Amgen, One Amgen Center Drive, Thousand Oaks, CA, 91320, USA.
Bayer AG, Berlin, Germany.
Ther Innov Regul Sci. 2024 Nov;58(6):1062-1070. doi: 10.1007/s43441-024-00682-x. Epub 2024 Aug 6.
TransCelerate BioPharma surveyed its member biopharmaceutical companies to understand current practices and identify opportunities to complement safety signal assessment with rapid real-world data (RWD) analysis.
A voluntary 30-question questionnaire regarding the use of RWD in safety signal assessment was disseminated to subject matter experts at all TransCelerate member companies in July 2022. Responses were blinded, aggregated, summarized, and presented.
Eighteen of 20 member companies provided responses to the questionnaire. Sixteen (89%) companies reported actively leveraging RWD in their signal assessment processes. Of 18 respondent companies, 8 (44%) routinely use rapid approaches to RWD analysis, 7 (39%) utilize rapid RWD analysis non-routinely or in a pilot setting, 2 (11%) are considering using rapid RWD analysis, and 1 (6%) has no plans to use rapid RWD analysis for their signal assessment. Most companies reported that RWD adds context to and improves quality of signal assessments. To conduct RWD analysis for signal assessment, 16 of 17 (94%) respondent companies utilize or plan to utilize internally available data, 8 (47%) utilize both internal and external data, and 3 (18%) utilize data networks. Respondents identified key challenges to rapidly performing RWD analyses, including data access/availability, time for analysis execution, and uncertainties regarding acceptance of minimal or non-protocolized approaches by health authorities.
Biopharmaceutical companies reported that they see value in the use of rapid RWD analyses for complementing signal assessments. Future work is recommended to offer a framework and process for use of rapid use of RWD analyses in signal assessment.
TransCelerate BioPharma 对其成员生物制药公司进行了调查，以了解当前的实践，并确定利用快速真实世界数据（RWD）分析来补充安全性信号评估的机会。
2022 年 7 月，向所有 TransCelerate 成员公司的主题专家分发了一份关于在安全性信号评估中使用 RWD 的自愿 30 个问题的调查问卷。答复被掩盖、汇总、总结并呈现。
20 家成员公司中有 18 家对问卷做出了答复。16 家（89%）公司报告称正在积极利用 RWD 进行信号评估。在 18 家答复公司中，8 家（44%）公司常规使用快速方法进行 RWD 分析，7 家（39%）公司在试点或非常规情况下使用快速 RWD 分析，2 家（11%）公司正在考虑使用快速 RWD 分析，而 1 家（6%）公司没有计划将快速 RWD 分析用于其信号评估。大多数公司报告称，RWD 为信号评估提供了背景并提高了其质量。为了进行信号评估的 RWD 分析，16 家（94%）答复公司利用或计划利用内部可用数据，8 家（47%）利用内部和外部数据，3 家（18%）利用数据网络。受访者确定了快速进行 RWD 分析的关键挑战，包括数据访问/可用性、分析执行时间以及对健康当局接受最小或非协议化方法的不确定性。
生物制药公司报告称，他们认为快速 RWD 分析在补充信号评估方面具有价值。建议未来开展工作，提供一个用于信号评估中快速使用 RWD 分析的框架和流程。
