真实世界数据和证据在2018-2019年欧盟批准的肿瘤学药物中的作用

IF 2 Q3 HEALTH POLICY & SERVICES
Sini M. Eskola , Hubertus G.M. Leufkens , Andrew Bate , Marie Louise De Bruin , Helga Gardarsdottir
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引用次数: 0

摘要

现实世界数据(RWD)的使用已经引起了癌症护理的不同利益相关者的兴趣。本研究的目的是确定和描述2018年和2019年EMA授权产品(n=111)在预授权阶段RWD/RWE的使用情况,重点是肿瘤学药物(n=24)。从欧洲公共评估报告(EPAR)摘要中提取信息,并记录药物开发生命周期的5个阶段(11类)(发现、早期开发、临床开发、注册/上市、生命周期管理)。对完整EPAR的具体章节进行了审查,以证实RWD/RWE在临床试验设计、疗效、安全性和有效性评估中的使用结果。RWD/RWE存在于肿瘤药物开发的各个阶段;发现占100.0%,早期开发占37.5%,临床开发占58.3%,注册决定占62.5%,授权后生命周期管理占100.0%。实例表明,RWD/RWE支持的试验设计包括使用开放标签/单臂研究;疗效是使用结果与历史对照的比较,提供临床试验之外获得的调查数据,或利用专家小组的建议;将文献发现纳入证据的安全性;以及将给定产品的试验结果与历史数据或现有护理标准进行比较的有效性。这项研究的结果为RWD/RWE如何用于癌症治疗方法的开发、它如何有助于监管决策提供了具体的见解,并可以指导该领域的进一步政策发展。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The role of Real-World Data and evidence in oncology medicines approved in EU in 2018–2019

Use of Real-World Data (RWD) has gained the interest of different stakeholders in cancer care. The aim of this study was to identify and describe the use of RWD/RWE during the pre-authorization phase of products authorized by the EMA in 2018 and 2019 (n = 111), with the focus on oncology medicines (n = 24). Information was extracted from the European Public Assessment Report (EPAR) summaries and recorded for 5 stages (11 categories) of the drug development lifecycle (discovery, early development, clinical development, registration/market launch, lifecycle management). Specific chapters of full EPAR were reviewed to substantiate the findings on RWD/RWE use in clinical trial design, efficacy, safety, and effectiveness evaluation. RWD/RWE is present in all stages of the oncology drug development; 100.0 % in discovery, 37.5 % early development, 58.3 % in clinical development, 62.5 % in registration decision and 100.0 % in post-authorization lifecycle management. Examples showed that trial design supported by RWD/RWE included use of open label/single arm studies; efficacy was about using either comparison of results to historical controls, supplying survey data obtained outside the clinical trial or utilizing expert panel advice; safety about including literature findings in evidence; and effectiveness on comparison of trial results of the given product to historical data or existing standard of care. The findings of this study provide specific insights into how RWD/RWE is used in development of cancer therapeutics, how it contributes to regulatory decision making and can guide further policy developments in this field.

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来源期刊
Journal of Cancer Policy
Journal of Cancer Policy Medicine-Health Policy
CiteScore
2.40
自引率
7.70%
发文量
47
审稿时长
65 days
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