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利用真实世界数据进行药品卫生技术评估:优势、难点及实用推进策略。

Using Real-World Data in the Health Technology Assessment of Pharmaceuticals: Strengths, Difficulties, and a Pragmatic Way Forward.

机构信息

Lumanity Inc, Sheffield, England, UK.

Lumanity Inc, Sheffield, England, UK.

出版信息

Value Health. 2023 Apr;26(4S):11-19. doi: 10.1016/j.jval.2023.01.010. Epub 2023 Jan 24.


DOI:10.1016/j.jval.2023.01.010
PMID:36706952
Abstract

In the past decade, there have been increasing calls for greater use of real-world evidence (RWE) and data (RWD), with the explicit goal of enabling faster provision of effective medicines to patients in need. The push for decision makers to accept RWE is especially noticeable in the pursuit of regulatory approval, but RWE, particularly when used to estimate the relative effectiveness of interventions, is not always readily accepted by agencies responsible for reimbursement and pricing of new pharmaceuticals and, to a varying degree, is not accepted across jurisdictions. This lack of trust hampers the use of RWE despite a very large and growing literature base on the principles of how RWE should be used. In this article, we suggest an important part of the explanation of why this situation has arisen and make suggestions for its alleviation. Given that problems commonly arise that are particular to the question being asked and the data sources being used, general guidance on the principles of how to use RWD cannot cover all eventualities. Therefore, we are suggesting the creation of an archive, or repository, to record uses of RWD in support of decisions by funding bodies or their advisors. This article introduces a proposed, structured classification of decision types using RWE, around which evidence can be assembled in a curated source (RWD/RWE taxonomy) and thus facilitate judgments on when evidence is "good enough." This article is part of a series in a special issue of this journal that looks at the barriers to optimal use of RWE in health technology assessment and how to overcome them. We begin significantly to populate our "taxonomy" with examples in an accompanying article. We also propose recommendations for international standards of evaluating the acceptability of RWD governance practices.

摘要

在过去的十年中,人们越来越呼吁更多地使用真实世界证据(RWE)和数据(RWD),其明确目标是使有需要的患者能够更快地获得有效的药物。决策者尤其希望接受 RWE,这在追求监管批准方面尤为明显,但 RWE(尤其是用于估计干预措施的相对有效性时)并不总是被负责新药品报销和定价的机构所接受,而且在不同程度上也不被所有司法管辖区所接受。尽管关于如何使用 RWE 的原则已经有大量且不断增长的文献基础,但由于缺乏信任,RWE 的使用受到了阻碍。在本文中,我们提出了造成这种情况的一个重要原因,并提出了缓解这种情况的建议。鉴于通常会出现特定于所提出问题和所使用数据源的问题,关于如何使用 RWD 的一般指导原则无法涵盖所有情况。因此,我们建议创建一个档案库或存储库,以记录 RWD 在支持资助机构或其顾问做出决策方面的使用情况。本文提出了一种使用 RWE 进行决策的分类建议,围绕该分类建议可以在经过策划的来源(RWD/RWE 分类法)中收集证据,从而便于判断证据是否“足够好”。本文是该期刊特刊中一系列文章的一部分,这些文章探讨了在健康技术评估中充分利用 RWE 的障碍以及如何克服这些障碍。我们在一篇相关文章中开始显著地用实例来填充我们的“分类法”。我们还提出了评估 RWD 治理实践可接受性的国际标准建议。

相似文献

[1]
Using Real-World Data in the Health Technology Assessment of Pharmaceuticals: Strengths, Difficulties, and a Pragmatic Way Forward.

Value Health. 2023-4

[2]
Can we use existing guidance to support the development of robust real-world evidence for health technology assessment/payer decision-making?

Int J Technol Assess Health Care. 2022-11-2

[3]
Real-world data for health technology assessment for reimbursement decisions in Asia: current landscape and a way forward.

Int J Technol Assess Health Care. 2020-10

[4]
Data Governance for Real-World Data Management: A Proposal for a Checklist to Support Decision Making.

Value Health. 2023-4

[5]
What, Where, and How to Collect Real-World Data and Generate Real-World Evidence to Support Drug Reimbursement Decision-Making in Asia: A reflection Into the Past and A Way Forward.

Int J Health Policy Manag. 2023

[6]
Real-world data: a comprehensive literature review on the barriers, challenges, and opportunities associated with their inclusion in the health technology assessment process.

J Pharm Pharm Sci. 2024

[7]
Improving transparency to build trust in real-world secondary data studies for hypothesis testing-Why, what, and how: recommendations and a road map from the real-world evidence transparency initiative.

Pharmacoepidemiol Drug Saf. 2020-11

[8]
Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing-Why, What, and How: Recommendations and a Road Map from the Real-World Evidence Transparency Initiative.

Value Health. 2020-9

[9]
The maze of real-world evidence frameworks: from a desert to a jungle! An environmental scan and comparison across regulatory and health technology assessment agencies.

J Comp Eff Res. 2024-9

[10]
Real-world evidence: perspectives on challenges, value, and alignment of regulatory and national health technology assessment data collection requirements.

Int J Technol Assess Health Care. 2021-2-24

引用本文的文献

[1]
APPRAISE: A Tool for Appraising Potential for Bias in Real-world Evidence Studies on Medication Effectiveness or Safety.

Value Health. 2025-8-5

[2]
Core Concepts in Pharmacoepidemiology: Multi-Database Distributed Data Networks.

Pharmacoepidemiol Drug Saf. 2025-7

[3]
Advancements in Electronic Medical Records for Clinical Trials: Enhancing Data Management and Research Efficiency.

Cancers (Basel). 2025-5-2

[4]
Real-world evidence to support health technology assessment and payer decision making: is it now or never?

Int J Technol Assess Health Care. 2025-3-31

[5]
NON-SMALL-CELL LUNG CANCER: Real-World Population-Based Cohorts' Study.

Cancers (Basel). 2025-2-14

[6]
A Comprehensive Framework for Evaluating the Value Created by Real-World Evidence for Diverse Stakeholders: The Case for Coordinated Registry Networks.

Ther Innov Regul Sci. 2024-11

[7]
Paving the way for patient centricity in real-world evidence (RWE): Qualitative interviews to identify considerations for wider implementation of patient-reported outcomes in RWE generation.

Heliyon. 2023-9-14

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