Xia Andrew D, Schaefer Caroline P, Szende Agota, Jahn Elke, Hirst Matthew J
Mundibiopharma Limited, Cambridge, UK.
Covance Market Access Services, Gaithersburg, MD, USA.
Drugs Real World Outcomes. 2019 Dec;6(4):193-203. doi: 10.1007/s40801-019-00167-6.
Real-world evidence (RWE) is essential for the development of pharmaceutical and medical technologies and informs treatment-related decisions by regulatory agencies, payers, healthcare providers, and patients. Given that planning RWE studies present diverse challenges, we developed the RWE Framework, a concise, visual, interactive tool designed to align multidisciplinary stakeholders toward common goals and encourage a methodical approach to RWE study planning.
A search of published literature and internet-based resources was performed to identify guidance on RWE study planning with decision and/or visual aids. A conceptual framework for a study design tool was developed based on best practices for RWE studies, enhanced with an infographic design, and refined by multidisciplinary input from RWE researchers.
The searches confirmed an unmet need for a concise tool to support a broad range of RWE study designs: only two sources with decision/visual aids were identified. The novel RWE Framework comprises sequential decision steps with instructions to guide users through consideration of research objectives, product approval status, study setting, outcomes of interest, data availability in routine practice, need for primary data collection and/or randomization, study type and methodology, and applicable regulatory standards. Pilot testing using case studies of pharmaceutical assets demonstrated the utility of RWE Framework and applicability for use in team environments.
The RWE Framework is a novel, concise, visual, and interactive tool to inform RWE study planning. It addresses a broad range of real-world study types and research objectives and was found to enhance RWE decision-making by multidisciplinary teams. Further validation is warranted.
真实世界证据（RWE）对于制药和医疗技术的发展至关重要，并为监管机构、支付方、医疗服务提供者和患者提供与治疗相关的决策依据。鉴于开展RWE研究面临各种挑战，我们开发了RWE框架，这是一种简洁、直观、交互式的工具，旨在使多学科利益相关者朝着共同目标保持一致，并鼓励以有条不紊的方式进行RWE研究规划。
对已发表的文献和基于互联网的资源进行检索，以确定有关RWE研究规划的决策和/或视觉辅助工具的指南。基于RWE研究的最佳实践，开发了一个研究设计工具的概念框架，并通过信息图设计进行强化，再由RWE研究人员进行多学科投入进行完善。
检索结果证实，对于支持广泛的RWE研究设计的简洁工具存在未满足的需求：仅识别出两个带有决策/视觉辅助工具的来源。新颖的RWE框架包括一系列决策步骤及说明，以指导用户考虑研究目标、产品批准状态、研究环境、感兴趣的结果、常规实践中的数据可用性、原始数据收集和/或随机化的需求、研究类型和方法以及适用的监管标准。使用药品案例进行的试点测试证明了RWE框架的实用性及其在团队环境中的适用性。
RWE框架是一种新颖、简洁、直观且交互式的工具，可用于指导RWE研究规划。它涵盖了广泛的真实世界研究类型和研究目标，并被发现可增强多学科团队的RWE决策。有必要进行进一步验证。
