的应用，通过将这些模型与模拟数据拟合以充分评估治疗效果估计的偏倚和准确性来探索两部分模型的性能，并通过生成模拟数据集来评估其性能。两部分模型的灵活性增加，预计将导致更好的拟合模型的疼痛数据，从而，最重要的是，更可靠的推理。