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真实世界证据:在美国医保支付方决策的现实世界中有用吗?如何有用?何时有用?以及哪些研究有用?

Real-World Evidence: Useful in the Real World of US Payer Decision Making? How? When? And What Studies?

作者信息

Malone Daniel C, Brown Mary, Hurwitz Jason T, Peters Loretta, Graff Jennifer S

机构信息

College of Pharmacy, The University of Arizona, Tucson, AZ, USA.

College of Pharmacy, The University of Arizona, Tucson, AZ, USA.

出版信息

Value Health. 2018 Mar;21(3):326-333. doi: 10.1016/j.jval.2017.08.3013. Epub 2017 Oct 18.

DOI:10.1016/j.jval.2017.08.3013
PMID:29566840
Abstract

OBJECTIVES

To examine how real-world evidence (RWE) is currently perceived and used in managed care environments, especially to inform pharmacy and therapeutic (P&T) committee decisions, to assess which study factors (e.g., data, design, and funding source) contribute to RWE utility in decisions, and to identify barriers to consideration of RWE studies in P&T decision making.

METHODS

We conducted focus groups/telephone-based interviews and surveys to understand perceptions of RWE and assess awareness, quality, and relevance of two high-profile examples of published RWE studies. A purposive sample comprised 4 physicians, 15 pharmacists, and 1 researcher representing 18 US health plans and health system organizations.

RESULTS

Participants reported that RWE was generally used, or useful, to inform safety monitoring, utilization management, and cost analysis, but less so to guide P&T decisions. Participants were not aware of the two sample RWE studies but considered both studies to be valuable. Relevant research questions and outcomes, transparent methods, study quality, and timely results contribute to the utility of published RWE. Perceived organizational barriers to the use of published RWE included lack of skill, training, and timely study results.

CONCLUSIONS

Payers recognize the value of RWE, but use of such studies to inform P&T decisions varies from organization to organization and is limited. Relevance to payers, timeliness, and transparent methods were key concerns with RWE. Participants recognized the need for continuing education on evaluating and using RWE to better understand the study methods, findings, and applicability to their organizations.

摘要

目的

探讨在管理式医疗环境中,当前对真实世界证据(RWE)的认知和使用情况,尤其是为药学与治疗学(P&T)委员会的决策提供信息;评估哪些研究因素(如数据、设计和资金来源)有助于RWE在决策中的应用;并确定在P&T决策中考虑RWE研究的障碍。

方法

我们开展了焦点小组/电话访谈和调查,以了解对RWE的认知,并评估已发表的RWE研究的两个备受瞩目的案例的知晓度、质量和相关性。一个有目的的样本包括4名医生、15名药剂师和1名代表18家美国健康计划和卫生系统组织的研究人员。

结果

参与者报告称,RWE通常被用于或有助于进行安全监测、利用管理和成本分析,但在指导P&T决策方面作用较小。参与者不了解这两个样本RWE研究,但认为这两项研究都有价值。相关的研究问题和结果、透明的方法、研究质量和及时的结果有助于已发表的RWE的应用。对已发表的RWE使用的感知组织障碍包括缺乏技能、培训和及时的研究结果。

结论

支付方认识到RWE的价值,但此类研究在为P&T决策提供信息方面的使用因组织而异且有限。与支付方的相关性、及时性和透明的方法是RWE的关键问题。参与者认识到需要开展关于评估和使用RWE的继续教育,以更好地理解研究方法、结果及其对各自组织的适用性。

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