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定量证据综合方法在评估临床和经济决策中治疗序列有效性的应用:简化假设的回顾与分类。

Quantitative Evidence Synthesis Methods for the Assessment of the Effectiveness of Treatment Sequences for Clinical and Economic Decision Making: A Review and Taxonomy of Simplifying Assumptions.

机构信息

North Wales Centre for Primary Care Research, College of Health and Behavioural Sciences, Bangor University, CAMBRIAN 2, Wrexham Technology Park, Wrexham, LL13 7YP, UK.

Centre for Health Economics and Medicines Evaluation, Bangor University, Bangor, UK.

出版信息

Pharmacoeconomics. 2021 Jan;39(1):25-61. doi: 10.1007/s40273-020-00980-w. Epub 2020 Nov 26.


DOI:10.1007/s40273-020-00980-w
PMID:33242191
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7790782/
Abstract

Sequential use of alternative treatments for chronic conditions represents a complex intervention pathway; previous treatment and patient characteristics affect both the choice and effectiveness of subsequent treatments. This paper critically explores the methods for quantitative evidence synthesis of the effectiveness of sequential treatment options within a health technology assessment (HTA) or similar process. It covers methods for developing summary estimates of clinical effectiveness or the clinical inputs for the cost-effectiveness assessment and can encompass any disease condition. A comprehensive review of current approaches is presented, which considers meta-analytic methods for assessing the clinical effectiveness of treatment sequences and decision-analytic modelling approaches used to evaluate the effectiveness of treatment sequences. Estimating the effectiveness of a sequence of treatments is not straightforward or trivial and is severely hampered by the limitations of the evidence base. Randomised controlled trials (RCTs) of sequences were often absent or very limited. In the absence of sufficient RCTs of whole sequences, there is no single best way to evaluate treatment sequences; however, some approaches could be re-used or adapted, sharing ideas across different disease conditions. Each has advantages and disadvantages, and is influenced by the evidence available, extent of treatment sequences (number of treatment lines or permutations), and complexity of the decision problem. Due to the scarcity of data, modelling studies applied simplifying assumptions to data on discrete treatments. A taxonomy for all possible assumptions was developed, providing a unique resource to aid the critique of existing decision-analytic models.

摘要

序贯治疗慢性疾病是一种复杂的干预途径;先前的治疗和患者特征会影响后续治疗的选择和效果。本文批判性地探讨了在健康技术评估(HTA)或类似过程中对序贯治疗方案有效性进行定量证据综合的方法。它涵盖了用于汇总临床有效性估计值或成本效益评估的临床投入的方法,并且可以包含任何疾病状况。本文全面回顾了当前的方法,包括用于评估治疗序列临床有效性的荟萃分析方法和用于评估治疗序列有效性的决策分析建模方法。估计一系列治疗方法的有效性并不简单或微不足道,并且受到证据基础的限制严重阻碍。序列的随机对照试验(RCT)往往不存在或非常有限。在缺乏整个序列的充分 RCT 的情况下,评估治疗序列没有一种最佳方法;然而,可以重复使用或调整某些方法,在不同疾病状况之间共享想法。每种方法都有其优缺点,并且受到可用证据、治疗序列的程度(治疗线或排列的数量)以及决策问题的复杂性的影响。由于数据稀缺,建模研究对离散治疗的数据应用了简化假设。开发了一个适用于所有可能假设的分类法,为现有的决策分析模型提供了一个独特的资源,以帮助进行批判性评估。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f5d1/7790782/00314de2f253/40273_2020_980_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f5d1/7790782/00314de2f253/40273_2020_980_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f5d1/7790782/00314de2f253/40273_2020_980_Fig1_HTML.jpg

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Development of a Decision Model to Estimate the Outcomes of Treatment Sequences in Advanced Melanoma.

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本文引用的文献

[1]
Economic Evaluation of Sequences of Biological Treatments for Patients With Moderate-to-Severe Rheumatoid Arthritis and Inadequate Response or Intolerance to Methotrexate in France.

Value Health. 2020-2-13

[2]
Cost-Effectiveness Analysis of Sequential Biologic Therapy with Ixekizumab Versus Secukinumab in the Treatment of Active Psoriatic Arthritis with Concomitant Moderate-to-Severe Psoriasis in the UK.

Pharmacoecon Open. 2020-12

[3]
Systematic Literature Review of Economic Evaluations of Biological Treatment Sequences for Patients with Moderate to Severe Rheumatoid Arthritis Previously Treated with Disease-Modifying Anti-rheumatic Drugs.

Pharmacoeconomics. 2020-5

[4]
Determining the Baseline Strategy in a Cost-Effectiveness Analysis with Treatment Sequences.

Appl Health Econ Health Policy. 2020-2

[5]
Treatment sequences for advanced renal cell carcinoma: A health economic assessment.

PLoS One. 2019-8-29

[6]
Improving Decision-Making Processes in Health: Is It Time for (Disease-Specific) Reference Models?

Appl Health Econ Health Policy. 2020-2

[7]
Development of the ACTIVE framework to describe stakeholder involvement in systematic reviews.

J Health Serv Res Policy. 2019-10

[8]
Practical Guidance for Involving Stakeholders in Health Research.

J Gen Intern Med. 2018-12-18

[9]
Stakeholder involvement in systematic reviews: a scoping review.

Syst Rev. 2018-11-24

[10]
Working with Patients and Members of the Public: Informing Health Economics in Child Health Research.

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