Global Database Studies Team, Real World Solutions IQVIA, London, UK.
Epidemiology & Real World Science, Parexel International, Gothenberg, Sweden.
Pharmacoepidemiol Drug Saf. 2024 Sep;33(9):e5862. doi: 10.1002/pds.5862.
There has been rapid growth in the variety and number of real-world data (RWD) sources, as well as the number of regulatory documents that provide guidance for assessing the suitability of RWD sources for pharmacoepidemiology studies. This study aims to assess differences in RWD guidance and variability in current practice for identifying and assessing RWD for studies with regulatory purpose.
Key criteria for feasibility assessment were mapped against relevant regulatory guidance documents across US, EU, and Asia-Pacific regions. An online survey was designed and deployed to International Society for Pharmacoepidemiology members to understand current practice. Findings were summarized and used to inform key considerations and recommendations.
Eleven RWD guidance documents were identified and mapped against 14 RWD assessment criteria. Variability was seen across these documents in guidance for these criteria. Between December 2022 and January 2023, 37 survey respondents reported having used RWD for post-marketing commitments (34, 92%) and/or background epidemiology (28, 76%). RWD were mostly identified through literature (33, 89%) and data landscaping (26, 70%); guidance documents referenced included: Food and Drug Administration (20, 54%), European Network for Centres for Pharmacoepidemiology and Pharmacovigilance (17, 46%), European Medical Agency (16, 43%), and Structured Process to Identify Fit-For-Purpose Data (11, 30%). Challenges for conducting feasibility assessments included RWD accessibility, ability to complete validation, and RWD provider responsiveness.
Existing guidelines are used extensively by researchers, but key criteria for RWD identification and feasibility assessment are not reflected consistently and challenges remain. Recommendations have been made reflecting study findings.
真实世界数据（RWD）来源的种类和数量以及为评估 RWD 来源是否适合进行药物流行病学研究提供指导的监管文件数量都在迅速增加。本研究旨在评估用于评估具有监管目的的研究的 RWD 的适用性的 RWD 指南之间的差异以及当前实践中的可变性。
将可行性评估的关键标准与美国、欧盟和亚太地区的相关监管指南文件进行了对照。设计并部署了一份在线调查，以了解国际药物流行病学学会成员的当前实践情况。总结了调查结果，并将其用于提供关键考虑因素和建议。
确定了 11 份 RWD 指导文件，并将其与 14 项 RWD 评估标准进行了对照。这些文件在这些标准的指导方面存在差异。在 2022 年 12 月至 2023 年 1 月期间，37 名调查受访者报告说已经使用 RWD 进行了上市后承诺（34 名，92%）和/或背景流行病学（28 名，76%）。RWD 主要通过文献（33 名，89%）和数据景观（26 名，70%）识别；引用的指南文件包括：美国食品和药物管理局（20 名，54%）、欧洲药物流行病学和药物警戒网络（17 名，46%）、欧洲药品管理局（16 名，43%）和结构化流程以确定适合目的的数据（11 名，30%）。进行可行性评估的挑战包括 RWD 的可访问性、完成验证的能力以及 RWD 提供商的响应能力。
研究人员广泛使用现有的指南，但 RWD 识别和可行性评估的关键标准没有得到一致反映，仍然存在挑战。根据研究结果提出了建议。
