真实世界证据加强卫生技术评估中的临床试验证据:对七个国家真实世界证据要求的严格审查和提高接受度的建议

Katia Thokagevistk, Céline Coppo, Laetitia Rey, Amanda Carelli, Veronica Díez, Sarah Vaselenak, Liana Oliveira, Ajay Patel, Emilia Sicari, Teresa Ramos, Susanne Schach, Erika Schirghuber, Alex Simpson, Remy Choquet, Katell Le Lay
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引用次数: 0

摘要

背景:真实世界证据(RWE)可加强卫生技术评估(HTA)中的临床试验证据。目标:回顾七个国家(巴西、加拿大、法国、德国、意大利、西班牙和英国)的卫生技术评估机构(HTAbs)对真实世界证据的要求、真实用途和接受程度,并概述可提高疗效/有效性评估和评价过程中对真实世界证据接受程度的建议。方法:根据 HTAbs 指南总结了 RWE 要求。根据行业经验和案例研究对 HTAbs 的接受程度进行评估。结果:截至 2022 年 6 月,七个国家中有三个国家制定了 RWE 方法指南。HTAbs 通常要求根据当地数据源进行分析,但首选的研究设计和数据源各不相同。HTAbs 有各自的提交、评估和鉴定流程;一些 HTAbs 允许尽早召开方案和/或结果验证会议,但很少有外部专家或医学会参与评估和鉴定。提交、评估和鉴定要求的程度并不一定反映接受程度。结论所有接受审查的国家在使用 RWE 方面都面临着共同的挑战。我们的建议涉及促进与行业和监管机构合作与沟通的必要性,以及在整个评估和鉴定过程中描述 RWE 设计和接受标准的具体准则的必要性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance
Background: Real-world evidence (RWE) can reinforce clinical trial evidence in health technology assessment (HTA). Objectives: Review HTA bodies’ (HTAbs) requirements for RWE, real uses, and acceptance across seven countries (Brazil, Canada, France, Germany, Italy, Spain, and the United Kingdom) and outline recommendations that may improve acceptance of RWE in efficacy/effectiveness assessments and appraisals processes. Methods: RWE requirements were summarized based on HTAbs’ guidelines. Acceptance by HTAbs was evaluated based on industry experience and case studies. Results: As of June 2022, RWE methodological guidelines were in place in three of the seven countries. HTAbs typically requested analyses based on local data sources, but the preferred study design and data sources differed. HTAbs had individual submission, assessment, and appraisal processes; some allowed early meetings for the protocol and/or results validation, though few involved external experts or medical societies to provide input to assessment and appraisal. The extent of submission, assessment, and appraisal requirements did not necessarily reflect the degree of acceptance. Conclusion: All the countries reviewed face common challenges regarding the use of RWE. Our proposals address the need to facilitate collaboration and communication with industry and regulatory agencies and the need for specific guidelines describing RWE design and criteria of acceptance throughout the assessment and appraisal processes.
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