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癌症研究中理解真实世界研究方法的实用途径:视频播客

A Practical Approach to Understanding Real-World Study Methodology in Cancer Research: A Vodcast.

作者信息

Brufsky Adam, Cheung Winson, Ryan Joanne C

机构信息

UPMC Hillman Cancer Center, University of Pittsburgh Medical Center, Pittsburgh, PA, USA.

Department of Oncology, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.

出版信息

Oncol Ther. 2025 Sep 11. doi: 10.1007/s40487-025-00366-y.

DOI:10.1007/s40487-025-00366-y
PMID:40935907
Abstract

Real-world studies have become more common in clinical literature in recent years, but many clinicians remain unfamiliar with real-world study design and statistical approaches. This vodcast intends to be a practical guide for clinicians by clarifying aspects of real-world study methodology. As both practicing oncologists and researchers with extensive real-world data experience, the hosts discuss types of study designs and real-world data source considerations. An overview of statistical techniques for mitigating treatment-selection bias is also provided, including propensity score matching, inverse probability of treatment weighting, and multivariable analysis. By combining high-quality data sources, careful sample size considerations, and rigorous statistical techniques, real-world studies can offer valuable insights into therapeutic effectiveness in routine clinical practice that supplement learnings from randomized clinical trials. This vodcast is designed to equip clinicians with the knowledge to critically evaluate real-world evidence and potentially apply it to their practice.Vodcast and infographic available for this article. Vodcast (MP4 1207725 KB) INFOGRAPHIC.

摘要

近年来,真实世界研究在临床文献中变得越来越普遍,但许多临床医生仍然不熟悉真实世界研究设计和统计方法。本视频播客旨在通过阐明真实世界研究方法的各个方面,为临床医生提供一份实用指南。作为既有丰富真实世界数据经验的执业肿瘤学家又身为研究人员,主持人讨论了研究设计类型和真实世界数据源考量因素。还提供了减轻治疗选择偏倚的统计技术概述,包括倾向得分匹配、治疗权重逆概率和多变量分析。通过结合高质量数据源、仔细考虑样本量以及采用严格的统计技术,真实世界研究可以为常规临床实践中的治疗效果提供有价值的见解,以补充从随机临床试验中获得的知识。本视频播客旨在让临床医生具备批判性评估真实世界证据并可能将其应用于实践的知识。本文提供视频播客和信息图表。视频播客(MP4 1207725 KB)信息图表

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本文引用的文献

1
Real-World Evidence: A Primer.真实世界证据:基础篇。
Pharmaceut Med. 2023 Jan;37(1):25-36. doi: 10.1007/s40290-022-00456-6. Epub 2023 Jan 5.
2
Observational designs for real-world evidence studies.真实世界证据研究的观察性设计。
Perspect Clin Res. 2022 Jan-Mar;13(1):12-16. doi: 10.4103/picr.picr_217_21. Epub 2022 Jan 6.
3
Assessing and Interpreting Real-World Evidence Studies: Introductory Points for New Reviewers.评估和解读真实世界证据研究:新手评审员入门要点。
Clin Pharmacol Ther. 2022 Jan;111(1):145-149. doi: 10.1002/cpt.2398. Epub 2021 Sep 1.
4
Using Propensity Scores for Causal Inference: Pitfalls and Tips.使用倾向得分进行因果推断:陷阱与技巧。
J Epidemiol. 2021 Aug 5;31(8):457-463. doi: 10.2188/jea.JE20210145. Epub 2021 Jun 12.
5
Comparisons of Real-World Time-to-Event End Points in Oncology Research.肿瘤学研究中真实世界事件发生时间终点的比较。
JCO Clin Cancer Inform. 2021 Jan;5:45-46. doi: 10.1200/CCI.20.00125.
6
Real-world evidence: the devil is in the detail.真实世界证据:细节决定成败。
Diabetologia. 2020 Sep;63(9):1694-1705. doi: 10.1007/s00125-020-05217-1. Epub 2020 Jul 15.
7
Propensity score matching and inverse probability of treatment weighting to address confounding by indication in comparative effectiveness research of oral anticoagulants.倾向评分匹配和治疗反概率加权法解决口服抗凝药物比较有效性研究中的指示性混杂
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8
Moving towards best practice when using inverse probability of treatment weighting (IPTW) using the propensity score to estimate causal treatment effects in observational studies.在观察性研究中,利用倾向得分采用治疗权重的逆概率(IPTW)估计因果治疗效果时,朝着最佳实践迈进。
Stat Med. 2015 Dec 10;34(28):3661-79. doi: 10.1002/sim.6607. Epub 2015 Aug 3.
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The use of propensity score methods with survival or time-to-event outcomes: reporting measures of effect similar to those used in randomized experiments.倾向评分方法在生存或事件发生时间结局中的应用:报告与随机试验中使用的效应测量指标相似的指标。
Stat Med. 2014 Mar 30;33(7):1242-58. doi: 10.1002/sim.5984. Epub 2013 Sep 30.