Oehrlein Elisabeth M, Schoch Silke, Burcu Mehmet, McBeth Julia F, Bright Jennifer, Pashos Chris L, Willke Richard, Love T Rosie, Mattingly T Joseph, Perfetto Eleanor M
National Health Council, Washington, DC, USA.
National Health Council, Washington, DC, USA.
Value Health. 2023 Jan;26(1):28-38. doi: 10.1016/j.jval.2022.04.1738. Epub 2022 Jul 19.
The Joint ISPOR-ISPE Special Task Force on Real-World Evidence included patient/stakeholder engagement as a recommended good procedural practice when designing, conducting, and disseminating real-world evidence (RWE). However, there are no guidelines describing how patient experience data (PED) can be applied when designing real-world data (RWD) studies. This article describes development of consensus recommendations to guide researchers in applying PED to develop patient-centered RWE.
A multidisciplinary advisory board, identified through recommendations of collaborators, was established to guide development of recommendations. Semistructured interviews were conducted to identify how experienced RWD researchers (n = 15) would apply PED when designing a hypothetical RWD study. Transcripts were analyzed and emerging themes developed into preliminary methods recommendations. An eDelphi survey (n = 26) was conducted to refine/develop consensus on the draft recommendations.
We identified 13 recommendations for incorporating PED throughout the design, conduct, and translation of RWE. The recommendations encompass themes related to the development of a patient-centered research question, designing a study, disseminating RWE, and general considerations. For example, consider how patient input can inform population/subgroups, comparators, and study period. Researchers can leverage existing information describing PED and may be able to apply those insights to studies relying on traditional RWD sources and/or patient registries.
Applying these emerging recommendations may improve the patient centricity of RWE through improved relevance of RWE to patient communities of interest and foster greater multidisciplinary participation and transparency in RWD research. As researchers gather experience by applying the methods recommendations, further refinement of these consensus recommendations may lead to "best practices."
ISPOR - ISPE联合真实世界证据特别工作组将患者/利益相关者参与纳入设计、开展和传播真实世界证据（RWE）时推荐的良好程序实践。然而，尚无指南描述在设计真实世界数据（RWD）研究时如何应用患者体验数据（PED）。本文描述了共识性建议的制定过程，以指导研究人员应用PED来开发以患者为中心的RWE。
通过合作者的推荐确定了一个多学科咨询委员会，以指导建议的制定。进行了半结构化访谈，以确定经验丰富的RWD研究人员（n = 15）在设计假设的RWD研究时将如何应用PED。对访谈记录进行了分析，并将新出现的主题发展为初步的方法建议。开展了一项电子德尔菲调查（n = 26），以完善/达成对建议草案的共识。
我们确定了13条在RWE的整个设计、开展和转化过程中纳入PED的建议。这些建议涵盖了与以患者为中心的研究问题的制定、研究设计、RWE传播以及一般考虑相关的主题。例如，考虑患者的意见如何为研究人群/亚组、对照和研究周期提供信息。研究人员可以利用描述PED的现有信息，并可能能够将这些见解应用于依赖传统RWD来源和/或患者登记处的研究。
应用这些新出现的建议可能会通过提高RWE与感兴趣的患者群体的相关性来改善RWE的以患者为中心程度，并促进RWD研究中更大程度的多学科参与和透明度。随着研究人员通过应用这些方法建议积累经验，对这些共识性建议的进一步完善可能会产生“最佳实践”。
