Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making
Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making
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DOI：
10.1002/pds.4297
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发表时间：
2017-09-01
影响因子：
2.6
通讯作者：
Daniel Mullins, C.
中科院分区：
文献类型：
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作者：
Berger, Marc L.;Sox, Harold;Daniel Mullins, C.
目的真实世界证据 (RWE) 包括来自回顾性或前瞻性观察研究和观察登记的数据，并提供超出随机对照试验所解决的见解。 RWE 研究旨在改善医疗保健决策。方法国际药物经济学和结果研究学会 (ISPOR) 和国际药物流行病学学会 (ISPE) 成立了一个工作组，就良好的程序实践提出建议，以增强决策者对 RWD 研究证据的信心。 ISPOR/ISPE 成员和工作组参与者的同行评审为主题和建议框架提供了建立共识的迭代过程。结果 ISPOR/ISPE 工作组的建议涵盖七个主题，例如研究注册、可复制性和利益相关者参与 RWE 研究。这些建议与早期有关研究方法的建议相结合，为 RWE 在医疗保健决策中的广泛使用提供了值得信赖的基础。结论这些建议的重点是在特定人群中检验特定假设的研究的良好程序实践。我们认识到，如果没有决策者、期刊编辑和其他主要利益相关者的适当激励，本报告中的一些建议可能不会被广泛采纳。
PurposeReal-world evidence (RWE) includes data from retrospective or prospective observational studies and observational registries and provides insights beyond those addressed by randomized controlled trials. RWE studies aim to improve health care decision making.MethodsThe International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the International Society for Pharmacoepidemiology (ISPE) created a task force to make recommendations regarding good procedural practices that would enhance decision makers' confidence in evidence derived from RWD studies. Peer review by ISPOR/ISPE members and task force participants provided a consensus-building iterative process for the topics and framing of recommendations.ResultsThe ISPOR/ISPE Task Force recommendations cover seven topics such as study registration, replicability, and stakeholder involvement in RWE studies. These recommendations, in concert with earlier recommendations about study methodology, provide a trustworthy foundation for the expanded use of RWE in health care decision making.ConclusionThe focus of these recommendations is good procedural practices for studies that test a specific hypothesis in a specific population. We recognize that some of the recommendations in this report may not be widely adopted without appropriate incentives from decision makers, journal editors, and other key stakeholders.