目的:本手稿为评估现实世界证据(RWE)在医疗保健决策中的价值提供了一个全面的框架。虽然RWE已经被提出来克服传统的一些限制,一次性研究,没有系统的框架来衡量RWE是否真的降低了负担。该框架旨在通过提供评估RWE的时间和成本效率的概念方法来填补这一空白,从而指导对RWE基础设施的战略投资。
方法:该框架由四个组成部分组成:(第114届国会。21世纪治愈法。;2015年。https://www.congress.gov/114/plaws/publ255/PLAW-114publ255。PDF。)识别使用和生产RWE的利益相关者,(国家卫生委员会。患者参与术语词汇表。已发布2019年。5月18日访问2021年。https://nationalhealthcouncil.org/glossary-of-patient-engagement-terms/。)了解RWE如何使利益相关者受益的价值主张,(药物评价和研究中心。CDER以患者为中心的药物开发。美国食品和药物管理局。)定义关键绩效指标(KPI),和(美国卫生与人类服务部-食品和药物管理局:设备和放射健康中心以及生物制品评估和研究中心。使用真实世界的证据来支持医疗器械的监管决策-工业和食品和药品管理局工作人员指南。2017.http://www。fda.gov/生物制品血液疫苗/指南合规性监管信息/Guida。)建立指标和案例研究来评估价值。KPI被归类为更好,更快,或更便宜的\"作为价值的指标:更好地关注高质量的可操作证据;\'更快,\'表示在证据生成中节省时间,和\'更便宜,与不涉及临床实践中常规收集的数据的方法相比,强调成本效益决策。指标和相关案例研究是根据利益相关者价值主张和选定的KPI量身定制的,这些KPI可用于评估使用RWE与传统证据生成方法相比并比较不同的RWE来源所创造的价值。
结果:通过文献中的指标和案例研究进行操作,RWE的价值被记录为改善治疗效果异质性评估,扩大医疗产品标签,并加快上市后的合规。与传统的一次性方法相比,RWE还可以减少产生证据所需的成本和时间。基于对国家心血管疾病登记处的分析,提出了度量RWE方法检测产品故障信号所节省的时间的度量的原始示例。
结论:本手稿中提出的框架为评估RWE的价值提供了一种全面的方法,适用于所有参与利用RWE进行医疗保健决策的利益相关者。通过提出的指标和举例说明的案例研究,提供了对提高效率的宝贵见解,成本效益,RWE促进了临床和监管领域的决策。虽然这个框架主要集中在医疗设备上,它可能有助于确定其他医疗产品中的RWE价值。通过辨别成本的变化,时间,以及各种证据生成方法中的数据效用,利益相关者有权对RWE基础设施进行战略性投资,并塑造未来的研究工作。
OBJECTIVE: This manuscript presents a comprehensive framework for the assessment of the value of real-world evidence (RWE) in healthcare decision-making. While RWE has been proposed to overcome some limitations of traditional, one-off studies, no systematic framework exists to measure if RWE actually lowers the burden. This framework aims to fill that gap by providing conceptual approaches for evaluating the time and cost efficiencies of RWE, thus guiding strategic investments in RWE infrastructure.
METHODS: The framework consists of four components: (114th Congress. 21st Century Cures Act.; 2015. https://www.congress.gov/114/plaws/publ255/PLAW-114publ255.pdf .) identification of stakeholders using and producing RWE, (National Health Council. Glossary of Patient Engagement Terms. Published 2019. Accessed May 18. 2021. https://nationalhealthcouncil.org/glossary-of-patient-engagement-terms/ .) understanding value propositions on how RWE can benefit stakeholders, (Center for Drug Evaluation and Research. CDER Patient-Focused Drug Development. U.S. Food & Drug Administration.) defining key performance indicators (KPIs), and (U.S. Department of Health and Human Services - Food and Drug Administration: Center for Devices and Radiological Health and Center for Biologics Evaluation and Research. Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices - Guidance for Industry and Food and Drug Administration Staff. 2017. http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guida .) establishing metrics and
case studies to assess value. KPIs are categorized as \'better, faster, or cheaper\" as an indicator of value: better focusing on high-quality actionable evidence; \'faster,\' denoting time-saving in evidence generation, and \'cheaper,\' emphasizing cost-efficiency decision compared to methodologies that do not involve data routinely collected in clinical practice. Metrics and relevant
case studies are tailored based on stakeholder value propositions and selected KPIs that can be used to assess what value has been created by using RWE compared to traditional evidence-generation approaches and comparing different RWE sources.
RESULTS: Operationalized through metrics and
case studies drawn from the literature, the value of RWE is documented as improving treatment effect heterogeneity evaluation, expanding medical product labels, and expediting post-market compliance. RWE is also shown to reduce the cost and time required to produce evidence compared to traditional one-off approaches. An original example of a metric that measures the time saved by RWE methods to detect a signal of a product failure was presented based on analysis of the National Cardiovascular Disease Registry.
CONCLUSIONS: The framework presented in this manuscript offers a comprehensive approach for evaluating the value of RWE, applicable to all stakeholders engaged in leveraging RWE for healthcare decision-making. Through the proposed metrics and illustrated
case studies, valuable insights are provided into the heightened efficiency, cost-effectiveness, and improved decision-making within clinical and regulatory domains facilitated by RWE. While this framework is primarily focused on medical devices, it could potentially inform the determination of RWE value in other medical products. By discerning the variations in cost, time, and data utility among various evidence-generation methods, stakeholders are empowered to invest strategically in RWE infrastructure and shape future research endeavors.