MRC-NIHR Trials Methodology Research Partnership, Department of Health Data Science, University of Liverpool, Liverpool, UK.
Health Services Research Unit, University of Aberdeen, Aberdeen, AB25 2ZD, UK.
Trials. 2024 Jan 29;25(1):94. doi: 10.1186/s13063-024-07926-z.
Healthcare system data (HSD) are increasingly used in clinical trials, augmenting or replacing traditional methods of collecting outcome data. This study, PRIMORANT, set out to identify, in the UK context, issues to be considered before the decision to use HSD for outcome data in a clinical trial is finalised, a methodological question prioritised by the clinical trials community.
The PRIMORANT study had three phases. First, an initial workshop was held to scope the issues faced by trialists when considering whether to use HSDs for trial outcomes. Second, a consultation exercise was undertaken with clinical trials unit (CTU) staff, trialists, methodologists, clinicians, funding panels and data providers. Third, a final discussion workshop was held, at which the results of the consultation were fed back, case studies presented, and issues considered in small breakout groups.
Key topics included in the consultation process were the validity of outcome data, timeliness of data capture, internal pilots, data-sharing, practical issues, and decision-making. A majority of consultation respondents (n = 78, 95%) considered the development of guidance for trialists to be feasible. Guidance was developed following the discussion workshop, for the five broad areas of terminology, feasibility, internal pilots, onward data sharing, and data archiving.
We provide guidance to inform decisions about whether or not to use HSDs for outcomes, and if so, to assist trialists in working with registries and other HSD providers to improve the design and delivery of trials.
医疗保健系统数据（HSD）越来越多地用于临床试验，补充或替代传统的收集结果数据的方法。本研究 PRIMORANT，旨在确定在英国背景下，在最终决定将 HSD 用于临床试验的结果数据之前需要考虑的问题，这是临床试验界优先考虑的方法学问题。
PRIMORANT 研究分为三个阶段。首先，举行了一次初始研讨会，以确定试验人员在考虑是否将 HSD 用于试验结果时面临的问题。其次，与临床试验单位（CTU）工作人员、试验人员、方法学家、临床医生、资助小组和数据提供者进行了磋商。第三，举行了一次最终讨论研讨会，会上反馈了磋商的结果，介绍了案例研究，并在小组讨论中讨论了小组成员考虑的问题。
磋商过程中涉及的关键主题包括结果数据的有效性、数据采集的及时性、内部试点、数据共享、实际问题和决策。大多数磋商受访者（n=78，95%）认为为试验人员制定指南是可行的。在讨论研讨会之后，为五个广泛领域制定了指南，包括术语、可行性、内部试点、后续数据共享和数据存档。
我们提供了指导，以帮助决策者决定是否使用 HSD 来获取结果，以及如果使用，协助试验人员与登记处和其他 HSD 提供者合作，以改进试验的设计和实施。
