Analytical Methods for Comparing Uncontrolled Trials With External Controls From Real-World Data: A Systematic Literature Review and Comparison With European Regulatory and Health Technology Assessment Practice.

IF 4.9 2区 医学 Q1 ECONOMICS Value in Health Pub Date : 2024-09-04 DOI:10.1016/j.jval.2024.08.002
Milou A Hogervorst, Kanaka V Soman, Helga Gardarsdottir, Wim G Goettsch, Lourens T Bloem
{"title":"Analytical Methods for Comparing Uncontrolled Trials With External Controls From Real-World Data: A Systematic Literature Review and Comparison With European Regulatory and Health Technology Assessment Practice.","authors":"Milou A Hogervorst, Kanaka V Soman, Helga Gardarsdottir, Wim G Goettsch, Lourens T Bloem","doi":"10.1016/j.jval.2024.08.002","DOIUrl":null,"url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials with external controls from individual patient data real-world data (IPD-RWD) and to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.</p><p><strong>Methods: </strong>A systematic literature review (until March 1, 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively with methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and 4 European HTA organizations (2015-2023).</p><p><strong>Results: </strong>Thirty-four identified scientific articles described analytical methods for comparing uncontrolled trial data with IPD-RWD-based external controls. The various methods covered controlling for confounding and/or dependent censoring, correction for missing data, and analytical comparative modeling methods. Seven guidelines also focused on research design, RWD quality, and transparency aspects, and 4 of those recommended analytical methods for comparisons with IPD-RWD. The methods discussed in regulatory (n = 15) and HTA (n = 35) assessment reports were often based on aggregate data and lacked transparency owing to the few details provided.</p><p><strong>Conclusions: </strong>Literature and guidelines suggest a methodological approach to comparing uncontrolled trials with external controls from IPD-RWD similar to target trial emulation, using state-of-the-art methods. External controls supporting regulatory and HTA decision making were rarely in line with this approach. Twelve recommendations are proposed to improve the quality and acceptability of these methods.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9000,"publicationDate":"2024-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Value in Health","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.jval.2024.08.002","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"ECONOMICS","Score":null,"Total":0}
引用次数: 0

Abstract

Objectives: This study aimed to provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials with external controls from individual patient data real-world data (IPD-RWD) and to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.

Methods: A systematic literature review (until March 1, 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively with methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and 4 European HTA organizations (2015-2023).

Results: Thirty-four identified scientific articles described analytical methods for comparing uncontrolled trial data with IPD-RWD-based external controls. The various methods covered controlling for confounding and/or dependent censoring, correction for missing data, and analytical comparative modeling methods. Seven guidelines also focused on research design, RWD quality, and transparency aspects, and 4 of those recommended analytical methods for comparisons with IPD-RWD. The methods discussed in regulatory (n = 15) and HTA (n = 35) assessment reports were often based on aggregate data and lacked transparency owing to the few details provided.

Conclusions: Literature and guidelines suggest a methodological approach to comparing uncontrolled trials with external controls from IPD-RWD similar to target trial emulation, using state-of-the-art methods. External controls supporting regulatory and HTA decision making were rarely in line with this approach. Twelve recommendations are proposed to improve the quality and acceptability of these methods.

查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
从真实世界数据中比较无对照试验与外部对照的分析方法:系统性文献综述及与欧洲监管和健康技术评估实践的比较。
目的:概述科学文献中利用患者个人真实世界数据(IPD-RWD)将无对照药物试验与外部对照进行比较的分析方法。此外,还将这些方法与欧洲监管机构和卫生技术评估(HTA)机构在指南中提出的建议及其在评估报告中描述的评价进行比较:方法:在PubMed和Connected Papers上进行了一次系统的文献综述(截至2023年3月1日),以确定将IPD-RWD中的非对照试验与外部对照进行比较的分析方法。将这些方法与方法指南中推荐的方法以及欧洲药品管理局(2015-2020 年)和四个欧洲 HTA 组织(2015-2023 年)的评估报告中出现的方法进行了描述性比较:34 篇已确定的科学文章介绍了将非对照试验数据与基于 IPD-RWD 的外部对照进行比较的分析方法。各种方法涵盖了混杂控制和/或从属删减、缺失数据校正以及分析比较建模方法。七份指南还侧重于研究设计、RWD 质量和透明度方面,其中四份指南推荐了与 IPD-RWD 进行比较的分析方法。监管报告(n=15)和 HTA 评估报告(n=35)中讨论的方法通常基于综合数据,并且由于提供的细节较少而缺乏透明度:文献和指南建议采用最先进的方法,将非对照试验与IPD-RWD的外部对照进行比较,类似于目标试验模拟。支持监管和 HTA 决策的外部对照很少符合这种方法。为提高这些方法的质量和可接受性,我们提出了 12 项建议。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 去求助
来源期刊
Value in Health
Value in Health 医学-卫生保健
CiteScore
6.90
自引率
6.70%
发文量
3064
审稿时长
3-8 weeks
期刊介绍: Value in Health contains original research articles for pharmacoeconomics, health economics, and outcomes research (clinical, economic, and patient-reported outcomes/preference-based research), as well as conceptual and health policy articles that provide valuable information for health care decision-makers as well as the research community. As the official journal of ISPOR, Value in Health provides a forum for researchers, as well as health care decision-makers to translate outcomes research into health care decisions.
期刊最新文献
Value Attribution for Combination Treatments: Two Potential Solutions for an Insoluble Problem. Evaluating the health and economic impacts of return-to-work interventions: a modelling study. Exploring social preferences for health and wellbeing across the digital divide. A qualitative investigation based on tasks taken from an online discrete choice experiment. Quantifying low-value care in Germany: An observational study using statutory health insurance data from 2018 to 2021. Indirect Costs of Alzheimer's Disease: Unpaid Caregiver Burden and Patient Productivity Loss.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1