目标:监管和卫生技术评估(HTA)机构越来越多地发布框架,指导方针,以及在医疗保健决策中使用真实世界证据(RWE)的建议。这些文件的范围和内容的变化,更新并行运行,可能会给它们的实施带来挑战,尤其是在药品生命周期的市场授权和报销阶段。此环境扫描旨在全面识别和总结大多数完善的监管和报销机构为RWE制定的指导文件,以及其他专注于医疗保健决策的组织,并呈现它们的异同。方法:RWE指导文件,包括监管机构和HTA机构的白皮书,在2024年3月进行了审查。由两名审核员提取了每个机构的范围和建议数据,并在四个主题上总结了异同:研究计划,选择适合目的的数据,研究行为,和报告。排除授权后或非药物指导。结果:在多个机构中确定了46份文件;美国FDA制定了与RWE相关的指南。所有机构都解决了与研究设计有关的特定且通常类似的方法问题,数据适合目的,可靠性,和再现性,尽管注意到这些主题的术语不一致。两个HTA机构(国家健康与护理卓越研究所[NICE]和加拿大药品局)各自将所有相关的RWE指导集中在一个统一的框架下。RWE质量工具和清单的命名不一致,并注意到偏好的一些差异。欧洲药品管理局,Nice,高级自动驾驶,卫生保健质量和效率研究所包括关于使用分析方法来解决RWE复杂性并增加对其结果的信任的具体建议。结论:机构对RWE研究设计的期望相似,质量元素,和报告将促进制造商面临的证据生成策略和活动,包括全球,监管和报销提交和重新提交。决策机构对本地现实世界数据生成的强烈偏好可能会阻碍数据共享和来自国际联合数据网络的输出的机会。决策机构之间更紧密的合作,以实现统一的RWE路线图,可以集中保存在生活模式中,将为制造商和研究人员提供最低验收要求和期望的清晰度,特别是作为RWE一代的新方法正在迅速出现。
Aim:
Regulatory and health technology assessment (HTA) agencies have increasingly published frameworks, guidelines, and recommendations for the use of real-world evidence (RWE) in healthcare decision-making. Variations in the scope and content of these documents, with updates running in parallel, may create challenges for their implementation especially during the market authorization and reimbursement phases of a medicine\'s life cycle. This environmental scan aimed to comprehensively identify and summarize the guidance documents for RWE developed by most well-established
regulatory and reimbursement agencies, as well as other organizations focused on healthcare decision-making, and present their similarities and differences. Methods: RWE guidance documents, including white papers from
regulatory and HTA agencies, were reviewed in March 2024. Data on scope and recommendations from each body were extracted by two reviewers and similarities and differences were summarized across four topics: study planning, choosing fit-for-purpose data, study conduct, and reporting. Post-authorization or non-pharmacological guidance was excluded. Results: Forty-six documents were identified across multiple agencies; US FDA produced the most RWE-related guidance. All agencies addressed specific and often similar methodological issues related to study design, data fitness-for-purpose, reliability, and reproducibility, although inconsistency in terminologies on these topics was noted. Two HTA bodies (National Institute for Health and Care Excellence [NICE] and Canada\'s Drug Agency) each centralized all related RWE guidance under a unified framework. RWE quality tools and checklists were not consistently named and some differences in preferences were noted. European Medicines Agency, NICE, Haute Autorité de Santé, and the Institute for Quality and Efficiency in Health Care included specific recommendations on the use of analytical approaches to address RWE complexities and increase trust in its findings. Conclusion: Similarities in agencies\' expectations on RWE studies design, quality elements, and reporting will facilitate evidence generation strategy and activities for manufacturers facing multiple, including global,
regulatory and reimbursement submissions and re-submissions. A strong preference by decision-making bodies for local real-world data generation may hinder opportunities for data sharing and outputs from international federated data networks. Closer collaboration between decision-making agencies towards a harmonized RWE roadmap, which can be centrally preserved in a living mode, will provide manufacturers and researchers clarity on minimum acceptance requirements and expectations, especially as novel methodologies for RWE generation are rapidly emerging.