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
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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.

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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。
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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.
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