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计算生存数据抽样的预期净收益:教程与案例研究。

Calculating the Expected Net Benefit of Sampling for Survival Data: A Tutorial and Case Study.

机构信息

Department of Health Management and Health Economics, University of Oslo, Oslo, Norway.

Clinical Trial Unit, Oslo University Hospital, Oslo, Norway.

出版信息

Med Decis Making. 2024 Oct;44(7):719-741. doi: 10.1177/0272989X241279459. Epub 2024 Sep 20.

DOI:10.1177/0272989X241279459
PMID:39305058
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11490075/
Abstract

The net value of reducing decision uncertainty by collecting additional data is quantified by the expected net benefit of sampling (ENBS). This tutorial presents a general-purpose algorithm for computing the ENBS for collecting survival data along with a step-by-step implementation in R.The algorithm is based on recently published methods for simulating survival data and computing expected value of sample information that do not rely on the survival data to follow any particular parametric distribution and that can take into account any arbitrary censoring process.We demonstrate in a case study based on a previous cancer technology appraisal that ENBS calculations are useful not only for designing new studies but also for optimizing reimbursement decisions for new health technologies based on immature evidence from ongoing trials.

摘要

通过收集额外数据来减少决策不确定性的净价值是通过抽样的预期净收益(ENBS)来量化的。本教程介绍了一种用于计算生存数据采集的 ENBS 的通用算法,并在 R 中逐步实现了该算法。该算法基于最近发表的用于模拟生存数据和计算样本信息期望价值的方法,这些方法不依赖于生存数据遵循任何特定的参数分布,并且可以考虑到任何任意的删失过程。我们在一个基于先前癌症技术评估的案例研究中证明,ENBS 计算不仅对于设计新研究有用,而且对于基于正在进行的试验中的不成熟证据对新的卫生技术进行报销决策优化也有用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0dbf/11490075/507e5a1de352/10.1177_0272989X241279459-fig2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0dbf/11490075/98e6eb59ead3/10.1177_0272989X241279459-fig1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0dbf/11490075/507e5a1de352/10.1177_0272989X241279459-fig2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0dbf/11490075/98e6eb59ead3/10.1177_0272989X241279459-fig1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0dbf/11490075/507e5a1de352/10.1177_0272989X241279459-fig2.jpg

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本文引用的文献

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The impact of different types of NHS expenditure on health: Marginal cost per QALY estimates for England for 2016/17.不同类型的国民保健制度支出对健康的影响:2016/17 年英格兰每质量调整生命年的边际成本估算。
Health Policy. 2023 Jun;132:104800. doi: 10.1016/j.healthpol.2023.104800. Epub 2023 Mar 21.
2
General-Purpose Methods for Simulating Survival Data for Expected Value of Sample Information Calculations.通用模拟生存数据的方法,用于计算样本信息的期望值。
Med Decis Making. 2023 Jul;43(5):595-609. doi: 10.1177/0272989X231162069. Epub 2023 Mar 27.
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Extrapolation beyond the end of trials to estimate long term survival and cost effectiveness.
试验结束后进行外推以估计长期生存率和成本效益。
BMJ Med. 2022 Mar 10;1(1):e000094. doi: 10.1136/bmjmed-2021-000094. eCollection 2022.
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Projecting overall survival in health-economic models: uncertainty and maturity of data.在健康经济模型中预测总生存期:数据的不确定性与成熟度
Curr Med Res Opin. 2023 Mar;39(3):367-374. doi: 10.1080/03007995.2023.2167442. Epub 2023 Jan 23.
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Exploring uncertainty and use of real-world data in the National Institute for Health and Care Excellence single technology appraisals of targeted cancer therapy.探索英国国家卫生与保健优化研究所靶向癌症治疗单一技术评估中不确定性和真实世界数据的应用。
BMC Cancer. 2022 Dec 5;22(1):1268. doi: 10.1186/s12885-022-10350-8.
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"Don't Think Twice, It's All Right": Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England.“别再犹豫,一切都好”:利用额外数据降低英国通过管理准入协议提供的肿瘤药物的不确定性
Pharmacoecon Open. 2023 Jan;7(1):77-91. doi: 10.1007/s41669-022-00369-9. Epub 2022 Sep 20.
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